Replimune's RP1 Melanoma Therapy Nears FDA Decision with July PDUFA Date as Commercial Infrastructure Readies for Launch
• Replimune's BLA for RP1 (vusolimogene oderparepvec) plus nivolumab in advanced melanoma is proceeding on schedule with a PDUFA date of July 22, 2025, following completed manufacturing inspections and late cycle review.
• The company has fully established its commercial infrastructure ahead of potential launch, targeting approximately 13,000 annual U.S. patients who progress on PD-1 treatment, with an estimated 80% eligible for RP1 therapy.
• Replimune maintains a strong financial position with $483.8 million in cash, cash equivalents and short-term investments as of March 31, 2025, providing runway into Q4 2026 to support commercialization efforts.
Samsung Biologics to Spin Off Biosimilar Business to Focus on Core CDMO Services
• Samsung Biologics announced plans to establish Samsung Epis Holdings, which will incorporate Samsung Bioepis as a wholly owned subsidiary, separating its biosimilar development from CDMO operations.
• The strategic spinoff aims to streamline operations by allowing each business to focus on their different revenue models, with final shareholder approval scheduled for September 16.
• As one of the world's leading CDMO firms, Samsung Biologics reported impressive financial performance with $3.3 billion in sales and $1 billion in net profit in 2024.
Medtronic Plans to Separate Diabetes Business into Standalone Company
• Medtronic has announced plans to separate its Diabetes business into an independent, publicly traded company within the next 18 months, enabling both entities to focus on their core strengths and growth opportunities.
• The separation is expected to improve Medtronic's financial metrics, with projected increases of approximately 50 basis points in adjusted gross margin and 100 basis points in adjusted operating margins.
• The new Diabetes Company, to be led by current EVP Que Dallara, will be positioned as the only company offering a complete ecosystem for intensive insulin management, serving patients with diabetes globally.
FDA Advisory Committee Votes 6-2 in Favor of Daratumumab for High-Risk Smoldering Multiple Myeloma
• The FDA's Oncologic Drug Advisory Committee (ODAC) voted 6-2 that daratumumab (Darzalex Faspro) demonstrates a favorable benefit-risk profile for patients with high-risk smoldering multiple myeloma, potentially offering the first approved therapy for this precursor condition.
• The phase 3 AQUILA trial showed daratumumab significantly delayed progression to active multiple myeloma with a 51% reduction in risk of progression or death compared to active monitoring, with 5-year PFS rates of 63.1% versus 40.8%.
• Committee members expressed concerns about risk classification accuracy and potential overtreatment, but ultimately determined the benefits outweighed risks for this malignant condition that has an 80% five-year progression risk to symptomatic multiple myeloma.
FDA Advisory Committee Rejects Genentech's Columvi Expansion for Transplant-Ineligible DLBCL Patients
• The FDA's Oncologic Drugs Advisory Committee voted 8-1 against expanding Genentech's bispecific antibody Columvi for transplant-ineligible patients with relapsed/refractory diffuse large B-cell lymphoma.
• Committee members expressed concerns about the applicability of the Phase III STARGLO study data to the U.S. population, as half of the participants were Asian patients with only 25 enrolled from North America.
• Columvi, which targets CD20 and CD3 proteins, was initially approved in June 2023 for DLBCL patients who had undergone at least two prior lines of systemic therapy.
Glofitamab-GemOx Shows Survival Benefit in Phase 3 STARGLO Trial for R/R DLBCL, But FDA Advisory Committee Questions US Applicability
• The phase 3 STARGLO trial demonstrated that glofitamab combined with gemcitabine and oxaliplatin nearly doubled median overall survival to 25.5 months compared with 12.9 months for rituximab-GemOx in transplant-ineligible R/R DLBCL patients.
• Despite showing a 41% reduction in death risk and 63% reduction in disease progression, the FDA's Oncologic Drugs Advisory Committee voted 8-to-1 against the applicability of the trial data to US patients, citing regional outcome differences.
• Glofitamab, a CD20xCD3 bispecific antibody, is currently approved in over 30 countries and has accelerated approval in the US, with an FDA decision on full approval expected by July 20, 2025.
KaliVir Advances Novel Oncolytic Immunotherapy VET3-TGI in Phase 1/1b Trial for Advanced Solid Tumors
• KaliVir Immunotherapeutics has successfully completed the first cohort of its STEALTH-001 Phase 1/1b trial evaluating VET3-TGI in patients with advanced solid tumors.
• The Data Safety Committee has reviewed safety data and cleared dosing for the next intratumoral and intravenous cohorts, allowing the trial to progress to higher dose levels.
