This Week in Biotech #55
Catch up on the latest biotech breakthroughs and upcoming trends (May 30 - June 5, 2025).
Hi and welcome to This Week in Biotech by Biotech Blueprint, edition 55.
🎙️ Biotech Blueprint is now a podcast. You can find us on YouTube, Spotify, and Apple Podcasts.
THIS WEEK’S KEY TAKEAWAYS 🔑
ASCO 2025 delivered clinical progress and strategic oncology dealmaking. AstraZeneca’s camizestrant demonstrated the power of precision medicine, cutting progression risk by 56% in ESR1-mutant breast cancer patients identified through circulating tumor DNA. This was the first trial to successfully use the tumor DNA fragments in patient blood for treatment selection. Summit’s ivonescimab demonstrated a 48% reduction in progression risk for EGFR-mutant lung cancer patients, though the overall survival trend missed statistical significance. More promising were results from Gilead and Merck’s collaboration as their Trodelvy/Keytruda combination became the first TROP-2 antibody-drug conjugate to beat standard care in frontline triple-negative breast cancer, extending progression-free survival by 3.4 months. Amgen’s Imdelltra cut death risk by 40% in small cell lung cancer, validating the DLL3-targeting approach in this difficult to treat population.
ASCO momentum also drove strategic acquisitions. BioNTech secured a $1.5B upfront deal with Bristol Myers for its PD-L1/VEGF bispecific, following strong mesothelioma data. Sanofi’s $9.1B acquisition of Blueprint Medicines locked in Ayvakit’s fast-growing mastocytosis franchise, which hit $479M in 2024 sales.
Beyond oncology, Regeneron advanced its obesity strategy on two fronts. Their muscle-preserving antibody combination retained 80% of lean mass typically lost with semaglutide (GLP-1) alone while enhancing fat reduction. This positions body composition (not just weight loss) as the next competitive frontier in metabolic medicine. The company also licensed a dual GLP-1/GIP agonist from Hansoh for $2B to further expand its metabolic pipeline.
Gene therapy continues delivering technical wins but facing market skepticism. Regenxbio hit 118% of normal microdystrophin levels in Duchenne patients, yet shares dropped 11%, highlighting persistent concerns about commercialization timelines and manufacturing scalability. Sarepta, however, received an FDA Platform Technology designation for its viral vector, rAAVrh74, a milestone that could streamline future gene therapy reviews.
Despite scientific breakthroughs, gene therapies, including gene editing are struggling to deliver commercially. I’ll unpack what’s holding them back in a special podcast episode coming this summer, featuring Hartaj Singh from the Biotech Capital Compass. Stay tuned and thanks for being part of Biotech Blueprint.
AI SUMMARY 🎙️
Listen to an AI-generated summary of this episode below.
MARKET UPDATES 📈
🔹 Biotech stocks have experienced a slight rebound after previous downturns. The SPDR Biotech ETF (XBI) is trading at $82.56, up 3.5% over the past five days, while the iShares Nasdaq Biotechnology ETF (IBB) stands at $125.06, reflecting a 2.2% increase in the same period. Despite these gains, both ETFs remain below their peak levels, indicating ongoing investor caution.
🔹 Omada Health, a virtual-first provider of care for chronic conditions like diabetes and hypertension, priced its IPO at $19 per share, offering 7.9M shares with an option for underwriters to buy an additional 1.185M. The stock will begin trading on Nasdaq under the ticker “OMDA” on June 6, 2025. The IPO is backed by Morgan Stanley, Goldman Sachs, and J.P. Morgan as lead bookrunners. Omada serves over 2,000 customers, including health plans and Fortune 500 employers, artiand leverages behavior change programs and clinical data to improve long-term health outcomes.
BIOTECH/PHARMA NEWS 🧬
🔹 Shares of 23andMe jumped 50% this week after the bankrupt genetic testing company asked to reopen bidding on its assets, following a surprise $305M offer from TTAM Research Institute - a nonprofit led by former 23andMe co-founder Anne Wojcicki. 23andMe had previously accepted an offer from Regeneron Pharmaceuticals for $256M in May. Regeneron has indicated that it is still interest in acquiring the company and is expected to submit a new offer, although they will seek a $10M consolation fee if its bid is ultimately rejected.
