HIV Breakthrough & Gene Editing Deals — This Week in Biotech #57
Gilead’s HIV breakthrough, Lilly’s $1.3B gene editing deal, and FDA reform efforts (June 13–19, 2025).
Hi and welcome to This Week in Biotech by Biotech Blueprint, edition 57.
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THIS WEEK IN BIOTECH VIDEO SUMMARY
THIS WEEK’S KEY TAKEAWAYS 🔑
🧪 Six months, one shot: The FDA approved Yeztugo (lenacapavir), Gilead’s long-acting HIV PrEP injection offering six months of protection. It’s a scientific breakthrough two decades in the making. But at $14k per dose, equitable access might be a challenge.
🧬 Lilly deepens its commitment to gene editing: Eli Lilly is acquiring Verve Therapeutics for up to $1.3B, securing full rights to VERVE-102, a one-time gene editing therapy for high cholesterol. It’s a bold bet on on solving heart disease at the DNA level instead of managing symptoms day-to-day.
⚠️ Sarepta’s gene therapy hard stumble: A second patient death linked to Elevidys raises serious safety questions for Sarepta’s $3.2M Duchenne therapy. Commercial shipments and a key trial were paused, shares plummeted, and the spotlight may now shift to rival capsid platforms, which could benefit as the field re-evaluates safety and delivery.
🏛️ FDA outlines drug approval reforms: Commissioner Makary unveiled plans to modernize drug approvals, including synthetic control arms, disease-specific standards, and generative AI. If realized, the reforms could cut review times to as little as one month. The FDA wants to move faster, collaborate more openly, and stop expecting companies to “mind read” expectations.
💪 Biotech’s weight loss arms race gets leaner: Scholar Rock’s apitegromab helped patients preserve lean muscle mass when paired with Lilly’s tirzepatide. As GLP-1s sweep obesity treatment, the next battle is for quality of weight loss.
🔬 AstraZeneca quietly scores in rare disease: Ultomiris delivered strong survival data in a lethal transplant complication (HSCT-TMA). With no approved treatments and survival rates historically under 20%, AZ may have a first-in-class winner.
MARKET UPDATES 📈
🔹 Short interest in the S&P 500 Healthcare sector (XLV) edged down slightly in May to 1.90%, from 1.95% in April, signaling a modest shift in sentiment. Moderna (MRNA) remained the most shorted stock by far, with short interest rising to 19.2%, driven by declining COVID-19 vaccine sales, a withdrawn combo vaccine, and a major funding setback in bird flu development. In contrast, Medtronic (MDT), Johnson & Johnson (JNJ), and AbbVie (ABBV) held the lowest short interest, each under 1%. Despite the easing shorts, the sector remains down 0.55% YTD, lagging the broader S&P 500.
BIOTECH/PHARMA NEWS 🧬
🔹❗On June 18, the FDA approved Gilead’s Yeztugo (lenacapavir), making it the first and only HIV prevention option in the U.S. that offers six months of protection per injection. Backed by phase 3 PURPOSE 1 and 2 trials showing ≥99.9% of participants remained HIV-negative, Yeztugo represents a major scientific milestone nearly two decades in the making. Gilead’s capsid inhibitor is designed to overcome adherence challenges associated with daily PrEP and could significantly expand access, particularly for underserved populations. However, its $14k per dose price tag and proposed federal cuts to HIV prevention programs may hinder equitable rollout. Whether Yeztugo can fulfill its promise will depend not just on the science, but on access, awareness, and infrastructure.
🔹 FDA Commissioner Marty Makary is signaling a major shift in how the agency approaches drug approvals. Speaking at the BIO International Convention, Makary laid out plans to streamline the FDA’s review process by tailoring requirements to specific diseases, using synthetic control arms where appropriate, and embracing AI tools like the agency’s new generative model “Elsa” to accelerate safety assessments and labeling. He emphasized that collaboration with industry doesn’t compromise scientific rigor, but rather reflects a healthier, faster path to approval. The FDA also launched a new priority voucher program aiming to cut drug review times to just 1–2 months, reinforcing its push to modernize and bring innovative therapies to patients more quickly.
🔹❗Eli Lilly will acquire gene editing company Verve Therapeutics for up to $1.3B, including a $10.50/share cash offer and an additional $3.00/share tied to milestone payments. The deal gives Lilly full rights to VERVE-102, a phase 1b in vivo gene editing therapy targeting PCSK9, aimed at delivering long-term cholesterol reduction with a single dose. The acquisition signals Lilly’s bet on transforming cardiovascular care from chronic drug regimens to one-time genetic interventions. Shares of Verve surged 78% on the news, with other gene editing stocks, including CRISPR, Intellia, Editas, and Beam, also trading higher in a rare bright spot for the sector.
🔹 Supernus Pharmaceuticals announced it will acquire Sage Therapeutics for up to $795M in cash, including $8.50/share upfront and up to $3.50/share in milestone payments tied to sales of Zurzuvae, the first oral treatment approved for postpartum depression. The deal adds a fourth growth product to Supernus’ CNS portfolio and could generate up to $200M in cost synergies. Closing is expected in Q3.
🔹 KalVista’s FDA decision on sebetralstat, an oral on-demand therapy for hereditary angioedema, is delayed past the original June 17 PDUFA date due to internal FDA workload, not drug-related issues. A decision is expected within four weeks. Shares fell ~3%.