• VET3-TGI is a novel oncolytic immunotherapy designed to selectively kill tumor cells while delivering an immuno-stimulatory payload of interleukin-12 and a TGFbeta inhibitor.
Pfizer Enters $6 Billion Licensing Deal with China's 3SBio for Novel Cancer Drug
• Pfizer has secured global rights (excluding China) to 3SBio's experimental cancer drug SSGJ-707 for $1.25 billion upfront, with potential additional payments of up to $4.8 billion based on developmental milestones.
• SSGJ-707 is currently being evaluated for multiple cancer types including non-small cell lung cancer, metastatic colorectal cancer, and gynecological tumors, with Phase III trials in China planned to begin this year.
• The deal includes a $100 million equity investment in 3SBio by Pfizer, with manufacturing planned at Pfizer's facilities in North Carolina and Kansas following FDA clearance of the Investigational New Drug application.
FDA Approves Teal Wand: First At-Home HPV Self-Collection Device for Cervical Cancer Screening
• Teal Health's Wand device has received FDA approval as the first prescription device for at-home self-collection of vaginal samples for HPV-based cervical cancer screening.
• The pivotal SELF-CERV study published in JAMA Network Open demonstrated 95% positive agreement for high-risk HPV detection and 96% sensitivity for precancerous lesions, matching clinician-collected samples.
• With an estimated 21 million women under- or unscreened for cervical cancer in the US, the Teal Wand aims to increase screening rates by offering a preferred at-home alternative without compromising clinical accuracy.
36-Month Results of OpRegen Cell Therapy for Geographic Atrophy to Be Presented at CTS 2025
• Long-term 36-month data from the Phase 1/2a trial of RG6501 (OpRegen) for geographic atrophy secondary to AMD will be presented at Clinical Trials at the Summit in June 2025.
• OpRegen, an allogeneic retinal pigment epithelial cell therapy developed by Lineage Cell Therapeutics in collaboration with Roche and Genentech, aims to counteract RPE cell loss in geographic atrophy.
• The therapy is currently advancing in a separate Phase 2a clinical study, potentially offering a novel treatment approach for geographic atrophy, a leading cause of adult blindness affecting over 5 million people globally.
Leap Therapeutics Halves Workforce and Refocuses Cancer Drug Development Amid Market Challenges
• Leap Therapeutics has announced a significant restructuring, reducing its workforce by approximately 50% and narrowing the development focus of its lead cancer drug candidate in response to challenging market conditions.
• The strategic pivot aims to extend the company's cash runway while concentrating resources on the most promising clinical applications of its lead oncology asset, potentially improving its chances for regulatory success.
• This move follows similar restructuring trends across the biotech sector, with companies like Arcturus, NGM Bio, and Erasca all recently announcing staff reductions and pipeline reprioritizations to navigate the difficult funding environment.
Prime Medicine Advances Gene Editing Pipeline with Key Programs for CGD, Wilson's Disease, and AATD
• Prime Medicine expects to release initial clinical data from its Phase 1/2 trial of PM359 for p47phox chronic granulomatous disease in 2025, potentially demonstrating the curative potential of its Prime Editing technology.
• The company recently unveiled its alpha-1 antitrypsin deficiency (AATD) program, which has shown promising preclinical results with high editing efficiency and full restoration of wild-type AAT protein to normal human range.
• Prime Medicine is advancing its Wilson's Disease program (PM577) through IND-enabling studies, with regulatory filings planned for first half of 2026, as part of its expanding liver disease franchise.
Novartis Announces $23 Billion Investment to Expand US Manufacturing and R&D Footprint
• Novartis plans to invest $23 billion over five years to establish seven new facilities and expand three existing ones across the United States, enabling 100% end-to-end US production of key medicines.
• The expansion includes a $1.1 billion research hub in San Diego and new radioligand therapy manufacturing facilities in Florida and Texas, creating approximately 5,000 jobs directly and indirectly.
• This strategic investment reverses Novartis' recent US pullback and comes amid trade policy uncertainties, positioning the company to maintain its projected sales growth of +5% CAGR through 2029.
Roche Expands Molecular Glue Portfolio with $2 Billion Monte Rosa Partnership
• Roche has signed a $2 billion partnership with Monte Rosa Therapeutics, including a $50 million upfront payment, to develop molecular glue therapies targeting previously "undruggable" proteins in cancer and neurological diseases.
• This marks Roche's second major molecular glue deal in a month, following a similar $2 billion alliance with Orionis Biosciences, demonstrating the company's strategic push to establish leadership in this emerging therapeutic category.
