Category Archives: Biotech Companies

OverT Bio Raises $16 Million, Tapping Innovative Reprogramming of Immune Cells to Deliver Next-Generation Therapies

Posted on by

OverT combines cutting-edge cell engineering technologies with big data approaches to address major unmet medical needs in solid tumors

NEW YORK–(BUSINESS WIRE)–OverT Bio, a data-driven company working to unlock the curative potential of cell therapies in solid tumors, announced today that it has raised $16 million in seed funding. The round was co-led by ARTIS Ventures and Wing VC, with participation from Fusion Fund, OMX Ventures, Alexandria Venture Investments, Gaingels, Civilization Ventures, Hawktail, and Cancer Research Institute. The funding will go toward expanding discovery platforms focused on addressing the primary barriers for cell therapy, leading to durable and curative treatments for advanced solid tumors.

OverT is the first to use patients’ immune response to cancer to find new receptors and targets that can be applied across the human population. The team searches the entire human genome to find new ways to make cell therapies more durable and capable of destroying cancers. The foundational science has been featured in leading scientific and medical journals, such as Nature and The New England Journal of Medicine.

“Cell therapies have shown, at least in blood cancers, that we can aim to completely cure patients that have even the most advanced diseases rather than just prolong their survival,” said Dr. Mat Legut, co-founder and CEO of OverT. “With OverT’s ability to rapidly screen and engineer thousands of genes, we are building next-generation therapies to cure advanced cancers of the solid tissues.”

OverT Bio combines cutting-edge cell engineering technologies with big data approaches to address major unmet medical needs in solid tumors. OverT has developed unique massively parallel genetic screening, single-cell multiomic, and synthetic biology platforms to build next-generation cell therapies. OverT’s core platform searches every gene in the genome to identify the best genetic modifications to endow immune cells with novel properties. The company has also built a similarly high-throughput approach to discover new receptors and targets that are both safe and efficacious.

“The OverT team is supercharging cell therapies and finding new cures for cancer,” said Sara Choi, partner at Wing VC. “They’re a company that thinks differently and is guided by cutting-edge science – new ways to reprogram T cells, a new class of safe and broadly cancer-specific receptors often ignored by most immunologists. We look forward to supporting the team as they explore the edges of what’s possible in biotech.”

“Everything OverT does is based on a patient-centric mission and focused on developing therapies that extend and enhance the lives of cancer patients,” said Dr. Vasudev Bailey, partner at ARTIS Ventures. “OverT’s approaches to modifying cell behavior and for receptor discovery are highly differentiated from everything else out there. Instead of making incremental tweaks to existing therapies, OverT is making bold bets on how to create truly transformative cell therapies.”

Leveraging decades of experience in cell therapy and immunology, OverT’s founding team has built a large intellectual property portfolio encompassing multiple discovery platforms and preclinical assets. Co-founder and CEO, Dr. Mat Legut, is an immunologist and entrepreneur with a track record of translating discoveries into clinical products, and co-founder, Dr. Neville Sanjana, is a faculty member at the New York Genome Center and NYU, and a pioneer in CRISPR and high-throughput functional genomics.

To learn more about OverT, please visit overt.bio.

About OverT Bio

OverT Bio is developing effective and safe cellular therapies for solid tumors. Founded in 2022 by Dr. Mat Legut and Dr. Neville Sanjana, OverT has pioneered new pooled functional screening and synthetic genomics platforms (OverTarget™ and OverTCR™) to develop next-generation engineered immune cells. OverT is based in New York and has raised $16 million in total funding from investors, including Alexandria Venture Investments, ARTIS Ventures, Cancer Research Institute, Civilization Ventures, Fusion Fund, Gaingels, Hawktail, OMX Ventures and Wing VC. For more information, visit overt.bio.

Contacts

Moxie Communications Group

wing@moxiegrouppr.com

Inmagene Reports Positive Interim Results from Phase 2a Trial of IMG-007, a Nondepleting Anti-OX40 Monoclonal Antibody with an Extended Half-life, for the Treatment of Atopic Dermatitis

Posted on by
  • Treatment with IMG-007, the only clinical-stage nondepleting anti-OX40 monoclonal antibody (mAb), led to rapid, marked, and durable improvement of skin signs in patients with atopic dermatitis (AD).
  • Overall, IMG-007 was well-tolerated, without any reports of pyrexia or chills.
  • IMG-007 is also being evaluated for the treatment of alopecia areata (AA).
  • Final results are anticipated in Q3 2024.

SAN DIEGO, May 06, 2024 (GLOBE NEWSWIRE) — Inmagene Biopharmaceuticals (“Inmagene” or the “Company”), a clinical-stage biotechnology company developing innovative and differentiated therapies for immunological and inflammatory (I&I) diseases, today announced positive interim data from Phase 2a trial of IMG-007 in patients with AD.

Inmagene

IMG-007 is a nondepleting anti-OX40 mAb, bioengineered to have a silenced antibody-dependent cellular cytotoxicity (ADCC) function and a prolonged half-life. In a prior Phase 1 single-dose study in healthy adults, IMG-007 demonstrated a favorable safety and tolerability profile, with no reports of pyrexia or chills, which is consistent with the abolished ADCC function. Furthermore, IMG-007 showed a slow antibody clearance and a 31-day half-life at anticipated therapeutic dose levels, which could enable it to be dosed every 12 weeks (Q12W) for induction therapy, and even less frequently for maintenance therapy in AD treatment.

The Phase 2a trial (NCT05984784) evaluates the safety, pharmacokinetics, and efficacy of IMG-007 in adult patients with moderate-to-severe AD who had inadequate response to and/or intolerant of topical therapies. Patients who had received prior systemic agents, such as biologics, were also included in the trial. Topical or systemic AD medications were not permitted during the study. Eligible patients were to receive three intravenous infusions of 300 mg IMG-007 over 4 weeks (Baseline, Week 2 and 4) and to be followed up for up to 24 weeks. Key study endpoints include safety and percent change from baseline in eczema area and severity index (EASI) over time.

A total of 13 patients were enrolled from 6 centers in the U.S. and Canada. Baseline key disease characteristics included mean EASI of 29.5, mean body surface area (BSA) of 52.0%, and 61.5% patients with investigator’s global assessment (IGA=3) and 38.5% with IGA=4.

IMG-007 treatment resulted in a rapid and marked improvement from baseline in EASI score as early as Week 1 and continued improvement over time after the last dose of IMG-007 at Week 4. The mean percent improvement from baseline in EASI was 23%, 29%, 47%, 66%, 68%,77%, and 87% at Weeks 1, 2, 4, 8, 12, 16, and 20, respectively.

By Week 20, a total of 69%, 54%, and 31% of patients achieved EASI improvement of at least 50% (EASI-50), at least 75% (EASI-75), and at least 90% (EASI-90), respectively.

There were no serious adverse events (SAEs), and no adverse events (AEs) leading to treatment discontinuation, and no treatment-related AEs. There were no reports of pyrexia or chills.

“Inhibiting OX40-OX40L signaling is an exciting potential therapeutic approach to treating AD,” said Jonathan Silverberg, M.D., Ph.D., Professor of Dermatology at The George Washington University School of Medicine and Health Sciences. “By uniquely targeting the OX40 receptor without depleting T cells and with its long half-life, IMG-007 presents a best-in-class potential to not only minimize safety risks associated with T cell depletion, but also provide patients with a more convenient dosing regimen such as Q12W.”

“We are highly encouraged by the remarkable efficacy seen with a short 4-week treatment period. Future studies of continuous treatment with IMG-007 in patients with AD could potentially drive more robust efficacy than seen in this proof-of-concept study,” said Yufang Lu, M.D., Ph.D., Chief Medical Officer of Inmagene. “Given the important role of the OX40-OX40L axis in the pathogenesis of a spectrum of I&I diseases, IMG-007 could be suitable for a number of indications. We are working hard to accelerate the clinical development of IMG-007 in AD with our subcutaneous formulation.”

In addition to the Phase 2a trial in patients with AD for which final results are anticipated in Q3 2024, IMG-007 is also being evaluated in a global study in adult patients with AA with anticipated initial data readout in Q4 2024.

About Inmagene

Inmagene is a global clinical-stage biotechnology company developing novel therapeutics for immunological and inflammatory (I&I) diseases. The company’s highly differentiated clinical-stage pipeline has multiple candidates with best-in-class potential. The lead asset IMG-007, a nondepleting anti-OX40 mAb, is in two global Phase 2a clinical trials in atopic dermatitis and alopecia areata (AA). IMG-004, a non-covalent reversible BTK inhibitor with an extended half-life and pharmacodynamic effect, enabling its potential for once-daily dosing, is completing a Phase 1 multiple-dose study. IMG-008, a long-acting anti-IL-36R mAb is entering global Phase 1 clinical development.

