Summit Therapeutics Reports Financial Results and Operational Progress for the Second Quarter Ended June 30, 2025




Summit Therapeutics Reports Financial Results and Operational Progress for the Second Quarter Ended June 30, 2025

Ivonescimab in Combination with Chemotherapy Showed Statistically Significant and Clinically Meaningful Improvement in Progression-Free Survival in Global Phase III HARMONi Trial Evaluating Patients with EGFRm NSCLC after EGFR TKI Therapy; Positive Trend Observed in Overall Survival

Interim Overall Survival Analysis Requested from Chinese Health Authorities Shows a Positive Trend Favoring Ivonescimab Compared to Pembrolizumab in PD-L1 Positive Advanced NSCLC from HARMONi-2 Study Conducted by Akeso in China; Ivonescimab Monotherapy Approved by NMPA in China for 1L PD-L1 Positive Advanced NSCLC

Ivonescimab in Combination with Chemotherapy Achieves Statistically Significant, Clinically Meaningful Superiority in PFS vs. Tislelizumab (PD-1 Inhibitor) Plus Chemotherapy in 1L Treatment of Patients with Squamous NSCLC in HARMONi-6 Study Conducted by Akeso in China

Enrollment Continues in Summit’s Global Phase III Trials HARMONi-3 in 1L NSCLC and HARMONi-7 in 1L PD-L1 High NSCLC

Summit and Revolution Medicines Enter Clinical Collaboration Evaluating Ivonescimab in Combination with Three RAS(ON) Inhibitors in RAS Mutant Tumors

MIAMI–(BUSINESS WIRE)–$SMMT–Summit Therapeutics Inc. (NASDAQ: SMMT) (“Summit,” “we,” or the “Company”) today reports its financial results and provides an update on operational progress for the second quarter ended June 30, 2025.

Operational & Corporate Updates

Operational progress continues with ivonescimab (SMT112), an investigational, potentially first-in-class bispecific antibody combining the effects of immunotherapy via a blockade of PD-1 with the anti-angiogenesis effects associated with blocking VEGF into a single molecule:

• Since in-licensing ivonescimab (SMT112), from Akeso Inc. (Akeso, HKEX Code: 9926.HK) in January 2023, over 2,800 patients have been treated in clinical studies globally. Summit has rights to develop and commercialize ivonescimab in the United States, Canada, Europe, Japan, Latin America, including Mexico and all countries in Central America, South America, and the Caribbean, the Middle East, and Africa while Akeso retains development and commercialization rights for the rest of the world, including China.

• Summit is developing ivonescimab in non-small cell lung cancer (“NSCLC”), specifically conducting Phase III clinical trials in the following proposed indications:

• HARMONi: Ivonescimab combined with chemotherapy in patients with epidermal growth factor receptor (EGFR)-mutated, locally advanced or metastatic non-squamous NSCLC who have progressed after treatment with a third-generation EGFR tyrosine kinase inhibitor (TKI)
• HARMONi-3: Ivonescimab combined with chemotherapy in first-line patients with metastatic NSCLC
• HARMONi–7: Ivonescimab monotherapy in patients with first-line metastatic NSCLC whose tumors have high PD-L1 expression

• In May 2025, we announced topline results from our multiregional, double-blinded, placebo-controlled, Phase III study, HARMONi.

• At the prespecified primary data analysis, ivonescimab in combination with chemotherapy demonstrated a statistically significant and clinically meaningful improvement in progression-free survival (PFS), with a hazard ratio of 0.52 (95% CI: 0.41 – 0.66; p<0.00001). PFS was measured by blinded independent central radiology review committee (BICR) compared to placebo in combination with chemotherapy.

• A clinically meaningful hazard ratio was observed in both Asia and ex-Asia sub-populations. The primary analysis demonstrated the consistency of the magnitude of the PFS benefit between patients randomized in Asia and ex-Asia, as well as the consistency in a single-region study (HARMONi-A) with this multiregional study.

• Ivonescimab in combination with chemotherapy showed a positive trend in overall survival (OS) in the primary analysis without achieving a statistically significant benefit with a hazard ratio of 0.79 (95% CI: 0.62 – 1.01; p=0.057). This trend provides further support for its use in 2L+ EGFRm NSCLC, a setting where high unmet need continues to exist with limited approved options in the United States and other western territories. Currently there are no FDA-approved regimens that have demonstrated a statistically significant OS benefit in this patient setting. The median follow-up time for western patients was less than the median OS at the time of the analysis, and these patients may continue to be followed for long-term outcomes. Both Asian and North American patients demonstrated a positive trend in OS. The results of the primary analysis in this multiregional study were consistent with that of the single-region HARMONi-A study, which demonstrated an OS hazard ratio of 0.80 at 52% data maturity in a similar patient population.

• The safety profile of ivonescimab in combination with chemotherapy was acceptable and manageable in the context of the observed clinical benefit.

• Based on the results of the HARMONi clinical trial, Summit, at present time, intends to file a Biologics License Application (BLA) in order to seek approval for ivonescimab plus chemotherapy in this setting. Based on discussions with the United States Food & Drug Administration (FDA), under our determination and subject to our review, Summit will consider the timing of the filing of this BLA.

• A more complete data presentation from HARMONi is intended to be shared at a future major medical conference.

• In April 2025, Akeso announced that HARMONi-6 met its primary endpoint of PFS. This trial, conducted in China by our partners at Akeso with all relevant data exclusively generated, managed, and analyzed by Akeso, evaluated ivonescimab combined with platinum-based chemotherapy against tislelizumab, a PD-1 inhibitor, with the same chemotherapy in patients with locally advanced or metastatic squamous NSCLC, regardless of PD-L1 expression. HARMONi-6 showed statistically significant and clinically meaningful improvement in PFS for ivonescimab plus chemotherapy, and no new safety signals were identified. This marks the first known Phase III trial in NSCLC to show significant improvement over PD-(L)1 inhibitor therapy combined with chemotherapy in a head-to-head setting. Following the success of Akeso’s HARMONi-2 study in China, this is the second instance where ivonescimab-based regimens have demonstrated a statistically significant benefit compared to standard-of-care PD-(L)1 inhibitor-based regimens in a Phase III. The full data set for HARMONi-6 is planned to be presented at an upcoming major medical conference.

• Also in April 2025, Akeso announced that ivonescimab was approved by the Chinese Health Authorities, the National Medical Products Administration (NMPA), for a second indication based on the results of the Phase III clinical trial, HARMONi-2. HARMONi-2 evaluated monotherapy ivonescimab against monotherapy pembrolizumab in patients with locally advanced or metastatic NSCLC whose tumors have positive PD-L1 expression. In conjunction with the approval announcement, Akeso announced that the results of a NMPA-requested interim OS analysis included a hazard ratio of 0.777. The analysis was conducted at 39% data maturity, with a nominal alpha level of 0.0001. HARMONi-2 is a single region, multi-center, Phase III study conducted in China sponsored by Akeso with all relevant data exclusively generated, managed, and analyzed by Akeso.

• Clinical trial collaborations and investigator sponsored trials with leading organizations, including MD Anderson, the Memorial Sloan Kettering Cancer Center, and the Dana Farber Cancer Institute, among others, continue to progress and expand evaluating ivonescimab in solid tumor settings outside of metastatic NSCLC.

• In June 2025, we announced a clinical collaboration with Revolution Medicines to evaluate ivonescimab in combination with three RAS(ON) inhibitors, including the multi-selective inhibitor daraxonrasib (RMC-6236), G12D-selective inhibitor zoldonrasib (RMC-9805), and G12C-selective inhibitor elironrasib (RMC-6291), in solid tumor settings with RAS mutations.

• Enrollment continues in Summit’s global Phase III trials, HARMONi-3 and HARMONi-7. In addition to the enrollment in multiregional studies conducted and sponsored by Summit, our partners at Akeso are also enrolling several single-region Phase III studies exclusively in China in multiple indications, including biliary-tract cancer, triple-negative breast cancer, head and neck squamous cell carcinoma, microsatellite stable colorectal cancer, and pancreatic cancer.

Financial Highlights

Cash and Cash Equivalents and Short-term Investments

• Aggregate cash and cash equivalents and short-term investments were $297.9 million and $412.3 million at June 30, 2025 and December 31, 2024, respectively.
• On August 11, 2025, the Company amended its Distribution Agreement with J.P. Morgan Securities LLC, (the “Sales Agent”), pursuant to which the Company may offer and sell, in an at-the-market (ATM) offering, from time to time, through the Sales Agent, additional shares of the Company’s common stock, having an aggregate offering price of up to $360.0 million. The Company filed a prospectus supplement with the SEC on August 11, 2025 in connection with this offer and sale of the shares pursuant to the Distribution Agreement. The Company has no obligation to sell any of the shares under the Distribution Agreement and may at any time suspend solicitations and offers under the Distribution Agreement.

Stock-Based Compensation Modification Expense

• On April 29, 2025, the compensation committee of the board of directors approved a modification to the Company’s outstanding unvested performance-based stock option awards for certain employees and executives in order to require only service-based vesting requirements to continue vesting considering the overall performance of the company including achievement of the performance goals related to market capitalization of the company for a sustained period of time. As a result, certain options immediately vested on the date of modification, and the remaining options continue to vest over a designated period of time.

• On the modification date, 44.5 million options were valued. These 44.5 million options which were modified represent approximately 6% of total shares outstanding as of June 30, 2025. There had been no prior expense recognized for these unvested performance-based stock options. Based on generally accepted accounting principles in the U.S. (US GAAP), total non-cash stock-based compensation expense for this modification was calculated based on the closing share price of $23.62 on the date of modification.
• Non-cash stock-based compensation expense for the stock options which were immediately vested on the modification date was calculated based on their intrinsic value. For the options which will continue to vest over the future service period, non-cash stock-based compensation expense was calculated using the Black-Scholes valuation methodology.
• For this modification, total non-cash stock-based compensation expense of $466.6 million was recognized during the three months ended June 30, 2025. The unrecognized non-cash stock-based compensation expense of $454.6 million will be recognized over the future remaining service period.

GAAP and Non-GAAP Operating Expenses

• GAAP operating expenses were $568.4 million for the second quarter of 2025, compared to $59.6 million for the same period of the prior year. The increase in GAAP operating expenses was primarily due to the increase in stock-based compensation expense of approximately $466.6 million as a result of the stock option modification noted above.

• Non-GAAP operating expenses were $89.6 million for the second quarter of 2025, compared to $48.5 million for the same period of the prior year. The increase in Non-GAAP operating expenses due to expansion of clinical studies and development costs related to ivonescimab.

GAAP and Non-GAAP Research and Development (R&D) Expenses

• GAAP R&D expenses were $208.0 million for the second quarter of 2025, compared to $30.8 million for the same period of the prior year. This increase was primarily due to the increase in stock-based compensation expense of approximately $123.7 million as a result of the stock option modification noted above.

• Non-GAAP R&D expenses were $79.4 million for the second quarter of 2025, compared to $27.3 million for the same period of the prior year. The increase is primarily related due to expansion of clinical studies and development costs related to ivonescimab.

GAAP and Non-GAAP General and Administrative (G&A) Expenses

• GAAP G&A expenses were $360.4 million for the second quarter of 2025, compared to $13.8 million for the same period of the prior year. The increase was primarily due to the increase in stock-based compensation expense of approximately $342.9 million as a result of the stock option modification noted above.

• Non-GAAP G&A expenses were $10.2 million for the second quarter of 2025, compared to $6.2 million for the same period of the prior year. The increase is related to building our infrastructure to support development of ivonescimab.

GAAP and Non-GAAP Net Loss

• GAAP net loss in the second quarter of 2025 and 2024 was $565.7 million or $(0.76) per basic and diluted share, and $60.4 million or $(0.09) per basic and diluted share, respectively.