🔹 Alvotech and Dr. Reddy’s Laboratories have announced a global partnership to co-develop and commercialize a biosimilar of Merck’s blockbuster cancer immunotherapy, Keytruda (pembrolizumab), which generated $29.5B in global sales in 2024. Under the agreement, both companies will share development and manufacturing responsibilities, with each retaining commercialization rights in most markets. The collaboration leverages Alvotech’s biologics platform and Dr. Reddy’s global reach to accelerate development and expand access to a cost-effective alternative in the high-value immuno-oncology space.
🔹 Cullinan Therapeutics is set to pay $20M upfront to acquire the rights to velinotamig, a BCMAxCD3 bispecific T cell engager, in all territories outside of China. Genrix Bio will retain the rights within China and could earn up to $700M in milestones and royalties. Velinotamig has previously been tested in phase 2 trials for multiple myeloma and has shown potential best-in-class efficacy. Cullinan aims to pair velinotamig with it’s existing CD19xCD3 asset, CLN-978, in a broad range of autoimmune indications, including their current pipeline for lupus.
🔹 The FDA has granted Platform Technology Designation to Sarepta’s rAAVrh74 viral vector, used in its investigational gene therapy SRP-9003 for limb-girdle muscular dystrophy type 2E/R4 (LGMD2E). This marks one of the first designations of its kind and signals confidence in the vector’s reproducibility and applicability across multiple gene therapies. The designation allows Sarepta to streamline future applications by leveraging existing data. SRP-9003 is designed to deliver a full-length beta-sarcoglycan gene to muscle tissue and has shown promise for treating LGMD2E, a progressive and often fatal neuromuscular disease.
🔹 BioNTech jumped 16% after announcing a major partnership with Bristol Myers Squibb to co-develop and commercialize BNT327, a bispecific antibody targeting PD-L1 and VEGF-A. The candidate is in phase 3 for lung and breast cancers and just posted promising phase 2 data in malignant mesothelioma at ASCO 2025, where 16 of 31 patients showed significant tumor shrinkage. The dual-action therapy disrupts key cancer growth and resistance pathways, offering potential advantages over traditional checkpoint inhibitors. BMS is paying $1.5B upfront, with up to $7.6B in milestones, and both companies will share costs and profits equally while retaining rights to explore additional uses.
🔹 Agenus signed a $141M strategic partnership with India’s Zydus Lifesciences to accelerate the development and manufacturing of its immunotherapy combo, botensilimab & balstilimab (BOT/BAL). The deal includes $75M upfront for the transfer of Agenus’ biologics manufacturing sites in California, up to $50M in production-based milestone payments, and a $16M equity investment at $7.50/share. Zydus also gains exclusive rights to commercialize BOT/BAL in India and Sri Lanka. Agenus will remain a manufacturing customer as it prepares for BLA submission and global commercialization.
🔹 Sanofi will acquire Blueprint Medicines for $129 per share in cash, valuing the deal at $9.1B, with up to $400M in milestone payments. Blueprint shares surged nearly 30% on the news. The deal adds Ayvakit, the only approved treatment for advanced and indolent systemic mastocytosis, along with early-stage assets elenestinib and BLU-808. Ayvakit generated $479M in 2024 and $150M in Q1 2025, up 60% year-over-year. The acquisition supports Sanofi’s push into immunology and is expected to close in Q3 2025.
🔹 On May 31, the FDA approved Moderna’s next-generation COVID-19 vaccine, mNEXSPIKE (mRNA-1283), for adults 65+ and individuals aged 12–64 with at least one high risk condition. The vaccine, which targets a portion of the spike protein, offers a more compact 10 μg dose (just one-fifth the size of the original Spikevax) and demonstrated 9.3% higher efficacy overall and 13.5% in older adults in a phase 3 trial involving >11k participants. With a potentially refrigerator-stable formulation, mNEXSPIKE could simplify distribution and is expected to be available for the 2025/2026 respiratory virus season.
