Oculis Announces Positive Topline Results of Phase 2b RELIEF Trial with Licaminlimab, Designed to Transform the Treatment Paradigm of Dry Eye Disease with a Precision Medicine Strategy

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ZUG, Switzerland, June 10, 2024 (GLOBE NEWSWIRE) —

Improvements in multiple sign efficacy endpoints were observed in full population and with predictive and more pronounced effects in the TNFR1 genetic biomarker population as identified in prior successful Phase 2 symptoms trial
Rapid treatment effect on corneal inflammation was observed in TNFR1 genetic biomarker patients as early as Day 15 and was statistically significant at final efficacy visit on Day 43
Licaminlimab was well tolerated similar to vehicle
Company plans to finalize Phase 3 development plans following an End-of-Phase 2 (EoP2) meeting with the U.S. Food and Drug Administration (FDA)
An investor and analyst webcast will be held today at 8:30am US Eastern Time

Oculis Holding AG (Nasdaq: OCS) (“Oculis”), a global biopharmaceutical company purposefully driven to save sight and improve eye care, today announced positive topline results from its Phase 2b RELIEF trial with licaminlimab, a novel anti-TNFα biologic eye drop with an established dual anti-inflammatory and anti-apoptotic mechanism of action in patients with dry eye disease (DED).

The Phase 2b RELIEF trial is a multi-center, randomized, double-masked, vehicle-controlled trial evaluating the efficacy and safety of licaminlimab in subjects with signs of DED (NCT05896670). The trial also evaluated the efficacy and safety of licaminlimab in a subpopulation of subjects with a TNFR1-related genotype as prespecified in the protocol. One hundred and twenty-two (122) patients were randomized 1:1 to either licaminlimab (n=62) or vehicle (n=60) across 4 sites for a 6-week treatment period and a 2-week follow up. A total of 23 patients carried a specific TNFR1-related genotype. Patients were evaluated for efficacy endpoints at baseline, Day 15 and Day 43. The prespecified investigational efficacy measures in this trial included multiple signs of DED that are accepted by the FDA as efficacy endpoints.

Phase 2b RELIEF trial showed positive effects on multiple signs of DED

For the full trial population (n=122):
- Treatment effect favoring licaminlimab was observed in multiple sign endpoints including fluorescein staining in the total cornea, inferior corneal, central corneal and nasal conjunctival regions, and in the Schirmer’s test.
For the subpopulation of patients with the TNFR1 genetic biomarker (n=23):
- Treatment effect favoring licaminlimab was observed in multiple sign endpoints including fluorescein staining in the total cornea, inferior corneal, central corneal, nasal conjunctival, total conjunctival and total ocular surface regions, in the Schirmer’s test, and in conjunctival redness.
- Rapid and favorable treatment effect in favor of licaminlimab on corneal inflammation was observed as early as Day 15 that was significant at Day 43, as measured by the difference in mean change from baseline versus vehicle for inferior corneal fluorescein staining score: -0.59 (CI: -1.165, -0.017). The treatment effect also increased over time. See attached Figure.
- Licaminlimab was well tolerated. The incidence of ocular TEAEs in the study eye was 11.5% in the licaminlimab group and 10.2% in the vehicle group. TEAEs in the fellow eye were similar to the study eye. All ocular TEAEs were mild and transient, and there were no serious ocular adverse events observed with licaminlimab in the study. Drop comfort was also evaluated and was similar to artificial tears.

Riad Sherif, MD, Chief Executive Officer of Oculis, commented: “We are pleased that we achieved all of our objectives for the trial, and extremely encouraged to see licaminlimab’s profound results with a precision medicine approach which has the potential to transform the way we develop drugs and treat patients in ophthalmology. With this and prior positive results on signs and symptoms, we look forward to discussing these encouraging data with the FDA and advancing this program into Phase 3.”

Eric Donnenfeld, M.D., Clinical Professor of Ophthalmology at New York University and Chair of Oculis’ Cornea Scientific Advisory Board, added: “The precision medicine approach with licaminlimab could be a groundbreaking paradigm shift in ophthalmology and the treatment of DED. The current approach of ‘trial and error’ and our inability to predict response for this highly heterogenous population leads to a low level of patient satisfaction. To my knowledge, Licaminlimab is the first dry eye disease medication to demonstrate in a clinical trial a predictive treatment effect in patients with a common genetic biomarker to potentially solve this problem.”

Christophe Baudouin, M.D., Ph.D., Professor of Ophthalmology and Chairman of Ophthalmology III at Quinze-vingts National Ophthalmology Hospital, Paris, and member of Oculis Scientific Advisory Board, added: “I am very excited to see that licaminlimab, with its dual anti-inflammatory and anti-apoptotic mechanism of action, targets the origin of DED and has the potential to be truly disease modifying as shown by improvements in several clinical signs of DED, including corneal staining.”

The Company is planning to conduct an end-of-Phase 2 meeting with the FDA to discuss the registrational path for licaminlimab in DED and finalize the Phase 3 development plan.

Analyst and investor call
The Oculis management team will host an analyst and investor call today at 8:30 am US Eastern Time, to review the trial results.

Interested parties may participate in the call via the following webcast here.

A replay of the webcast and accompanying slides will be available for 90 days following the event through the “Events and Presentations” page of the “Investors and Media” section of the company’s website.

About Dry Eye Disease (DED)
DED is a common condition estimated to impact nearly 40 million people in 2023 in the US alone¹. It is a multifactorial disease in which ocular surface inflammation plays a central role in sustaining the pathological state^2,3. It usually affects both eyes and patients may experience a stinging, burning or scratchy sensation. In addition, some patients experience sensitivity to light, eye redness, difficulty wearing contact lenses, difficulty with nighttime driving, and blurred vision which can greatly affect their quality of life.

Of the approximately 20 million patients who are diagnosed with DED in the U.S., about half or 10 million are considered to have moderate to severe disease¹. However, only 13% receive prescription treatment, primarily with an anti-inflammatory medications¹. Despite currently available treatments, with 87% of chronic patients still unsatisfied⁴ highlighting the tremendous unmet need remaining in this underserved patient population. Furthermore, given the heterogenicity of the DED patient population, there is a need for more personalized treatment approaches to improve outcomes for patients.

About licaminlimab (OCS-02)
Licaminlimab is an anti-TNFα eye drop candidate developed with a single chain antibody fragment (scFv) technology specifically designed to treat ocular inflammatory diseases. The dual anti-inflammatory and anti-necrotic mechanism of action of TNF-α inhibition has been well-established in inflammatory disorders where the systemic use of TNF-α inhibitors has led to marked improvements in the disease management and treatment outcomes. In multiple Phase 2 trials, licaminlimab has shown positive effects on treating both the signs and symptoms of DED and has been well tolerated. In addition, a genetic biomarker was identified which showed a clear correlation between this variant in the TNFR1 gene and improved response to licaminlimab.

Licaminlimab is an investigational drug and has not received regulatory approval for commercial use in any country. For more information, please visit: www.oculis.com

About Oculis

Oculis is a global biopharmaceutical company (Nasdaq: OCS; XICE: OCS) purposefully driven to save sight and improve eye care. Oculis’ highly differentiated pipeline comprises multiple innovative product candidates in development. It includes OCS-01, a topical eye drop candidate for diabetic macular edema (DME) and for the treatment of inflammation and pain following cataract surgery; licaminlimab (OCS-02), a topical biologic anti-TNFα eye drop candidate for dry eye disease (DED) and for non-infectious anterior uveitis; and OCS-05, a neuroprotective candidate for acute optic neuritis (AON). Headquartered in Switzerland and with operations in the U.S. and Iceland, Oculis’ goal is to improve the health and quality of life of patients worldwide. The company is led by an experienced management team with a successful track record and is supported by leading international healthcare investors.

Oculis Contacts
Ms. Sylvia Cheung, CFO
sylvia.cheung@oculis.com

Investor & Media Relations
LifeSci Advisors
Corey Davis, Ph.D.
cdavis@lifesciadvisors.com
1-212-915-2577

Cautionary Statement Regarding Forward Looking Statements

This press release contains forward-looking statements and information. For example, statements regarding the potential benefits of licaminlimab, including patient impact and market opportunity; the potential of licaminlimab for treating DED; expected future milestones and catalysts; the initiation, timing, progress and results of Oculis’ clinical and preclinical studies; Oculis’ research and development programs, regulatory and business strategy, future development plans, and management; Oculis’ ability to advance product candidates into, and successfully complete, clinical trials; and the timing or likelihood of regulatory filings and approvals, are forward-looking. The clinical trial results presented in this press release are topline and preliminary and subject to change, as analysis is ongoing. These topline results may not be reproduced in subsequent patients and clinical trials. All forward-looking statements are based on estimates and assumptions that, while considered reasonable by Oculis and its management, are inherently uncertain and are inherently subject to risks, variability, and contingencies, many of which are beyond Oculis’ control. These forward-looking statements are provided for illustrative purposes only and are not intended to serve as, and must not be relied on by an investor as, a guarantee, assurance, prediction or definitive statement of a fact or probability. Actual events and circumstances are difficult or impossible to predict and will differ from assumptions. All forward-looking statements are subject to risks, uncertainties and other factors that may cause actual results to differ materially from those that we expected and/or those expressed or implied by such forward-looking statements. Forward-looking statements are subject to numerous conditions, many of which are beyond the control of Oculis, including those set forth in the Risk Factors section of Oculis’ annual report on Form 20-F and any other documents filed with the U.S. Securities and Exchange Commission (the “SEC”). Copies of these documents are available on the SEC’s website, www.sec.gov. Oculis undertakes no obligation to update these statements for revisions or changes after the date of this release, except as required by law.

