This Week in Biotech #45
Catch up on the latest biotech breakthroughs and upcoming trends (Mar 21-27).
Welcome or welcome back to This Week in Biotech by Biotech Blueprint, edition 45.
THIS WEEK’S KEY TAKEAWAYS 🔑
🦠 FDA approves GSK’s Blujepa (gepotidacin), first in class oral antibiotic for UTIs, Blujepa is the first new class of oral antibiotics in nearly 30 years. It targets drug-resistant bacteria and showed strong results in two phase 3 trials. U.S. launch is expected in late 2025.
🧬 23andMe files for bankruptcy. After a data breach and declining demand, 23andMe is restructuring, and has court approval to sell its user DNA data, raising privacy red flags. The CA attorney general is urging users to delete their data.
🏛️ RFK Jr. announces sweeping cuts at HHS concerning ~10k jobs, including thousands at the FDA, CDC, and NIH, and shutter half of its regional offices in a massive restructuring to streamline federal health agencies.
🧑⚕️ Marty Makary and Jay Bhattacharya confirmed to lead the FDA & NIH. Both known for their criticism of mainstream medical policies, Makary (FDA) and Bhattacharya (NIH) are now leading the agencies amid health system shakeups under the Trump administration.
🍽️ Soleno’s Vykat XR was approved for Prader-Willi Syndrome. First ever FDA approved therapy for hyperphagia in PWS, a rare genetic disorder marked by extreme hunger. Launch expected in April 2025.
🧪 Regulus reports promising kidney drug results. In ADPKD, farabursen halted kidney volume growth in a phase 1b trial. A phase 3 trial starts in Q3 2025, and the stock surged over 30%.
✂️ Arbutus restructures and cuts 57% of staff. Arbutus cut its workforce down to just 19 employees and exited its HQ to conserve capital and focus on advancing its lead HBV drug, imdusiran.
💊 Tonix Pharma gains on fibromyalgia drug progress. TNX-102 SL moves closer to approval with no advisory committee required by the FDA. If approved, it would be the first new fibromyalgia drug in over 15 years. PDUFA date: Aug. 15, 2025.
🎙️ PODCAST
BIOTECH NEWS
🔹 23andMe has filed for bankruptcy and received court approval to sell customer DNA data, raising serious privacy concerns. The company, once valued at nearly $6 billion, saw a sharp decline due to waning demand and fallout from a 2023 data breach that exposed personal information from 14,000 users. Despite assurances that customer data will remain protected, critics warn that genetic information could end up in the hands of employers, insurers, or pharmaceutical companies. Since HIPAA doesn’t cover direct-to-consumer DNA testing, users have limited protections. The California Attorney General is urging users to delete their genetic data and has issued guidance on how to do so. Though 23andMe states it will not share data with law enforcement without valid legal orders, users remain vulnerable if the company is sold. CEO Anne Wojcicki has resigned, and interim CEO Joe Selsavage is overseeing operations during the sale process.
🔹 Marty Makary has been confirmed as FDA Commissioner, and Jay Bhattacharya as NIH Director, placing two critics of mainstream medical policy in top health roles under the Trump administration. Makary, a Johns Hopkins surgeon and health policy expert, was confirmed in a bipartisan vote and is known for his focus on patient safety and cost transparency. Bhattacharya, confirmed along party lines, is known for opposing COVID-19 lockdowns. Their appointments come amid major federal health agency cutbacks and restructuring efforts.
🔹 U.S. Health Secretary Robert F. Kennedy Jr. announced a sweeping overhaul of the Department of Health and Human Services (HHS), including the elimination of 10k jobs and the closure of half of its regional offices. The restructuring aims to reduce bureaucracy and centralize functions across key agencies like the FDA, CDC, and NIH, which will all see major staffing cuts: 3,500 at the FDA, 2,400 at the CDC, and 1,200 at the NIH. The CMS will be less affected, with just 300 cuts. The total HHS workforce will shrink from 82,000 to 62,000 employees, factoring in voluntary departures.