• Non-GAAP net loss in the second quarter of 2025 and 2024 was $86.9 million or $(0.12) per basic and diluted share, and $49.3 million or $(0.07) per basic and diluted share, respectively.

Use of Non-GAAP Financial Measures

This release includes measures that are not in accordance with U.S. generally accepted accounting principles (“Non-GAAP measures”). These Non-GAAP measures should be viewed in addition to, and not as a substitute for, Summit’s reported GAAP results, and may be different from Non-GAAP measures used by other companies. In addition, these Non-GAAP measures are not based on any comprehensive set of accounting rules or principles. Summit management uses these non-GAAP measures for internal budgeting and forecasting purposes and to evaluate Summit’s financial performance. Summit management believes the presentation of these Non-GAAP measures is useful to investors for comparing prior periods and analyzing ongoing business trends and operating results. For further information regarding these Non-GAAP measures, please refer to the tables presenting reconciliations of our Non-GAAP results to our U.S. GAAP results and the “Notes on our Non-GAAP Financial Information” that accompany this press release.

About Ivonescimab

Ivonescimab, known as SMT112 in Summit’s license territories, North America, South America, Europe, the Middle East, Africa, and Japan, and as AK112 in China and Australia, is a novel, potential first-in-class investigational bispecific antibody combining the effects of immunotherapy via a blockade of PD-1 with the anti-angiogenesis effects associated with blocking VEGF into a single molecule. Ivonescimab displays unique cooperative binding to each of its intended targets with multifold higher affinity to PD-1 when in the presence of VEGF.

This could differentiate ivonescimab as there is potentially higher expression (presence) of both PD-1 and VEGF in tumor tissue and the tumor microenvironment (TME) as compared to normal tissue in the body. Ivonescimab’s tetravalent structure (four binding sites) enables higher avidity (accumulated strength of multiple binding interactions) in the TME (Zhong, et al, SITC, 2023). This tetravalent structure, the intentional novel design of the molecule, and bringing these two targets into a single bispecific antibody with cooperative binding qualities have the potential to direct ivonescimab to the tumor tissue versus healthy tissue. The intent of this design, together with a half-life of 6 to 7 days after the first dose (Zhong, et al, SITC, 2023), is to improve upon previously established efficacy thresholds, in addition to side effects and safety profiles associated with these targets.

Ivonescimab was engineered by Akeso Inc. (HKEX Code: 9926.HK) and is currently engaged in multiple Phase III clinical trials. Over 2,800 patients have been treated with ivonescimab in clinical studies globally.

Summit began its clinical development of ivonescimab in non-small cell lung cancer (NSCLC), commencing enrollment in 2023 in two multiregional Phase III clinical trials, HARMONi and HARMONi-3. Additionally, in early 2025 the Company began enrolling clinical trial sites in the United States for HARMONi-7.

HARMONi is a Phase III clinical trial which intends to evaluate ivonescimab combined with chemotherapy compared to placebo plus chemotherapy in patients with EGFR-mutated, locally advanced or metastatic non-squamous NSCLC who have progressed after treatment with a 3rd generation EGFR TKI (e.g., osimertinib). Enrollment in HARMONi was completed in the second half of 2024, and top-line results were announced in May of 2025.

HARMONi-3 is a Phase III clinical trial which is intended to evaluate ivonescimab combined with chemotherapy compared to pembrolizumab combined with chemotherapy in patients with first-line metastatic, squamous or non-squamous NSCLC, irrespective of PD-L1 expression.

HARMONi-7 is a Phase III clinical trial which is intended to evaluate ivonescimab monotherapy compared to pembrolizumab monotherapy in patients with first-line metastatic NSCLC whose tumors have high PD-L1 expression.

In addition, Akeso has recently had positive read-outs in three single-region (China), randomized Phase III clinical trials for ivonescimab in NSCLC: HARMONi-A, HARMONi-2, and HARMONi-6.

HARMONi-A was a Phase III clinical trial which evaluated ivonescimab combined with chemotherapy compared to placebo plus chemotherapy in patients with EGFR-mutated, locally advanced or metastatic non-squamous NSCLC who have progressed after treatment with an EGFR TKI.

HARMONi-2 is a Phase III clinical trial evaluating monotherapy ivonescimab against monotherapy pembrolizumab in patients with locally advanced or metastatic NSCLC whose tumors have positive PD-L1 expression.

HARMONi-6 is a Phase III clinical trial evaluating ivonescimab in combination with platinum-based chemotherapy compared with tislelizumab, an anti-PD-1 antibody, in combination with platinum-based chemotherapy in patients with locally advanced or metastatic squamous NSCLC, irrespective of PD-L1 expression.

Ivonescimab is an investigational therapy that is not approved by any regulatory authority in Summit’s license territories, including the United States and Europe. Ivonescimab was initially approved for marketing authorization in China in May 2024. Ivonescimab was granted Fast Track designation by the US Food & Drug Administration (FDA) for the HARMONi clinical trial setting.

About Summit Therapeutics

Summit Therapeutics Inc. is a biopharmaceutical oncology company focused on the discovery, development, and commercialization of patient-, physician-, caregiver- and societal-friendly medicinal therapies intended to improve quality of life, increase potential duration of life, and resolve serious unmet medical needs.

Summit was founded in 2003 and our shares are listed on the Nasdaq Global Market (symbol “SMMT”). We are headquartered in Miami, Florida, and we have additional offices in Menlo Park, California, and Oxford, UK.

For more information, please visit https://www.smmttx.com and follow us on X @SMMT_TX.

Summit Forward-looking Statements

Any statements in this press release about the Company’s future expectations, plans and prospects, including but not limited to, statements about the clinical and preclinical development of the Company’s product candidates, entry into and actions related to the Company’s partnership with Akeso Inc., the Company’s anticipated spending and cash runway, the therapeutic potential of the Company’s product candidates, the potential commercialization of the Company’s product candidates, the timing of initiation, completion and availability of data from clinical trials, the potential submission of applications for marketing approvals, potential acquisitions, statements about the previously disclosed At-The-Market equity offering program (“ATM Program”), the expected proceeds and uses thereof, the Company’s estimates regarding stock-based compensation, and other statements containing the words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “would,” and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including the Company’s ability to sell shares of our common stock under the ATM Program, the conditions affecting the capital markets, general economic, industry, or political conditions, the results of our evaluation of the underlying data in connection with the development and commercialization activities for ivonescimab, the outcome of discussions with regulatory authorities, including the Food and Drug Administration, the uncertainties inherent in the initiation of future clinical trials, availability and timing of data from ongoing and future clinical trials, the results of such trials, and their success, global public health crises, that may affect timing and status of our clinical trials and operations, whether preliminary results from a clinical trial will be predictive of the final results of that trial or whether results of early clinical trials or preclinical studies will be indicative of the results of later clinical trials, whether business development opportunities to expand the Company’s pipeline of drug candidates, including without limitation, through potential acquisitions of, and/or collaborations with, other entities occur, expectations for regulatory approvals, laws and regulations affecting government contracts and funding awards, availability of funding sufficient for the Company’s foreseeable and unforeseeable operating expenses and capital expenditure requirements and other factors discussed in the “Risk Factors” and “Management’s Discussion and Analysis of Financial Condition and Results of Operations” sections of filings that the Company makes with the Securities and Exchange Commission. Any change to our ongoing trials could cause delays, affect our future expenses, and add uncertainty to our commercialization efforts, as well as to affect the likelihood of the successful completion of clinical development of ivonescimab. Accordingly, readers should not place undue reliance on forward-looking statements or information. In addition, any forward-looking statements included in this press release represent the Company’s views only as of the date of this release and should not be relied upon as representing the Company’s views as of any subsequent date. The Company specifically disclaims any obligation to update any forward-looking statements included in this press release.

Summit Therapeutics and the Summit Therapeutics logo are trademarks of Summit Therapeutics Inc.

Copyright 2025, Summit Therapeutics Inc. All Rights Reserved

Contacts

Contact Summit Investor Relations:
Dave Gancarz

Chief Business & Strategy Officer

Nathan LiaBraaten

Senior Director, Investor Relations

investors@smmttx.com
media@smmttx.com

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Neurogene Reports Second Quarter 2025 Financial Results and Highlights Recent Updates




Neurogene Reports Second Quarter 2025 Financial Results and Highlights Recent Updates

Announced design of Embolden™ registrational clinical trial of NGN-401 gene therapy for Rett syndrome; trial initiation activities underway

Completed dosing in Phase 1/2 NGN-401 trial, and remains on track to report updated clinical efficacy and safety data in the second half of 2025

Cash runway into early 2028

NEW YORK–(BUSINESS WIRE)–Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced second quarter 2025 financial results and highlighted recent corporate updates.

“In the first half of 2025, we made significant progress in our NGN-401 program for Rett syndrome. We completed dosing of the last five participants in the Phase 1/2 trial and received written agreement from the U.S. FDA on the key elements of the Embolden™ registrational trial, in which we have already begun initiation activities,” stated Rachel McMinn, Ph.D., Founder and Chief Executive Officer of Neurogene. “We are pleased to be moving forward with Embolden, which was purposefully and rigorously designed to differentiate NGN-401 by evaluating participants as young as three years of age with a primary endpoint that incorporates measures that KOLs, caregivers and payors believe to be clinically meaningful. Our previously announced reallocation of capital provides us runway into early 2028, enabling us to focus our resources on advancement of this potential therapy for the patients and families who urgently need new treatment options.”

Dr. McMinn continued, “In leveraging the continual dialogue under the START program, the FDA encouraged the analysis of the Embolden primary endpoint to remain at 12 months as the basis for full approval, noting that a 6-month endpoint may not be considered clinically meaningful. In further maintaining the rigorous design of the Embolden trial, we are electing to dose the last planned participant from the Phase 1/2 trial as part of the registrational Embolden trial and add one more participant to complete the proposed sample size at 20 patients.”

Second Quarter 2025 and Recent Highlights, and Anticipated Milestones

NGN-401 Gene Therapy for Treatment of Rett Syndrome

• Received written agreement from the U.S. Food and Drug Administration (FDA) on key elements of the Embolden registrational trial of NGN-401 and refined the sample size to propose 20 participants
• Initiated Embolden clinical trial activities to support the conversion of the Phase 1/2 trial to a registrational trial
• Completed enrollment in the Phase 1/2 trial, with the last 5 additional participants dosed in the first half of 2025
• Remains on track to report updated clinical efficacy and safety data from the Phase 1/2 trial in the second half of 2025
• Presented at scientific conferences the hemophagocytic lymphohistiocytosis (HLH) monitoring and treatment algorithm incorporated into the NGN-401 clinical trial, which has been acknowledged as valuable information by the Rett syndrome and gene therapy communities
  • There has been no evidence of HLH/hyperinflammatory syndrome in any NGN-401 trial participant at the 1E15 vg dose level, as of the date of this press release

Upcoming Events

• Stifel Biotech Summer Summit: Management will participate in a fireside chat at 12:00 p.m. ET on August 12 (not webcast)
• H.C. Wainwright Annual Global Investment Conference: Management will participate in a fireside chat at 2:30 p.m. ET on September 8 and participate in 1×1 meetings

Second Quarter 2025 Financial Results

• Cash, Cash Equivalents and Short-Term Investments: Cash, cash equivalents and short-term investments as of June 30, 2025 were $274.5 million. We currently expect cash, cash equivalents and short-term investments to fund planned operations into early 2028.
• Research & Development (R&D) Expenses: R&D expenses were $19.4 million for the three months June 30, 2025 compared to $15.7 million for the three months ended June 30, 2024. The increase in R&D expenses for the three months ended June 30, 2025 was primarily driven by an increase in Rett syndrome clinical trial costs and employee-related expenses due to an increase in R&D headcount.
• General & Administrative (G&A) Expenses: G&A expenses were $6.7 million for the three months ended June 30, 2025 compared to $5.3 million for the three months ended June 30, 2024. The increase in G&A expenses for the three months ended June 30, 2025 was primarily driven by an increase in employee-related expenses due to an increase in stock-based compensation, headcount and other corporate expenses.
• Net Loss: Net loss was $22.0 million for the three months ended June 30, 2025 compared to $18.5 million for the three months ended June 30, 2024.