¹DRG (part of Clarivate) – Dry Eye Disease Landscape and Forecast report 2020
²TFOS DEWS II The Ocular Surface 15 (2017)
³ Baudouin C. Dry Eye Disease, the complex interactions of vicious cycles. EuDES European Dry Eye Society
https://www.dryeye-society.com/resources/dry-eye-disease-complex-interactions-vicious-cycles
⁴Mukamal, R. Why is Dry Eye So Difficult to Treat? 2021 https://www.aao.org/eye-health/tips-prevention/fix-dry-eye-treatment-eyedrops

Attachment

Figure 1 – RELIEF Topline Results PR

Macomics Announces Appointment of Simon Dew as New Chief Business Officer

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Macomics continues to expand its leadership team to accelerate the development of first-in-class macrophage-targeted therapies
Simon Dew brings over 25 years of experience in pharmaceutical business development to advance partnerships for Macomics’ macrophage ENIGMAC™ platform

EDINBURGH, United Kingdom and CAMBRIDGE, United Kingdom, June 10, 2024 (GLOBE NEWSWIRE) — Macomics Ltd, a leader in macrophage drug discovery has announced the appointment of Simon Dew in the newly created position of Chief Business Officer (CBO). He will be instrumental in advancing Macomics’ mission to develop first-in-class medicines targeting macrophage-driven diseases of high unmet medical need, in oncology and chronic inflammatory diseases.

With an extensive background in pharmaceutical business development, corporate strategy, Simon will play a pivotal role in maximizing the potential of Macomics’ proprietary macrophage ENIGMAC™ drug discovery platform through strategic partnerships and collaborations.

Simon brings over 25 years of experience in the pharmaceutical industry, having held senior leadership roles in Pharma and Biotech. He is currently on the Business Development Board of Sunstone Capital, a Danish Venture Capital Company and, most recently served as CBO at ReNeuron Group. His career also includes significant roles at Gyroscope Therapeutics, Astellas Pharmaceuticals, IQVIA, Parexel, Phytopharm plc, and GSK/SB, where he demonstrated exceptional expertise in business development, corporate strategy, and operational leadership. He has a BSc in Pharmacy and has completed the INSEAD Managing Partnerships and Strategic Alliances and M&A Executive Program.

Dr Stephen Myatt, CEO of Macomics said, “I am delighted to welcome Simon to the company. Business development is a critical role to our leadership team and Simon’s extensive experience and successful track record in the biotech and pharmaceutical industry will be invaluable as we continue to advance our macrophage-targeted therapies and expand our strategic partnerships. Macrophages are key to multiple diseases of high unmet medical need, including as key mediators of solid tumour immunosuppression and pathological inflammation in chronic inflammatory disorders.”

Simon Dew, new CBO of Macomics said, “The potential of the ENIGMAC platform to discover and develop novel macrophage-targeted therapies is unique, and I look forward to working with the talented team at Macomics to deliver on strategic partnerships that will accelerate the development of transformative medicines for patients with high unmet medical needs.”

Image: Simon Dew, Chief Business Officer, Macomics

About Macomics – www.macomics.com

Macomics Ltd is a macrophage drug discovery company with a world-leading macrophage drug discovery platform, developing first-in-class medicines to deliver transformational impact for patients with macrophage-driven diseases: Macrophages are key to multiple diseases of high unmet medical need, including as key mediators of solid tumour immunosuppression and pathological inflammation in chronic inflammatory disorders.

The company is progressing a diversified portfolio of therapies targeting disease specific macrophages towards the clinic. Its ENIGMAC macrophage drug discovery platform enables identification and validation of novel macrophage therapeutic targets and provides a translationally relevant path to clinic through the development of more physiologically relevant human macrophage models combined with proprietary gene editing technology.

The company was co-founded in 2020 by Prof. Jeffrey Pollard and Dr. Luca Cassetta, University of Edinburgh, internationally recognised leaders in macrophage biology. It has R&D and office facilities in Edinburgh and Cambridge, UK. The company’s financing was led by Epidarex Capital and the company is backed by Scottish Enterprise, LifeLink Ventures and Caribou Property Limited.

For further information please contact:

Macomics

At the Company – Steve Myatt, CEO, Macomics E: myatt@macomics.com

Media enquiries (for Macomics) – Sue Charles, Charles Consultants T: +44 (0)7968 726585 E: sue@charles-consultants.com

Geron Announces FDA Approval of RYTELO™ (imetelstat), a First-in-Class Telomerase Inhibitor, for the Treatment of Adult Patients with Lower-Risk MDS with Transfusion-Dependent Anemia

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Approval across ESA ineligible and ESA relapsed/refractory patients with LR-MDS with transfusion-dependent anemia, regardless of ring sideroblast (RS) status
Durable and sustained red blood cell transfusion independence, increases in hemoglobin levels and reduction in transfusion burden observed across key LR-MDS subgroups in the IMerge Phase 3 clinical trial; the most common Grade 3/4 adverse reactions were thrombocytopenia and neutropenia, which were generally manageable and short-lived
Lower-risk MDS is a progressive blood cancer with high unmet need, where many patients with anemia become dependent on red blood cell transfusions, which can be associated with clinical consequences and decreased quality of life
Conference call with Geron management scheduled at 8am ET on Friday, June 7, 2024

FOSTER CITY, Calif.–(BUSINESS WIRE)–Geron Corporation (Nasdaq: GERN), a commercial-stage biopharmaceutical company aiming to change lives by changing the course of blood cancer, today announced that the U.S. Food and Drug Administration (FDA) has approved RYTELO™ (imetelstat) for the treatment of adult patients with low- to intermediate-1 risk myelodysplastic syndromes (MDS) with transfusion-dependent (TD) anemia requiring four or more red blood cell units over eight weeks who have not responded to or have lost response to or are ineligible for erythropoiesis-stimulating agents (ESA).

“With the approval and availability of RYTELO, we believe eligible patients with lower-risk MDS can potentially experience meaningful clinical benefit, particularly the potential for greater than 24 weeks of freedom from the burden of red blood cell transfusions and symptomatic anemia,” said John A. Scarlett, M.D., Geron’s Chairman and Chief Executive Officer. “The approval of RYTELO as the first telomerase inhibitor is a testament to the power of our science and the passion of our people to innovate in the field of blood cancer. As we celebrate today’s momentous milestone, I would like to thank the patients and families, advocates, clinicians, study coordinators and site personnel, scientists, and Geron employees and collaborators past and present whose participation was integral to this achievement and to supporting our transformation into a commercial company.”

Lower-risk myelodysplastic syndromes (LR-MDS) is a blood cancer that often progresses to require increasingly intensified management of key symptoms such as anemia and resulting fatigue¹. These symptomatic LR-MDS patients frequently become red blood cell transfusion dependent, which has been shown to be associated with short- and long-term clinical consequences that reduce quality of life and shorten survival^2,3. There is a high unmet need for many LR-MDS patients, particularly those with characteristics having poorer prognosis. Current treatment options for those failing ESA are limited to select sub-populations and there is an unmet need for treatments that can provide extended and continuous red blood cell transfusion independence.

Approval Based on Results from IMerge Phase 3 Clinical Trial

“For patients with lower-risk MDS and anemia who are transfusion dependent, we have very few options today and often cycle through available therapies, making the approval of RYTELO potentially practice changing for us,” said Rami Komrokji, MD, Vice Chair, Malignant Hematology Department, Moffitt Cancer Center, who was an investigator of the pivotal IMerge clinical trial. “What is exciting about RYTELO is the totality of the clinical benefit across LR-MDS patients irrespective of ring sideroblast status or high transfusion burden, including sustained and durable transfusion independence and increases in hemoglobin levels, all within a well-characterized safety profile of generally manageable cytopenias. The treatment goal for patients with LR-MDS and anemia is transfusion-independence and before today, this wasn’t possible for many patients.”

The FDA approval of RYTELO is based on results from the IMerge Phase 3 clinical trial, published in The Lancet⁴. The IMerge trial met its primary and key secondary endpoints, with RYTELO demonstrating significantly higher rates of red blood cell transfusion independence (RBC-TI) versus placebo for at least eight consecutive weeks (RYTELO 39.8% [95% CI 30.9–49.3]; placebo 15.0% [7.1–26.6]; p<0.001) and for at least 24 weeks (RYTELO 28.0% [95% CI 20.1-37.0]; placebo 3.3% [95% CI 0.4-11.5]; p<0.001). RBC-TI was durable and sustained in the RYTELO treated population, with a median RBC-TI duration for 8-week responders and 24-week responders of approximately 1 year and 1.5 years, respectively.

In an exploratory analysis of RYTELO-treated patients achieving ≥8-week RBC-TI, median increases in hemoglobin were 3.6 g/dL for RYTELO and 0.8 g/dL for placebo. Clinically meaningful efficacy results were observed across key MDS subgroups irrespective of ring sideroblast (RS) status, baseline transfusion burden and IPSS risk category.

