Viking Falls on Obesity Pill, Rocket’s Gene Therapy Resumes, FDA Greenlights Papzimeos — This Week in Biotech #66
Precigen soars on RRP approval, Rocket rebounds after clinical hold, Viking disappoints on tolerability, and Gilead makes a $350M CAR-T bet with Interius (August 15–21, 2025).
Hi and welcome to This Week in Biotech by Biotech Blueprint, edition 66, covering biotech & pharma news from August 15th to 21st, 2025.
🎙️ Biotech Blueprint brings you weekly video updates on the latest biotech and pharma news, plus in-depth podcast interviews with industry leaders. You can find us on YouTube, Spotify, and Apple Podcasts.
In the latest episode, I teamed up with the Biotech Capital Compass to break down Sarepta Therapeutics’s recent safety crisis, and what the FDA’s partial green light means for Elevidys, investor confidence, and the broader AAV field. We dig into the science, regulatory dynamics, and what might come next.
THIS WEEK IN BIOTECH VIDEO SUMMARY
THIS WEEK’S KEY TAKEAWAYS 🔑
Obesity pill letdown: Viking’s once-daily oral GLP-1/GIP drug met its goals, but the data underwhelmed compared to rivals. A 40% stock plunge reflected doubts about its ability to compete in the current obesity market.
Gene therapy revival: Rocket Pharma’s rare disease trial is back on track after a brief FDA hold. Revised protocols for immune safety cleared the way, and shares surged 30% on the green light.
Precigen’s breakout moment: The FDA approved Papzimeos, the first treatment for recurrent respiratory papillomatosis. The decision, made without a confirmatory trial, signals growing confidence in virus-specific T cell therapies. Shares soared 83%.
Gilead goes in vivo: Kite Pharma will acquire Interius for $350M to gain in vivo CAR-T capabilities. The platform eliminates the need for cell harvesting or chemo, a major move as big pharma races to simplify cell therapy.
FDA nod for Ionis: The agency approved Dawnzera (donidalorsen) for hereditary angioedema. Delivered via autoinjector every 4-8 weeks, it cut attack rates by over 80%, which is a win for Ionis’s solo launch ambitions.
MASH milestone in Europe: Madrigal’s Rezdiffra gets conditional EU approval for MASH with fibrosis. No biopsy required, which is a big shift for streamlined access in a population of 370k+.
Altimmune enters AUD race: Pemvidutide, a GLP-1/glucagon dual agonist, earned FDA Fast Track for alcohol use disorder.
BIOTECH/PHARMA NEWS 🧬
🔹 Gilead’s Kite Pharma will acquire Interius BioTherapeutics for $350M to gain access to its in vivo CAR-T platform, which reprograms immune cells inside the body with no cell harvesting or chemotherapy needed. A lead candidate (INT2104) is in a phase 1 blood cancer trial. The deal positions Gilead alongside AstraZeneca and AbbVie, both of which also made recent moves in the in vivo cell therapy space. Interius will form a new center of excellence under Kite in Philadelphia.
🔹 The FDA approved Ionis’s Dawnzera (donidalorsen) to prevent hereditary angioedema (HAE) attacks in patients 12 and up. Dawnzera, delivered via autoinjector every 4 or 8 weeks, cut monthly attack rates by over 80% in phase 3 trials. Patients switching from other therapies also saw improved control, with 84% preferring Dawnzera. This marks Ionis’s second solo launch in under a year, following TRYNGOLZA. Shares rose 10% on the news.
🔹 Madrigal Pharmaceuticals received conditional European Commission approval for Rezdiffra (resmetirom) as the first authorized treatment for MASH (metabolic dysfunction-associated steatohepatitis) with moderate to advanced fibrosis. The once-daily oral drug earned the green light based on the phase 3 MAESTRO-NASH trial, which showed fibrosis reduction, MASH resolution, and improvements in noninvasive biomarkers like liver stiffness and fat. Rezdiffra is already included in European treatment guidelines and launches in Germany later this year. The approval notably does not require a liver biopsy, streamlining access for the estimated 370,000 eligible patients across Europe.
🔹 FDA lifts clinical hold on Rocket Pharma’s gene therapy. Shares of Rocket Pharma surged over 30% after the FDA lifted a clinical hold on its pivotal phase 2 trial of RP-A501 for Danon disease. The hold, imposed in May after a patient death linked to immune complications, was resolved in under three months. Rocket will resume dosing at a reduced level with revised immune suppression protocols, dropping C3 inhibitors and adjusting criteria for C5 inhibitor use. RP-A501, an AAV9-based gene therapy delivering a functional LAMP2B gene, aims to improve cardiac biomarkers and structure in this rare, life-threatening condition. The updated trial will treat three patients sequentially under the new safety framework.
🔹 Altimmune’s pemvidutide gets Fast Track designation for alcohol use disorder (AUD), a novel use for a GLP-1/glucagon dual agonist. The once-weekly injectable is now in phase 2, testing its ability to reduce heavy drinking days and improve biomarkers like PEth. With GLP-1s known to reduce cravings and glucagon targeting liver inflammation and fat, pemvidutide could tackle both the behavioral and metabolic drivers of AUD, an area with limited treatment options and high unmet need. Just 2% of the 28M Americans with AUD receive medication today. Altimmune believes pemvidutide’s dual-action mechanism may also benefit patients with comorbidities like fatty liver and obesity, which are common in AUD.