About Neurogene

The mission of Neurogene is to treat devastating neurological diseases to improve the lives of patients and families impacted by these rare diseases. Neurogene is developing novel approaches and treatments to address the limitations of conventional gene therapy in central nervous system disorders. This includes selecting a delivery approach to maximize distribution to target tissues and designing products to maximize potency and purity for an optimized efficacy and safety profile. The Company’s novel and proprietary EXACT™ transgene regulation platform technology allows for the delivery of therapeutic levels while limiting transgene toxicity associated with conventional gene therapy. Neurogene has constructed a state-of-the-art gene therapy manufacturing facility in Houston, Texas. CGMP production of NGN-401 was conducted in this facility and will support pivotal clinical development activities. For more information, visit www.neurogene.com.

About NGN-401

NGN-401 is an investigational AAV9 gene therapy being developed as a one-time treatment for Rett syndrome. It is the first clinical candidate to deliver the full-length human MECP2 gene under the control of Neurogene’s EXACT™ transgene regulation technology. EXACT technology is an important advancement in gene therapy for Rett syndrome, specifically because the disorder requires a treatment approach that enables targeted levels of MECP2 transgene expression without causing overexpression-related toxic effects associated with conventional gene therapy.

NGN-401 was selected by the U.S. Food and Drug Administration (FDA) for its START Pilot Program and has also received Regenerative Medicine Advance Therapy (RMAT) designation, orphan drug designation, Fast Track designation and rare pediatric designation from the FDA. Neurogene was previously granted an INTERACT meeting with the FDA regarding the EXACT technology. NGN-401 also received Priority Medicines (PRIME) designation, orphan designation and advanced therapy medicinal product designation from the European Medicines Agency (EMA) and the Innovative Licensing and Application Pathway (ILAP) designation from the United Kingdom (UK) Medicines and Healthcare products Regulatory Agency (MHRA).

Cautionary Note Regarding Forward-Looking Statements

Statements in this press release are made as of the date of this press release. Neurogene does not undertake any obligation to make any updates to these statements to reflect events that occur or circumstances that arise after the date of this press release, except as may be required under applicable U.S. securities law.

Statements in this press release which are not historical in nature are intended to be, and hereby are identified as, forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may discuss goals, intentions and expectations as to future plans, trends, events, results of operations or financial condition, or otherwise, based on current expectations and beliefs of the management of Neurogene, as well as assumptions made by, and information currently available to, management of Neurogene, including, but not limited to, statements regarding: the therapeutic potential and utility, efficacy and clinical benefits of NGN-401; trial designs, clinical development plans and timing for NGN-401, including elements of the registrational clinical study trial design subject to final approval of the FDA, such as the proposed number of participants in the Embolden trial, and the timing of the conversion of the NGN-401 Phase 1/2 clinical trial to a registrational clinical trial, anticipated timing of additional updates for the Embolden registrational trial of NGN-401 for Rett syndrome; expected timing for additional interim data from the Company’s NGN-401 Phase 1/2 trial for Rett Syndrome, expected future interactions with or positions of the FDA; and the time period over which existing cash resources may be sufficient to fund the Company’s operations. Forward-looking statements generally include statements that are predictive in nature and depend upon or refer to future events or conditions, and include words such as “may,” “will,” “should,” “would,” “expect,” “anticipate,” “plan,” “likely,” “believe,” “estimate,” “project,” “intend,” “on track,” and other similar expressions or the negative or plural of these words, or other similar expressions that are predictions or indicate future events or prospects, although not all forward-looking statements contain these words. Forward-looking statements are based on current beliefs and assumptions that are subject to risks, uncertainties and assumptions that are difficult to predict with regard to timing, extent, likelihood, and degree of occurrence, which could cause actual results to differ materially from anticipated results and many of which are outside of Neurogene’s control. Such risks, uncertainties and assumptions include, among other things: the expected timing of additional results from the NGN-401 clinical trial; the potential for negative impacts to participants in the Phase 1/2 clinical trial of NGN-401 for the treatment of Rett syndrome; the risk that the Company may not be able to report data on the predicted timeline; risks related to the Company’s ability to obtain regulatory approval for, and ultimately commercialize, its product candidates, including NGN-401; risks related to timing of initiating the Embolden trial of NGN-401 for Rett syndrome; and other risks and uncertainties identified under the heading “Risk Factors” included in Neurogene’s Quarterly Report on Form 10-Q for the quarter ended June 30, 2025, filed with the Securities and Exchange Commission (“SEC”) on August 11, 2025, and other filings that the Company has made and may make with the SEC in the future. Nothing in this communication should be regarded as a representation by any person that the forward-looking statements set forth herein will be achieved or that the contemplated results of any such forward-looking statements will be achieved. Forward-looking statements in this communication speak only as of the day they are made and are qualified in their entirety by reference to the cautionary statements herein. Except as required by applicable law, Neurogene undertakes no obligation to revise or update any forward-looking statement, or to make any other forward-looking statements, whether as a result of new information, future events or otherwise.

This communication contains hyperlinks to information that is not deemed to be incorporated by reference into this communication.

– Financial Tables Follow –

Contacts

Company Contact:
Cara Mayfield

Vice President, Corporate Affairs

cara.mayfield@neurogene.com

Investor Contact:
Melissa Forst

Argot Partners

Neurogene@argotpartners.com

Quince Therapeutics Provides Business Update and Reports Second Quarter 2025 Financial Results




Quince Therapeutics Provides Business Update and Reports Second Quarter 2025 Financial Results

Marks major milestone with completion of enrollment in pivotal Phase 3 NEAT clinical trial evaluating lead asset, eDSP, for the treatment of A-T; topline results expected in first quarter of 2026

Closed financing priced at a premium bringing existing cash position to approximately $35 million; expected to provide runway through Phase 3 topline results and into at least second quarter of 2026

Entered into strategic relationship with Option Care Health to support commercial launch of eDSP in the U.S.

SOUTH SAN FRANCISCO, Calif.–(BUSINESS WIRE)–$QNCX #biotech–Quince Therapeutics, Inc. (Nasdaq: QNCX), a late-stage biotechnology company dedicated to unlocking the power of a patient’s own biology for the treatment of rare diseases, today provided an update on the company’s development pipeline and reported financial results for the second quarter ended June 30, 2025.

Dirk Thye, M.D., Quince’s Chief Executive Officer and Chief Medical Officer, said, “We achieved many critical milestones over the last quarter that significantly advance our research programs and strengthen our business model. Specifically, we completed enrollment in our pivotal Phase 3 NEAT clinical trial, secured additional financing to extend our operating runway sufficiently beyond topline results, and solidified our commercial development planning through our strategic partnership with Option Care Health. Quince remains confident in our ability to deliver topline results in the first quarter of 2026 and subsequent NDA submission in the second half of 2026, assuming positive study results.”

Pivotal Phase 3 NEAT Clinical Trial

• Quince completed enrollment in its pivotal Phase 3 NEAT (Neurological Effects of eDSP on Subjects with A–T; NCT06193200/IEDAT-04-2022) clinical trial with a total of 105 participants, including 83 participants in the six to nine year-old primary analysis population and 22 participants aged 10 years and older.

• Quince expects to report topline results from its Phase 3 NEAT clinical trial in the first quarter of 2026.

• Concluding the NEAT study with 83 enrolled participants in the six to nine year-old primary analysis population reflects powering of approximately 90% to determine statistical significance of the primary endpoint.

• All 50 NEAT participants to date have elected to transition to the open label extension (OLE) study (NCT06664853/IEDAT-04-2022). Participants who complete the full treatment period, complete study assessments, and provide informed consent are eligible to transition to the OLE study.

• The Phase 3 NEAT clinical trial is being conducted under a Special Protocol Assessment (SPA) agreement with the U.S. Food and Drug Administration (FDA).

• Assuming positive study results, the company plans to submit a New Drug Application (NDA) to the FDA in the second half of 2026.

• Quince was granted FDA Fast Track designation for the company’s eDSP System for the treatment of patients with A-T based on the potential to address a high unmet medical need.

• NEAT is an international, multicenter, randomized, double-blind, placebo-controlled clinical trial to evaluate the neurological effects of Quince’s lead asset, eDSP (dexamethasone sodium phosphate [DSP] encapsulated in autologous red blood cells; previously referred to as EryDex) in patients with A-T.

• Participants are randomized (1:1) between eDSP or placebo and treatment consists of six infusions scheduled once every 21 to 30 days. The primary efficacy endpoint will be measured by the change from baseline to last efficacy visit using the Rescored modified International Cooperative Ataxia Rating Scale (RmICARS) compared to placebo.

Pipeline and Corporate Updates

• Announced a strategic relationship with Option Care Health, Inc. (Nasdaq: OPCH), the nation’s largest independent provider of home and ambulatory infusion services, to support the commercial development and efficient launch of Quince’s lead asset, eDSP, in the U.S. The strategic relationship will leverage Option Care’s robust network of specialty pharmacy and ambulatory infusion suites to provide for the administration of eDSP in an effective and efficient way while delivering this innovative treatment to patients with greater geographic flexibility.

• Closed a private placement of common stock and accompanying warrants in June 2025 led by healthcare-focused institutional investor Nantahala Capital with participation from existing Quince stockholders including ADAR1 Capital Management, Legend Capital Partners, and Lagfin S.C.A., new stockholder Second Line Capital, along with members of Quince’s senior management. Priced at a more than a 10% premium to the market price of Quince’s common stock, the financing resulted in approximately $11.5 million in upfront proceeds and potential additional proceeds of up to $10.4 million, if the accompanying warrants are exercised in full, before deducting placement agent fees and other private placement expenses.

• Finalized Phase 2 clinical trial study designs to evaluate eDSP for the potential treatment of patients with Duchenne muscular dystrophy (DMD), the company’s second targeted indication for its lead asset, eDSP. Quince plans to prioritize capital efficient study approaches, including potential investigator-initiated trials (IITs), to advance the evaluation of DMD as a second targeted eDSP indication.

• Initiated Study #3 in the company’s European Union pediatric investigational plan (PIP) – named the Pediatric Encapsulated Dexamethasone Sodium Phosphate (PeD) study – to evaluate the safety and pharmacokinetics of eDSP in younger patients with A-T who weigh between nine and 15 kilograms.

• Participated at the 2025 A-T Clinical Research Conference organized by the A-T Society, a leading A-T patient advocacy group based in the United Kingdom, where key opinion leaders (KOLs) presented post hoc data analyses from the company’s prior Phase 3 ATTeST clinical trial, in addition to Quince management providing an overview of the Phase 3 NEAT clinical trial.

• Appointed Dr. Hassan Abolhassani, Assistant Professor of Clinical Immunology and Research Specialists in the Department of Medical Biochemistry and Biophysics at the Karolinska Institutet in Stockholm, Sweden, to the company’s Scientific Advisory Board (SAB). Dr. Abolhassani becomes the ninth member to join Quince’s SAB, which is comprised of leading experts in A-T, biochemistry, neurology, immunology, genetic, hematology, pharmacology, and clinical practice.