In the IMerge trial, the safety profile of RYTELO was well-characterized with generally manageable and short-lived thrombocytopenia and neutropenia, which are familiar side effects for hematologists who are experienced with managing cytopenias. The most common Grade 3/4 adverse reactions were neutropenia (72%) and thrombocytopenia (65%), which lasted a median duration of less than two weeks, and in more than 80% of patients were resolved to Grade < 2 in under four weeks. Cytopenias were generally manageable with dose modifications. The intravenous administration of RYTELO every four weeks aligns to routine blood count monitoring for these patients.

The most common adverse reactions (incidence ≥10% with a difference between arms of >5% compared to placebo), including laboratory abnormalities, were decreased platelets (thrombocytopenia), decreased white blood cells, decreased neutrophils (neutropenia), increased aspartate aminotransferase (AST), increased alkaline phosphatase (ALP), increased alanine aminotransferase (ALT), fatigue, prolonged partial thromboplastin time, arthralgia/myalgia, COVID-19 infections, and headache. Clinically relevant adverse reactions in < 5% of patients who received RYTELO included febrile neutropenia, sepsis, gastrointestinal hemorrhage, and hypertension.

Conference Call Details

A conference call with Geron management is scheduled at 8am Eastern Time on Friday, June 7, 2024, to discuss the FDA approval and launch of RYTELO. To access the webcast and slides, please visit the Investors & Media page. Participants may access the webcast by registering online using the following link, https://events.q4inc.com/attendee/923992744.

About RYTELO™ (imetelstat)

RYTELO™ (imetelstat) is an FDA-approved oligonucleotide telomerase inhibitor for the treatment of adult patients with low-to-intermediate-1 risk myelodysplastic syndromes (LR-MDS) with transfusion-dependent anemia requiring four or more red blood cell units over eight weeks who have not responded to or have lost response to or are ineligible for erythropoiesis-stimulating agents (ESAs). It is indicated to be administered as an intravenous infusion over two hours every four weeks.

RYTELO is a first-in-class treatment that works by inhibiting telomerase enzymatic activity. Telomeres are protective caps at the end of chromosomes that naturally shorten each time a cell divides. In LR-MDS, abnormal bone marrow cells often express the enzyme telomerase, which rebuilds those telomeres, allowing for uncontrolled cell division. Developed and exclusively owned by Geron, RYTELO is the first and only telomerase inhibitor approved by the U.S. Food and Drug Administration.

Geron aims to ensure broad access to RYTELO for eligible patients. Accordingly, our REACH4RYTELO™ Patient Support Program provides a range of resources that support access and affordability to eligible patients prescribed RYTELO.

IMPORTANT SAFETY INFORMATION

WARNINGS AND PRECAUTIONS

Thrombocytopenia

RYTELO can cause thrombocytopenia based on laboratory values. In the clinical trial, new or worsening Grade 3 or 4 decreased platelets occurred in 65% of patients with MDS treated with RYTELO.

Monitor patients with thrombocytopenia for bleeding. Monitor complete blood cell counts prior to initiation of RYTELO, weekly for the first two cycles, prior to each cycle thereafter, and as clinically indicated. Administer platelet transfusions as appropriate. Delay the next cycle and resume at the same or reduced dose, or discontinue as recommended.

Neutropenia

RYTELO can cause neutropenia based on laboratory values. In the clinical trial, new or worsening Grade 3 or 4 decreased neutrophils occurred in 72% of patients with MDS treated with RYTELO.

Monitor patients with Grade 3 or 4 neutropenia for infections, including sepsis. Monitor complete blood cell counts prior to initiation of RYTELO, weekly for the first two cycles, prior to each cycle thereafter, and as clinically indicated. Administer growth factors and anti-infective therapies for treatment or prophylaxis as appropriate. Delay the next cycle and resume at the same or reduced dose, or discontinue as recommended.

Infusion-Related Reactions

RYTELO can cause infusion-related reactions. In the clinical trial, infusion-related reactions occurred in 8% of patients with MDS treated with RYTELO; Grade 3 or 4 infusion-related reactions occurred in 1.7%, including hypertensive crisis (0.8%). The most common infusion-related reaction was headache (4.2%). Infusion-related reactions usually occur during or shortly after the end of the infusion.

Premedicate patients at least 30 minutes prior to infusion with diphenhydramine and hydrocortisone as recommended and monitor patients for one hour following the infusion as recommended. Manage symptoms of infusion-related reactions with supportive care and infusion interruptions, decrease infusion rate, or permanently discontinue as recommended.

Embryo-Fetal Toxicity

RYTELO can cause embryo-fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during treatment with RYTELO and for 1 week after the last dose.

ADVERSE REACTIONS

Serious adverse reactions occurred in 32% of patients who received RYTELO. Serious adverse reactions in >2% of patients included sepsis (4.2%) and fracture (3.4%), cardiac failure (2.5%), and hemorrhage (2.5%). Fatal adverse reactions occurred in 0.8% of patients who received RYTELO, including sepsis (0.8%).

Most common adverse reactions (≥10% with a difference between arms of >5% compared to placebo), including laboratory abnormalities, were decreased platelets, decreased white blood cells, decreased neutrophils, increased AST, increased alkaline phosphatase, increased ALT, fatigue, prolonged partial thromboplastin time, arthralgia/myalgia, COVID-19 infections, and headache.

Please see RYTELO (imetelstat) full Prescribing Information, including Medication Guide, available at https://pi.geron.com/products/US/pi/rytelo_pi.pdf.

About Geron

Geron is a commercial-stage biopharmaceutical company aiming to change lives by changing the course of blood cancer. Our first-in-class telomerase inhibitor RYTELO™ (imetelstat) is FDA-approved for the treatment of adult patients with lower-risk MDS with transfusion dependent anemia. We are also conducting a pivotal Phase 3 clinical trial of imetelstat in JAK-inhibitor relapsed/refractory myelofibrosis (R/R MF), as well as studies in other hematologic malignancies. Inhibiting telomerase activity, which is increased in malignant stem and progenitor cells in the bone marrow, aims to potentially reduce proliferation and induce death of malignant cells. To learn more, visit www.geron.com or follow us on LinkedIn.

Use of Forward-Looking Statements

Except for the historical information contained herein, this press release contains forward-looking statements made pursuant to the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995. Investors are cautioned that such statements, include, without limitation, those regarding: (i) Geron’s belief that eligible patients with lower-risk MDS can potentially experience meaningful clinical benefit with RYTELO, particularly the potential for greater than 24 weeks of freedom from the burden of red blood cell transfusions and symptomatic anemia; (ii) an unmet need for new treatments for patients with LR-MDS that can provide extended and continuous red blood cell transfusion independence; (iii) that RYTELO could be practice-changing for hematologists who treat patients with lower-risk MDS and anemia who are transfusion dependent; (iv) the potential for RYTELO to offer a totality of clinical benefit across LR-MDS patients irrespective of ring sideroblast status or high transfusion burden, including sustained and durable transfusion independence and increases in hemoglobin levels, all within a well-characterized safety profile of generally manageable cytopenias; (v) that inhibiting telomerase activity aims to potentially reduce proliferation and induce death of malignant cells; (vi) that Geron aims to ensure broad access to RYTELO; (vii) that imetelstat has the potential to demonstrate disease-modifying activity in patients; (viii) that IMpactMF has registrational intent; and (ix) other statements that are not historical facts, constitute forward-looking statements. These forward-looking statements involve risks and uncertainties that can cause actual results to differ materially from those in such forward-looking statements. These risks and uncertainties, include, without limitation, risks and uncertainties related to: (a) whether Geron is successful in commercializing RYTELO (imetelstat) for the treatment of patients with LR-MDS with transfusion dependent anemia; (b) whether Geron overcomes potential delays and other adverse impacts caused by enrollment, clinical, safety, efficacy, technical, scientific, intellectual property, manufacturing and regulatory challenges in order to have the financial resources for and meet expected timelines and planned milestones; (c) whether regulatory authorities permit the further development of imetelstat on a timely basis, or at all, without any clinical holds; (d) whether any future safety or efficacy results of imetelstat treatment cause the benefit-risk profile of imetelstat to become unacceptable; (e) whether imetelstat actually demonstrates disease-modifying activity in patients and the ability to target the malignant stem and progenitor cells of the underlying disease; (f) that Geron may seek to raise substantial additional capital in order to continue the development and commercialization of imetelstat; (g) whether Geron meets its post-marketing requirements and commitments in the U.S. for RYTELO for the treatment of patients with LR-MDS with transfusion dependent anemia; (h) whether there are failures or delays in manufacturing or supplying sufficient quantities of imetelstat or other clinical trial materials that impact commercialization of RYTELO for the treatment of patients with LR-MDS with transfusion dependent anemia or the continuation of the IMpactMF trial; (i) that the projected timing for the interim and final analyses of the IMpactMF trial may vary depending on actual enrollment and death rates in the trial; and (j) whether the EMA will approve RYTELO for the treatment of patients with LR-MDS with transfusion dependent anemia and whether the FDA and EMA will approve imetelstat for other indications on the timelines expected, or at all. Additional information on the above risks and uncertainties and additional risks, uncertainties and factors that could cause actual results to differ materially from those in the forward-looking statements are contained in Geron’s filings and periodic reports filed with the Securities and Exchange Commission under the heading “Risk Factors” and elsewhere in such filings and reports, including Geron’s quarterly report on Form 10-Q for the quarter ended March 31, 2024, and subsequent filings and reports by Geron. Undue reliance should not be placed on forward-looking statements, which speak only as of the date they are made, and the facts and assumptions underlying the forward-looking statements may change. Except as required by law, Geron disclaims any obligation to update these forward-looking statements to reflect future information, events, or circumstances.