For more information, please visit www.inmagenebio.com.

About IMG-007

IMG-007 is a humanized anti-OX40 IgG1 mAb, with a silenced ADCC function and an extended half-life. The OX40-OX40L axis is important in T cell activation, expansion, and survival, thereby having an important role in the pathogenesis of a spectrum of I&I diseases. In nonclinical studies, IMG-007 potently blocked the signaling between OX40 and OX40L. Phase 1 single dose study demonstrated a 31-day half-life at anticipated therapeutic dose levels, enabling the potential for once every 12 weeks (Q12W) dosing for induction therapy and even less frequently for maintenance therapy, and a favorable safety profile, without any reports of pyrexia or chills. IMG-007 is being evaluated for the treatment of moderate-to-severe atopic dermatitis and alopecia areata in two Phase 2a studies.

Forward-Looking Statements

This press release contains forward-looking statements. While Inmagene believes the projections to be based on reasonable assumptions, these forward-looking statements may be called into question by a number of hazards and uncertainties, so that actual results may differ materially from those anticipated in such forward-looking statements.

For further information, please contact:
Inmagene:
Anna Vardanyan, MD, PhD
Vice President of Business Development
public.relations@inmagenebio.com

Investor Relations:
Bruce Mackle
LifeSci Advisors, LLC
bmackle@lifesciadvisors.com

TAR-210 results show 90 percent recurrence-free survival and 90 percent complete response in patients with high-risk and intermediate-risk non-muscle-invasive bladder cancer, respectively

Posted on by

Updated results reinforce the potential of TAR-210 to transform treatment of non-muscle-invasive bladder cancer with fibroblast growth factor receptor (FGFR) alterations1

BEERSE, BELGIUM, May 05, 2024 (GLOBE NEWSWIRE) — Janssen-Cilag International NV, a Johnson & Johnson company, announced today updated results from an open-label, multicentre, multi-cohort Phase 1 study of the safety and efficacy of TAR-210, an intravesical targeted releasing system designed to provide sustained, local release of erdafitinib into the bladder, in patients with non-muscle-invasive bladder cancer (NMIBC) with select FGFR alterations.1 These data were featured today in an Oral Presentation Session (Abstract #PD48-02)1 at the 2024 American Urological Association (AUA) Annual Meeting taking place 3-6 May, 2024, in San Antonio, Texas.

Results featured updated data from Cohort 1 (C1); patients with recurrent, Bacillus Calmette-Guérin (BCG)-unresponsive high-risk (HR) NMIBC (high-grade Ta/T1; papillary only) who refused or were ineligible for radical cystectomy and Cohort 3 (C3); patients with recurrent, intermediate-risk (IR) NMIBC (Ta/T1) low-grade papillary disease left in situ as tumour marker lesions.1 First results were featured at the European Society for Medical Oncology 2023 Congress, with interim results presented at the European Association of Urology (EAU) 2024 Annual Congress.2,3

“Advancement in the treatment landscape of high or intermediate-risk non-muscle-invasive bladder cancer has remained stagnant for more than 50 years,” said Antoni Vilaseca*, M.D., Ph.D., of the Hospital Clínic de Barcelona, presenting author of the Phase 1 TAR-210 study. “Results presented today further underscore that TAR-210 for the localised treatment of bladder cancer may offer a promising alternative for patients with limited treatment options.”

At the data cutoff of 22 March, 2024, 64 patients had been treated with TAR-210 across the two cohorts.1 Of the 21 patients in C1 with HR-NMIBC, the 12-month recurrence free (RF) survival rate was 90 percent (95 percent confidence interval (CI), 66-97).1 In C3, 31 patients were efficacy evaluable with 28/31 achieving a complete response (CR) rate of 90 percent (95 percent CI, 74-98).1

The most common treatment-related emergent adverse events (TEAEs) were Grade 1/2 lower urinary tract events.1 There were no dose-limiting toxicities and no deaths.1 Two patients (3 percent) discontinued the study due to TEAEs of low-grade urinary symptoms and two patients had serious TEAEs with pyelonephritis and sepsis or UTI (urinary tract infection) and sepsis, respectively.1

“FGFR genetic alterations are most common in NMIBC,” said Sabine Brookman-May, M.D., Vice President, Late Development Oncology, Johnson & Johnson Innovative Medicine. “These results further support the potential of TAR-210 with quarterly administration as a bladder-sparing and BCG-free treatment option, underscoring our deep commitment to pioneering novel therapies for patients who face limited treatment avenues.”

“At Johnson & Johnson, we are committed to transforming bladder cancer treatment with novel drug delivery technology and precision-based therapies,” said Henar Hevia, Senior Director, EMEA Therapeutic Area Lead, Oncology at Johnson and Johnson Innovative Medicine. “As the data continue to mature, it is encouraging to see sustained positive responses to treatment. We look forward to investigating the full potential of TAR-210 in patients with FGFR-altered non-muscle invasive bladder cancer through an ongoing and comprehensive clinical development programme.”

Europe has one of the highest rates of bladder cancer in the world4 with nearly 225,000 patients diagnosed in 2022,5 a 10 percent increase from 2020.6 NMIBC constitutes approximately 75 percent of all newly diagnosed bladder cancers.7 Currently, adjuvant intravesical immunotherapy with BCG or intravesical chemotherapy is the standard of care for patients with intermediate- and high-risk NMIBC.8 Between 30 to 40 percent of patients do not respond to BCG, facing disease recurrence or progression.9 In such scenarios of HR-NMIBC, radical cystectomy (removal of the bladder) emerges as the primary treatment option.9 This major abdominal procedure requires a urinary diversion to be created to collect and store urine.10

About TAR-210
TAR-210 is an investigational erdafitinib intravesical targeted releasing system.11 The safety and efficacy of TAR-210 is being evaluated in a Phase 1 study (NCT05316155)12 in patients with muscle-invasive bladder cancer (MIBC) and NMIBC. The study categorises patients into four cohorts based on their disease presentation.13 Cohort 1 (C1) includes patients with recurrent, BCG-unresponsive HR-NMIBC with concomitant high-grade papillary disease who have refused or are ineligible for radical cystectomy (RC).13 Cohort 2 (C2) includes patients with the same presentation, but who are scheduled for RC.13 Cohort 3 (C3) includes patients with recurrent, intermediate-risk NMIBC with a history of low-grade papillary disease.13 To be eligible for C3, the presence of visible tumour(s) is required. Cohort 4 (C4) includes patients with MIBC.13 The primary endpoint of the study is safety (adverse events, including dose-limiting toxicity).13 Secondary endpoints include pharmacokinetics (PK), RF survival in patients in C1 and C2, CR rate and duration of CR in patients in C3 and pathologic CR rate in C4.13

About Erdafitinib
Erdafitinib is a once-daily, oral pan-fibroblast growth factor receptor (FGFR) tyrosine kinase inhibitor being evaluated by Johnson & Johnson Innovative Medicine in Phase 2 and 3 clinical trials in patients with advanced urothelial cancer.14 In addition to the Phase 1 study (NCT05316155)12 in patients with MIBC and NMIBC, erdafitinib is also being studied in the Phase 3 THOR study (NCT03390504),15 a study assessing whether erdafitinib provided a survival advantage versus chemotherapy in patients with advanced or metastatic urothelial cancer (mUC) with select FGFR alterations; the Phase 2 THOR-2/BLC2003 (NCT04172675)16 study examining erdafitinib versus investigator choice of intravesical chemotherapy in participants who received BCG and recurred with HR-NMIBC; the Phase 1b/2 NORSE (NCT03473743)17 study of erdafitinib in combination with cetrelimab in patients with locally advanced or mUC and FGFR3 or FGFR2 gene alterations; the Phase 2 RAGNAR (NCT04083976)18 study evaluating the safety and efficacy of erdafitinib in patients with advanced solid tumours, regardless of cancer type or tumour location (tumour-agnostic), driven by FGFR1–4 alterations.