Second Quarter 2025 Financial Results

• Reported cash, cash equivalents, and short-term investments of $34.7 million for the second quarter ended June 30, 2025. Quince expects its existing cash runway to be sufficient to fund the company’s capital efficient development plan through Phase 3 NEAT topline results into the second quarter of 2026. If warrants related to the company’s recent financing are exercised in full for cash, Quince’s cash runway would extend into the second half of 2026.

• Reported research and development (R&D) expenses of $6.6 million for the second quarter ended June 30, 2025. R&D expenses primarily included costs related to ongoing Phase 3 NEAT clinical trial activities and related manufacturing costs.

• Reported general and administrative (G&A) expenses of $3.3 million for the second quarter ended June 30, 2025. G&A expenses primarily included personnel-related and stock-based compensation expenses, commercial planning and new product planning expenses, and other professional administrative costs.

• Reported a net loss of $16.1 million, or a net loss of $0.34 per basic and diluted share, for the second quarter ended June 30, 2025. Weighted average shares outstanding for the year were 46.7 million.

• Reported net cash used in operating activities of $21.0 million for the six months ended June 30, 2025. Cash used in operating activities was primarily due to net loss of $31.1 million for the period, adjusted for $9.9 million of non-cash items, including $4.5 million change in the fair value of warrants, $2.7 million in stock-based compensation, $2.5 million change in the fair value of contingent consideration liabilities, $0.8 million change in the fair value of the European Investment Bank loan, and a net decrease in operating assets of $0.5 million, offset by a net increase in accounts payable, and accrued expenses, and other current liabilities of $0.3 million.

About Quince Therapeutics

Quince Therapeutics, Inc. (Nasdaq: QNCX) is a late-stage biotechnology company dedicated to unlocking the power of a patient’s own biology for the treatment of rare diseases. For more information on the company and its latest news, visit www.quincetx.com and follow Quince on social media platforms LinkedIn, Facebook, X, and YouTube.

Forward-looking Statements

Statements in this news release contain “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995 as contained in Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, which are subject to the “safe harbor” created by those sections. All statements, other than statements of historical facts, may be forward-looking statements. Forward-looking statements contained in this news release may be identified by the use of words such as “believe,” “may,” “should,” “expect,” “anticipate,” “plan,” “believe,” “estimated,” “potential,” “intend,” “will,” “can,” “seek,” or other similar words. Examples of forward-looking statements include, among others, statements relating to the timing, success, and reporting of results of the clinical trials and related data, including expected timing of Phase 3 NEAT topline results and submission of a related NDA; expected cash position and operating runway, including cash potentially receivable upon the exercise of warrants; current and future clinical development of eDSP, including for the potential treatment of Ataxia-Telangiectasia (A-T), Duchenne muscular dystrophy (DMD), and other potential indications; the strategic development path for eDSP, including the anticipated benefits of the strategic partnership with Option Care Health; planned regulatory agency submissions and clinical trials and timeline, prospects, and milestone expectations; and the potential benefits of eDSP and the company’s market opportunity. Forward-looking statements are based on Quince’s current expectations and are subject to inherent uncertainties, risks, and assumptions that are difficult to predict and could cause actual results to differ materially from what the company expects. Further, certain forward-looking statements are based on assumptions as to future events that may not prove to be accurate. Factors that could cause actual results to differ include, but are not limited to, the risks and uncertainties described in the section titled “Risk Factors” in the company’s Annual Report on Form 10-K filed with the Securities and Exchange Commission (SEC) on March 24, 2025, Quarterly Report on Form 10-Q filed with the SEC on May 13, 2025, and other reports as filed with the SEC. Forward-looking statements contained in this news release are made as of this date, and Quince undertakes no duty to update such information except as required under applicable law.

Contacts

Media & Investor Contact:
Stacy Roughan

Quince Therapeutics, Inc.

Vice President, Corporate Communications & Investor Relations

ir@quincetx.com

4 Day Conflict Management for Pharmaceutical Executives Training Course | Vital Strategies and Tools to Identify, Address, and Resolve Workplace Conflicts (ONLINE EVENT: September 15-18, 2025) – ResearchAndMarkets.com




4 Day Conflict Management for Pharmaceutical Executives Training Course | Vital Strategies and Tools to Identify, Address, and Resolve Workplace Conflicts (ONLINE EVENT: September 15-18, 2025) – ResearchAndMarkets.com

DUBLIN–(BUSINESS WIRE)–The “Conflict Management for Pharmaceutical Executives Training Course” training has been added to ResearchAndMarkets.com’s offering.

Effective conflict management is essential for pharmaceutical executives to navigate the complexities of a dynamic, globalised industry.

This four-module webinar equips participants with vital strategies and tools to identify, address, and resolve workplace conflicts. Key topics include understanding the roots and types of conflict, developing communication strategies, managing diverse relationships, and employing the innovative Seven Lens Toolkit for decision-making and problem-solving. Participants will gain practical experience through workshops, case studies, and role-play exercises. The course concludes with a personalised conflict management profile and a tailored development plan.

The webinar will be delivered over four half days to suit the time frames of individuals in the UK, Europe, East Coast United States and Canada. After the webinar, participants will be offered an opportunity to attend a 45-minute confidential coaching session with the course tutor where they will be able to share and discuss their PDP (Personal Development Plan).

Benefits of attending

• Explore the concept of conflict in the workplace (pharmaceutical organisations)
• Understand why and how conflict occurs and how we can limit and prevent it
• Learn how to deal with difficult people and situations
• Discover ourselves and others – people with whom we need to build relationships
• See how to modify behaviour – develop effective communication strategies
• Become more effective in managing and handling conflict – using tools and techniques
• Manage upwards, sideways and downwards (bosses, clients, staff and colleagues)
• Practice using tools and techniques that help us to prevent, mitigate and resolve conflict in the workplace

Key Topics Covered:

Day 1

Introduction

• How do we define conflict in the organisation?
• What causes conflict?
• What types of conflict issues exist within the organisation (comparison of scenarios)
• Defining problem-solving and decision-making related to conflict
• Identifying typical challenges within the Pharma industry at the current time
• Skill sets and competencies needed for conflict management, problem solving, and resolution (self-assessment tool)

Exploring and categorising the different types of conflict

• Intrapersonal
• Interpersonal
• Intragroup
• Intergroup
• Understanding the key challenges and obstacles to managing and resolving conflict in the pharmaceutical industry
• Self- and other-awareness (how I as an individual perceive a situation and how others relate to the same issue)
• Thinking styles
• Influencing styles (strategic versus tactical) when it comes to dealing with conflict
• Cross-cultural differences and attitudes

Day 2

The ‘Seven Lens Toolkit’ for managing conflict

• Assessing (researching facts, data and important information)
• Envisioning (encouraging strategic thinking)
• Testing (engaging in risk analysis and what if scenarios)
• Approving (building trust and buy-in across cultures and functions)
• Innovating (formulating creative ideas and solutions)
• Delivering (developing and executing the action plan for dealing with conflict)
• Self-assessment questionnaire – understanding your strengths and weaknesses

Day 3

Communication

• Exploring the various methods of communication in the contemporary business world
• Remote versus face-to-face communication challenges
• Learning how to deploy communication tools such as Zoom, Microsoft Teams, Google Meet etc. for dealing with conflict (intragroup and intergroup)
• How to navigate organisational structures when dealing with conflict e.g. hierarchical versus matrix structures
• Solving problems and making decisions with people from different cultural backgrounds
• Managing expectations when dealing with conflict
• Understanding how conflicting values and ideas can affect conflict resolution

Day 4

Practical workshops with case studies and role play

• Modifying our behaviour to suit conflict challenges
• Understanding the differences between aggressive, assertive and passive behaviour
• How to plan communication sessions prior to engaging in conflict resolution (one to one, one to group or within a group)
• Exploring typical scenarios that occur within the pharmaceutical regarding conflict resolution case studies and role plays)
• Learning, acquiring and practising effective behaviours that help to resolve conflict
• Learning assertive techniques that help to deal with potential conflict
• How to minimise negativity and acrimonious consequences when dealing with difficult people (role-playing exercises)

Conclusion

• Participants will be given the opportunity to identify their own personal conflict management profile, as well as a 5-10 point Personal Development Plan (PDP)

Speakers:

Robert Hersowitz

Director

Falconbury Ltd

Robert Hersowitz is director of his own business consultancy specialising in organisational and management development and works closely with top management as a consultant and executive coach on change management, human resources and leadership issues. He has established an international reputation over the past 21 years, working with blue chip companies worldwide. Well-known for his work in designing and delivering management workshops and seminars across many sectors, he regularly contributes as a key-note speaker at international conferences and has written numerous articles.

For more information about this training visit https://www.researchandmarkets.com/r/48zvx4

About ResearchAndMarkets.com

ResearchAndMarkets.com is the world’s leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends.

Contacts

ResearchAndMarkets.com

Laura Wood, Senior Press Manager

press@researchandmarkets.com

For E.S.T Office Hours Call 1-917-300-0470

For U.S./ CAN Toll Free Call 1-800-526-8630

For GMT Office Hours Call +353-1-416-8900

North America Clinical Trials Market Growth Trends Report 2025-2033 | Lengthy Clinical Trials, Rising Viral Disorders, Demand for New Treatments, and Government R&D Support – ResearchAndMarkets.com




North America Clinical Trials Market Growth Trends Report 2025-2033 | Lengthy Clinical Trials, Rising Viral Disorders, Demand for New Treatments, and Government R&D Support – ResearchAndMarkets.com

DUBLIN–(BUSINESS WIRE)–The “North America Clinical Trials Market Size and Forecast Report 2025-2033” report has been added to ResearchAndMarkets.com’s offering.

The North America Clinical Trials Market is expected to reach US$ 33.91 billion by 2033 from US$ 20.07 billion in 2024, with a CAGR of 6.00% from 2025 to 2033. This is explained by longer clinical trial cycles, the demand for new treatments, and an increase in viral disorders as COVID-19 and diabetes, as well as advantageous government R&D spending.

North America Clinical Trials Industry Overview

The growing need for novel medicines in a variety of therapeutic areas, including neurology, cardiology, and oncology, is the main factor propelling the North American clinical trials market’s constant expansion. As a market leader, the United States enjoys the advantages of strong infrastructure, top-notch research facilities, and substantial investments in innovative healthcare.

Drug development procedures are becoming faster and more efficient because to technological developments like big data, artificial intelligence, and decentralized clinical trials. Government financing and support for clinical research also contribute to the market’s expansion. Nonetheless, the sector is still affected by issues including regulatory barriers, trouble recruiting patients, and expensive trial operating expenses. The market is nevertheless vibrant and is anticipated to continue evolving in spite of these obstacles.

The market will expand quickly as a result of the growing number of clinical trials in North America, the pharmaceutical industry’s expensive R&D costs, and the rising incidence of illnesses. Clinical trials for new or uncommon diseases are anticipated to benefit from the diverse disease profiles that are found to be growing with time due to the growing population in the North American region. Therefore, biopharmaceutical companies would be encouraged to increase their investment in clinical trials for a particular disease segment based on the number of patients with that ailment.

As of September 2022, there were over 13,323 ongoing clinical studies in various stages for cancer indications in the US, according to the US National Library of Medicine. Over the past few years, pharmaceutical corporations have likewise been spending more and more on research and development (R&D). This was mostly caused by a large number of patents expiring, which leaves many pharmaceutical companies with no choice except to create new medications. As a result, businesses are investing more in R&D to speed up the creation of medications through clinical trials, which will increase the market as a whole.

Growth Drivers for the North America Clinical Trials Market

Rising Prevalence of Chronic Diseases

One of the main factors propelling the North American clinical trials market’s expansion is the growing incidence of chronic illnesses including diabetes, cancer, and cardiovascular disorders. Clinical trials are being conducted by pharmaceutical corporations and research institutions due to the growing demand for new treatments and therapies as these diseases proliferate.