CLINICAL TRIAL UPDATES 📊
🔹 Regenxbio reported new positive interim data from its phase 1/2 AFFINITY DUCHENNE trial for RGX-202, its investigational gene therapy for Duchenne muscular dystrophy. At 12 months post-treatment, participants showed clinically meaningful gains, with one 2-year-old achieving microdystrophin expression exceeding 118% of normal. The therapy continues to show a favorable safety profile, with no serious adverse events reported. Regenxbio shares fell 11% on the news. The drop likely reflects investor caution around the small sample size, delayed commercialization timeline, and broader safety concerns in the gene therapy space, particularly following recent adverse events in competing programs.
🔹 AnaptysBio reported positive 6 month results from its phase 2b trial of rosnilimab in rheumatoid arthritis (RA), highlighting strong efficacy and a favorable safety profile. The PD-1+ T cell-targeting antibody demonstrated JAK-like activity, durable responses lasting at least two months off drug, and the potential for extended maintenance dosing. However, shares reversed premarket gains on Wednesday after disappointing phase 2 data for J&J’s similar PD-1-targeting drug, JNJ-67484703, which faced issues in its ulcerative colitis arm.
🔹 Vigil Neuroscience announced disappointing results from its phase 2 IGNITE trial evaluating iluzanebart (VGL101) in patients with ALSP, a rare neurodegenerative disease. While the drug showed a favorable safety and pharmacokinetic profile, it failed to demonstrate meaningful clinical or biomarker improvements. As a result, Vigil is discontinuing its long-term extension study. Notably, iluzanebart was excluded from Sanofi’s $470M acquisition of Vigil, announced in May.
🔹 Amgen’s Imdelltra significantly extended survival in patients with small cell lung cancer who progressed after platinum chemo, reducing the risk of death by 40%. The phase 3 trial also met its progression-free survival endpoint and improved patient-reported symptoms. Safety was manageable, with lower rates of severe adverse events compared to chemo. Results were presented at ASCO 2025 and published in NEJM.
🔹 Immatics (IMTX) shares climbed nearly 12% after updated phase 1b results showed its IMA203 PRAME-targeting cell therapy led to a 56% confirmed response rate in heavily pretreated metastatic melanoma patients. The one-time infusion therapy also demonstrated durable responses and a strong safety profile, with manageable side effects typical of lymphodepletion. These results support Immatics’ ongoing phase 3 SUPRAME trial, aiming for a potential FDA submission in early 2027.
🔹 Regeneron released interim results from its phase 2 COURAGE trial showing that combining semaglutide with trevogrumab, with or without garetosmab, helped preserve up to 80% of the lean muscle mass typically lost with semaglutide alone, while boosting fat loss. The dual combo was generally well-tolerated, though the triplet regimen led to more side effects and two deaths, with no clear link to the treatment. In a separate move, Regeneron licensed global rights (ex-China) to Hansoh’s dual GLP-1/GIP agonist HS-20094, now in phase 3, expanding its obesity pipeline alongside muscle-sparing strategies.
🔹 Kymera Therapeutics announced strong phase 1 data for KT-621, its first-in-class oral STAT6 degrader. The once-daily drug achieved >90% STAT6 degradation in blood at doses as low as 1.5 mg and complete degradation in both blood and skin at doses ≥50 mg. Biomarker reductions (TARC down 37%, Eotaxin-3 down 63%) rival or exceed those reported for dupilumab, despite being tested in healthy volunteers. KT-621 was well-tolerated, with a safety profile indistinguishable from placebo. A phase 1b trial in atopic dermatitis is underway, with two phase 2b trials in AD and asthma slated for late 2025 and early 2026.
🔹 Gilead’s Trodelvy combined with Merck’s Keytruda has become the first TROP-2 antibody-drug conjugate to show superiority over standard of care in a pivotal phase 3 trial for first-line treatment of PD-L1+ metastatic triple-negative breast cancer (mTNBC). The combination reduced the risk of disease progression or death by 35% compared to Keytruda plus chemotherapy, extending median progression-free survival to 11.2 months vs. 7.8 months. Importantly, the safety profile was manageable and did not worsen with the dual regimen.