In addition to the marketing authorisation application submitted to the European Medicines Agency (EMA) in September 2023, Johnson & Johnson also submitted a Supplemental New Drug Application to the U.S. Food and Drug Administration (FDA), in August 2023, based upon the Phase 3 THOR study.19,20

In 2008, Janssen Pharmaceuticals entered into an exclusive worldwide licence and collaboration agreement with Astex Pharmaceuticals to develop and commercialise erdafitinib.21

About Urothelial Carcinoma
Urothelial carcinoma (UC), also known as transitional cell carcinoma, starts in the innermost lining of the bladder.22 Almost all bladder cancers – more than 90 percent – are UCs.23 Up to one in five patients (20 percent) diagnosed with mUC have a FGFR genetic alteration.24 FGFRs are a family of receptor tyrosine kinases that can be activated by genetic alterations in a variety of tumour types, and these alterations may lead to increased tumour cell growth and survival.25 FGFRs play a key role in several biological processes including tissue repair, inflammatory response and metabolism.26 Fusions or mutations in the genes that control FGFR (known as FGFR1–4 alterations) may lead to the development and progression of certain cancers by increasing tumour cell growth and survival.27 Patients with advanced UC, including FGFR-driven tumours, face a poor prognosis and the need for innovative therapies remains high.28 The five-year survival rate for patients with metastatic bladder cancer that has spread to distant parts of the body is currently eight percent.29

About High-Risk Non-Muscle-Invasive Bladder Cancer
High-risk non-muscle-invasive bladder cancer (HR-NMIBC) is a type of non-invasive bladder cancer that is more likely to recur or spread beyond the lining of the bladder, called the urothelium, and progress to invasive bladder cancer compared to low-risk NMIBC.30 HR-NMIBC is characterised by a combination of high-grade, large tumour size, presence of multiple tumours, and carcinoma in situ (CIS).30 Radical cystectomy is currently recommended for HR-NMIBC patients who fail BCG therapy, with over 90 percent cancer-specific survival if performed before muscle-invasive progression.30 Given that NMIBC typically affects older patients, many may be unwilling or unfit to undergo radical cystectomy.30 The high rates of recurrence and progression can pose significant morbidity and distress for these patients.30

About Johnson & Johnson
At Johnson & Johnson, we believe health is everything. Our strength in healthcare innovation empowers us to build a world where complex diseases are prevented, treated, and cured, where treatments are smarter and less invasive, and solutions are personal. Through our expertise in Innovative Medicine and MedTech, we are uniquely positioned to innovate across the full spectrum of healthcare solutions today to deliver the breakthroughs of tomorrow, and profoundly impact health for humanity.

Learn more at https://www.jnj.com/emea. Follow us at https://twitter.com/JNJInnovMedEMEA and https://www.linkedin.com/company/jnj-innovative-medicine-emea/ for our latest news. Janssen-Cilag International NV, Janssen Pharmaceutica NV, and Janssen Research & Development, LLC are Johnson & Johnson companies.

Cautions Concerning Forward-Looking Statements
This press release contains “forward-looking statements” as defined in the Private Securities Litigation Reform Act of 1995 regarding product development and the potential benefits and treatment impact of TAR-210 or erdafitinib. The reader is cautioned not to rely on these forward-looking statements. These statements are based on current expectations of future events. If underlying assumptions prove inaccurate or known or unknown risks or uncertainties materialize, actual results could vary materially from the expectations and projections of Janssen-Cilag International NV, Janssen Pharmaceutica NV, and Janssen Research & Development, LLC and Johnson & Johnson. Risks and uncertainties include, but are not limited to: challenges and uncertainties inherent in product research and development, including the uncertainty of clinical success and of obtaining regulatory approvals; uncertainty of commercial success; competition, including technological advances, new products and patents attained by competitors; challenges to patents; changes in behavior and spending patterns of purchasers of health care products and services; changes to applicable laws and regulations, including global health care reforms; and trends toward health care cost containment. A further list and descriptions of these risks, uncertainties and other factors can be found in Johnson & Johnson’s Annual Report on Form 10-K for the fiscal year ended December 31, 2023, including in the sections captioned “Cautionary Note Regarding Forward-Looking Statements” and “Item 1A. Risk Factors,” and in Johnson & Johnson’s subsequent Quarterly Reports on Form 10-Q and other filings with the Securities and Exchange Commission. Copies of these filings are available online at www.sec.gov , www.jnj.com or on request from Johnson & Johnson. None of Janssen-Cilag International NV, Janssen Pharmaceutica NV, and Janssen Research & Development, LLC nor Johnson & Johnson undertakes to update any forward-looking statement as a result of new information or future events or developments.

© Janssen-Cilag International NV, Inc. 2024. All rights reserved.

*Dr. Vilaseca has not been paid for any media work.

1 Vilaseca A, et al. First-in-Human Study of TAR-210 Erdafitinib Intravesical Delivery System in Patient With Non-Muscle-Invasive Bladder Cancer With Select FGFR Alterations. 2024 Annual Urological Association. Oral presentation, 2024 American Urological Association Annual Meeting. May 2024.
2 Vilaseca A, et al. First Safety and Efficacy Results of the TAR-210 Erdafitinib Intravesical Delivery System in Patients with Non–Muscle-Invasive Bladder Cancer (NMIBC) With Select FGFR Alterations. 2023 European Society for Medical Oncology. Oral presentation, 2023 ESMO Annual Meeting. October 22, 2023. Available at: https://www.jnj.com/media-center/press-releases/first-results-with-erdafitinib-releasing-intravesical-delivery-system-tar-210-show-early-evidence-of-positive-clinical-activity-in-patients-with-non-muscle-invasive-bladder-cancer-with-select-fibroblast-growth-factor-receptor-alterations. Last accessed May 2024.
3 Guerrero-Ramos F, et al. First Safety and Efficacy Results of the TAR-210 Erdafitinib Intravesical Delivery System in Patients With Non–Muscle-Invasive Bladder Cancer With Select FGFR Alterations. Poster presented at 2024 EAU Congress. Last accessed May 2024.
4 Wong MCS, et al. The global epidemiology of bladder cancer: a joinpoint regression analysis of its incidence and mortality Itrends and projection. Scientific Reports. 2018;8:11.29.
5 Globocan 2022. Europe Cancer Factsheet. Available at: https://gco.iarc.who.int/media/globocan/factsheets/cancers/30-bladder-fact-sheet.pdf. Last accessed May 2024.
6 Globocan 2020. Europe Cancer Factsheet. Available at: https://gco.iarc.fr/today/data/factsheets/populations/908-europe-fact-sheets.pdf. Last accessed May 2024.
7 Jubber I, et al. Epidemiology of Bladder Cancer in 2023: A Systematic Review of Risk Factors. Europ Urol. 2023; 84:176–190. Available at: https://www.europeanurology.com/article/S0302-2838(23)02707-0/pdf. Last accessed May 2024.
8 Laukhtina E, et al. Urothelial carcinoma working group. Intravesical Therapy in Patients with Intermediate-risk Non-muscle-invasive Bladder Cancer: A Systematic Review and Network Meta-analysis of Disease Recurrence. Eur Urol Focus. 2022 Mar;8(2):447-456. doi: 10.1016/j.euf.2021.03.016. Epub 2021 Mar 21. PMID: 33762203.
9 Zlotta AR, et al. The management of BCG failure in non-muscle-invasive bladder cancer: an update. Can Urol Assoc J. 2013 May 1;3(6-S4):199
10 Bcan.org. Bladder removal surgery: What is a radical cystectomy? https://bcan.org/bladder-removal-surgery/. Last accessed May 2024.
11 Clinicaltrials.gov. Study of Erdafitinib Intravesical Delivery System for Localized Bladder Cancer. Available at: https://classic.clinicaltrials.gov/ct2/show/NCT05316155. Last accessed May 2024.
12 Vilaseca A, et al. Safety and efficacy of the erdafitinib (erda) intravesical delivery system, TAR-210, in patients (pts) with non–muscle-invasive bladder cancer (NMIBC) or muscle-invasive bladder cancer (MIBC) harboring select FGFR mutations or fusions: Phase 1 first-in-human study. Poster presented at ASCO GU 2023 Congress. Abstract available at: https://ascopubs.org/doi/abs/10.1200/JCO.2023.41.6_suppl.TPS583. Last accessed May 2024.
13 Tabernero J, et al. Phase I dose-escalation study of JNJ-42756493, an oral pan-fibroblast growth factor receptor inhibitor, in patients with advanced solid tumours. J Clin Oncol. 2015;33:3401–3408
14 Clinicaltrials.gov. A Study of Erdafitinib Compared With Vinflunine or Docetaxel or Pembrolizumab in Participants With Advanced Urothelial Cancer and Selected Fibroblast Growth Factor Receptor (FGFR) Gene Aberrations (THOR). Available at: https://clinicaltrials.gov/study/NCT03390504. Last accessed May 2024.
15 Clinicaltrials.gov. A Study of Erdafitinib Versus Investigator Choice of Intravesical Chemotherapy in Participants Who Received Bacillus Calmette-Guérin (BCG) and Recurred With High Risk Non-Muscle-Invasive Bladder Cancer (NMIBC). Available at: https://classic.clinicaltrials.gov/ct2/show/NCT04172675. Last accessed May 2024.
16 Clinicaltrials.gov. A Study of Erdafitinib in Participants With Metastatic or Locally Advanced Urothelial Cancer. Available at: https://clinicaltrials.gov/study/NCT03473743. Last accessed May 2024.
17 Clincaltrials.gov. A Study of Erdafitinib in Participants With Advanced Solid Tumors and Fibroblast Growth Factor Receptor (FGFR) Gene Alterations (RAGNAR). Available at: https://clinicaltrials.gov/study/NCT04083976. Last accessed May 2024.
19 Jnj.com. Janssen Submits Supplemental New Drug Application to the U.S. Food and Drug Administration Seeking Full Approval of BALVERSA® (erdafitinib) for the Treatment of Patients with Locally Advanced or Metastatic Urothelial Carcinoma and Selected Fibroblast Growth Factor Receptor Gene Alterations. Available at: https://www.jnj.com/media-center/press-releases/janssen-submits-supplemental-new-drug-application-to-the-u-s-food-and-drug-administration-seeking-full-approval-of-balversa-erdafitinib-for-the-treatment-of-patients-with-locally-advanced-or-metastatic-urothelial-carcinoma-and-selected-fibroblast-growth-factor-receptor-gene-alterations. Last accessed May 2024.
20 Janssen.com/EMEA. Janssen Submits Marketing Authorisation Application to the European Medicines Agency Seeking Approval of Erdafitinib for the Treatment of Patients with Locally Advanced or Metastatic Urothelial Cancer with Susceptible FGFR Alterations. Available at: https://www.janssen.com/emea/sites/www_janssen_com_emea/files/balversa_filing_press_release_september_2023_0.pdf. Last accessed May 2024.
21 Astex Therapeutics Limited. Astex Announces New Drug Discovery Alliance with Janssen Pharmaceutica N.V. 2008. Available at: https://astx.com/wpcontent/uploads/2016/11/ASTX_News_2008_6_9_General_Releases.pdf. Last accessed May 2024.
22 Tanaka M & Sonpavde G. Diagnosis and Management of Urothelial Carcinoma of the Bladder. Postgraduate Medicine. 2011;123(3):43-55.
23 Milojevic B, et al. Urothelial carcinoma: Recurrence and risk factors. J BUON. 2015;20(2):391-8.
24 Montazeri K & Bellmunt J. Erdafitinib for the treatment of metastatic bladder cancer. Expert Rev Clin Pharmacol. 2020 Jan;13(1):1-6. doi: 10.1080/17512433.2020.1702025. Epub 2019 Dec 22.
25 Presta M et al. Fibroblast growth factors (FGFs) in cancer: FGF traps as a new therapeutic approach. Pharmacol Ther. 2017;179:171-187.
26 Xie Y, et al. FGF/FGFR signaling in health and disease. Sig Transduct Target Ther 5, 181 (2020). https://doi.org/10.1038/s41392-020-00222-7.
27 Katoh M. Fibroblast growth factor receptors as treatment targets in clinical oncology. Nat Rev Clin Oncol. 2019;16(2):105-122.
28 Loriot Y, et al. Erdafitinib in Locally Advanced or Metastatic Urothelial Carcinoma. N Engl J Med 2019; 381(4):338-348.
29 American Cancer Society. Survival Rates for Bladder Cancer. Available at: https://www.cancer.org/cancer/types/bladder-cancer/detection-diagnosis-staging/survival-rates.html. Last accessed May 2024.
30 Brooks NA, O’Donnell MA. Treatment options in non-muscle-invasive bladder cancer after BCG failure. Indian J Urol. 2015;31(4):312-319. doi:10.4103/0970-1591.166475. Last accessed May 2024.