These studies are essential for assessing the efficacy and security of possible treatments. Clinical trials are crucial for expanding medical knowledge and enhancing patient outcomes since chronic diseases frequently call for long-term management and innovative treatment choices. An older population, which is more likely to suffer chronic illnesses, supports this trend and increases the need for ongoing clinical research and innovative therapy development.

Advancements in Technology

Technological developments are drastically changing the clinical trials environment in North America. Clinical study design, management, and execution are being improved by emerging technologies including artificial intelligence (AI), machine learning (ML), and big data analytics. These technologies aid in the real-time analysis of massive volumes of trial data, the more accurate identification of qualified applicants, and the prediction of patient outcomes.

They thereby shorten trial durations, cut down on mistakes, and enhance decision-making. AI and ML are also being used to remotely monitor patient adherence and optimize protocol design, which lowers costs and increases trial efficiency. These developments make technology a potent growth engine in the dynamic clinical research environment since they not only speed up drug development but also increase trial success rates.

Increased Investment in Oncology Research

One of the main factors propelling the growth of the clinical trials market is the rising incidence of cancer in North America, which has greatly increased funding for oncology research. The creation of novel cancer treatments is receiving a significant amount of support from public and private institutions as well as pharmaceutical firms.

Clinical trials focusing on cancer, such as immunotherapies, targeted medicines, and personalized medical methods, have increased as a result of this financial boom. Research efforts have been sped up by the need to find efficient treatments and raise survival rates, which has prompted quicker trial initiation and increased cooperation between sponsors and research institutes. Since cancer is still one of the top causes of mortality, the region’s clinical trial activity is growing in scope and size due to the strong emphasis on oncology research.

Challenges in the North America Clinical Trials Market

High Operational Costs

One major issue facing the clinical trials sector in North America is high operating costs. Significant costs are associated with conducting a clinical trial, such as hiring highly qualified personnel, investing in cutting-edge technology, building out the facility, adhering to regulations, and continuously gathering and tracking data. Complex trial designs, multi-site coordination, and longer study durations can all result in further increases in these expenses.

Financial limitations can make it difficult for smaller biotech companies and research institutes to start or continue trials, frequently forcing them to rely on collaborations or outside funding. There is still a lot of pressure to strike a balance between cost effectiveness, data quality, and legal requirements. The cost of trials only goes up as they get more creative and customized, which is a major obstacle to larger research and development initiatives.

Regulatory Complexities

One of the biggest obstacles facing the clinical trials sector in North America is the complexity of regulations. To guarantee the safety and effectiveness of novel medications and therapies, organizations such as the U.S. Food and Drug Administration (FDA) implement strict and ever-changing regulations. Although these rules are necessary, following them can cause delays in trial approvals and raise compliance expenses dramatically.

The procedure, which can be time- and resource-intensive, entails thorough documentation, ethical evaluations, and adherence to stringent criteria. Conducting multinational or multi-site studies also adds another level of complexity because different regulatory requirements in different locations need to be carefully maintained. These elements may cause delays in the start of trials, cause schedule disruptions, and increase the administrative load on sponsors and research institutions.

Key Players Analyzed: Overviews, Key Persons, Recent Developments, Revenue

• ICON Plc
• Wuxi AppTec
• SGS SA
• Syneos Health
• PRA Health Sciences Inc
• Pfizer Inc.
• IQVIA
• Medpace
• Stryker Corporation
• Zimmer Biomet Holdings
• Orthofix Medical Inc.
• NuVasive Inc.
• Globus Medical Inc.
• Bejo Zaden BV
• Corteva Agriscience

Key Attributes:

Key Topics Covered:

1. Introduction

2. Research & Methodology

2.1 Data Source

2.1.1 Primary Sources

2.1.2 Secondary Sources

2.2 Research Approach

2.2.1 Top-Down Approach

2.2.2 Bottom-Up Approach

2.3 Forecast Projection Methodology

3. Executive Summary

4. Market Dynamics

4.1 Growth Drivers

4.2 Challenges

5. North America Clinical Trials Market

5.1 Historical Market Trends

5.2 Market Forecast

6. Market Share

6.1 By Phases

6.2 By Indications

6.3 By Study Designs

6.4 By Countries

7. Phases

7.1 Phase 1

7.2 Phase 2

7.3 Phase 3

7.4 Phase 4

8. Indications

8.1 Autoimmune/Inflammation

8.2 Pain management

8.3 Oncology

8.4 CNS Condition

8.5 Diabetes

8.6 Obesity

8.7 Cardiovascular

8.8 Others

9. Study Designs

9.1 Interventional

9.2 Observational

9.3 Expanded Access

10. Countries

10.1 United States

10.2 Canada

10.3 Mexico

10.4 Rest of North America

11. Porter’s Five Forces Analysis

11.1 Bargaining Power of Buyers

11.2 Bargaining Power of Suppliers

11.3 Degree of Rivalry

11.4 Threat of New Entrants

11.5 Threat of Substitutes

12. SWOT Analysis

12.1 Strength

12.2 Weakness

12.3 Opportunity

12.4 Threat

13. Key Players Analysis

For more information about this report visit https://www.researchandmarkets.com/r/8xu5mh

About ResearchAndMarkets.com

ResearchAndMarkets.com is the world’s leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends.

Contacts

ResearchAndMarkets.com

Laura Wood, Senior Press Manager

press@researchandmarkets.com

For E.S.T Office Hours Call 1-917-300-0470

For U.S./ CAN Toll Free Call 1-800-526-8630

For GMT Office Hours Call +353-1-416-8900

Quantum-Si to Participate in the UBS Precision Medicine Frontiers Summit




Quantum-Si to Participate in the UBS Precision Medicine Frontiers Summit

BRANFORD, Conn.–(BUSINESS WIRE)–Quantum-Si Incorporated (Nasdaq: QSI) (“Quantum-Si,” “QSI” or the “Company”), a proteomics technology company redefining protein analysis through single-molecule detection, today announced that it will be participating in the UBS Precision Medicine Frontiers Summit taking place at the Waldorf Astoria Monarch Beach Resort & Club in Dana Point, CA, August 13-14, 2025.

Jeff Hawkins, President and Chief Executive Officer, and Jeff Keyes, Chief Financial Officer, will be available for one-on-one meetings throughout the day on Thursday, August 14, 2025, and Jeff Hawkins will participate in the New Dimensions in Proteomics and Cellular Research panel on the same day from 8:15 – 8:50 AM Pacific Daylight Time.

About Quantum-Si Incorporated

Quantum-Si is transforming proteomics with a benchtop platform that brings single-molecule protein analysis to every lab, everywhere. The Company’s platform enables real-time kinetic-based detection and allows researchers to move beyond traditional, multistep workflows and directly access dynamic, functional protein insights with unparalleled resolution. By making protein analysis simpler, faster, and more informative, Quantum-Si is accelerating proteomic discoveries to improve the way we live. Learn more at quantum-si.com or follow us on LinkedIn or X.

Contacts

Investors & Media
Jeff Keyes

Chief Financial Officer

ir@quantum-si.com

Repare Therapeutics Provides Business Update and Reports Second Quarter 2025 Financial Results




Repare Therapeutics Provides Business Update and Reports Second Quarter 2025 Financial Results

Entered into worldwide licensing agreement with Debiopharm for lunresertib

Evaluating strategic alternatives to maximize shareholder value

Initial data for LIONS and POLAR trials expected to be reported in Q4 2025

$109.5 million in cash and cash equivalents and marketable securities

CAMBRIDGE, Mass. & MONTREAL–(BUSINESS WIRE)–Repare Therapeutics Inc. (“Repare” or the “Company”) (Nasdaq: RPTX), a clinical-stage precision oncology company, today reported financial results for the second quarter ended June 30, 2025.

“We remain focused on exploring strategic alternatives and partnerships across our portfolio to enhance long-term shareholder value, as exemplified by our recent worldwide licensing agreement with Debiopharm for lunresertib and out-licensing of early-stage discovery platforms to DCx,” said Steve Forte, President, Chief Executive Officer and Chief Financial Officer of Repare. “In parallel to evaluating these strategic opportunities for our remaining programs, we expect to deliver initial data from the LIONS and POLAR trials in the fourth quarter.”

Second Quarter 2025 and Recent Portfolio Highlights:

• Entered into a worldwide licensing agreement with Debiopharm for lunresertib
  • In July 2025, Repare entered into an exclusive worldwide licensing agreement with Debiopharm International S.A. (“Debiopharm”) for lunresertib, a first-in-class precision oncology PKMYT1 inhibitor. Under the terms of the agreement, Repare will receive a $10 million upfront payment, and is eligible to receive up to $257 million in potential clinical, regulatory, commercial and sales milestones, including up to $5 million in potential near-term payments, and single-digit royalties on global net sales. This agreement builds on the success of Repare and Debiopharm’s clinical study and collaboration agreement to explore the synergy between lunresertib and Debio 0123, a potential best-in-class, brain penetrant and highly selective WEE1 inhibitor. Debiopharm will assume sponsorship of the MYTHIC study and take over existing and future development activities related to lunresertib.

• Announced out-licensing of its discovery platforms to DCx Biotherapeutics
  • In May 2025, Repare out-licensed its early-stage discovery platforms, including certain platform and program intellectual property, to DCx Biotherapeutics Corporation (“DCx”), a newly-launched Canadian biotechnology company developing next generation precision drug conjugates supported by Amplitude Ventures. In connection with this agreement, Repare received a $1 million upfront payment and is expected to receive $3 million in near-term payments. In addition, Repare received a 9.99% equity position in DCx, including certain dilution protection rights, and is eligible to receive potential future out-licensing, clinical and commercial milestone payments, as well as low single-digit sales royalties for the development of certain products by DCx. In connection with this transaction, Repare recognized a $5.7 million gain during the quarter.

• RP-3467: Potential best-in-class, oral Polθ ATPase/helicase inhibitor
  • Repare is conducting a Phase 1 clinical trial of RP-3467 (POLAR), dosing patients alone and in combination with the poly-ADP ribose polymerase (PARP) inhibitor, olaparib. POLAR is a multicenter, open-label, dose-escalation Phase 1 clinical trial designed to investigate the safety, pharmacokinetics, pharmacodynamics, and preliminary clinical activity of RP-3647 alone or in combination with olaparib in adults with locally advanced or metastatic epithelial ovarian cancer, metastatic breast cancer, metastatic castration-resistant prostate cancer, or pancreatic adenocarcinoma.
  • Upcoming expected milestone:
    • Q4 2025: Topline safety, tolerability and early efficacy data from the POLAR trial in monotherapy and in combination with olaparib.

• RP-1664: First-in-class, oral selective PLK4 Inhibitor
  • Repare completed enrolment of 29 patients in its Phase 1 LIONS clinical trial, evaluating RP-1664 as a monotherapy in adult and adolescent patients with TRIM37-high solid tumors. LIONS is a first-in-human, multicenter, open-label Phase 1 clinical trial designed to investigate safety, pharmacokinetics, pharmacodynamics and the preliminary efficacy of RP-1664.
  • Upcoming expected milestone:
    • Q4 2025: Initial topline safety, tolerability and early efficacy data from the LIONS trial.

• Amended our collaboration and license agreement with Bristol-Myers Squibb Company to include an additional druggable target in the collaboration
  • Repare recognized $0.3 million during the quarter as revenue related to druggable targets, reflecting this option fee payment.
• Exploring strategic alternatives to maximize shareholder value
  • Repare continues to actively explore strategic alternatives, partnerships and sale opportunities across its portfolio to maximize shareholder value.