• Monte Rosa will lead discovery and preclinical activities using its QuEEN platform, with Roche taking over late-stage development of candidates that can potentially address the 80% of human proteins currently inaccessible to traditional drug development approaches.
Roche and Zealand Pharma Forge $5.3 Billion Partnership to Develop Novel Obesity Treatments
• Roche has entered into a $5.3 billion collaboration with Zealand Pharma to co-develop petrelintide, a promising amylin analog for obesity treatment, both as monotherapy and in combination with Roche's incretin asset CT-388.
• The partnership includes upfront payments of $1.65 billion to Zealand Pharma, with profits and losses to be shared 50/50 in the US and Europe, while Roche gains exclusive commercialization rights for the rest of the world.
• Clinical data suggests petrelintide could deliver weight loss comparable to GLP-1 receptor agonists but with improved tolerability, potentially addressing unmet needs in the obesity market that is projected to affect 4 billion people globally by 2035.
Spear Bio and Beckman Coulter Receive FDA Breakthrough Device Designations for Alzheimer's Blood Tests
• Spear Bio's pTau 217 blood test received FDA Breakthrough Device Designation, offering a less invasive method for early Alzheimer's diagnosis.
• Beckman Coulter's Access p-Tau217/β-Amyloid 1-42 plasma ratio test also gained FDA Breakthrough Device Designation, aiding in identifying amyloid pathology.
• Both tests address the critical need for accessible, early Alzheimer's diagnosis, potentially improving patient outcomes and treatment access.
• These designations expedite the development and review process, bringing innovative diagnostic tools to market faster for Alzheimer's disease.
Metsera's MET-097i Demonstrates Significant Weight Loss in Phase 2a Trial, Supporting Monthly Dosing Potential
• Metsera's MET-097i, an ultra-long acting GLP-1 receptor agonist, showed substantial placebo-adjusted weight loss of up to 11.3% after 12 weeks in a Phase 2a trial.
• The study indicated a dose-dependent weight reduction with individual responses reaching ~20% in the 1.2mg dose cohort, and no weight loss plateau was observed.
• MET-097i demonstrated a favorable tolerability profile, with mostly mild and transient gastrointestinal adverse events, particularly in the dose-escalated cohort.
• Data support the feasibility of monthly dosing, with a four-fold increase in drug exposure over 12 weeks and well-tolerated step-up doses, paving the way for further studies.
Sarepta Seeks Accelerated Approval for DMD Gene Therapy SRP-9001
• Sarepta Therapeutics has submitted SRP-9001 (delandistrogene moxeparvovec) to the FDA for accelerated approval to treat ambulatory Duchenne muscular dystrophy (DMD) patients.
• The filing is based on positive data from early-stage studies, showing improvements in clinical function and a consistent safety profile, while awaiting Phase 3 EMBARK results.
• SRP-9001, a one-time gene therapy, delivers a shortened dystrophin gene via an AAV vector, addressing the underlying genetic defect in DMD patients.
• If approved, SRP-9001 would offer a one-time treatment option for DMD, contrasting with Sarepta's existing chronic exon-skipping therapies.
FDA Approves Roche's Companion Diagnostic for HER2-Ultralow Metastatic Breast Cancer
• The FDA has approved Roche's PATHWAY HER2 (4B5) test to identify HER2-ultralow metastatic breast cancer patients for targeted treatment.
• This approval expands treatment options for approximately 20-25% of HR-positive, HER2-negative breast cancer patients with HER2-ultralow status.
• The DESTINY-Breast06 trial demonstrated that ENHERTU improved median progression-free survival compared to chemotherapy in HER2-low and HER2-ultralow patients.
• Roche's diagnostic test standardizes HER2 assessment, reduces errors, and helps clinicians make informed treatment decisions for improved patient outcomes.
BioNTech's Bispecific Antibody BNT-327 Shows Promise in Triple-Negative Breast Cancer
• BioNTech's BNT-327, a bispecific antibody targeting PD-L1 and VEGF, has demonstrated positive early results in patients with triple-negative breast cancer.
• The bispecific antibody builds on the success of checkpoint inhibitors like Keytruda, potentially representing the next generation of immunotherapy drugs.
• Early trial data, presented at the San Antonio Breast Cancer Symposium, suggest BNT-327 could become a critical component in treating triple-negative breast cancer.
• The development of BNT-327 aligns with a broader interest in PD1/PD-L1 and anti-VEGF bispecifics, following promising results from Summit Therapeutics in lung cancer.
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