🔹 The FDA has approved Blujepa (gepotidacin), the first new class of oral antibiotic for uncomplicated urinary tract infections (UTIs) in nearly 30 years, for use in female adults and pediatric patients aged 12 and up. Developed by GSK, Blujepa targets common uncomplicated UTI-causing bacteria and is designed to combat rising antibiotic resistance. Approval was based on positive results from phase 3 EAGLE-2 and EAGLE-3 trials, where Blujepa showed non-inferiority and superiority over nitrofurantoin. The treatment was generally well tolerated, with the most common side effects being mild gastrointestinal issues. U.S. launch is planned for the second half of 2025.
🔹 CureVac announced a key win in its patent dispute with BioNTech, as the European Patent Office (EPO) upheld the validity of CureVac’s patent in amended form. The EPO largely dismissed BioNTech’s opposition, filed in 2023, affirming the strength of CureVac’s claims around its split poly-A tail mRNA technology, which enhances protein expression. This ruling is a major step in ongoing German litigation between the companies, with an infringement hearing set for Jul. 1, 2025. A favorable infringement ruling could lead to damages proceedings. CureVac’s CEO called the decision a milestone recognizing its pioneering role in mRNA technology used in COVID-19 vaccines.
🔹 Arbutus Biopharma announced a significant corporate restructuring in Q1 2025, including a 57% reduction in workforce, cutting its team down to 19 employees, as part of efforts to streamline operations and preserve capital. This move follows a broader review of the company’s HBV programs under newly appointed CEO Lindsay Androski, and aims to support the advancement of its lead candidate, imdusiran, into phase 2b trials. The company also exited its Warminster, PA headquarters, ceased all in-house research, and terminated its ATM offering program. In addition, three senior executives, including the CFO, CMO, and General Counsel, have departed.
🔹 Soleno Therapeutics announced FDA approval of Vykat XR (diazoxide choline extended-release tablets), the first approved treatment for hyperphagia in individuals with Prader-Willi syndrome (PWS) aged 4+. Hyperphagia, the most debilitating aspect of PWS, causes uncontrollable hunger and food-seeking behavior. The approval follows strong results from a phase 3 trial where patients on Vykat XR experienced significantly less worsening of symptoms compared to those switched to placebo. With over four years of safety data, common side effects included hypertrichosis, edema, hyperglycemia, and rash. Soleno will launch Vykat XR in April 2025.
🔹 Humacyte CEO Dr. Laura Niklason addressed a recent New York Times piece raising questions about the FDA’s approval of Symvess, a bioengineered vessel for vascular trauma repair. She emphasized that Symvess underwent a rigorous 13-month BLA review, including additional scrutiny beyond the original PDUFA date. While the FDA considered convening an external Advisory Committee after consultant Dr. Robert Lee expressed concerns, it ultimately opted for further internal review and outside surgical expertise. Niklason clarified that Dr. Lee was not a formal FDA reviewer and had resigned prior to the product’s approval. She also refuted claims that synthetic graft ruptures are unheard of or that shrapnel wounds rarely occur in combat, citing both personal experience and FDA data. Humacyte is conducting a post-approval study to confirm the product’s safety and effectiveness. Niklason noted that, over 12 years and in nine clinical studies with more than 600 patients, Humacyte’s engineered vessels have not prompted any study halts. The company’s stock fell 13% after the NYT article was published.
🔹 Palatin Technologies announced that the FDA granted orphan drug designation for PL7737, an oral melanocortin-4 receptor (MC4R) agonist aimed at treating leptin receptor deficiency, a rare genetic cause of severe, early-onset obesity. Currently, the only approved therapy for this condition involves daily injections, so PL7737 could offer a more convenient option. Palatin expects to file its IND in late 2025 and begin phase 1 studies soon after, with clinical data anticipated in the first half of 2026.