Second Quarter 2025 Financial Results

• Cash, cash equivalents and marketable securities: Cash, cash equivalents and marketable securities as of June 30, 2025 were $109.5 million.
• Revenue from collaboration agreements: Revenue from collaboration agreements were $0.3 million for the three and six months ended June 30, 2025, respectively, as compared $1.1 million and $53.5 million for three and six months ended June 30, 2024.
• Research and development expense, net of tax credits (Net R&D): Net R&D expenses were $14.3 million and $34.6 million for the three and six months ended June 30, 2025, respectively, as compared to $30.1 million and $63.1 for the three and six months ended June 30, 2024.
• General and administrative (G&D) expenses: G&A expenses were $6.0 million and $13.7 million for the three and six months ended June 30, 2025, respectively, compared to $8.3 million and $16.9 million for the three and six months ended June 30, 2024.
• Net loss: Net loss was $16.7 million, or $0.39 per share, and $46.8 million, or $1.09 per share, in the three and six months ended June 30, 2025, respectively, compared to $34.8 million, or $0.82 per share, and $21.6 million, or $0.51 per share, in the three and six months ended June 30, 2024, respectively.

About Repare Therapeutics Inc.

Repare Therapeutics is a clinical-stage precision oncology company enabled by its proprietary synthetic lethality approach to the discovery and development of novel therapeutics. Repare Therapeutics has developed highly targeted cancer therapies focused on genomic instability, including DNA damage repair. The Company’s clinical-stage pipeline includes RP-3467, a Phase 1 Polθ ATPase inhibitor; and RP-1664, a Phase 1 PLK4 inhibitor. For more information, please visit www.reparerx.com and follow @Reparerx on X (formerly Twitter) and LinkedIn.

Forward-Looking Statements

This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995 and securities laws in Canada. All statements in this press release other than statements of historical facts are “forward-looking statements. These statements may be identified by words such as “aims,” “anticipates,” “believes,” “could,” “estimates,” “expects,” “forecasts,” “goal,” “intends,” “may,” “plans,” “possible,” “potential,” “seeks,” “will” and variations of these words or similar expressions that are intended to identify forward-looking statements, although not all forward-looking statements contain these words. Forward-looking statements in this press release include, but are not limited to, statements regarding: the Company’s licensing arrangements with Debiopharm and DCx, including the potential benefits of such transactions and the receipt of clinical and commercial milestone payments and royalties under such agreements; the Company’s plans for exploring strategic alternatives and partnerships across the clinical portfolio; and the design, objectives, initiation, timing, progress and results of current and future clinical trials of the Company’s product candidates including the advancement of its two ongoing clinical trials. These forward-looking statements are based on the Company’s expectations and assumptions as of the date of this press release. Each of these forward-looking statements involves risks and uncertainties that could cause the Company’s clinical development programs, future results or performance to differ materially from those expressed or implied by the forward-looking statements. Many factors may cause differences between current expectations and actual results, including: the Company’s ability to successfully pursue a strategic transaction on attractive terms, or at all; the potential that success in preclinical testing and earlier clinical trials does not ensure that later clinical trials will generate the same results or otherwise provide adequate data to demonstrate the efficacy and safety of a product candidate; the impacts of macroeconomic conditions, including tariffs and other trade policies, the conflict in Ukraine and the conflict in the Middle East, fluctuations in inflation and uncertain credit and financial markets, on the Company’s business, clinical trials and financial position; unexpected safety or efficacy data observed during preclinical studies or clinical trials; clinical trial site activation or enrollment rates that are lower than expected; the Company’s ability to realize the benefits of its collaboration and license agreements; changes in expected or existing competition; changes in the regulatory environment; the uncertainties and timing of the regulatory approval process; and unexpected litigation or other disputes. Other factors that may cause the Company’s actual results to differ from those expressed or implied in the forward-looking statements in this press release are identified in the section titled “Risk Factors” in the Company’s Annual Report on Form 10-K for the year ended December 31, 2024 filed with the Securities and Exchange Commission (“SEC”) and the Québec Autorité des Marchés Financiers (“AMF”) on March 3, 2025, and in other filings made with the SEC and AMF from time to time, including the Company’s Quarterly Report on Form 10-Q for the quarter ended June 30, 2025. The Company expressly disclaims any obligation to update any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise, except as otherwise required by law. For more information, please visit reparerx.com and follow Repare on X (formerly Twitter) at @RepareRx and on LinkedIn at https://www.linkedin.com/company/repare-therapeutics/.

Contacts

Investor Relations & Media Contact:
Matthew DeYoung

Investor Relations and Media

Argot Partners

investors@reparerx.com

Bicycle Therapeutics Reports Recent Business Progress and Second Quarter 2025 Financial Results




Bicycle Therapeutics Reports Recent Business Progress and Second Quarter 2025 Financial Results

Continued advancement across research and development pipeline, with key program updates expected in 2H 2025

Phase 1/2 Duravelo-4 trial for zelenectide pevedotin in NECTIN4-amplified non-small cell lung cancer open and actively recruiting patients

Strengthened clinical leadership and bolstered roster of scientific advisors with additions to Board of Directors and creation of Research and Innovation Advisory Board

Strategic cost realignment of approximately 30%, primarily through a workforce reduction

Cash and cash equivalents of $721.5 million as of June 30, 2025, with expected financial runway extended into 2028

CAMBRIDGE, England & BOSTON–(BUSINESS WIRE)–Bicycle Therapeutics plc (NASDAQ: BCYC), a pharmaceutical company pioneering a new and differentiated class of therapeutics based on its proprietary bicyclic peptide (Bicycle^®) technology, today reported financial results for the second quarter ended June 30, 2025, and provided recent corporate updates.

“We continue to execute on our strategy, which is grounded in scientific rigor and focused on fulfilling our mission to develop next-generation precision-guided therapeutics that have the potential to help patients live longer and live well,” said Bicycle Therapeutics CEO Kevin Lee, Ph.D. “We are energized by the progress we are making across our pipeline, and with this momentum, we are pleased to welcome our new Research and Innovation Advisory Board members, as well as new Board member Charles Swanton, to further our innovation and strategic growth.”

Dr. Lee continued: “As we advance our various pipeline programs that hold strong potential for changing the treatment paradigm for patients with cancer and creating value for shareholders, Bicycle remains committed to disciplined capital allocation. Today we announced organizational streamlining efforts that provide us with operational flexibility to deliver potentially value-generating datasets while strengthening our financial position in uncertain market conditions. Saying goodbye to talented team members is very difficult, and we sincerely thank them for their dedication to our company. We believe Bicycle is strongly positioned to realize our strategic priorities and milestones and look forward to providing key program updates over the second half of this year.”

Second Quarter 2025 and Recent Events

• Presented additional human imaging data for an early Bicycle Radioconjugate^® (BRC^®) molecule targeting MT1-MMP at the American Association for Cancer Research (AACR) Annual Meeting 2025. A poster presentation included new data from a second patient who underwent MT1-MMP-PET/CT imaging that build on previously announced data. Altogether, the data continue to validate the potential of MT1-MMP as a novel cancer target and demonstrate the positive properties of BRC molecules for radiopharmaceutical imaging. Imaging data from these two patients are representative of the data generated to date in 12 out of 14 patients with various solid tumors.

  Bicycle Therapeutics continues to advance its emerging BRC pipeline, with initial EphA2 human imaging data expected in 2H 2025 and company-sponsored clinical trials planned for 2026.

• Presented two abstracts highlighting the development of Bicycle^® Drug Conjugate (BDC^®) zelenectide pevedotin for metastatic urothelial cancer (mUC) at the 2025 American Society for Clinical Oncology (ASCO) Annual Meeting. The abstracts outlined previously disclosed topline combination data for zelenectide pevedotin plus pembrolizumab in first-line mUC from the Phase 1/2 Duravelo-1 trial and provided an overview of the ongoing Phase 2/3 Duravelo-2 registrational trial for zelenectide pevedotin in mUC.

  Bicycle Therapeutics is on track to provide an update on dose selection from the Duravelo-2 trial and the accelerated approval pathway for zelenectide pevedotin in mUC following a meeting with the U.S. Food and Drug Administration planned for 4Q 2025.

• Phase 1/2 Duravelo-4 trial for zelenectide pevedotin in NECTIN4-amplified non-small cell lung cancer (NSCLC) open and actively recruiting patients. Duravelo-4 is Bicycle Therapeutics’ second trial to leverage NECTIN4 gene amplification as a biomarker for patient selection and to expand the development of zelenectide pevedotin for additional solid tumors.

  With several trials underway assessing the potential for zelenectide pevedotin to treat mUC, breast cancer and lung cancer, the company has decided to pause the previously announced Phase 1/2 Duravelo-5 trial in multiple tumors.

• Expanded Board of Directors with the addition of Charles Swanton, M.D., Ph.D., FRS, FMedSci, FRCP, current chair of Bicycle Therapeutics’ Clinical Advisory Board. Dr. Swanton leads the Cancer Evolution and Genome Instability Laboratory at the Francis Crick Institute. His research focuses on how tumors evolve over space and time, developing an understanding of branching evolutionary histories of solid tumors, processes that drive cancer cell-to-cell variation and the impact of cancer diversity on effective immune surveillance and clinical outcomes. Dr. Swanton is a fellow of the Royal Society, a fellow of the Royal College of Physicians and a fellow of the Academy of Medical Sciences. He completed his M.D. and Ph.D. training at the Imperial Cancer Research Fund Laboratories.

• Formed Research and Innovation Advisory Board (RAB) to support scientific advancement and strategic growth across preclinical programs. The RAB replaces Bicycle’s Scientific Advisory Board. Inaugural RAB members include:

• Jose-Carlos Gutierrez-Ramos, Ph.D., is a director on the Bicycle Therapeutics Board of Directors. He also serves as the chief science officer at Danaher Corporation, leading the Danaher Innovation Centers and the Danaher Scientific Advisory Board. Previously, Dr. Gutierrez-Ramos was head of global drug discovery at AbbVie Inc., group senior vice president of biotherapeutics research and development (R&D) at Pfizer Inc., and senior vice president and CEDD head of immuno-inflammation at GlaxoSmithKline plc. He was also the founding CEO and president of Repertoire Immune Medicine, where he built and led a team focused on decoding the human immunome. Prior to that, he served as president and CEO of Synlogic, Inc. Dr. Gutierrez-Ramos earned a Ph.D. from the immunology department of the Center for Molecular Biology at the Universidad Autonoma de Madrid, and a B.S., summa cum laude, in chemistry with a minor in biochemistry from the Universidad Complutense de Madrid.
• Jason Lewis, Ph.D., is the Emily Tow Chair at Memorial Sloan Kettering Cancer Center (MSKCC) and currently serves as the deputy director at the Sloan Kettering Institute, overseeing the Office of Scientific Education and Training. He is also the scientific director of the Radiochemistry and Molecular Imaging Probe Core Facility at MSKCC. Dr. Lewis is a laboratory head in Sloan-Kettering Institute’s molecular pharmacology program and serves as a professor at the Gerstner Sloan-Kettering Graduate School of Biomedical Sciences and at Weill-Cornell Medical College. He earned a Ph.D. in biochemistry from the University of Kent and an M.S. and B.S. in chemistry from the University of Essex.
• Robert Lutz, Ph.D., is a consultant/advisor to biotech and pharma with more than 30 years of experience with a significant focus on the development of antibody-drug conjugates (ADCs). He currently serves as chief scientific officer of Iksuda Therapeutics and is a board member and chief development officer of Synthis Therapeutics. Prior to his consulting practice, Dr. Lutz was vice president of translational research and development at ImmunoGen, where he was responsible for the advancement of multiple ADC programs, including KADCYLA^® (ado-trastuzumab emtansine), the first ADC to be approved for solid tumor indications, and ELAHERE^® (mirvetuximab soravtansine). He earned a Ph.D. in biochemistry from Brandeis University and a B.S. in biochemistry from the University of New Hampshire.
• Michael Hofman, MBBS, FRACP, FAANMS, FICIS, GAICD, is a nuclear medicine physician and professor at the Sir Peter MacCallum Department of Oncology at the University of Melbourne in Australia. His research has been instrumental in advancing PSMA PET imaging and PSMA radioligand therapy, helping to revolutionize the diagnosis and treatment of prostate cancer. He was named Australia’s top researcher in nuclear medicine, radiotherapy and molecular imaging in both 2024 and 2025. Professor Hofman leads the PET/CT program and the Prostate Cancer Theranostics and Imaging Centre of Excellence at Peter MacCallum Cancer Centre. He earned a degree in medicine and surgery from Monash University in Australia and undertook a PET/CT fellowship at St. Thomas’ Hospital in London.