May 2024
CP-449918

CONTACT: Media contact:
Zayn Qureshi
zqureshi@its.jnj.com
+44 7760 334666 

Investor contact:
Raychel Kruper
investor-relations@its.jnj.com

Epredia and NovaScan Announce Intent to Enter U.S. Distribution Agreement for MarginScan™ Device for Non-Melanoma Skin Cancer Detection

Posted on by

Avantik to Serve as Primary Distribution Partner

PORTSMOUTH, N.H.–(BUSINESS WIRE)–#CancerDiagnostics–Epredia, a global leader in precision cancer diagnostics and subsidiary of PHC Holdings Corporation (TSE: 6523), and NovaScan, Inc., a company that develops breakthrough technology for cancer detection and stratification, announce that they have signed a letter of intent for a U.S. exclusive commercial distribution agreement for MarginScan™, a medical device that will support physicians in real-time detection of non-melanoma skin cancer. Epredia has engaged Avantik, a company specializing in supporting diagnostic labs with their operations, with getting this new device into the hands of Mohs surgeons.

Skin cancer is the most common group of cancers diagnosed worldwide1, and it is estimated that one in five Americans will develop skin cancer in their lifetime2. Non-melanoma skin cancers (NMSC) are the most common cancers in the United States, affecting more than 3 million Americans a year3. More than 20 percent of Americans are expected to develop NMSC before reaching age 70. Most surgeries for NMSC are simple excisions, which may result in removal of substantial healthy tissue around the suspected cancer.

MarginScan™ is designed to address this challenge by supporting Mohs surgery procedures for skin cancer treatment. Mohs Micrographic Surgery (MMS) is an operative method used to detect the presence or absence of a tumor in the margins of a surgical excision. In an MMS procedure, cancer tissue is excised in stages, then flash-frozen and assessed histologically in an onsite lab until clean margins are obtained. Previously, MMS was the only modality for skin cancer treatment that involved comprehensive margin assessment during a procedure. MarginScan™ is designed to use an electrical assessment to confirm cancer-free margins without the intraoperative histological assessment. This has the potential to allow for faster excisions that spare more healthy tissue, leading to improved outcomes for both patients and clinicians.

With this planned agreement, Epredia will serve as the primary distribution partner of MarginScan™ and will have exclusive distribution rights in the United States. Along with distribution, Epredia will be responsible for all marketing and commercial activities related to the launch and sale of MarginScan™. Epredia will work closely with Avantik to market and sell the new device to Mohs surgeons.

MarginScan™ will be the latest addition to Epredia’s world-class portfolio of precision cancer diagnostics products and services that provide a seamless end-to-end laboratory workflow, including slide scanners and printers, cryostats, tissue processors, and consumables such as slides and reagents. In addition to offering MarginScan™, Epredia will also provide consumable products that are used with the device, such as the MarginScan™ electrode and proprietary electrolytic gel used during excisions.

The two companies anticipate a 2025 U.S. launch for MarginScan™. This distribution agreement builds on a previous partnership between NovaScan and PHC Corporation, another subsidiary of PHC Holdings Corporation that is focused on the development and manufacturing of innovative medical testing devices. PHC Holdings Corporation is referred to collectively with its subsidiaries as PHC Group.

Steven Lynum, President of Epredia, said, “With more than 85 years supporting precision cancer diagnostics, we strive to continuously serve the changing needs of our customers by working with innovative companies like NovaScan to deliver the best solutions. Helping providers make faster, more accurate diagnoses and ultimately improving patient care is what makes this initiative so important.”

Craig Davis, CEO of NovaScan, remarked, “NovaScan is thrilled to be partnering with Epredia to bring MarginScan™ to clinical healthcare providers in the U.S. We are proud for MarginScan™ to join Epredia’s portfolio of market-leading cancer diagnostics products. We believe that MarginScan™ will play an important role in skin cancer detection and treatment.”

Mark Zacur, CEO of Avantik, added, “Our team is continually sourcing the best solutions to expand our portfolio of solutions supporting Mohs clinics with their critical work. We are honored that Epredia selected Avantik as their partner to bring this breakthrough device to our network of providers and their patients.”

About Epredia

Epredia is a global leader in the anatomical pathology field, providing comprehensive solutions for precision cancer diagnostics and tissue diagnostics. Powered by key brands, including Erie Scientific, Menzel-Gläser, Microm, Shandon, and Richard-Allan Scientific, Epredia’s portfolio includes microscope slides, instruments and consumables. Epredia was established following the acquisition of Thermo Fisher Scientific’s Anatomical Pathology business by PHC Holdings in 2019. Epredia has operations in major sites in the United States, the United Kingdom, Germany, Switzerland and China with a total of around 1,200 employees. Epredia is committed to achieving its mission to improve lives by enhancing cancer diagnostics for patients around the world. For further information on Epredia and its products, please visit www.epredia.com.

