KalVista’s FDA Win, Merck’s $10B Deal, and CRISPR Momentum — This Week in Biotech #60
FDA nods, big M&A, and major trial wins (July 3–10, 2025).
Hi and welcome to This Week in Biotech by Biotech Blueprint, edition 60, covering biotech & pharma news from July 3rd to July 10th, 2025.
🎙️ Biotech Blueprint is now a podcast. You can find us on YouTube, Spotify, and Apple Podcasts.
THIS WEEK IN BIOTECH VIDEO SUMMARY
THIS WEEK’S KEY TAKEAWAYS 🔑
Merck is acquiring Verona Pharma for $10B to add Ohtuvayre, a first-in-class COPD drug, to its portfolio. AbbVie, meanwhile, licensed a trispecific T cell engager from Ichnos for multiple myeloma in a deal that could total nearly $2B. Both moves highlight continued pipeline investments as companies prepare for major patent expirations.
Vertex shared six year follow up data for Casgevy in sickle cell disease and beta thalassemia, reinforcing CRISPR’s durability and long-term safety. In chronic kidney disease, ProKidney’s autologous cell therapy showed a 78% improvement in eGFR decline, leading to a 600% spike in its share price this week.
KalVista secured FDA approval for Ekterly, the first oral on-demand therapy for hereditary angioedema. Moderna’s Spikevax was granted full approval for children 6 months to 11 years at risk for severe Covid. The update comes as RFK Jr. rolls back federal Covid vaccine recommendations for children and pregnant women.
Tonix advanced two programs: TNX-102 SL, with a PDUFA date next month for fibromyalgia, and TNX-801, a single-dose smallpox/mpox vaccine candidate. Rhythm, Apogee, and Cogent also posted strong trial results in obesity, atopic dermatitis, and systemic mastocytosis, respectively.
The U.S. is experiencing its worst measles outbreak since 1992, driven by declining vaccination rates. Medical associations have filed suit against RFK Jr. over the rollback of federal Covid vaccine recommendations, citing risks to public trust and immunization coverage.
BIOTECH/PHARMA NEWS 🧬
🔹 AbbVie has secured exclusive global rights to ISB 2001, a first-in-class trispecific T-cell engager targeting BCMA, CD38, and CD3, currently in phase 1 for relapsed/refractory multiple myeloma, via a $700M upfront licensing deal with Ichnos Glenmark Innovation. The agreement could generate up to $1.23B in development, regulatory, and commercial milestones, plus double-digit royalties. Early data from 35 patients reported at ASCO showed a 79% overall response rate and 30% complete responses, with a favorable safety profile.
🔹 Moderna’s COVID-19 vaccine, Spikevax, has received full FDA approval for use in children aged 6 months to 11 years who are at increased risk for severe disease, expanding beyond its previous emergency use authorization. This makes Spikevax fully approved across all at-risk age groups, from infants to the elderly. The update comes amid shifting federal guidance under Health Secretary RFK Jr., who removed routine vaccination recommendations for healthy children and pregnant women. Moderna plans to make its updated vaccine available ahead of the 2025/2026 respiratory virus season. Shares rose 2% on the news.
🔹 Merck announced a $10B acquisition of Verona Pharma, adding newly launched Ohtuvayre to its portfolio, a first-in-class inhaled maintenance therapy for COPD and the first new inhaled mechanism in over 20 years. Approved in mid-2024, Ohtuvayre combines bronchodilation and anti-inflammatory effects, with analysts projecting peak annual sales exceeding $3B. The deal bolsters Merck’s cardio-pulmonary pipeline as it prepares for Keytruda’s looming patent cliff and marks the company’s largest M&A move since its 2023 Prometheus buyout. Verona shares jumped 20% on the news.
🔹 KalVista Pharmaceuticals scored an FDA approval for Ekterly (sebetralstat), the first oral on demand treatment for hereditary angioedema (HAE), offering patients a needle-free option to manage sudden, potentially life threatening swelling attacks. The approval follows a brief but controversial delay, reportedly tied to internal tensions at the FDA, including allegations that Martin Makary pushed for a rejection. The agency ultimately greenlit Ekterly based on strong phase 3 data showing rapid symptom relief and a favorable safety profile. KalVista is launching the therapy immediately. The stock is up nearly 40% on a five day basis.