• Welcomed Michael Method, M.D., as senior vice president of clinical development. Dr. Method is an academic and clinical gynecologic oncologist with extensive drug development experience. He most recently served as a senior vice president of clinical development at Karyopharm Therapeutics, Inc., after his time as an executive medical director at ImmunoGen, Inc. where he led global clinical development for gynecologic and female malignancies. Previously, Dr. Method was a senior medical advisor for global medical affairs at Eli Lilly, focused on breast cancer. He earned his M.D. and MPH from Northwestern University, and his B.S. in biochemistry and MBA from the University of Notre Dame.

Participation in Upcoming Investor Conferences

Bicycle Therapeutics management will participate in the following investor conferences in September:

• Cantor Global Healthcare Conference on Thursday, Sept. 4; fireside chat at 3:55 p.m. ET
• Morgan Stanley 23^rd Annual Global Healthcare Conference on Tuesday, Sept. 9; fireside chat at 7:45 a.m. ET

Live webcasts of the fireside chats will be accessible in the Investor section of the company’s website at www.bicycletherapeutics.com. Archived replays of the webcasts will be available following the fireside chat dates.

Second Quarter 2025 Financial Results

• Cash and cash equivalents were $721.5 million as of June 30, 2025, compared to $879.5 million as of December 31, 2024. The decrease in cash and cash equivalents is primarily due to cash used in operations, including increased cash payments for clinical program activities.
• R&D expenses were $71.0 million for the three months ended June 30, 2025, compared to $40.1 million for the three months ended June 30, 2024. The increase in expense of $30.9 million was primarily due to increased clinical program expenses for zelenectide pevedotin development, increased discovery, platform and other expenses, and increased personnel-related costs, offset by decreased clinical program expenses for Bicycle Tumor-Targeted Immune Cell Agonist^® (Bicycle TICA^®) molecules as well as higher U.K. R&D tax credits period over period.
• General and administrative expenses were $18.5 million for the three months ended June 30, 2025, compared to $15.9 million for the three months ended June 30, 2024. The increase in expense of $2.6 million was primarily due to increased personnel-related costs, as well as increased professional and consulting fees.
• Net loss was $79.0 million, or $(1.14) basic and diluted net loss per share, for the three months ended June 30, 2025, compared to net loss of $39.8 million, or $(0.77) basic and diluted net loss per share, for three months ended June 30, 2024.

In recognition of the evolving macroeconomic environment and the importance of preserving capital, Bicycle Therapeutics is implementing a workforce reduction and taking other steps to optimize its operations and extend the company’s expected financial runway. These strategic cost realignment efforts are being implemented to prioritize potentially high-impact, value-generating programs, which include the advancement of zelenectide pevedotin, BT5528, next-generation Bicycle^® Drug Conjugates and the company’s wholly owned pipeline of Bicycle^® Radioconjugates. Bicycle Therapeutics anticipates total operational savings of approximately 30% over the course of the financial runway period. These actions are expected to extend the financial runway into 2028 and strengthen the company’s ability to weather continued market uncertainty as it advances clinical programs through key milestones.

About Bicycle Therapeutics

Bicycle Therapeutics is a clinical-stage pharmaceutical company developing a novel class of medicines, referred to as Bicycle^® molecules, for diseases that are underserved by existing therapeutics. Bicycle molecules are fully synthetic short peptides constrained with small molecule scaffolds to form two loops that stabilize their structural geometry. This constraint facilitates target binding with high affinity and selectivity, making Bicycle molecules attractive candidates for drug development. The company is evaluating zelenectide pevedotin (formerly BT8009), a Bicycle^® Drug Conjugate (BDC^®) targeting Nectin-4, a well-validated tumor antigen; BT5528, a BDC molecule targeting EphA2, a historically undruggable target; and BT7480, a Bicycle Tumor-Targeted Immune Cell Agonist^® (Bicycle TICA^®) targeting Nectin-4 and agonizing CD137, in company-sponsored clinical trials. Additionally, the company is developing Bicycle^® Radioconjugates (BRC^®) for radiopharmaceutical use and, through various partnerships, is exploring the use of Bicycle^® technology to develop therapies for diseases beyond oncology.

Bicycle Therapeutics is headquartered in Cambridge, UK, with many key functions and members of its leadership team located in Cambridge, Mass. For more information, visit bicycletherapeutics.com.

Forward Looking Statements

This press release may contain forward-looking statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These statements may be identified by words such as “aims,” “anticipates,” “believes,” “could,” “estimates,” “expects,” “forecasts,” “goal,” “intends,” “may,” “plans,” “possible,” “potential,” “seeks,” “will” and variations of these words or similar expressions that are intended to identify forward-looking statements, although not all forward-looking statements contain these words. Forward-looking statements in this press release include, but are not limited to, statements regarding the validation of MT1-MMP as a cancer target and BRC molecules having positive properties for radiopharmaceutical imaging; the initiation of new clinical trials, the progress of Bicycle’s ongoing clinical trials and the timing of EphA2 human imaging data and updates on dose selection in the Duravelo-2 clinical trial and accelerated approval pathway; the outcome of Bicycle’s strategic cost realignment efforts and Bicycle’s expected financial runway; and the use of Bicycle Therapeutics’ technology through various partnerships to develop therapies for diseases beyond oncology. Bicycle Therapeutics may not actually achieve the plans, intentions or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements as a result of various factors, including: uncertainties inherent in research and development and in the initiation, progress and completion of clinical trials and clinical development of Bicycle Therapeutics’ product candidates; the risk that Bicycle Therapeutics may not realize the intended benefits of its cost realignment efforts; the risk that Bicycle’s projections regarding its expected cash runway are inaccurate or that its conduct of its business requires more cash than anticipated; and other important factors, any of which could cause Bicycle Therapeutics’ actual results to differ from those contained in the forward-looking statements, are described in greater detail in the section entitled “Risk Factors” in Bicycle Therapeutics’ Annual Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) on May 1, 2025, as well as in other filings Bicycle Therapeutics may make with the SEC in the future. Any forward-looking statements contained in this press release speak only as of the date hereof, and Bicycle Therapeutics expressly disclaims any obligation to update any forward-looking statements contained herein, whether because of any new information, future events, changed circumstances or otherwise, except as otherwise required by law.

Contacts

Investors:
Stephanie Yao

SVP, Investor Relations and Corporate Communications

stephanie.yao@bicycletx.com
857-523-8544

Matthew DeYoung

Argot Partners

ir@bicycletx.com
212-600-1902

Media:
Jim O’Connell

Weber Shandwick

media@bicycletx.com
312-988-2343

Revelation Biosciences, Inc. Announces Financial Results for the Three and Six Months Ended June 30, 2025




Revelation Biosciences, Inc. Announces Financial Results for the Three and Six Months Ended June 30, 2025

SAN DIEGO–(BUSINESS WIRE)–$REVB #AKI—Revelation Biosciences, Inc. (NASDAQ: REVB) (the “Company” or “Revelation”), a clinical-stage life sciences company that is focused on rebalancing inflammation to optimize health, today reported its financial results for the three and six months ended June 30, 2025.

Corporate Highlights

• Completed dosing of patients in PRIME Phase 1b Clinical Study of Gemini in CKD Patients
• Received gross proceeds of $4 million from public offering in May 2025

“The Revelation team continues to strategically maximize its financial resources to achieve our stated objectives and advance the Gemini program,” said James Rolke, Chief Executive Officer of Revelation. “We look forward to announcing data from the Phase 1b study later this quarter and engaging with the FDA later this year to further the clinical development of Gemini and enhance shareholder value.”

Results of Operations

As of June 30, 2025, Revelation had $5.2 million in cash and cash equivalents, compared to $6.5 million as of December 31, 2024. The decrease in cash and cash equivalents was primarily due to cash used for operating activities. Based on current operating plans and projections, Revelation believes that its current cash and cash equivalents are sufficient to fund operations through December 2025.

Revelation’s net cash used for operating activities for the three months ended June 30, 2025 was $4.7 million compared to net cash used for operating activities of $5.3 million for the same period in 2024. Revelation’s net loss for the three months ended June 30, 2025 was $2.4 million, or $(7.01) basic and diluted net loss per share compared to a net loss of $8.4 million, or $(246.27) basic and diluted net loss per share for the same period in 2024. Revelation’s net loss for six months ended June 30, 2025 was $4.5 million, or $(13.60) basic and diluted net loss per share compared to net loss of $11.1 million, or $(390.02) basic and diluted net loss per share for the same period in 2024.

About Gemini

Gemini is an intravenously administered, proprietary formulation of phosphorylated hexaacyl disaccharide (PHAD^®) that reduces the damage associated with inflammation by reprogramming the innate immune system to respond to stress (trauma, infection, etc.) in an attenuated manner.

Gemini is being developed for multiple indications including as a pretreatment to prevent or reduce the severity and duration of acute kidney injury (GEMINI-AKI program), and as pretreatment to prevent or reduce the severity and duration of post-surgical infection (GEMINI-PSI program), or infection post severe burn (GEMINI-PBI). Gemini may also be a treatment to stop or slow the progression of chronic kidney disease (GEMINI-CKD program).

Revelation has conducted multiple preclinical studies demonstrating the therapeutic potential of Gemini in the target indications. Revelation previously announced positive Phase 1 clinical data for intravenous treatment with Gemini: the primary safety endpoint was met in the Phase 1 study, and results demonstrated statistically significant pharmacodynamic activity, as observed through expected changes in multiple biomarkers including upregulation of IL-10.

About Revelation Biosciences, Inc.

Revelation Biosciences, Inc. is a clinical stage life sciences company focused on harnessing the power of trained immunity for the prevention and treatment of disease using its proprietary formulation Gemini. Revelation has multiple ongoing programs to evaluate Gemini, including as a prevention for post-surgical infection, as a prevention for acute kidney injury, and for the treatment of chronic kidney disease.

For more information on Revelation, please visit www.RevBiosciences.com.