🔹 PTC Therapeutics has received a Complete Response Letter from the FDA, rejecting its New Drug Application for vatiquinone as a treatment for Friedreich’s ataxia in both children and adults. Despite earlier being granted priority review, the agency concluded that PTC did not present sufficient evidence of efficacy and requested an additional well-controlled study to support resubmission. Vatiquinone, a first-in-class 15-lipoxygenase inhibitor, is designed to reduce oxidative stress and support neuronal survival, mechanisms relevant to the mitochondrial dysfunction underlying Friedreich’s ataxia. PTC expressed disappointment in the decision and plans to consult with the FDA to determine next steps.
🔹 Tonix Pharmaceuticals has received FDA approval for Tonmya, the first new fibromyalgia treatment in over a decade. The once daily, non-opioid tablet is taken at bedtime and absorbed under the tongue, targeting the condition’s chronic pain and nonrestorative sleep. Approval followed two phase 3 trials showing significant pain reduction versus placebo. Side effects were mostly mild, including mouth numbness and drowsiness. Tonmya’s formulation bypasses the liver, which may support better long term safety. Fibromyalgia affects more than 10M Americans, mostly women, and patients have long called for better options. Despite the approval, Tonix shares fell 20% Monday, as analysts flagged safety concerns and the likely need for additional fundraising to support a commercial launch.
🔹 The FDA has granted approval to Precigen’s Papzimeos, the first and only treatment that targets the root cause of recurrent respiratory papillomatosis (RRP), debilitating HPV-driven disease. RRP, caused by chronic HPV 6/11 infection, often requires repeated surgeries. Papzimeos is a non-replicating adenoviral vector immunotherapy designed to generate HPV-specific T cell responses. In a pivotal study, over half of patients required no surgeries in the 12 months following treatment. The approval bypassed the need for a confirmatory trial, a rare move highlighting FDA’s confidence in the data. Shares of Precigen soared 83% following the news, with analysts projecting peak sales could hit $1.1B by 2033. With ~27k adults affected in the U.S. alone, this therapy has the potential to significantly reduce surgical burden and improve quality of life.
CLINICAL TRIAL UPDATES 📊
🔹 AbbVie announced positive topline results from the second pivotal phase 3 study of its UP-AA clinical program, showing that once-daily Rinvoq (upadacitinib) led to significant scalp hair regrowth in patients with severe alopecia areata. At 24 weeks, 45.2% (15 mg) and 55.0% (30 mg) of patients achieved ≥80% scalp coverage, versus 1.5% in placebo. Key secondary endpoints were also met, including ≥90% and complete (100%) scalp hair regrowth, along with improvements in eyebrows and eyelashes. The safety profile was consistent with Rinvoq’s approved uses and showed no new safety signals. These results mirror findings from the first pivotal study, strengthening the case for Rinvoq as a potential treatment for this chronic autoimmune condition. Regulatory submissions are planned.
🔹 Dynavax reported promising topline results from part 1 of its phase 1/2 trial for Z-1018, a next gen shingles vaccine aiming to rival GSK’s Shingrix. The candidate matched Shingrix on immune response, achieving a 100% humoral response rate at the selected dose, while showing significantly better tolerability, with far fewer grade 2/3 local and systemic side effects. Z-1018 combines Dynavax’s proprietary CpG 1018 adjuvant with glycoprotein E and is now advancing to part 2 of the trial in adults 70+, set to begin in the second half of 2025. With the global shingles vaccine market dominated by a single product, Z-1018’s early success positions Dynavax as a potential disruptor in a multi billion dollar space.
🔹 Viking Therapeutics tumbled over 40% after reporting topline results from its phase 2 VENTURE trial of VK2735, a once-daily oral GLP-1/GIP dual agonist for obesity. The drug achieved statistically significant weight loss of up to 12.2% over 13 weeks and met all primary and secondary endpoints. However, 20% of patients on VK2735 discontinued treatment, compared to 13% on placebo, raising concerns about tolerability. Analysts noted the results fell short of expectations when stacked against Eli Lilly’s orforglipron (~11% placebo-adjusted weight loss over 72 weeks) and Novo Nordisk’s oral Wegovy (~14% over 64 weeks). While some doubt VK2735’s ability to compete with these frontrunners, others see it as one of the few viable oral contenders in a massive, fast growing market.
PUBLIC HEALTH SPOTLIGHT
🔹 The American Academy of Pediatrics (AAP) now recommends Covid-19 vaccination for all children aged 6 to 23 months, emphasizing their heightened risk of severe illness and lack of prior exposure to the virus. For older children and teens, a single COVID-19 dose is advised if they are high risk, previously unvaccinated, or if their families request it. These updated recommendations, outlined in a 2025 policy statement in Pediatrics, highlight the ongoing burden of COVID-19 in children, including risks of hospitalization, post-acute sequelae, and MIS-C. The AAP’s guidance departs from current CDC policy, shaped by HHS Secretary RFK Jr., which leaves pediatric Covid vaccination to shared decision-making between families and doctors. HHS responded with sharp criticism, accusing the AAP of politicizing immunization. The AAP defended its stance, citing the importance of offering clear, evidence-based guidance amid rising vaccine misinformation and ongoing viral threats.
🔹 The U.S. Department of Health and Human Services has reinstated a federal panel focused on childhood vaccine safety that had been inactive for over two decades. Originally created in 1986 and disbanded in 1998, the Task Force on Safer Childhood Vaccines will now be led by NIH Director Jay Bhattacharya and include senior officials from the NIH, FDA, and CDC. Its mission is to reduce adverse reactions, improve oversight, and support safer vaccine development. The move aligns with HHS Secretary RFK Jr.’s broader push to scrutinize the current vaccination schedule, following pressure from his former group, Children’s Health Defense. Some see this as a step toward better safety monitoring, while others fear it may erode trust by re-litigating settled science. The first report is due in two years.
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