🔹 Vertex shared long-term follow up data for Casgevy at EHA 2025, showing durable efficacy and safety up to 6 years in patients with sickle cell disease and transfusion-dependent beta thalassemia. Nearly all evaluable patients remained free of vaso-occlusive crises or transfusion needs. Improvements in iron overload markers suggest deeper correction, reinforcing confidence in Casgevy’s potential as a lasting, one-time CRISPR cure.
CLINICAL TRIAL UPDATES 📊
🔹 Tonix Pharmaceuticals rose nearly 9% after announcing two significant developments. First, Pain Medicine published positive phase 3 data for TNX-102 SL in fibromyalgia, showing meaningful pain reduction and a favorable safety profile. With a PDUFA date set for Aug. 15, the therapy could become the first centrally acting, non-opioid fibromyalgia treatment approved in over a decade. Second, at the 2025 Vaccine Congress, Tonix unveiled promising preclinical results for TNX-801, a single-dose mpox/smallpox vaccine. The data showed strong durability and comparable efficacy with subcutaneous versus traditional delivery, supporting plans to enter clinical trials.
🔹 Rhythm Pharmaceuticals announced promising phase 2 results for its oral MC4R agonist, bivamelagon, in patients with acquired hypothalamic obesity. The drug achieved statistically significant BMI reductions of 9.3% and 7.7% in the 600mg and 400mg groups, respectively, over 14 weeks, along with meaningful reductions in hunger scores. Rhythm plans to meet with the FDA and EMA to chart a registrational path for phase 3 development. The stock jumped over 35% on the news.
🔹 ProKidney reported compelling phase 2 results for its autologous cell therapy, rilparencel, in patients with chronic kidney disease and diabetes, showing a 78% improvement in annual eGFR decline in the higher-dose group, both statistically and clinically significant. The therapy was well tolerated, with no serious adverse events. The data strengthens confidence in the ongoing phase 3 trial and sets the stage for a key FDA meeting this summer on using eGFR slope as a surrogate endpoint for accelerated approval. ProKidney shares are up more than 600% over the past five days.
🔹 Apogee Therapeutics announced strong phase 2 results from part A of its APEX trial evaluating the anti-IL-13 antibody, APG777, in moderate-to-severe atopic dermatitis (AD). APG777 achieved 71% mean reduction in Eczema Area Severity Index (EASI) score after 16 weeks, compared to 33.8% in the placebo cohort. Part A of the trial supports a potential best in class 3- or 6-month maintenance dosing schedule and informs part B, which is testing higher doses. These result position the drug well to compete with Regeneron and Sanofi’s Dupixent which is the current standard of care. Despite the promising data, Apogee (APGE) shares fell 16%.
🔹 Cogent Biosciences announced positive data from part 2 of its phase 3 SUMMIT trial, in which bezuclastinib met all primary and key secondary endpoints in patients with non-advanced systemic mastocytosis. In patients on bezuclastinib, mean symptom score was reduced by 24.3 points compared to 15.4 points in the placebo group (p=0.0002). Additionally, 87.4% of patients on bezuclastinib achieved >50% reduction in a key disease marker, serum tryptase. Cogent will submit a New Drug Application to the FDA by late 2025. Shares rose 14% on the news.
PUBLIC HEALTH SPOTLIGHT 🦠
🔹 Measles cases have surged to a 30-year high in the U.S., with over 1,200 confirmed across 39 states and a major outbreak in Texas accounting for hundreds of hospitalizations and three deaths. The resurgence follows declining vaccination rates, many falling below herd immunity thresholds, and growing vaccine hesitancy. At the same time, top medical groups have sued Health Secretary RFK Jr. for removing federal COVID vaccine recommendations for children and pregnant women, arguing the rollback endangers public health and undermines scientific guidance.
AI SUMMARY 🎙️
Listen to an AI-generated summary of this episode below.
ON THE HORIZON 🔭
🔹 July 2025 FDA PDUFAs:
July 10: Regeneron’s Lynozyfic ✅ APPROVED
July 22: Replimune Group Inc.’s Vusolimogene oderparepvec for advanced melanoma.
July 23: GSK’s Blenrep for the treatment of relapsed/refractory multiple myeloma.
July 27: Royalty Pharma’s aficamten for the treatment of obstructive hypertrophic cardiomyopathy.
July 28: Apellis Pharmaceuticals’ Pegcetacoplan for kidney disease.
July 29: PTC Therapeutics’ Sepiapterin for phenylketonuria.
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, including AI automation tools.
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.