About PHC Holdings Corporation (PHC Group)

PHC Holdings Corporation (TSE 6523) is a global healthcare company with a mission of contributing to the health of society through healthcare solutions that have a positive impact and improve the lives of people. Its subsidiaries (referred to collectively as PHC Group) include PHC Corporation, Epredia, Ascensia Diabetes Care, LSI Medience Corporation, Mediford, and Wemex. Together, these companies develop, manufacture, sell and service solutions across diabetes management, healthcare solutions, life sciences and diagnostics. PHC Group’s consolidated net sales in FY2022 were JPY 356.4 billion with global distribution of products and services in more than 125 countries and regions. www.phchd.com

About NovaScan

About NovaScan: Based in Chicago, NovaScan (www.novascaninc.com) is a clinical stage oncology diagnostic and stratification company that has developed a low cost, point of care platform for real time cancer detection and stratification. NovaScan’s platform is active in skin, GI, lung and breast.

About Avantik

Founded in 1971, Avantik specializes in supporting diagnostic laboratories with maintaining their critical operations. The company offers a national network of more than 250 engineers, technicians and service personnel that assist diagnostic labs with increasing their productivity, enhancing their products and streamlining their processes. It also manages a comprehensive inventory of high-quality lab equipment and consumables that it continually expands to provide labs with new products that advance their goals for fast and accurate diagnoses. In 2023, Avantik partnered with Water Street Healthcare Partners, an investment firm dedicated to building market leaders in health care, to advance its goals for expansion and building on its legacy as a trusted partner to diagnostic laboratories. To learn more about Avantik, visit www.avantik-us.com.

1 Source: World Health Organization.

2 Source: World Health Organization.

3 Source: American Academy of Dermatology.

Contacts

Julia Cottrill

PHC Group Corporate Communications

julia.cottrill@phchd.com

Karius Raises $100M Co-Led by Khosla Ventures, 5AM Ventures and Gilde Healthcare, to Expand Access to Advanced Genomic Diagnostics in Infectious Disease, Addressing Antimicrobial Resistance Crisis

Posted on by

New Board Members Appointed to Support Expansion

REDWOOD CITY, Calif.–(BUSINESS WIRE)–Karius®, Inc., a world leader in genomic diagnostics for infectious disease, today announced it has secured $100 million in Series C funding. The round was co-led by Khosla Ventures and new investors 5AM Ventures and Gilde Healthcare. Also investing was new investor Seventure Partners, and existing investors Softbank Vision Fund 2, General Catalyst, HBM Healthcare Investments, Blue Water Life Sciences, Innovation Endeavors, Waycross Ventures, and others.

This investment will enable Karius to extend its reach beyond the 400 U.S. hospitals currently using the Karius Test®, an infectious disease diagnostic test that utilizes genomic analysis and artificial intelligence to detect over 1,000 pathogens from a single blood sample. The funding enables Karius to address increasing demand from healthcare providers to expand access to the Karius Test beyond the hospital setting. Additionally, it will support research into the broader health implications of Karius’ microbial cell-free DNA technology beyond infectious diseases.

“Every minute in the U.S., five cancer patients are admitted to the hospital due to infections—conditions that are often overshadowed by their primary diagnosis but are equally lethal, leading to nearly 1,000 deaths daily,” said Alec Ford, CEO of Karius. “This worrying reality emphasizes a critical and frequently overlooked gap in our healthcare system: the urgent need for faster diagnostic solutions. At Karius, we confront this challenge head-on. The Karius Test can reduce the time required to identify the cause of infections, as every minute counts for cancer patient survival. This additional $100 million will significantly enhance our capacity to deliver rapid diagnostic testing to more patients, where faster treatment saves more lives and significant healthcare resources.”

New Board Appointments

Karius also announced the appointment of three new board members joining recent additions, Dr. Norman Sharpless M.D., former National Cancer Institute Head, and Elizabeth O’Farrell, former Eli Lilly executive:

  • Alex Morgan, M.D., Ph.D., Partner at Khosla Ventures
  • Joep Muijrers, Ph.D., General Partner at Gilde Healthcare
  • Andrew Booth, Venture Advisor to 5AM Ventures, and CFO of AbCellera Biologics

“Investing in Karius addresses critical gaps in healthcare diagnostics,” stated Alex Morgan, Partner at Khosla Ventures. “Traditional methods can be slow and can contribute to overuse of antibiotics, particularly risky for immunocompromised patients and fueling Antimicrobial Resistance (AMR). Precisely identifying pathogens allows for targeted treatment, minimizing the use of broad-spectrum antibiotics and reducing the risk of AMR proliferation. This investment aligns with our commitment to advancing technologies for better public health outcomes.”

“We are thrilled to partner with Karius and to support the company in its journey to help deliver better care at lower cost to some of the most vulnerable patients out there,” added Joep Muijrers, General Partner at Gilde Healthcare.

“Karius’ strong revenue and growth illustrates how impactful the Karius Test is to clinical care. We are proud to co-lead the company’s Series C as Karius expands into new markets and moves to profitability,” added Andrew Booth, Venture Advisor, at 5AM Ventures.

“Over the years, we’ve witnessed firsthand the repeat impact Karius has made on patient management across numerous clinical settings,” added Vali Barsan, M.D., Karius Board Member and investor for SoftBank Investment Advisers. “Alongside our new as well as existing investors, we’re proud to extend our partnership with the Karius team as they continue to scale precision metagenomics for more rapid and definitive diagnostics that enable better patient outcomes.”

Karius’ Series C financing coincides with the positive results from the PICKUP1 trial, a landmark multi-center study enrolling 257 hospitalized adults with pneumonia and active hematologic malignancies across 10 leading medical centers, including Duke University Health, MD Anderson Cancer Center, Memorial Sloan Kettering Cancer Center, and University of California, San Francisco Medical Center. When added to the standard of care workup, the Karius Test detected 40% (21/52) more infections with a potential to change medical management for 81% (17/21) of these cases, demonstrating a significant additive diagnostic value in patients with blood cancers or bone marrow transplants. This first-of-its-kind study underscores the Karius Test’s potential to transform the diagnostic journey and patient care.

“We’ve done extensive due diligence, and the value that Karius brings to patients and the healthcare system at large is abundantly clear,” said Isabelle De Cremoux, CEO and Managing Partner of Seventure Partners. “The clinical evidence clearly shows its potential to help more patients. We’re excited to join Karius at such a key strategic time for the company as they grow to support the wider commercialization of the Karius Test.”

Learn more about Karius’ solutions for infectious disease diagnostics by visiting kariusdx.com.

About Karius

Karius Inc., a global leader in liquid biopsy for infectious diseases, harnesses metagenomics, next-generation sequencing, and artificial intelligence (AI) to help enhance the precision and speed of pathogen diagnosis. The Karius Test®, used in over 400 healthcare institutions–including 90+ transplant centers and 40+ children’s hospitals across the United States–identifies more than 1,000 pathogens, including viruses, bacteria, fungi, and parasites from a single blood draw typically within a day of sample receipt.

A landmark study in the Journal of Clinical Microbiology found that the Karius Test detected 701 unique microbial taxa across a cohort of 15,000 patients, making it the largest study of its kind, demonstrating the capability of the Karius Test in pathogen identification. The Karius Test also has been incorporated into the diagnostics recommendations in the 2023 Duke-ISCVID Criteria for Infective Endocarditis.

References:

  1. Bergin SP, et al. Clinical Infectious Diseases. October 2023

 

Contacts

Media Contact
Consort Partners for Karius

kariusdx@consortpartners.com

Nvelop Therapeutics Announces Members of Its Scientific Advisory Board

Posted on by

SAB includes Pioneers in Gene and Cell Therapy, Gene Editing and Drug Delivery; Also Appoints Gene Therapy Veteran Luca Biasco, Ph.D., Head of Translational Research

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Nvelop Therapeutics, a biotechnology company engineering programmable, non-viral vehicles for the in vivo delivery of therapeutic cargo, today announced the members of its scientific advisory board, bringing together a world-class group of experts from across the fields of gene therapy, gene editing and drug delivery.

“It’s a privilege to have this distinguished group of visionary leaders join our scientific advisory board as we work to advance two potentially breakthrough platforms for the delivery of genetic medicines, and develop our own pipeline of new treatments,” said Melissa Bonner, Nvelop’s chief scientific officer. “The board’s collective expertise and experience will be invaluable in our efforts to develop in vivo genetic medicines for a wide range of diseases.”