🔹❗Sarepta Therapeutics has suspended commercial shipments and paused a key clinical trial of Elevidys, its $3.2M gene therapy for Duchenne muscular dystrophy (DMD), after a second non-ambulatory patient died from acute liver failure. Both fatalities, within months of each other, raise serious safety and regulatory concerns, particularly since Elevidys received accelerated approval based on dystrophin expression, not clinical benefit. Internal FDA dissent had already cast doubt on the approval decision, and this latest death may reignite debate over the agency’s standards for fast-tracking rare disease therapies. While Elevidys remains Sarepta’s flagship asset with over 900 patients treated, the setback threatens its broader adoption in advanced DMD.
Sarepta shares fell over 45% on the news, with at least four banks, including Bank of America, Piper Sandler, and H.C. Wainwright, downgrading the company. The company also suspended its 2025 revenue guidance, intensifying market uncertainty. Some investors are treating this as Sarepta-specific. UniQure, also developing a late-stage AAV-based therapy, saw minimal impact. But the setback reinforces the need for next-gen AAV capsids with improved safety and efficacy, potentially a long-term tailwind for capsid innovation plays like Sangamo.
CLINICAL TRIAL UPDATES 📊
🔹 Scholar Rock reported positive phase 2 results from its EMBRAZE trial, showing that its lead monoclonal antibody candidate, apitegromab, preserved significantly more lean mass when combined with Eli Lilly’s obesity drug, tirzepatide. Over 24 weeks, patients on the combination lost similar total weight to tirzepatide alone, but with a much healthier profile as 85% of weight loss came from fat compared to 70% with tirzepatide alone, and an additional 4.2 lbs of lean mass were preserved. The treatment was well tolerated, with no new safety signals. These results address a major concern with GLP-1 therapies, the loss of muscle mass, and suggest that apitegromab could become a key adjunct to improve the quality of weight loss in obesity treatment.
🔹 AbbVie reported positive phase 3 results in TEMPLE study of atogepant in migraine prevention: fewer patients discontinued treatment due to side effects (12.1% vs. 29.6% with topiramate), and more saw a ≥50% drop in monthly migraines (64.1% vs. 39.3%). Meanwhile, its VERONA trial of venetoclax + azacitidine in higher-risk myelodysplastic syndromes missed its survival endpoint, though no new safety issues emerged. AbbVie continues to pursue additional indications in partnership with Roche.
🔹 GRAIL announced positive top-line results from its phase 3 PATHFINDER 2 study evaluating Galleri multi-cancer early detection test in over 25k asymptomatic adults aged 50+. The study showed significantly improved cancer detection and positive predictive value (PPV) compared to the earlier PATHFINDER study, while maintaining high specificity (99.5%) and accuracy in predicting the cancer’s origin. These data are supportive of the use of Galleri alongside standard-of-care tests and will be submitted to the FDA as part of GRAIL’s ongoing premarket approval application. Detailed data will be presented later this year.
🔹 AstraZeneca’s Ultomiris (ravulizumab) delivered strong phase 3 results in pediatric patients with HSCT-associated thrombotic microangiopathy (HSCT-TMA), a rare and life-threatening post-transplant complication with no approved treatments. In the ALXN1210-TMA-314 trial, Ultomiris showed an 87.2% overall survival rate at 26 weeks, far above historical rates as low as 18%. While only 17.1% of patients met the full composite response criteria, 71% achieved at least one marker of clinical response. The safety profile was consistent with previous uses. These results position Ultomiris as a potential first-in-class therapy for HSCT-TMA.
🔹 Beam Therapeutics shared updated phase 1/2 data for BEAM-101: all 17 patients hit key targets, with no vaso-occlusive crises reported post-treatment. Patients experienced rapid engraftment, improved red blood cell health, and resolution of anemia. Safety remained in line with standard conditioning. With enrollment now complete, Beam expects to dose 30 patients by mid-year, strengthening the case for base editing in severe sickle cell disease.
PUBLIC HEALTH SPOTLIGHT 🦠
🔹 At its first meeting on June 25–26, the CDC’s newly overhauled vaccine advisory panel will vote on whether to recommend flu shots that contain thimerosal, a mercury-based preservative still used in multi-dose vials. While thimerosal has been deemed safe at low doses and removed from childhood vaccines, it remains in some adult flu vaccines. This vote will test the direction of the panel under Health Secretary RFK Jr., who dismissed all 17 previous members and appointed eight replacements, many of whom have expressed skepticism about vaccines or promoted discredited claims, including linking thimerosal to autism. Major manufacturers like GSK, Sanofi, and CSL could be affected by any shift in federal vaccine guidance.
AI SUMMARY 🎙️
Listen to an AI-generated summary of this episode below.
ON THE HORIZON 🔭
🔹 June 2025 FDA PDUFAs:
June 10: Merck’s BLA for clesrovimab. ✅ APPROVED
June 12: UroGen’s UGN-102. ✅ APPROVED
June 17: KalVista’s sebetralstat. ⏳ DELAYED to ~mid-July
June 19: Gilead’s lenacapavir. ✅ APPROVED
June 23: Nuvation Bio’s taletrectinib.
June 28: Unicycive’s oxylanthanum carbonate.
June 30: Verastem’s avutometinib + defactinib combo.
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DISCLOSURE: I have no business relationships with any company mentioned in this article.