¹ Lewis R, Bewersdorf JP, Zeidan AM. Clinical Management of Anemia in Patients with Myelodysplastic Syndromes: An Update on Emerging Therapeutic Options. Cancer Manag Res. 2021 Jan 25;13:645-657. doi: 10.2147/CMAR.S240600. PMID: 33531837; PMCID: PMC7846829.

² Cogle CR, Reddy SR, Chang E, et al. Early treatment initiation in lower-risk myelodysplastic syndromes produces an earlier and higher rate of transfusion independence. Leuk Res. 2017;60:123-128.

³ Balducci, L. (2006), Transfusion independence in patients with myelodysplastic syndromes. Cancer, 106: 2087-2094. https://doi.org/10.1002/cncr.21860
⁴ Platzbecker, U. et al. Imetelstat in patients with lower-risk myelodysplastic syndromes who have relapsed or are refractory to erythropoiesis-stimulating agents (IMerge): a multinational, randomised, double-blind, placebo-controlled, phase 3 trial. The Lancet. Volume 403, Issue 10423, P249-260. Jan 20, 2024.

Contacts

Aron Feingold

Vice President, Investor Relations and Corporate Communications

Kristen Kelleher

Associate Director, Investor Relations and Corporate Communications

investor@geron.com
media@geron.com

Tiziana Life Sciences Announces Six-Month Qualitative Improvement in Neuroimaging in 80% of Multiple Sclerosis Patients Receiving Intranasal Foralumab

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Qualitative improvements in PET imaging seen in 80% of non-active Secondary Progressive Multiple Sclerosis (na-SPMS) Expanded Access patients receiving intranasal foralumab for at least 6-months.
FDA Allowance for an additional 20 Patients to be enrolled in the intranasal foralumab Multiple Sclerosis Expanded Access Program will allow further data collection and analysis.
Applied for FDA Orphan Drug Designation of foralumab for na-SPMS

NEW YORK, June 06, 2024 (GLOBE NEWSWIRE) — Tiziana Life Sciences, Ltd. (Nasdaq: TLSA) (“Tiziana” or the “Company”), a biotechnology company developing breakthrough immunomodulation therapies via novel routes of drug delivery, today announced the qualitative results for all 10 non-active Secondary Progressive Multiple Sclerosis (na-SPMS) patients enrolled in the intermediate-size patient population Expanded Access (EA) Program receiving foralumab for at least six months.

Tarun Singhal, M.B.B.S., M.D., Director of the PET Imaging Program in Neurologic Diseases, associate neurologist and nuclear medicine physician at Brigham and Women’s Hospital, a founding member of Mass General Brigham Healthcare System, and Associate Professor of Neurology at Harvard Medical School, commented, “Based on currently available data from the latest cohort of four Expanded Access patients, three out of the four subjects had findings that suggest a qualitative reduction in the microglial PET signal over a period of six months of treatment with nasal foralumab. When combined with my assessment of the first six Expanded Access patients at six months, eight of the ten suggest a qualitative reduction in microglial PET signal. Further studies are needed to confirm these findings using additional cases and quantitative approaches.”

Gabriele Cerrone, Chairman, acting CEO, and founder of Tiziana Life Sciences, added, “I am thrilled that 80% of the na-SPMS patients who received intranasal foralumab treatment for at least 6-months have a qualitative reduction of microglial activity as confirmed in these latest PET images. I am also greatly appreciative of Dr. Singhal’s research and look forward to the additional quantitative analysis of the data. With the allowance of an additional 20 patients in the EA program, the application for Orphan Drug Designation for na-SPMS, and the ongoing Phase 2a trial, Tiziana is rapidly progressing its intranasal foralumab program in multiple sclerosis.”

About Foralumab

Activated T cells play an important role in the inflammatory process. Foralumab, the only fully human anti-CD3 monoclonal antibody (mAb), binds to the T cell receptor and dampens inflammation by modulating T cell function, thereby suppressing effector features in multiple immune cell subsets. This effect has been demonstrated in patients with COVID and with multiple sclerosis, as well as in healthy normal subjects. The non-active SPMS intranasal foralumab Phase 2 trial (NCT06292923) began screening patients in November of 2023. Immunomodulation by nasal anti-CD3 mAb represents a novel avenue for treatment of neuroinflammatory and neurodegenerative human diseases.^[¹^],[²^]

About Tiziana Life Sciences

Tiziana Life Sciences is a clinical-stage biopharmaceutical company developing breakthrough therapies using transformational drug delivery technologies to enable alternative routes of immunotherapy. Tiziana’s innovative nasal approach has the potential to provide an improvement in efficacy as well as safety and tolerability compared to intravenous (IV) delivery. Tiziana’s lead candidate, intranasal foralumab, which is the only fully human anti-CD3 mAb, has demonstrated a favorable safety profile and clinical response in patients in studies to date. Tiziana’s technology for alternative routes of immunotherapy has been patented with several applications pending and is expected to allow for broad pipeline applications.

For further inquiries:

Tiziana Life Sciences Ltd
Paul Spencer, Business Development and Investor Relations
+44 (0) 207 495 2379
email: info@tizianalifesciences.com

Investors:
Irina Koffler
LifeSci Advisors, LLC
646.970.4681
ikoffler@lifesciadvisors.com

[1] https://www.pnas.org/doi/10.1073/pnas.2220272120
[2] https://www.pnas.org/doi/10.1073/pnas.2309221120

Agomab Receives FDA Orphan Drug Designation for AGMB-447 in Idiopathic Pulmonary Fibrosis

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— AGMB-447 is an inhaled lung-restricted ALK5-inhibitor currently in a Phase 1 clinical trial —

ANTWERP, Belgium–(BUSINESS WIRE)–Agomab Therapeutics NV (‘Agomab’) today announced that it has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for AGMB-447, its inhaled, small molecule inhibitor of ALK5. Agomab is evaluating AGMB-447 as a potential treatment for Idiopathic Pulmonary Fibrosis (IPF) in a Phase 1 clinical trial (NCT06181370).

The FDA’s Orphan Drug Designation program is designed to facilitate development of medicinal treatments for rare diseases that affect fewer than 200,000 people in the U.S. The designation provides companies with various development and commercial benefits, including market exclusivity and a range of financial incentives, such as tax relief for clinical research costs.

“Receiving Orphan Drug Designation from the FDA provides further support that AGMB-447’s mechanism of action has the potential to achieve meaningful therapeutic benefits to IPF patients,” said Philippe Wiesel, Chief Medical Officer at Agomab Therapeutics. “As we progress through our ongoing first-in-human Phase 1 trial, we look forward to evaluating the data from the single ascending dose and multiple ascending dose evaluation of AGMB-447 in healthy subjects and IPF patients.”

AGMB-447 is an investigational drug and not approved by any regulatory authority. Its efficacy and safety have not been established.

About AGMB-447

AGMB-447 is a small molecule lung-restricted inhibitor of ALK5 (or TGFβRI) for the treatment of Idiopathic Pulmonary Fibrosis (IPF) and other fibrotic respiratory indications. IPF is a devastating disease affecting 100,000 patients in the U.S. IPF is characterized by unregulated production of fibrotic, scar-like tissue that builds up in the lungs. As a result, the fibrotic lung becomes stiff which hampers breathing and reduces the absorption of inhaled oxygen in the blood. Even though some medicinal treatments are available, without a lung transplant, the average survival following diagnosis is only three to five years. TGFβ is a known master regulator of fibrosis in IPF and preliminary clinical data supports targeting the pathway. AGMB-447 is specifically designed to potently and safely inhibit ALK5 only in the lung due to its rapid metabolism through hydrolysis in plasma, which prevents clinically relevant systemic exposure.

About Agomab

Agomab is focused on achieving disease modification by modulating fibrosis and regeneration in chronic indications such as Fibrostenosing Crohn’s Disease and Idiopathic Pulmonary Fibrosis. We do this by targeting biologically validated pathways – including Transforming Growth Factor β and Hepatocyte Growth Factor – and by applying specialized capabilities in organ-restricted small molecules and high affinity antibodies. With a differentiated clinical pipeline across several fibrotic disorders, end-to-end research and development capabilities, a proven BD track-record and a strong investor base, Agomab is building a leading European biopharma company.

Contacts

For Agomab Therapeutics

Tim Knotnerus, CEO

E-Mail: tim.knotnerus@agomab.com

Media Requests for Agomab
Dr. Stephanie May or Gretchen Schweitzer

Trophic Communications

Phone: + 49 (0) 172 861 8540

E-Mail: agomab@trophic.eu

Osteal Therapeutics Closes $50M Series D Financing to Support Approval and Commercial Launch of Lead Candidate

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Financing led by Zimmer Biomet with participation from existing investors Johnson & Johnson Innovation – JJDC, Inc., Gideon Strategic Partners, and HM Capital.

Proceeds will support NDA submission and commercial launch of lead candidate, VT-X7, a 7-day treatment for periprosthetic joint infection.