Inaugural members of the scientific advisory board include:

  • Aravind Asokan, Ph.D. : Dr. Asokan is a professor and director of gene therapy at Duke University, with appointments in the departments of Surgery, Biomedical Engineering, Molecular Genetics and Microbiology. His research group has pioneered several viral and RNA platform technologies to enable the translation of gene therapies. He is the director of the Danaher-Duke Beacon for Gene Therapy Innovation, a Novartis Global Scholar and has co-founded multiple biotech start-ups, including StrideBio (now part of Ginkgo Bioworks), Torque Bio and Lucidigm Therapeutics.
  • Keith Joung, M.D., Ph.D. : Dr. Joung is co-founder of Nvelop Therapeutics, and a pioneer in the development of targeted gene and epigenetic editing technologies. He is currently a lead translator at Arena BioWorks, and a professor of pathology (on leave 2023-2024) at Harvard Medical School. He was previously the Robert B. Colvin, M.D. Endowed Chair in Pathology at Massachusetts General Hospital. He has co-founded multiple additional biotechnology companies, including Beam Therapeutics, Chroma Medicine, Editas Medicine, Pairwise Plants, SeQure Dx and Verve Therapeutics.
  • Sadik Kassim, Ph.D. : Dr. Kassim is the chief technology officer (CTO) of genomic medicines for Danaher Corporation. Previously, he was CTO of Vor Bio, where he built the teams responsible for process development, analytical development, and supply chain and manufacturing support of a gene-edited product. Prior to Vor Bio, Dr. Kassim spent time at Kite Pharma, Mustang Bio and Novartis, where he directly contributed to the development of several first-in-human CAR T-, TCR- and TIL-based programs, and to the launch of KYMRIAH®, YESCARTA® and TESCARTUS®.
  • David Liu, Ph.D. : Dr. Liu is co-founder of Nvelop Therapeutics, and the Richard Merkin Professor and director of the Merkin Institute of Transformative Technologies in Healthcare, vice-chair of the faculty at the Broad Institute of MIT and Harvard, the Thomas Dudley Cabot Professor of the Natural Sciences at Harvard University, and a Howard Hughes Medical Institute investigator. His laboratory pioneered base editing, prime editing, phage-assisted continuous evolution (PACE) and DNA-encoded small-molecule libraries. He is also the founder or co-founder of several biotechnology and therapeutics companies, including Beam Therapeutics, Prime Medicine, Editas Medicine, Pairwise Plants, Exo Therapeutics and Chroma Medicine.
  • Jacob Giehm Mikkelsen, Ph.D. : Dr. Mikkelsen is a professor in the Department of Biomedicine at Aarhus University in Denmark. His research lab focuses on developing innovative viral and nonviral gene delivery technologies, along with novel protein delivery techniques to make in vivo gene editing safer. Dr. Mikkelsen is a frequent collaborator with clinicians and biopharma companies.
  • Luigi Naldini, M.D., Ph.D. : Dr. Naldini is a professor of cell and tissue biology and gene and cell therapy at the San Raffaele University School of Medicine, and scientific director of the San Raffaele Telethon Institute for Gene Therapy. He is a pioneer in the development and applications of lentiviral vectors for gene therapy, and co-founder of several biotechnology companies, including Genenta, Epsilen Bio (now part of Chroma Medicine) and Genespire.
  • Gabor Veres, Ph.D. : Dr. Veres is the senior vice president of translational research at Mammoth Biosciences. He has more than 25 years of experience leading gene therapy research and development for biopharma companies, including Vedere Bio II, BioMarin Pharmaceutical and bluebird bio. His experience includes guiding some of the first genetic medicines from preclinical research to clinical development, such as ZYNTEGLO® and SKYSONA™.

The company also announced today the appointment of Luca Biasco, Ph.D., as its head of translational research. Dr. Biasco brings nearly two decades of experience in the field of genetic medicine, and is recognized as a pioneer in clonal tracking of genetically engineered cells in vivo. He was previously executive director of hematopoietic stem cell (HSC) gene therapy at Sana Biotechnology, where he led the development of approaches for the in vivo delivery of genetic payloads to HSCs. Prior to Sana, he was the director of research and development at AVROBIO. Dr. Biasco will lead the particle development team at Nvelop.

“I’m very excited to be part of the Nvelop team during this time of growth and innovation,” said Dr. Biasco, “and to work with this exceptional group of talented and driven people to help overcome the limitations of today’s in vivo delivery approaches to ultimately meet the promise of genetic medicines for many patients that, today, have few or no treatment options.”

About Nvelop Therapeutics

Nvelop Therapeutics was founded in 2022 to address the challenge of efficiently delivering therapeutic cargoes to target cells in vivo. Nvelop’s next-generation platforms enable highly efficient and cell-specific in vivo delivery of a wide range of cargoes to many types of target cells. The two platforms were independently developed in the labs of scientific co-founders and gene editing pioneers Dr. David Liu of the Broad Institute of MIT and Harvard, and Dr. Keith Joung at Massachusetts General Hospital. With its own pipeline of therapeutics and through strategic collaborations, Nvelop aims to use these programmable, non-viral platforms to transform delivery for a broad range of genetic medicines and modalities in order to treat many previously undruggable diseases. Based in Cambridge, Mass., Nvelop is backed by top life science investors including Newpath Partners, Atlas Venture, F-Prime Capital, 5AM Ventures, GV, and ARCH Venture Partners. For more information, visit www.nveloptx.com or follow us on LinkedIn, X (Twitter), and Instagram.

Contacts

Media Contact
Mohana Ray

Scientific Director, HDMZ

Email: mohana.ray@hdmz.com
Phone: 312-506-5210

Corporate Contact
Jason Glashow

Glashow Strategic Communications for Nvelop

Email: jglashow@nveloptx.com
Phone: 617-510-1800

Medigene Presents Superior TCR-T Cell Functionality by Inclusion of a Costimulatory Switch Protein

Posted on by

Planegg/Martinsried, May 2, 2024. Medigene AG (Medigene or the “Company”, FSE: MDG1, Prime Standard), an immuno-oncology platform company focusing on the discovery and development of T cell immunotherapies for solid tumors, presented superior T cell receptor engineered T (TCR-T) cell functionality upon combination of optimal affinity 3S (sensitive, specific and safe) TCRs with the PD1-41BB costimulatory switch protein (CSP) at the 7th International Neoantigen Summit held in Amsterdam, Netherlands from April 29 – May 1, 2024.

The presentation with the title “KRAS Mutation-Specific TCR-T Cells are Empowered for Improved Multi-Functionality & Durability by Inclusion of a Costimulatory Switch Protein” is available on Medigene’s website: https://medigene.com/science/abstracts/

Overcoming the immunosuppressive solid tumor microenvironment (TME) stands as a major hurdle for durable TCR-T therapies in patients. The PD-1/PD-L1 axis suppresses T cell activation, proliferation, survival, cytokine secretion and cytotoxicity in a TME with tumor cells that express PD-L1. The Company´s PD1-41BB CSP effectively counters this tumor self-defense mechanism against T cell attack by replacing the inhibitory signaling domain of PD-1 expressed by TCR-T cells with the activating signaling domain of 4-1BB, thereby improving TCR-T cell functionality.

“Our PD1-41BB CSP, a proprietary component of our End-to-End (E2E) Platform, provides an innovative technology that can be paired with diverse optimal affinity 3S-TCRs that target cancer-testis antigens or neoantigens generated using our high-throughput TCR discovery process,” said Dr. Selwyn Ho, Chief Executive Officer at Medigene. “Extensive in vitro assessments underscore the significant advantages of armoring and enhancing 3S-TCRs with our PD1-41BB CSP, showcasing the potential to surmount the immunosuppressive TME of solid tumors. This breakthrough combination promises enhanced and persistent efficacy of TCR-T therapies in difficult-to-treat solid tumors. The PD1-41BB CSP can be used not only to improve TCR-T therapies but also holds potential to improve functions in other cell types as well as for application in chimeric antigen receptor T cell therapies.”
“Notably, our innovative TCR discovery method allowed us to identify TCRs possessing distinctive supplementary qualities, demonstrating functionality even in absence of the CD8 co-receptor, normally an important component of immune defense.“

The data presented displayed the exceptional specificity and sensitivity of TCR-T cells co-expressing the PD1-41BB CSP with three different 3S-TCRs that recognize the mKRAS (mutant Kirsten rat sarcoma viral oncogene homologue) G12V neoantigen. This was seen by elevated interferon gamma (IFNγ) secretion, which was solely observed following TCR-T cell stimulation with mKRAS G12V-positive tumor cells but not by stimulation with any tumor or healthy cell type expressing naturally occurring wild-type KRAS protein. Each of the three 3S TCRs displayed exceptionally high sensitivity for the mKRAS G12V neoantigen, as evidenced by their response to activation by exceedingly low levels of mKRAS-G12V peptide. Coexpression of the PD1-41BB CSP strongly enhanced TCR-T cell functionality and allowed sustained cytotoxicity to be directed against 3D tumor spheroids through multiple rounds of tumor exposure, underscoring the potent anti-cancer activity of the TCR-T cells.