Forward-Looking Statements

This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended. Forward-looking statements are statements that are not historical facts. These forward-looking statements are generally identified by the words “anticipate”, “believe”, “expect”, “estimate”, “plan”, “outlook”, and “project” and other similar expressions. We caution investors that forward-looking statements are based on management’s expectations and are only predictions or statements of current expectations and involve known and unknown risks, uncertainties and other factors that may cause actual results to be materially different from those anticipated by the forward-looking statements. Revelation cautions readers not to place undue reliance on any such forward looking statements, which speak only as of the date they were made. The following factors, among others, could cause actual results to differ materially from those described in these forward-looking statements: the ability of Revelation to meet its financial and strategic goals, due to, among other things, competition; the ability of Revelation to grow and manage growth profitability and retain its key employees; the possibility that the Revelation may be adversely affected by other economic, business, and/or competitive factors; risks relating to the successful development of Revelation’s product candidates; the ability to successfully complete planned clinical studies of its product candidates; the risk that we may not fully enroll our clinical studies or enrollment will take longer than expected; risks relating to the occurrence of adverse safety events and/or unexpected concerns that may arise from data or analysis from our clinical studies; changes in applicable laws or regulations; expected initiation of the clinical studies, the timing of clinical data; the outcome of the clinical data, including whether the results of such study is positive or whether it can be replicated; the outcome of data collected, including whether the results of such data and/or correlation can be replicated; the timing, costs, conduct and outcome of our other clinical studies; the anticipated treatment of future clinical data by the FDA, the EMA or other regulatory authorities, including whether such data will be sufficient for approval; the success of future development activities for its product candidates; potential indications for which product candidates may be developed; the ability of Revelation to maintain the listing of its securities on NASDAQ; the expected duration over which Revelation’s balances will fund its operations; and other risks and uncertainties described herein, as well as those risks and uncertainties discussed from time to time in other reports and other public filings with the SEC by Revelation.

Contacts

Mike Porter

Investor Relations
Porter LeVay & Rose Inc.

Email: mike@plrinvest.com

Chester Zygmont, III

Chief Financial Officer
Revelation Biosciences Inc.

Email: czygmont@revbiosciences.com

Medical Gas Market Trends and Forecast Report 2025-2033 | Home Healthcare Trends, Chronic Disease Burden, and Medical Tech Advancements Drive Growth – ResearchAndMarkets.com




Medical Gas Market Trends and Forecast Report 2025-2033 | Home Healthcare Trends, Chronic Disease Burden, and Medical Tech Advancements Drive Growth – ResearchAndMarkets.com

DUBLIN–(BUSINESS WIRE)–The “Medical Gas Market Share Analysis and Size – Growth Trends and Forecast Report 2025-2033” report has been added to ResearchAndMarkets.com’s offering.

The Global Medical Gas Market is expected to reach US$ 28.61 billion by 2033 from US$ 14.41 billion in 2024, with a CAGR of 7.92% from 2025 to 2033. Some of the key reasons driving the market are the growing tendency toward home healthcare and telemedicine, the prevalence of chronic illnesses including respiratory and cardiovascular diseases (CVDs), and many developments in medical technology.

Global Medical Gas Industry Overview

The aging population, rising rates of chronic respiratory conditions, and improvements in healthcare infrastructure are all contributing to the substantial expansion of the worldwide medical gas market. Medical gases, such as carbon dioxide, nitrous oxide, and oxygen, are necessary for a number of therapeutic and diagnostic uses in ambulatory surgery centers, home healthcare settings, and hospitals. The need for portable medical gas solutions, such oxygen concentrators, has increased due to the growing demand for home healthcare services.

Due to growing healthcare sectors and government expenditures in medical infrastructure, the Asia-Pacific region is expected to develop at the highest rate, while North America already has a significant market share. The availability and distribution of medical gases may be impacted by supply chain interruptions and strict regulatory restrictions, which provide difficulties for the sector.

The need for medicinal gases is also being fueled by the increasing incidence of asthma. The Australian Institute of Health and Welfare (AIHW) estimates that in 2023, asthma accounted for 35% of the entire burden related to all respiratory disorders and 2.5% of the overall illness burden. Nitrous oxide, carbon dioxide, nitrogen, oxygen, and medical air are some of the most often utilized medical gases in hospitals.

One of the leading companies, Air Liquide Healthcare, provides medicinal gases to 20,000 hospitals and new healthcare facilities, and assists over 2 million people in managing chronic illnesses. Medical gases including heliox, oxygen, and lung gas mixes are widely employed in the diagnosis and treatment of certain respiratory conditions.

Additionally, the need for portable medical gases, such as oxygen concentrators, is rising as more people choose home-based healthcare. In order to ensure that chronic patients receive the oxygen therapy they require at home, this change places an emphasis on convenience and continuity of care. Home healthcare has become increasingly popular in recent years and is predicted to continue to grow. Many medical illnesses may now be successfully treated at home, including those that require ventilator assistance, mixed gas therapies, and long-term oxygen therapy.

Additionally, it is anticipated that rising home healthcare reimbursement would support market expansion throughout the projection period. The Firesafe Cannula Valve, for example, was formally covered by Iowa Medicaid in November 2023 and was given the HCPCS number E0700 for reimbursement. In the event that the oxygen tubing downstream burns, this novel mechanism functions as a thermal fuse and instantly stops the oxygen supply. Thermal fuses must be installed in all home oxygen systems in the United Kingdom. Interestingly, the US has a 20-fold greater risk of death from oxygen-related flames than England, where installing firebreaks has been required since 2006.

Growth Drivers for the Medical Gas Market

Numerous Medical Technology Advancements

Laparoscopy and endoscopy are examples of minimally invasive (MI) surgical techniques that have been made possible by technological breakthroughs. Surgeons can execute treatments with fewer incisions, quicker patient recovery, and less tissue stress when medical gases like carbon dioxide are utilized to provide a clean operating field. Furthermore, more specialized and individualized treatment for respiratory problems is now possible because to developments in respiratory therapy equipment.

The World Health Organization reports that asthma and other chronic respiratory diseases (CRDs) are on the rise worldwide, with 3.2 million deaths from COPD and 262 million cases of asthma in 2019. Advanced oxygen treatment equipment, such as portable oxygen delivery systems and oxygen concentrators, are used to supply medical gases like oxygen. Additionally, a number of developments in dermatology and cryosurgery have increased the use of medicinal gases, such as liquid nitrogen, to freeze and remove sick or aberrant tissue. The market is being driven by the regulated and focused treatment that cryotherapy equipment provides, which minimizes harm to nearby healthy tissue.

Growing Preference for Telemedicine and Home Healthcare

Medical gases, such as oxygen, are frequently given to patients in their homes as part of home healthcare. To control their symptoms and enhance their quality of life, patients with long-term respiratory diseases like COPD need oxygen treatment. Medical oxygen gases are in greater demand as a result of the shift toward home healthcare, which enables patients to obtain oxygen therapy without the need for extended hospital stays. Furthermore, nebulization, pain relief, and respiratory therapies are only a few of the uses for medical gases that go beyond oxygen therapy. As a result of this trend, fewer extended hospital stays are required, which raises the requirement for medical oxygen.

Additionally, as cardiovascular disorders are thought to cause 17.9 million deaths worldwide each year, medicinal gases – such as oxygen – are essential for treating associated ailments, which raises the need for at-home therapies. The need for medical gases is further increased by the growth of telemedicine and home healthcare, which enables patients to receive a greater variety of medical gas treatments in the convenience of their own homes.

Rising Rates of Chronic Conditions, Including Heart and Respiratory Conditions

Medical gases like oxygen are necessary for respiratory support because to the rising prevalence of respiratory conditions such asthma, interstitial lung disorders, and chronic obstructive pulmonary disease (COPD). Chronic respiratory disorders (CRDs), such as asthma, interstitial lung diseases, and chronic obstructive pulmonary disease (COPD), are on the rise, according to the World Health Organization (WHO). According to the Global Asthma Report, asthma affects an estimated 262 million people globally, and COPD alone was responsible for almost 3.2 million deaths in 2019.

Oxygen treatment is necessary for patients who have trouble breathing in order to keep their blood oxygen levels sufficient and to relieve their symptoms. Furthermore, medicinal gases are frequently needed for diagnostic and therapeutic purposes in cardiovascular illnesses, such as heart failure, coronary artery disease, and hypertension. In order to ensure patient comfort and stability during cardiovascular procedures, nitrous oxide is used as an anesthetic agent. Additionally, medicinal gases are essential for palliative care for individuals with chronic illnesses that have progressed.

Challenges in the Medical Gas Market

Stringent Regulatory Compliance

Because medical gases are essential to patient care and safety, the market is subject to strict regulatory compliance. To guarantee that medical gases fulfill therapeutic needs, regulatory agencies impose stringent criteria for purity, labeling, packing, and transportation. Operational complexity can be further increased by the constant monitoring, certification procedures, and thorough paperwork that are frequently required to comply with these rules. To maintain compliance, manufacturers must spend more on qualified staff and sophisticated quality control systems, which raises prices. Furthermore, managing disparate regional restrictions might make international distribution plans more difficult. Although these regulations guarantee patient safety and product dependability, they also place a financial and logistical strain on manufacturers, particularly newly established smaller businesses.

High Production and Storage Costs

Complex infrastructure and procedures are needed to produce and store medicinal gases, which raises supply chain costs. To guarantee purity and safety, the gases need to be produced under exacting circumstances, which calls for cutting-edge technology and strict adherence to regulations. Energy-intensive systems are needed for compression and liquefaction, and storage facilities need to be built to withstand temperature changes, pollution, and leakage.

Specialized, frequently temperature-controlled containers that adhere to stringent rules are also necessary for the transportation of these gases. Long-term costs are further increased by continuing storage system monitoring and maintenance. These elements work together to make medical gases far more expensive to produce, handle, and distribute than many other medical supplies, which puts a strain on both healthcare providers and suppliers.

Key Players Analyzed: Overview, Key Persons, Recent Development & Strategies, Revenue Analysis

• Air Liquide
• Linde PLC
• Atlas Copco Group
• INOX-Air Products Inc.
• TAIYO NIPPON SANSO CORPORATION
• MATHESON TRI-GAS, INC.
• HORIBA Group
• SOL India Private Limited

Key Attributes:

Key Topics Covered:

1. Introduction

2. Research Methodology

2.1 Data Source

2.1.1 Primary Sources

2.1.2 Secondary Sources

2.2 Research Approach

2.2.1 Top-Down Approach

2.2.2 Bottom-Up Approach

2.3 Forecast Projection Methodology

3. Executive Summary

4. Market Dynamics

4.1 Growth Drivers

4.2 Challenges

5. Global Medical Gas Market

5.1 Historical Market Trends

5.2 Market Forecast

6. Medical Gas Market Share Analysis

6.1 By Product

6.2 By Application

6.3 By End Use

6.4 By Countries

7. Product

7.1 Pure Gases

7.2 Gas Mixtures

8. Application

8.1 Therapeutics

8.2 Diagnostics

8.3 Others

9. End Use

9.1 Hospitals

9.2 Pharmaceutical & Biotechnology Companies

9.3 Ambulatory Surgical Centers

9.4 Diagnostic & Research Laboratories

9.5 Academic & Research Institutes

9.6 Home Healthcare

10. Countries

10.1 North America

10.1.1 United States

10.1.2 Canada

10.2 Europe

10.2.1 France

10.2.2 Germany

10.2.3 Italy

10.2.4 Spain

10.2.5 United Kingdom

10.2.6 Belgium

10.2.7 Netherlands

10.2.8 Turkey

10.3 Asia-Pacific

10.3.1 China

10.3.2 Japan

10.3.3 India

10.3.4 South Korea

10.3.5 Thailand

10.3.6 Malaysia

10.3.7 Indonesia

10.3.8 Australia

10.3.9 New Zealand

10.4 Latin America

10.4.1 Brazil

10.4.2 Mexico

10.4.3 Argentina

10.5 Middle East & Africa

10.5.1 Saudi Arabia

10.5.2 UAE

10.5.3 South Africa

11. Porter’s Five Forces Analysis

11.1 Bargaining Power of Buyers

11.2 Bargaining Power of Suppliers

11.3 Degree of Rivalry

11.4 Threat of New Entrants

11.5 Threat of Substitutes

12. SWOT Analysis

12.1 Strength

12.2 Weakness

12.3 Opportunity

12.4 Threat

13. Key Players Analysis

For more information about this report visit https://www.researchandmarkets.com/r/er19ws

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