DALLAS, June 05, 2024 (GLOBE NEWSWIRE) — Osteal Therapeutics, Inc. (“Osteal”), a clinical-stage biopharmaceutical company developing a new category of combination drug/device therapies for orthopedic infections, announced today the completion of an oversubscribed $50 million Series D preferred stock equity financing. Zimmer Biomet led the round, joined by returning investors Johnson & Johnson Innovation – JJDC, Inc., Gideon Strategic Partners, and HM Capital. In conjunction with the financing, a representative of Zimmer Biomet will join Osteal’s Board of Directors.

The proceeds of the financing will be used to advance the development of Osteal’s portfolio of therapies, including the submission of a New Drug Application (NDA) for, and accelerated commercial launch of, VT-X7 for the treatment of periprosthetic joint infection (PJI) of the hip and knee. VT-X7 has been the subject of two multicenter, randomized, controlled trials, APEX and APEX-2. APEX-2 recently met its primary endpoint and will be completed in late 2024. The study builds upon the safety and efficacy evidence obtained from APEX, a similarly designed clinical study that successfully met its primary endpoint in 2023. Both studies were designed in close consultation with the FDA, which previously granted VT-X7 Breakthrough Therapy, Orphan Drug, Fast Track, and Qualified Infectious Disease Product designations. The company expects to seek FDA approval upon completion of APEX-2.

“This round of financing represents an inflection point in the company’s progress towards introducing VT-X7 to the market,” said David Thompson, Chairman and Chief Executive Officer. “From the beginning, Osteal has been extremely fortunate to have the support of a world-class group of financial and strategic partners who share our vision and recognize the tremendous impact our programs will have on patient care. I am delighted to welcome Zimmer Biomet as a partner and look forward to working closely with their exemplary team.”

About Periprosthetic Joint Infection (PJI)

Affecting over 40,000 people in the U.S. annually, PJI is a rare and potentially devastating complication of joint replacement surgery in which pathogenic bacteria colonize the joint prosthesis, forming difficult-to-remove structures called biofilms. Biofilm infections are challenging to resolve, requiring long, invasive and expensive treatments that are often unsuccessful, resulting in high rates of permanent disability and early death. Recent retrospective analyses demonstrate that the current gold standard for treatment of PJI, two-stage exchange arthroplasty, takes an average of 16 weeks and has a success rate under 50% after 12 months, highlighting the unmet need for faster and more efficacious treatment options.

About VT-X7

VT-X7 (vancomycin hydrochloride and tobramycin sulfate for irrigation/VT-X7 irrigation system) is a novel drug/device combination product designed to deliver therapeutic concentrations of vancomycin and tobramycin, well-established, broad-spectrum antibiotics, directly to the joint space and surrounding tissue to treat PJI. VT-X7 is a seven-day therapy designed to address the unmet clinical need for a rapid, reliable treatment for these challenging infections. In clinical studies of VT-X7, 100% of patients were treated and received a new permanent joint prosthesis in seven days with >90% remaining infection-free after one year. The US Food and Drug Administration has granted VT-X7 Breakthrough Therapy, Orphan Drug, Fast Track and Qualified Infectious Disease Product designations. This initial application of VT-X7 represents a first-of-its-kind, multibillion-dollar opportunity to dramatically improve outcomes for a serious unmet medical need.

About Osteal Therapeutics, Inc.

Osteal Therapeutics is a privately held, clinical-stage biopharmaceutical company developing novel musculoskeletal therapeutics to treat orthopedic infections and their consequences. The company is leveraging the ability of concentrated, locally delivered antimicrobials to treat the bacterial biofilms typically responsible for musculoskeletal infections while minimizing off-target tissue exposure and associated adverse effects. Osteal employs a low-risk development strategy by using approved drugs with long histories of safety and efficacy as candidates for new routes of local delivery. The company’s lead candidate, VT-X7, is in late-stage clinical development to treat periprosthetic joint infections, a serious complication of joint replacement surgery. For more information, please visit: www.ostealtx.com

Corporate Contact:

Todd Saunders
Vice President of Marketing
Osteal Therapeutics, Inc.
Phone: 469-809-2630
Email: info@ostealtx.com

QUANTRO Therapeutics reaches a milestone in the collaboration with Boehringer Ingelheim to develop first-in-class cancer treatments

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Vienna, Austria, 04 June 2024: QUANTRO Therapeutics (QUANTRO), a pioneer in the discovery of first-in-class transcription factor targeting cancer treatments, announced today that the company has reached a key milestone in its joint R&D program with Boehringer Ingelheim. In successfully applying QUANTRO’s proprietary QUANTROseq® Transcriptional Fingerprint technology, all pre-set goals for technical proof-of-concept, cross-validation of HTS screening technology and identification of high-quality hits for a previously undruggable transcription factor were fully accomplished.

For many cancer patients there are no or only limited treatment options, which is why cancer remains to be a leading cause of death worldwide, accounting for nearly one in six deaths. Transcription factors have emerged as a promising class of therapeutic targets to address currently undruggable cancers. Transcription factors are central regulators of gene expression that are commonly dysregulated in cancer and have remained largely unamenable to pharmacological intervention. The joint R&D program, which was initiated in 2022, aims at changing the implied status quo via development of transformational, first-in-class cancer treatments.

Michael Bauer, CEO of QUANTRO, commented: “We are committed to drive a positive change for people affected by cancer, and to that end are looking forward to developing new mode of action compounds jointly with Boehringer Ingelheim. In reaching this milestone, QUANTRO Therapeutics continues to advance its leading transcriptomic R&D platform to build a highly innovative pipeline of modulators, inhibitors or degraders of transcription factors, transcriptional regulators and cell signaling targets.“

Norbert Kraut, Global Head of Cancer Research at Boehringer Ingelheim said: “We are excited and encouraged to see a very convincing and robust technology proof of concept, and highly attractive hits from the first screening that we can directly take to the next discovery and development stages. We look forward to further expanding our pipeline of first-in-class treatments to address the remaining high unmet patient need in cancer in partnership with QUANTRO.”

Upon accomplishment of this milestone, QUANTRO will receive an undisclosed milestone payment in addition to previously received upfront payments and R&D funding. The total potential transaction value may exceed EUR 500 million in form of R&D funding and success driven discovery, development, regulatory and commercial milestones.

About QUANTRO:

QUANTRO Therapeutics is a transcriptomic Drug Discovery and R&D company focused on building a highly innovative pipeline of modulators, inhibitors or degraders of transcription factors, transcriptional regulators and cell signaling targets. QUANTRO´s transcriptomic discovery platform is using a novel and proprietary time-resolved gene expression profiling technology to target gene transcription factors thus far considered un-druggable.

The technology is uniquely positioned to quantify changes in gene expression over time with unprecedented precision and sensitivity, overcoming the deficiencies of traditional RTqPCR based technologies like DrugSeq, which are limited to only measure RNA abundance, without information on transcriptional activity and dynamics.

QUANTRO was founded in in 2019 as Spin-out from the prestigious research institutes IMBA and IMP in Vienna, Austria, and since 2020 supported by Boehringer Ingelheim Venture Fund BIVF and Evotec as SEED investors. In total EUR 11 million SEED financing were placed in milestone-driven tranches in 2020 and 2023.

Contact
QUANTRO Therapeutics GmbH
Dr. Michael Bauer, CEO
Mail: office@quantro-tx.com
Phone: +43 122 66001-20
www.quantro-tx.com

Media Contact
MC Services AG
Katja Arnold, Dr. Johanna Kobler, Shaun Brown
Phone: +49 89 210228 0
Mail: quantro@mc-services.eu

04.06.2024 CET/CEST Dissemination of a Corporate News, transmitted by EQS News – a service of EQS Group AG.
The issuer is solely responsible for the content of this announcement.

The EQS Distribution Services include Regulatory Announcements, Financial/Corporate News and Press Releases.
Archive at www.eqs-news.com

ExeVir Bio Announces Exceptional Virus Neutralization Potency of its Variant-proof XVR013m Antibody Against the SARS-CoV-2 Variant JN.1

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Developed to provide protection for millions of immunocompromised and elderly individuals who are most vulnerable to COVID-19

GHENT, Belgium, June 04, 2024 (GLOBE NEWSWIRE) — ExeVir Bio, a biotech company developing robust heavy chain-only antibody therapies for broad protection against infectious diseases, today announced new data demonstrating that its antibodies are exceptionally potent in neutralizing the SARS-CoV-2 variant JN.1, the parental strain of the currently most dominantly circulating variants worldwide.

ExeVir’s COVID-19 program focuses on addressing the high unmet medical need to protect and treat immunocompromised and elderly individuals that account for respectively 4% and 10% of the global population. Despite the availability of effective vaccines, the immunocompromised and elderly remain at a higher risk for severe COVID-19 as they are often not able to elicit an adequate immune response to vaccination and therefore would strongly benefit from additional protection in the form of antibody therapeutics.

ExeVir’s variant-proof lead clinical candidate XVR013m is a heavy chain-only antibody that targets a unique membrane-proximal epitope in the S2 subunit of the spike protein. This binding region remains completely unmutated across all variants of concern, variants of interest and variants under monitoring that have circulated to date. XVR013m demonstrates exceptional neutralization potency against JN.1, the parental strain of the currently most dominantly circulating variants worldwide, with an IC50 of 2.8 ng/mL in a pseudovirus neutralization assay, unchanged compared to all previously tested variants. The activity against new circulating variants under monitoring is continually being tested.

ExeVir is currently gearing up to start the clinical development of the variant-proof XVR013m in 2025 as a solution for the prevention of COVID-19 in the target populations of immunocompromised and elderly individuals.