Finally, all three 3S TCRs exhibited excellent safety profiles. TCR-T cells expressing each of the 3S TCRs, in combination with the PD1-41BB CSP, were not activated to secrete IFNγ nor to mediate killing upon exposure to healthy cells from major tissues or organs, confirming their selective cytotoxicity towards cancer cells without toxicity for healthy tissue.

About Medigene AG

Medigene AG (FSE: MDG1) is an immuno-oncology platform company dedicated to developing differentiated T cell therapies for treatment of solid tumors. Its End-to-End Platform is built on multiple proprietary and exclusive technologies that enable the Company to generate optimal T cell receptors against both cancer testis antigens and neoantigens, armor and enhance these T cell receptors engineered (TCR) -T cells to create best-in-class, differentiated TCR-T therapies, and optimize the drug product composition for safety, efficacy and durability. The End-to-End Platform provides product candidates for both its own therapeutics pipeline and partnering. Medigene’s lead TCR-T program MDG1015 is expected to receive IND/CTA approval in the second half of 2024. For more information, please visit https://medigene.com/

About Medigene’s TCR-T Cells

T cells are at the center of Medigene’s therapeutic approaches. Medigene’s immunotherapies help activate the patient’s own defense mechanisms, and harness T cells in the battle against cancer. Medigene’s therapies arm the patient’s own T cells with tumor-specific T cell receptors (TCRs) creating TCR-modified T cells with enhanced potential to detect and efficiently kill cancer cells.
Medigene’s approach to immunotherapy is designed to overcome the patient’s tolerance of cancer cells and tumor-induced immunosuppression. By activating the patient’s T cells outside the body, genetically modifying them with tumor-specific TCRs and expanding the resultant activated TCR-T cells, patients can rapidly be given large numbers of tumor-specific T cells to fight their cancer.

About Medigene’s PD1-41BB Costimulatory Switch Protein

Checkpoint inhibition via PD-1/PD-L1 pathway:

Cells of solid tumors are sensitive to killing by activated T cells but can escape this killing activity by producing inhibitory molecules known as ‘checkpoint proteins’, such as the Programmed Death Ligand 1 (PD-L1), on their surface. When this occurs, activated T cells which express PD-1, the natural receptor for PD-L1, are inactivated. The expression of PD-L1 is an adaptive immune resistance mechanism for tumors that can help them survive and grow.

The 4-1BB (CD137) costimulatory signaling pathway:

Effective T cell immune responses to antigens typically require both a primary antigenic stimulation via the T cell receptor (TCR) and costimulatory signals. The intracellular signaling domains of the 4-1BB protein offer a well-characterized pathway to costimulation and enhanced T cell responses.

Medigene’s PD1-41BB switch receptor turns the tumor’s attempted self-defense mechanism against the tumor by substituting the inhibitory signaling domain of PD-1 with the activating signaling domain of 4-1BB. Therefore, instead of inactivating T cells, the switch receptor delivers an activating signal to TCR-T cells. PD1-41BB-modified TCR-T cells proliferate strongly in the presence of PD-L1-positive tumor cells and kill more tumor cells upon repeated exposure. Additionally, switch receptor signals enable TCR-T cells to function better with low levels of glucose or high levels of TGFß, two conditions characteristic of strongly hostile tumor microenvironments.

About KRAS

KRAS (Kirsten rat sarcoma viral oncogene homologue) belongs to the group of small so-called Guanosine-5′-triphosphate (GTP)-binding proteins, known as RAS-like GTPases. Under physiological conditions KRAS tightly regulates cell proliferation and survival.
In cancer, KRAS is found frequently altered, in a wide variety of often fatal solid cancer types like pancreatic ductal adenocarcinoma, non-small-cell lung cancer, and colorectal cancer. Mutations in the KRAS gene result in the creation of neoantigens which drive uncontrolled proliferation of cancer cells. These mutations within the KRAS gene are unique to cancer cells and absent in healthy normal tissue, making KRAS an attractive target for TCR-T therapies. T cell receptor engineered T cell therapies offer a promising approach to targeting these mutations and addressing the challenges posed by solid tumors. Unlike CAR-T cells, which require surface antigens for recognition and may have limitations in target accessibility, TCR-T cells recognize a broader range of targets including intracellular proteins like neoantigens. This unique ability makes TCR-T therapies particularly well-suited for targeting KRAS mutations and other challenging neoantigens.

This press release contains forward-looking statements representing the opinion of Medigene as of the date of this release. The actual results achieved by Medigene may differ significantly from the forward-looking statements made herein. Medigene is not bound to update any of these forward-looking statements. Medigene® is a registered trademark of Medigene AG. This trademark may be owned or licensed in select locations only.

Medigene AG

Pamela Keck
Phone: +49 89 2000 3333 01
E-mail: investor@medigene.com

In case you no longer wish to receive any information about Medigene, please inform us by e-mail (investor@medigene.com). We will then delete your address from our distribution list.

PANCREACTIC CANCER: SUCCESFUL TRIAL AGAINST PANCREATIC CANCER WITH DEFENCE’S ARM-002 ANTI-CANCER VACCINE

Posted on by

Vancouver, BC, Canada, May 1st, 2024 – Defence Therapeutics Inc. (“Defence” or the “Company”), (CSE: DTC, OTCQB: DTCFF, FSE: DTC), a Canadian biopharmaceutical company developing novel immune-oncology therapeutics and drug delivery technologies, is pleased to announce the successful completion of a pre-clinical vaccination trial using its ARM-002TM vaccine against pancreatic cancer. The vaccine was shown to be therapeutically effective against pre-established pancreatic cancer especially when combined with the anti-PD-1 immune-checkpoint inhibitor.

In the context of an in vivo pre-clinical study, Defence tested its ARM-002TM vaccine pulsed with a pancreatic cancer lysate in combination with the anti-PD-1 immune-checkpoint in animals with pre-established Pan02 tumors. Animals’ follow-up revealed that the vaccine is indeed potent as all treated animals remained alive for over 40 days (equivalent to almost 5 years in the human scale) with tumor growth heavily impaired/blocked compared to other treatments/controls. Since Defence is interested in targeting “hard-to-treat” cancers, these results represent an important infliction point and an incentive to redirect its Phase I trial using the ARM-002TM anti-cancer vaccine to target pancreatic cancer.

Pancreatic cancer begins when uncontrolled cellular growth occurs in the pancreas. It is rarely diagnosed at early stages when the chance of curing the disease is the greatest as it often doesn’t cause symptoms until it metastasises to other organs. This classifies this type of cancer as a “hard-to-treat” cancer as patients at that point face limited treatment options.

Treatment for pancreatic cancer depends on the cancer stage as well as its location. Although the first goal of pancreatic cancer treatment is to get rid of the cancer, this unrealistic option often shifts to improving quality of life and keeping the cancer from growing or causing more harm. As such, treatments available for pancreatic cancer often involve surgery, radiation, chemotherapy or a combination, and are associated with limited clinical benefits or life-related complications. For instance, even if surgery to remove the whole pancreas is performed, patients would then rely on taking medicine their entire life to replace the hormones and enzymes made by the pancreas. Alternatively, chemotherapy (often combined with radiation therapy) can shrink the cancer, but it is often associated with resistance and/or relapse. As these standards of care fail in large set of patients, the next available option relies on immunotherapy, which uses the body’s immune system to kill cancer cells.

“Defence’s goal is to bring our proprietary and innovative immune therapies to the clinical stage for the benefit of the cancer patients. We often say that our Accum® platform is highly versatile as it can lead to the development of verticals promoting different products for multiple indications. This also applies to a single product as it can be adapted to various diseases as demonstrated with our ARM-002TM vaccine in vivo pre-clinical studies, which was shown to impair the growth of solid T-cell lymphoma, melanoma and now pancreatic cancer” says Mr. Plouffe, Chief Executive Officer of Defence Therapeutics.

Pancreatic Cancer Market size is estimated to surpass USD 36 Billion by the end of 2036, growing at a CAGR of 18% during the forecast period of 2024-2036. In 2023, the industry size of the pancreatic cancer was over USD 6 Billion. The growth of the market can be attributed to the rising prevalence of cancer cases among people across the world.

https://www.researchnester.com/reports/pancreatic-cancer-market/5299

About Defence:

Defence Therapeutics is a publicly-traded clinical-stage biotechnology company working on engineering the next generation vaccines and ADC products using its proprietary platform. The core of Defence Therapeutics platform is the ACCUM® technology, which enables precision delivery of vaccine antigens or ADCs in their intact form to target cells. As a result, increased efficacy and potency can be reached against catastrophic illness such as cancer and infectious diseases.