🔹 Trump has nominated Susan Monarez, the acting director of the U.S. Centers for Disease Control and Prevention (CDC), to the permanent position. Monarez, who has a background in microbiology and immunology, has been serving as the CDC’s acting director since Jan. 2025. Before her current role, she worked at the Advanced Research Projects Agency for Health (ARPA-H) and held various positions in federal health agencies, including the Department of Homeland Security and the White House’s Office of Science and Technology Policy. Monarez was chosen following the withdrawal of Trump’s earlier nominee, David Weldon.
🔹 Tonix Pharmaceuticals’ stock is up 45% on a 5-day basis, following the announcement that the FDA will not require an advisory committee meeting for its New Drug Application for TNX-102 SL. If approved, TNX-102 SL would be the first new treatment for fibromyalgia in over 15 years. The FDA has granted fast track designation to the drug, with the PDUFA goal date for approval set for Aug. 15, 2025.
CLINICAL TRIAL UPDATES
🔹 Regulus Therapeutics announced the successful completion of its phase 1b trial of farabursen for autosomal dominant polycystic kidney disease, showing strong biomarker response and a mean halt in kidney volume growth in patients treated with the 300 mg fixed dose. Exploratory analysis also showed statistically significant improvements in kidney volume growth compared to a historical placebo group, supporting the potential of farabursen to slow disease progression. The treatment was well tolerated with a favorable safety profile. Regulus plans to initiate a phase 3 single pivotal trial in Q3 2025. The company’s stock jumped over 30% following the announcement.
🔹 Equillium announced results from its phase 3 EQUATOR study evaluating itolizumab as a first-line treatment for acute graft versus host disease (aGVHD). The trial did not meet its primary endpoint of improving complete response at day 29, however, itolizumab achieved statistically significant and clinically meaningful longer-term outcomes, including complete response at day 99 and improved failure-free survival. The drug also showed a favorable safety profile, with no increase in infections or sepsis, key contributors to aGVHD mortality. Based on these results, Equillium has submitted a request for breakthrough therapy designation and a meeting with the FDA to explore accelerated approval, with feedback expected in May 2025. The company’s stock fell nearly 40% following the announcement.
🔹 Kezar Life Sciences reported topline data from its PORTOLA phase 2a trial evaluating zetomipzomib in autoimmune hepatitis (AIH) patients. The study enrolled 24 patients (2:1 randomization of zetomipzomib vs. placebo) and 31% of zetomipzomib-treated patients achieved a complete biochemical response, compared to 12.5% of placebo patients. Additionally, patients on zetomipzomib showed a durable response with no disease flares, and the safety profile was favorable.
🔹 UNITY Biotechnology announced topline results from its phase 2b ASPIRE study of UBX1325 for diabetic macular edema (DME). The treatment showed vision gains comparable to aflibercept, with UBX1325 being non-inferior to aflibercept at most time points. While the drug met the non-inferiority criterion in most cases, it did not achieve statistical non-inferiority at the average of weeks 20 and 24. As a result, the stock is down about 27%.
PUBLIC HEALTH SPOTLIGHT
🔹 The CDC’s vaccine advisory panel, originally scheduled to meet in February, has been rescheduled for April 15-16. The delay followed the appointment of Robert F. Kennedy Jr. as health secretary, with the official reason cited as allowing more time for public comment. The meeting will cover the ongoing measles outbreak and various vaccine-related topics, though influenza vaccine guidelines have been removed from the agenda. The postponement coincided with the FDA bypassing its usual advisory process for flu shot recommendations. Kennedy’s long-standing skepticism of vaccines, including the MMR vaccine, has raised concerns, especially as the U.S. faces a growing measles outbreak.