Jeanne Bolger, interim CEO of ExeVir, said: “COVID-19 is here to stay and is still the most important respiratory infectious disease causing substantially more hospitalizations and deaths compared to influenza and RSV. Today, no real seasonality is observed for COVID-19, in sharp contrast to influenza and RSV. Our antibody XVR013m is differentiated by targeting a unique and highly conserved epitope in the S2 subunit of the SARS-CoV-2 spike protein that makes it virtually variant-proof.”

Viki Bockstal, CSO of ExeVir, said: “XVR013m not only demonstrates exceptional virus neutralization potency against all variants tested to date, it is also anticipated to maintain its activity against future variants because of the unique nature of the epitope. There is still a strong and urgent medical need for novel antibodies that work against both the newest as well as future SARS-CoV-2 variants, and these new data again demonstrate that XVR013m has the potential to become a durable therapeutic solution to protect the most vulnerable patients from this serious infectious disease.”

About ExeVir Bio

ExeVir Bio is a clinical stage biotechnology company developing heavy-chain only antibody therapies focusing on infectious diseases. The company is harnessing its llama-derived antibody (VHH) technology platform to generate multi-specific antibodies for prophylaxis and treatment of infectious diseases. ExeVir’s initial focus is on prevention of COVID-19 for the immunocompromised patient population, including active chemotherapy, immunosuppressive drugs, solid organ transplantation, hematological malignancies, AIDS patients, and for the elderly, where there remains a high unmet need due to the limitations of current vaccines and therapeutic approaches.

ExeVir has demonstrated it can progress its candidates from research to the clinic in under one year, execute on Phase 1a and Phase 1b studies, and conduct scale-up manufacturing. Leveraging this extensive experience, its XVR013m asset is being developed to start a First in Human clinical trial in 2025.

VHHs are smaller in size than whole antibodies, giving them access to hidden epitopes that traditional monoclonal antibodies are unable to reach with potential for deeper tissue penetration and simpler, more cost-effective manufacturing. VHHs can be linked together like building blocks into single multispecific molecules to tackle different epitopes or act through different mechanisms of actions at once, to address the “arms race” in more complex and co-evolving infectious diseases.

ExeVir is a spin out of VIB, the leading Belgium-based life sciences research institute. It is backed by strong investors including Fund+, which led the series A of EUR 42 million, together with an international consortium including UCB Ventures, SFPIM, V-Bio Ventures, VIB, Wallonie Entreprendre, Noshaq, Vives IUF and SambrInvest. ExeVir has received support from VLAIO, the SPW-Recherche and the European Union, leading to a total of EUR 18 million in non-dilutive funding. In 2023, ExeVir secured an option for EUR 25 million venture debt financing from the European Investment Bank.

References

1. Impact of COVID-19 on immunocompromised populations during the Omicron era: insights from the observational population-based INFORM study, Evans et al, The Lancet, 2023.
https://www.thelancet.com/journals/lanepe/article/PIIS2666-7762%2823%2900166-7/fulltext.

2. World population above 65 years old: Population ages 65 and above, total | Data (worldbank.org)

For more information contact:

ExeVir Bio
Veronique Vandevoorde
vvandevoorde@exevir.com

Scius Communications (for media)
Katja Stout
+44 778 943 5990
katja@sciuscommunications.com

Daniel Gooch
+44 774 787 5479
daniel@sciuscommunications.com

Find out more on ExeVir’s LinkedIn or on ExeVir’s website

RS Oncology Announces Positive Data from a Phase 1 Clinical Trial of RSO-021, a First-in-Class Therapeutic for Malignant Pleural Mesothelioma

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First-in-human MITOPE clinical study meets primary objective of Phase 1 safety and tolerability
First-in-Class therapy shows early signs of efficacy and offers new hope to patients with malignant pleural mesothelioma with pleural effusion and other cancers.

CAMBRIDGE, Mass.–(BUSINESS WIRE)–#Lungcancer—RS Oncology, a privately held biopharmaceutical company focusing on novel treatments for rare and aggressive cancers presented positive results from its Phase 1 study in patients with Malignant Pleural Mesothelioma (MPM) with Malignant Pleural Effusion (MPE) or MPE associated with other solid tumors at the 2024 American Society of Clinical Oncology (ASCO) Annual Meeting.

The Phase 1 data was presented by Professor Dean Fennell, MRCP, FRCP (MD/PhD) on behalf of all the MITOPE investigators at an oral presentation session. In the 15 recruited patients safety data demonstrated weekly treatment of RSO-021 was well tolerated at 90 mg. The pharmacokinetic data showed minimal systemic exposure to RSO-021 after intrapleural administration. Efficacy data showed a long-term partial response in one patient as well as encouraging survival in 7 of the 10 evaluable patients. In addition to responses in mesothelioma, the drug showed promising responses in non-target lesions and other cancers with metastatic disease to the lung.

“The MITOPE trial would not have been possible without the support of the outstanding investigation teams throughout the UK who are dedicated to the treatment of patients with mesothelioma. We thank all of the MITOPE trial participants and their supportive families, and look forward to hearing about their continued benefit,” said George Naumov, PhD, RS Oncology Chief Operations Officer.

“RSO-021 represents a new class of drugs with a first-in-class anticancer mechanism. The safety and efficacy observed in the Phase 1 trial is supported by strong pre-clinical rationale” said Brian Cunniff, PhD, Chief Science Officer for RS Oncology.

MPM is a rare and aggressive form of cancer that typically develops years after asbestos inhalation and/or exposure. Most cases (70%) originate in the pleura, but it can also be found the peritoneum and the pericardium. A mesothelioma prognosis remains poor with a shorter life expectancy and decreased quality of life.

Phase 2 exploration of this novel agent is ongoing at two doses, as a single agent and in combination with chemotherapy. Clinical trial information: NCT05278975.

About RS0-021

RSO-021 is a naturally occurring, sulfur-rich, cyclic oligopeptide of the thiopeptide class, which covalently inactivates PRX3, leading to catastrophic oxidative stress and cell death.

The oral and poster presentations will be available for viewing on RSOncology.com.

About the MITOPE study

MITOPE is an open-label, non-randomized, multicenter, translational Phase 1/2 dose-escalation and expansion study. It is designed to determine the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary anti-tumor activity of RSO-021 after intrapleural (IP) administration in patients with MPE associated with either MPM or other solid tumors.

About RS Oncology

RS Oncology is a private, clinical-stage biopharmaceutical company leading scientific discoveries and global collaborations to improve the lives of patients with the most aggressive cancers. Taking a fundamentally different approach to drug development, we are advancing a pipeline of programs based on mitochondrial dynamics and metabolic function that dictate cell processes including cell migration and tumor cell metastasis. The oral presentation will be posted to RSOncology.com or follow us on LinkedIn and X.

Contacts

Corporate contact:

Jarrett Duncan, CEO

j.duncan@rsoncology.com

Promising Anti-tumor Activity of Novel Costimulatory Bispecific Antibody REGN7075 (EGFRxCD28) in Combination with Libtayo® (cemiplimab) to be Reported at ASCO

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Oral presentation to highlight activity of REGN7075 in combination with Libtayo from dose-escalation portion of trial in patients with microsatellite stable colorectal cancer, which has historically proven unresponsive to immunotherapy

Ongoing REGN7075 Phase 1/2 trial is investigating a potentially first-in-class combination across a range of advanced solid tumors

TARRYTOWN, N.Y., May 23, 2024 (GLOBE NEWSWIRE) — Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced positive new results from an ongoing Phase 1/2 trial evaluating its first-in-class costimulatory bispecific antibody, REGN7075 (EGFRxCD28), in combination with Libtayo^® (cemiplimab) in patients with advanced solid tumors. Data from the dose-escalation portion of the trial showed the investigational combination led to anti-tumor responses in patients with microsatellite stable colorectal cancer (MSS CRC). REGN7075 is one of the first immunotherapies to demonstrate clinical activity in MSS CRC, including in a patient with liver metastases. The results will be shared during an oral session at the American Society of Clinical Oncology (ASCO) 2024 Annual Meeting in Chicago.

“Microsatellite stable colorectal cancer has historically been unresponsive to immunotherapy,” said Neil H. Segal, M.D., Ph.D., Medical Oncologist and Research Director in the Division of Gastrointestinal Oncology at Memorial Sloan Kettering Cancer Center, and a trial investigator. “The early results for this novel investigational EGFRxCD28 costimulatory bispecific in combination with Libtayo are encouraging, showing anti-tumor responses in a highly difficult-to-treat cancer. This combination is one of the first immunotherapy regimens to show clinical activity in microsatellite stable colorectal cancer, and we are excited to advance this trial in additional tumor types.”

In the dose-escalation portion of the trial, patients with metastatic and locally advanced solid tumors – who had exhausted standard treatment options, and most of whom also had liver metastases – received combination therapy with REGN7075 and Libtayo, following a REGN7075 monotherapy lead-in dose. Among 94 patients treated as of data cutoff, 65% (n=61) had MSS CRC, of which 51 MSS CRC patients were treated at an active dose level. Efficacy results among these 51 patients were as follows:

6% (n=3) overall response rate (ORR) and 29% (n=15) disease control rate (DCR). This included one complete response (CR), two partial responses (PR), and 12 patients with stable disease. At data cutoff, all responders were without liver metastases.
Among the subset of 15 patients without liver metastases, there was a 20% ORR (n=3) and 80% DCR (n=12).
Among the subset of 36 patients with liver metastases, three patients had stable disease as of data cutoff, and one patient achieved a PR following data cutoff.