For further information:

Sebastien Plouffe, President, CEO and Director

P: (514) 947-2272

Splouffe@defencetherapeutics.com

www.defencetherapeutics.com

Cautionary Statement Regarding “Forward-Looking” Information

This release includes certain statements that may be deemed “forward-looking statements”. All statements in this release, other than statements of historical facts, that address events or developments that the Company expects to occur, are forward-looking statements. Forward-looking statements are statements that are not historical facts and are generally, but not always, identified by the words “expects”, “plans”, “anticipates”, “believes”, “intends”, “estimates”, “projects”, “potential” and similar expressions, or that events or conditions “will”, “would”, “may”, “could” or “should” occur. Although the Company believes the expectations expressed in such forward-looking statements are based on reasonable assumptions, such statements are not guarantees of future performance and actual results may differ materially from those in the forward-looking statements. Factors that could cause the actual results to differ materially from those in forward-looking statements include regulatory actions, market prices, and continued availability of capital and financing, and general economic, market or business conditions. Investors are cautioned that any such statements are not guarantees of future performance and actual results or developments may differ materially from those projected in the forward-looking statements. Forward-looking statements are based on the beliefs, estimates and opinions of the Company’s management on the date the statements are made. Except as required by applicable securities laws, the Company undertakes no obligation to update these forward-looking statements in the event that management’s beliefs, estimates or opinions, or other factors, should change.

Neither the CSE nor its market regulator, as that term is defined in the policies of the CSE, accepts responsibility for the adequacy or accuracy of this release.

show this

Remepy Launches with $15 Million in Funding To Bring First Hybrid Drug To Market

Posted on by

Merck Global Head of R&D and Chief Medical Officer Danny Bar Zohar joins Former Israeli Prime Minister Naftali Bennett on Board of Directors

NEW YORK–(BUSINESS WIRE)–Remepy, a pioneer in “hybrid drugs,” today announced that it has successfully closed a $10M seed investment round, which together with earlier funding totals $15M. The round was led by NFX, joined by Vine Ventures, PsyMed Ventures, Supernode Ventures and Firstime Ventures, joining previous pre-seed lead investor TechAviv as well as Fresh.fund, Samsung Next, StageNext Fund and 97212 Ventures.

Remepy’s new hybrid drugs combine traditional drugs with its “digital molecules.” Digital molecules are therapeutic interventions that trigger physiological effects (aka MOAs, Mechanisms of Action) through the brain. These physiological changes have been known to enhance the effectiveness of traditional drugs.

The digital molecules are designed for hybrid drug experiences. They are based on non-invasive cognitive, psychological, and behavioral interventions that follow principles of sensory integration, sensory substitution and sensory deprivation. The interventions trigger multiple unique mechanisms of actions, including: changes to brain connectivity in important brain areas; changes to immune system blood biomarkers, and important behavioral changes.

Remepy has already demonstrated the effects of its digital molecule interventions in clinical trials using extensive fMRI imaging, blood and saliva samples analysis and standardized questionnaires. It is among the first in the world to show that its patent–pending digital therapies can modulate blood biomarkers.

Many diseases are better treated by combining drugs with non-pharmacological interventions. Remepy is making such combinations standardized, accessible and personalized at scale with the use of its digital molecules.

Remepy is targeting neurodegenerative diseases, cancer, autoimmune diseases, and degenerative eye diseases. Remepy’s innovative approach creates a new market for hybrid medications, and a whole new world of data and IP assets for pharmaceutical companies.

Dr. Danny Bar Zohar, Merck’s Global head of R&D and Chief Medical Officer, who shares the company’s vision for hybrid drugs states: “As a physician and drug developer, it is fascinating for me to see Remepy’s clinical data, which shows how these unique non-invasive digital interventions can modulate the immune system, make significant changes to brain plasticity, and drive behavioral changes. Remepy’s product has the potential to enhance immunotherapy for cancer, or improve existing drug therapy for neurodegenerative diseases such as Parkinson’s Disease and MCI (Mild Cognitive Impairment). These proprietary digital-drug combinations could significantly improve patient outcomes and create tangible value for patients and healthcare systems.”

Dr. Michal Tsur, Remepy Co-CEO: “I am thrilled that Dr. Danny Bar Zohar has joined us as a board member. Many industries have transformed by adopting a hybrid product approach, integrating new technology with traditional products.” Co-CEO Or Shoval added: “I am particularly excited about the prospects of improving Parkinson’s Disease patients’ lives with a hybrid PD drug that will better address cognitive, movement, speech, sleeping and psychological symptoms and has the potential of disease modification with changes to brain connectivity.”

“Remepy’s vision of enhancing drugs with digital companions is groundbreaking and their clinical proof that such enhancement is possible is incredibly exciting,” adds Gigi Levy-Weiss, Managing Partner at NFX “Remepy has an experienced team of entrepreneurs and world-class scientists that are defining a new category in pharma.”

About Remepy:

Remepy is pioneering Hybrid Drugs: traditional drugs combined with digital therapeutics that are personalized and enhance the effect of pharmaceutical treatments. Remepy has already proven in human studies the effect of its products, using extensive brain imaging, as well as blood and saliva samples, with promising results for Oncology, MCI, and Parkinson’s Disease. Their approach offers an efficient method to create new proprietary pharma assets, unlocking an entire new market and world of data. The Remepy team includes top scientists, physicians, and technologists.

Contacts

Michal Tsur

Press@Remepy.com

SCHOTT Pharma appoints Tobias Erfurth as Head of Investor Relations

Posted on by
  • Tobias Erfurth will lead Investor Relations Team starting in August 2024.
  • He brings more than 20 years of experience in finance, capital markets and investor relations.

SCHOTT Pharma, a pioneer in pharma drug containment solutions and delivery systems, today announced the appointment of Tobias Erfurth as Head of Investor Relations effective August 2024. He will strengthen and expand the Investor Relations team at SCHOTT Pharma and will report to CFO Almuth Steinkühler. “Tobias joining our finance team is a real win for SCHOTT Pharma. With his extensive expertise and DAX40 experience, we look forward to further growing SCHOTT Pharma’s presence in the capital markets and showcasing our attractive financial profile,” said Dr. Almuth Steinkühler, CFO of SCHOTT Pharma.

Tobias Erfurth has gained more than 20 years of experience in finance, capital markets and investor relations, both from corporate positions and roles in banking. In his most recent position as Head of Investor Relations at Symrise AG for over 13 years, Erfurth was responsible for managing all investor activities during the company’s development from a small cap to a member of the DAX40. Prior to that, Erfurth accompanied the IPO of Crop Energies AG and subsequently built up the investor relations department. Erfurth also has extensive experience in corporate finance and equity capital markets gained in his previous role as analyst at Dresdner Bank.

“With its convincing equity story, dynamic growth, attractive margins, SCHOTT Pharma is a true success story, and I look forward to joining the team. Together, we will continue to deepen the established relationships with investors and analysts and help key stakeholders to understand SCHOTT Pharma’s strategy and its role as a pioneer in one of the fastest growing markets of the pharma industry,” said Tobias Erfurth.

For additional news about SCHOTT Pharma please visit our media center.

About SCHOTT Pharma

Human health matters. That is why SCHOTT Pharma designs solutions grounded in science to ensure that medications are safe and easy to use for people around the world. The portfolio comprises drug containment solutions and delivery systems for injectable drugs ranging from prefillable glass and polymer syringes to cartridges, vials, and ampoules. Every day, a team of over 4,600 people from over 60 nations works at SCHOTT Pharma to contribute to global healthcare. The company is represented in all main pharmaceutical hubs with 16 manufacturing sites in Europe, North and South America, and Asia. With over 1,000 patents and technologies developed in-house and a state-of-the-art R&D center in Switzerland, the company is focused on developing innovations for the future. SCHOTT Pharma AG & Co. KGaA is headquartered in Mainz, Germany and listed on the Frankfurt Stock Exchange as part of the SDAX. It is part of SCHOTT AG, which is owned by the Carl Zeiss Foundation. In light of this spirit, SCHOTT Pharma is committed to sustainable development for society and the environment and has the strategic goal of becoming climate-neutral by 2030. Currently, SCHOTT Pharma has over 1,800 customers including the top 30 leading pharma manufacturers for injectable drugs and generated revenue of EUR 899 million in the fiscal year 2023.

 

Press contact

Joana Kornblum

Media Relations

Tel.: +49 151/29223552

E-Mail: joana.kornblum@schott.com

Investor contact

Jasko Terzic, CFA

Senior Manager Investor Relations

E-Mail: ir.pharma@schott.com

 

show this