🔹 Summit Therapeutics announced that ivonescimab, a PD-1/VEGF bispecific antibody, met its primary endpoint in the phase 3 HARMONi trial, reducing the risk of progression or death by 48% in EGFR-mutant NSCLC patients. The trial showed a hazard ratio of 0.52 for PFS (p<0.00001). A positive trend in overall survival (HR 0.79) was also observed but did not reach statistical significance, causing SMMT stock to fall about 20%.
🔹 In the phase 3 SERENA-6 trial, AstraZeneca’s oral SERD camizestrant, combined with a CDK4/6 inhibitor, reduced the risk of disease progression or death by 56% versus standard aromatase inhibitor therapy in patients with HR-positive, HER2-negative advanced breast cancer and emerging ESR1 mutations. Median progression-free survival was 16.0 vs. 9.2 months, and time to deterioration in quality of life improved by nearly 18 months. As the first trial to use circulating tumor DNA (ctDNA) to guide early treatment switches, SERENA-6 may redefine first-line care. The FDA has granted Breakthrough Therapy Designation for camizestrant.
🔹 In the AERIFY-1 study, Sanofi and Regeneron’s itepekimab met its primary endpoint, reducing moderate or severe exacerbations by 27% at 52 weeks in former smokers with COPD, a statistically and clinically significant outcome. However, the parallel AERIFY-2 trial did not meet its primary endpoint, showing only a 2% reduction. The therapy was generally well tolerated across both studies. The companies are reviewing the data and plan to discuss next steps with regulators. Both stocks dipped on the news.
PUBLIC HEALTH SPOTLIGHT 🦠
🔹 Dr. Lakshmi Panagiotakopoulos, a pediatric infectious disease specialist at the CDC, has resigned from her role as co-leader of the agency’s COVID-19 vaccines working group and is leaving the CDC altogether, Reuters reports. In an email to colleagues, she cited concerns about no longer being able to effectively serve the most vulnerable populations.
🔹 U.S. Health and Human Services Secretary Robert F. Kennedy Jr. announced that Moderna has agreed to conduct a true placebo-controlled trial for its new COVID-19 vaccine. The trial comes amid concerns over the FDA’s limited approval of the mRNA shot for high-risk groups. Kennedy emphasized that the agency will monitor the study closely and collect data on all adverse events.
ON THE HORIZON 🔭
🔹 June 2025 FDA PDUFAs:
June 10: Merck’s BLA for clesrovimab, an investigational, long-acting monoclonal antibody designed to protect infants from RSV during their first season.
June 12: UroGen’s UGN-102, a novel, non-surgical intravesical therapy for low-grade intermediate-risk non-muscle invasive bladder cancer.
June 17: KalVista’s sebetralstat, an oral on-demand treatment for hereditary angioedema (HAE). If approved, it would be the first non-injectable option for managing acute HAE attacks.
June 19: Gilead’s lenacapavir, a twice-yearly injectable for HIV prevention. If approved, it would be the first long-acting PrEP option administered only twice a year.
June 23: Nuvation Bio’s taletrectinib, a next-generation ROS1 inhibitor for advanced ROS1-positive non-small cell lung cancer. If approved, it would offer a potential best-in-class treatment with activity against resistance mutations and brain metastases.
June 28: Unicycive’s oxylanthanum carbonate, a next-generation phosphate binder for hyperphosphatemia in dialysis patients. If approved, it could offer a lower pill burden and more convenient dosing compared to existing treatments.
June 30: Verastem’s avutometinib + defactinib combo in recurrent KRAS-mutant low-grade serous ovarian cancer. If approved, it would be the first FDA-approved treatment specifically for this population.
Have a great rest of your week and thanks for reading Biotech Blueprint!
👩🏻💻 BIOTECH BLUEPRINT CONSULTING
We have a new website: biotechblueprint.com!
We provide tailored consulting solutions designed to meet the unique challenges of both established companies and startups. The services span a wide range of strategic and technical needs.
BOOK A FREE 30-MINUTE CONSULTATION OR A MEET & GREET below.
DISCLAIMER: This content is for informational purposes only. It should not be taken as legal, tax, investment, financial, or other advice. The views expressed here are my own and do not reflect the opinions of any company or institution.
DISCLOSURE: I have no business relationships with any company mentioned in this article.