Safety was assessed in 84 patients across multiple solid tumor types at a variety of doses of REGN7075. REGN7075 and Libtayo showed an acceptable safety profile, and the maximum tolerated dose was not reached. Treatment-emergent adverse events (TEAEs) of any grade occurred in 98% of patients; Grade 3 and 4 TEAEs occurred in 35% of patients. Treatment-related adverse events (TRAEs) occurred in 90% of patients, with 7% of cases reported as grade 3 or 4. The majority of TRAEs were Grade 1 to 2 (83%), with the most common being infusion-related reactions (58%) that were manageable with premedication and dosing adjustments. TRAEs led to discontinuation in 5% of patients, and three patients discontinued treatment due to Grade 2 infusion-related reactions. As of data cutoff, there have been no dose-limiting toxicities, no reports of cytokine release syndrome, and no treatment-related deaths.

“Regeneron is focused on developing a unique investigational portfolio of oncology medicines including checkpoint inhibitors, CD3 bispecifics and CD28 costimulatory bispecifics. Over the past several years, we have made progress in our programs across checkpoint inhibitors and the CD3 class and are now showing promising activity with two costimulatory bispecific antibodies,” said Israel Lowy, M.D., Ph.D., Senior Vice President, Translational and Clinical Oncology at Regeneron. “Our costimulatory bispecifics were designed with the goal of turning cancer cells into antigen presenting cells, thereby converting historically immunotherapy unresponsive tumors from ‘cold’ to ‘hot’. These early data speak to the potential of REGN7075 in combination with Libtayo and add to a growing body of evidence supporting novel costimulatory bispecifics that are in clinical trials for a range of solid tumors and blood cancers.”

The combination of REGN7075 and Libtayo is currently under clinical development, and its safety and efficacy have not been fully evaluated by any regulatory authority. While the dose escalation portion of the trial across multiple solid tumor types including non-small cell lung cancer, colorectal cancer, head and neck cancer and other tumor types is ongoing, expansion cohorts in several tumor types have also been initiated.

About the Phase 1/2 Trial
The Phase 1/2, first-in-human, open-label trial investigating REGN7075 in combination with Libtayo is currently enrolling patients with metastatic and locally advanced solid tumors who have exhausted standard treatment options. The trial includes an ongoing Phase 1 dose-escalation portion and a Phase 2 dose-expansion period. In the Phase 1 dose-escalation portion, patients first receive a weekly lead-in dose of REGN7075 monotherapy for three weeks to assess its safety and efficacy alone. This is followed by treatment with combination therapy, with Libtayo dosed once every three weeks and REGN7075 dosed either every week or every three weeks. The primary endpoints are assessing safety and tolerability, while the secondary endpoints are assessing efficacy, pharmacokinetics and immunogenicity. Expansion cohorts in several tumor types have been initiated. For more information, visit the Regeneron clinical trials website, or contact via clinicaltrials@regeneron.com or 844-734-6643.

About Regeneron in Cancer
We aspire to turn revolutionary discoveries into medicines that can transform the lives of those impacted by cancer. Our team around the world is driven to solve the needs and challenges of those affected by one of the most serious diseases of our time.

Backed by our legacy of scientific innovation and a deep understanding of biology, genetics and the immune system, we’re pursuing potential therapies across more than 30 types of solid tumors and blood cancers. Our cancer strategy is powered by cutting-edge technologies and therapies that can be flexibly combined to investigate potentially transformative treatments for patients. Oncology assets in clinical development comprise nearly half of Regeneron’s pipeline, and include checkpoint inhibitors, bispecific antibodies and costimulatory bispecific antibodies. Our approved PD-1 inhibitor Libtayo serves as the backbone of many of our investigational combinations.

To complement our extensive in-house capabilities, we collaborate with patients, healthcare providers, governments, biopharma companies and each other to further our shared goals. Together, we are united in the mission to serve as a beacon of transformation in cancer care.

About Libtayo
Libtayo is a fully human monoclonal antibody targeting the immune checkpoint receptor PD-1 on T cells and was invented using Regeneron’s proprietary VelocImmune^® technology. By binding to PD-1, Libtayo has been shown to block cancer cells from using the PD-1 pathway to suppress T-cell activation. In the U.S. and other countries Libtayo is indicated in certain patients with advanced basal cell carcinoma (BCC), advanced cutaneous squamous cell carcinoma (CSCC) and advanced non-small cell lung cancer (NSCLC), as well as in advanced cervical cancer in the European Union, Canada and Brazil. Libtayo is developed and marketed globally by Regeneron.

In the U.S., the generic name for Libtayo in its approved indications is cemiplimab-rwlc, with rwlc as the suffix designated in accordance with Nonproprietary Naming of Biological Products Guidance for Industry issued by the U.S. Food and Drug Administration (FDA). Outside of the U.S., the generic name of Libtayo in its approved indication is cemiplimab.

The extensive clinical program for Libtayo is focused on difficult-to-treat cancers. Libtayo is currently being investigated in trials as a monotherapy, as well as in combination with either conventional or novel therapeutic approaches for other solid tumors and blood cancers. These potential uses are investigational, and their safety and efficacy have not been evaluated by any regulatory authority.

U.S. FDA-approved Indications
Libtayo is a prescription medicine used to treat:

People with a type of skin cancer called cutaneous squamous cell carcinoma (CSCC) that has spread or cannot be cured by surgery or radiation.
People with a type of skin cancer called basal cell carcinoma (BCC) when your BCC cannot be removed by surgery (locally advanced BCC) or when it has spread (metastatic BCC) and have received treatment with a hedgehog pathway inhibitor (HHI), or cannot receive treatment with a HHI.
Adults with a type of lung cancer called non-small cell lung cancer (NSCLC).
- LIBTAYO may be used in combination with chemotherapy that contains a platinum medicine as your first treatment when your lung cancer has not spread outside your chest (locally advanced lung cancer) and you cannot have surgery or chemotherapy with radiation, or your lung cancer has spread to other areas of your body (metastatic lung cancer), and your tumor does not have an abnormal “EGFR,” “ALK,” or “ROS1” gene.
- LIBTAYO may be used alone as your first treatment when your lung cancer has not spread outside your chest (locally advanced lung cancer) and you cannot have surgery or chemotherapy with radiation, or your lung cancer has spread to other areas of your body (metastatic lung cancer), and your tumor tests positive for high “PD-L1,” and your tumor does not have an abnormal “EGFR,” “ALK,” or “ROS1” gene.

It is not known if Libtayo is safe and effective in children.

IMPORTANT SAFETY INFORMATION FOR U.S. PATIENTS

What is the most important information I should know about LIBTAYO?
LIBTAYO is a medicine that may treat certain cancers by working with your immune system. LIBTAYO can cause your immune system to attack normal organs and tissues in any area of your body and can affect the way they work. These problems can sometimes become severe or life-threatening and can lead to death. You can have more than one of these problems at the same time. These problems may happen anytime during treatment or even after your treatment has ended.

Call or see your healthcare provider right away if you develop any new or worsening signs or symptoms, including:

Lung problems: cough, shortness of breath, or chest pain
Intestinal problems: diarrhea (loose stools) or more frequent bowel movements than usual, stools that are black, tarry, sticky or have blood or mucus, or severe stomach-area (abdomen) pain or tenderness
Liver problems: yellowing of your skin or the whites of your eyes, severe nausea or vomiting, pain on the right side of your stomach-area (abdomen), dark urine (tea colored), or bleeding or bruising more easily than normal
Hormone gland problems: headache that will not go away or unusual headaches, eye sensitivity to light, eye problems, rapid heartbeat, increased sweating, extreme tiredness, weight gain or weight loss, feeling more hungry or thirsty than usual, urinating more often than usual, hair loss, feeling cold, constipation, your voice gets deeper, dizziness or fainting, or changes in mood or behavior, such as decreased sex drive, irritability, or forgetfulness
Kidney problems: decrease in your amount of urine, blood in your urine, swelling of your ankles, or loss of appetite
Skin problems: rash, itching, skin blistering or peeling, painful sores or ulcers in mouth or nose, throat, or genital area, fever or flu-like symptoms, or swollen lymph nodes
Problems can also happen in other organs and tissues. These are not all of the signs and symptoms of immune system problems that can happen with LIBTAYO. Call or see your healthcare provider right away for any new or worsening signs or symptoms, which may include: chest pain, irregular heartbeat, shortness of breath or swelling of ankles, confusion, sleepiness, memory problems, changes in mood or behavior, stiff neck, balance problems, tingling or numbness of the arms or legs, double vision, blurry vision, sensitivity to light, eye pain, changes in eyesight, persistent or severe muscle pain or weakness, muscle cramps, low red blood cells, or bruising
Infusion reactions that can sometimes be severe or life-threatening. Signs and symptoms of infusion reactions may include: nausea, vomiting, chills or shaking, itching or rash, flushing, shortness of breath or wheezing, dizziness, feel like passing out, fever, back or neck pain, or facial swelling
Rejection of a transplanted organ. Your healthcare provider should tell you what signs and symptoms you should report and monitor you, depending on the type of organ transplant that you have had
Complications, including graft-versus-host disease (GVHD), in people who have received a bone marrow (stem cell) transplant that uses donor stem cells (allogeneic). These complications can be serious and can lead to death. These complications may happen if you underwent transplantation either before or after being treated with LIBTAYO. Your healthcare provider will monitor you for these complications

Getting medical treatment right away may help keep these problems from becoming more serious. Your healthcare provider will check you for these problems during your treatment with LIBTAYO. Your healthcare provider may treat you with corticosteroid or hormone replacement medicines. Your healthcare provider may also need to delay or completely stop treatment with LIBTAYO if you have severe side effects.