ON THE HORIZON
🔹 March 2025 FDA PDUFAs:
Mar. 18: NT-501, developed by Neurotech Pharmaceuticals, is a treatment designed for Macular Telangiectasia Type 2 (MacTel), a rare neurodegenerative eye disease that causes progressive central vision loss. NT-501 uses a cell-based delivery system, known as Encapsulated Cell Therapy, to provide sustained release of ciliary neurotrophic factor, a neuroprotective protein that supports photoreceptor survival and slows retinal degeneration. APPROVED ✅
Mar. 23: Vutrisiran is an investigational RNAi therapeutic being developed by Alnylam for the treatment of ATTR amyloidosis with cardiomyopathy (ATTR-CM), a serious and progressive disease characterized by misfolded transthyretin proteins that accumulate in tissues such as the heart. This therapy works by reducing both mutant and wild-type transthyretin (TTR) proteins, addressing the underlying cause of ATTR amyloidosis. If approved, vutrisiran would be the first therapy available to treat both the polyneuropathy and cardiomyopathy manifestations of this disease. APPROVED ✅
Mar. 26: Gepotidacin is a novel, oral antibiotic developed by GSK that is currently under investigation for the treatment of uncomplicated urinary tract infections (UTIs) and gonorrhea. APPROVED ✅
Mar. 27: Milestone’s Etripamil nasal spray, with a PDUFA date of Mar. 27, is designed to be self-administered as a nasal spray to rapidly treat episodes of paroxysmal supraventricular tachycardia (PSVT), a type of abnormal heart rhythm, allowing patients to manage their symptoms at home without immediate medical intervention; it works by blocking calcium channels in the heart to slow down the rapid heart rate.
In my January 2025 deep dive where I discussed some of the most exciting drugs that could potentially receive FDA approval this year, I covered both gepotidacin and etripamil:
Mar. 28: Sanofi’s Fitusiran is an investigational therapy for hemophilia A or B, used to prevent bleeding in patients, including those with inhibitors. It works by lowering antithrombin to enhance blood clotting, and is administered subcutaneously, potentially reducing treatment frequency to just six doses a year.
🔹 April 2025 FDA PDUFAs:
Apr. 2: Reproxalap is an investigational drug developed by Aldeyra Therapeutics for the treatment of dry eye disease. It functions as a small-molecule modulator of reactive aldehyde species, which are elevated in ocular and systemic inflammatory diseases.
Apr. 3: Cabozantinib, marketed by Exelixis under the brand name Cabometyx, is an oral tyrosine kinase inhibitor approved for treating various cancers, including advanced renal cell carcinoma, hepatocellular carcinoma, and differentiated thyroid cancer.
Apr. 3: Amgen's (Horizon Therapeutics) Inebilizumab (Uplizna) for IgG4-related disease (supplemental BLA for the CD19-targeted mAb’s new use).
Apr. 18: Dupilumab (Dupixent), developed by Regeneron and Sanofi, is an anti-inflammatory biologic already approved for several conditions like atopic dermatitis, asthma, and eosinophilic esophagitis. The FDA is currently reviewing two supplemental applications: one for bullous pemphigoid (PDUFA date: June 20, 2025) and another for chronic spontaneous urticaria (PDUFA date: April 18, 2025), which could further expand its use.
Apr. 26: Telix Pharma’s TLX101-CDx (Pixclara), imaging agent for recurrent glioma (new NDA for F-18 FET diagnostic tracer) awaiting its decision on Apr. 26.
Apr. 29: Elamipretide is an investigational peptide developed by Stealth BioTherapeutics, designed to target mitochondrial dysfunction by binding to cardiolipin in the inner mitochondrial membrane, thereby enhancing mitochondrial function. It is primarily being developed for the treatment of Barth syndrome, an ultra-rare genetic disorder characterized by cardiac abnormalities, muscle weakness, and reduced life expectancy.
Apr. 29: J&J’s Nipocalimab for generalized Myasthenia Gravis (gMG) in adults who are anti-AChR, anti-MuSK or anti-LRP4 antibody-positive (original BLA, anti-FcRn monoclonal antibody). The product has orphan designation and priority review.
Apr. 30: Dihydroergotamine (DHE) is used for the acute treatment of migraine with or without aura in adults. Satsuma Pharmaceuticals is developing STS101, a novel DHE nasal powder formulation designed for fast, easy self-administration and rapid absorption.
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