Before you receive LIBTAYO, tell your healthcare provider about all your medical conditions, including if you:

have immune system problems such as Crohn’s disease, ulcerative colitis, or lupus
have received an organ transplant
have received or plan to receive a stem cell transplant that uses donor stem cells (allogeneic)
have received radiation treatment to your chest area
have a condition that affects your nervous system, such as myasthenia gravis or Guillain-Barré syndrome
are pregnant or plan to become pregnant. LIBTAYO can harm your unborn baby
are breastfeeding or plan to breastfeed. It is not known if LIBTAYO passes into your breast milk. Do not breastfeed during treatment and for at least 4 months after the last dose of LIBTAYO

Females who are able to become pregnant:

Your healthcare provider will give you a pregnancy test before you start treatment
You should use an effective method of birth control during your treatment and for at least 4 months after your last dose of LIBTAYO. Talk to your healthcare provider about birth control methods that you can use during this time
Tell your healthcare provider right away if you become pregnant or think you may be pregnant during treatment with LIBTAYO

Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

The most common side effects of LIBTAYO when used alone include tiredness, muscle or bone pain, rash, diarrhea, and low levels of red blood cells (anemia). The most common side effects of LIBTAYO when used in combination with platinum-containing chemotherapy include hair loss, muscle or bone pain, nausea, tiredness, numbness, pain, tingling, or burning in your hands or feet, and decreased appetite. These are not all the possible side effects of LIBTAYO. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088. You may also report side effects to Regeneron Pharmaceuticals at 1-877-542-8296.

Please see full Prescribing Information, including Medication Guide.

About Regeneron’s VelocImmune Technology
Regeneron’s VelocImmune technology utilizes a proprietary genetically engineered mouse platform endowed with a genetically humanized immune system to produce optimized fully human antibodies. When Regeneron’s co-Founder, President and Chief Scientific Officer George D. Yancopoulos was a graduate student with his mentor Frederick W. Alt in 1985, they were the first to envision making such a genetically humanized mouse, and Regeneron has spend decades inventing and developing VelocImmune and related VelociSuite^® technologies. Dr. Yancopoulos and his team have used VelocImmune technology to create a substantial proportion of all original, FDA-approved or authorized fully human monoclonal antibodies. This includes REGEN-COV^® (casirivimab and imdevimab), Dupixent^® (dupilumab), Libtayo^®, Praluent^® (alirocumab), Kevzara^® (sarilumab), Evkeeza^® (evinacumab-dgnb), Inmazeb^® (atoltivimab, maftivimab and odesivimab-ebgn) and Veopoz^® (pozelimab-bbfg).

About Regeneron
Regeneron (NASDAQ: REGN) is a leading biotechnology company that invents, develops and commercializes life-transforming medicines for people with serious diseases. Founded and led by physician-scientists, our unique ability to repeatedly and consistently translate science into medicine has led to numerous approved treatments and product candidates in development, most of which were homegrown in our laboratories. Our medicines and pipeline are designed to help patients with eye diseases, allergic and inflammatory diseases, cancer, cardiovascular and metabolic diseases, neurological diseases, hematologic conditions, infectious diseases, and rare diseases.

Regeneron pushes the boundaries of scientific discovery and accelerates drug development using our proprietary technologies, such as VelociSuite^®, which produces optimized fully human antibodies and new classes of bispecific antibodies. We are shaping the next frontier of medicine with data-powered insights from the Regeneron Genetics Center^® and pioneering genetic medicine platforms, enabling us to identify innovative targets and complementary approaches to potentially treat or cure diseases.
For more information, please visit www.Regeneron.com or follow Regeneron on LinkedIn, Instagram, Facebook or X.

Dr. Segal has financial interests related to Regeneron Pharmaceuticals.

Forward-Looking Statements and Use of Digital Media
This press release includes forward-looking statements that involve risks and uncertainties relating to future events and the future performance of Regeneron Pharmaceuticals, Inc. (“Regeneron” or the “Company”), and actual events or results may differ materially from these forward-looking statements. Words such as “anticipate,” “expect,” “intend,” “plan,” “believe,” “seek,” “estimate,” variations of such words, and similar expressions are intended to identify such forward-looking statements, although not all forward-looking statements contain these identifying words. These statements concern, and these risks and uncertainties include, among others, the nature, timing, and possible success and therapeutic applications of products marketed or otherwise commercialized by Regeneron and/or its collaborators or licensees (collectively, “Regeneron’s Products”) and product candidates being developed by Regeneron and/or its collaborators or licensees (collectively, “Regeneron’s Product Candidates”) and research and clinical programs now underway or planned, including without limitation REGN7075 in combination with Libtayo^® (cemiplimab) and other of Regeneron’s Product Candidates discussed or referenced in this press release; the likelihood, timing, and scope of possible regulatory approval and commercial launch of Regeneron’s Product Candidates and new indications for Regeneron’s Products, such as REGN7075 in combination with Libtayo in patients with advanced solid tumors and the other clinical programs discussed or referenced in the press release; uncertainty of the utilization, market acceptance, and commercial success of Regeneron’s Products and Regeneron’s Product Candidates and the impact of studies (whether conducted by Regeneron or others and whether mandated or voluntary), including the studies discussed or referenced in this press release, on any of the foregoing or any potential regulatory approval of Regeneron’s Products and Regeneron’s Product Candidates (such as REGN7075 in combination with Libtayo); the ability of Regeneron’s collaborators, licensees, suppliers, or other third parties (as applicable) to perform manufacturing, filling, finishing, packaging, labeling, distribution, and other steps related to Regeneron’s Products and Regeneron’s Product Candidates; the ability of Regeneron to manage supply chains for multiple products and product candidates; safety issues resulting from the administration of Regeneron’s Products and Regeneron’s Product Candidates (such as REGN7075 in combination with Libtayo) in patients, including serious complications or side effects in connection with the use of Regeneron’s Products and Regeneron’s Product Candidates in clinical trials; determinations by regulatory and administrative governmental authorities which may delay or restrict Regeneron’s ability to continue to develop or commercialize Regeneron’s Products and Regeneron’s Product Candidates; ongoing regulatory obligations and oversight impacting Regeneron’s Products, research and clinical programs, and business, including those relating to patient privacy; the availability and extent of reimbursement of Regeneron’s Products from third-party payers, including private payer healthcare and insurance programs, health maintenance organizations, pharmacy benefit management companies, and government programs such as Medicare and Medicaid; coverage and reimbursement determinations by such payers and new policies and procedures adopted by such payers; competing drugs and product candidates that may be superior to, or more cost effective than, Regeneron’s Products and Regeneron’s Product Candidates; the extent to which the results from the research and development programs conducted by Regeneron and/or its collaborators or licensees may be replicated in other studies and/or lead to advancement of product candidates to clinical trials, therapeutic applications, or regulatory approval; unanticipated expenses; the costs of developing, producing, and selling products; the ability of Regeneron to meet any of its financial projections or guidance and changes to the assumptions underlying those projections or guidance; the potential for any license, collaboration, or supply agreement, including Regeneron’s agreements with Sanofi and Bayer (or their respective affiliated companies, as applicable), to be cancelled or terminated; the impact of public health outbreaks, epidemics, or pandemics (such as the COVID-19 pandemic) on Regeneron’s business; and risks associated with intellectual property of other parties and pending or future litigation relating thereto (including without limitation the patent litigation and other related proceedings relating to EYLEA^® (aflibercept) Injection), other litigation and other proceedings and government investigations relating to the Company and/or its operations (including the pending civil proceedings initiated or joined by the U.S. Department of Justice and the U.S. Attorney’s Office for the District of Massachusetts), the ultimate outcome of any such proceedings and investigations, and the impact any of the foregoing may have on Regeneron’s business, prospects, operating results, and financial condition. A more complete description of these and other material risks can be found in Regeneron’s filings with the U.S. Securities and Exchange Commission, including its Form 10-K for the year ended December 31, 2023 and its Form 10-Q for the quarterly period ended March 31, 2024. Any forward-looking statements are made based on management’s current beliefs and judgment, and the reader is cautioned not to rely on any forward-looking statements made by Regeneron. Regeneron does not undertake any obligation to update (publicly or otherwise) any forward-looking statement, including without limitation any financial projection or guidance, whether as a result of new information, future events, or otherwise.
Regeneron uses its media and investor relations website and social media outlets to publish important information about the Company, including information that may be deemed material to investors. Financial and other information about Regeneron is routinely posted and is accessible on Regeneron’s media and investor relations website (https://investor.regeneron.com) and its LinkedIn page (https://www.linkedin.com/company/regeneron-pharmaceuticals).

Contacts:Media Relations
Taylor Skott
Tel: +1 914-409-2381
taylor.ramsey@regeneron.com

Investor Relations
Vesna Tosic
Tel: +1 914-847-5443
vesna.tosic@regeneron.com

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