Sarepta’s $400M Pivot, MiNK’s 500% Surge, and AML Breakthroughs — This Week in Biotech #61
Layoffs, CRLs, and standout trial data from Sarepta, MiNK, SELLAS, DiaMedica, and more (July 11–17, 2025).
Hi and welcome to This Week in Biotech by Biotech Blueprint, edition 61, covering biotech & pharma news from July 11th to July 17th, 2025.
🎙️ Biotech Blueprint is now a podcast. You can find us on YouTube, Spotify, and Apple Podcasts.
In the last episode, I teamed up with the Biotech Capital Compass to explore the current state of gene editing. We dug into CRISPR’s first FDA approval, the regulatory frameworks shaping the field, and why delivery, durability, and manufacturing still stand between promising science and scalable medicine. It’s part 1 of a new series on gene therapies, told from both a scientific and investment perspective.
THIS WEEK IN BIOTECH VIDEO SUMMARY
THIS WEEK’S KEY TAKEAWAYS 🔑
Sarepta led the week with sweeping changes: the company laid off 500 employees and pivoted toward its siRNA platform after safety concerns with its Duchenne gene therapy, Elevidys, triggered an FDA-mandated black box warning. While painful, the restructuring was met with investor optimism, driving the stock up nearly 40%.
On the clinical front, SELLAS reported standout phase 2 data for its CDK9 inhibitor in relapsed AML, with response rates more than double historical expectations and a move into front-line trials now underway. Similarly, DiaMedica impressed with early data in preeclampsia, a space with no approved drugs, showing significant blood pressure reductions without crossing the placenta.
MiNK Therapeutics soared 500% after publishing a striking case study: a full remission in metastatic testicular cancer using its off-the-shelf iNKT cell therapy. While the data is from a single patient, the durability and safety profile suggest meaningful potential for solid tumors.
Not all news was positive. Capricor’s Duchenne therapy was rejected by the FDA despite priority review, sending shares tumbling over 50%. Ultragenyx also received a CRL for its Sanfilippo gene therapy, though the delay was attributed to manufacturing issues, not clinical concerns.
Elsewhere, AstraZeneca’s AL amyloidosis antibody failed its phase 3 primary endpoint but showed signs of efficacy in a subset of patients, setting up a potential niche strategy. And in a surprise twist, 23andMe was acquired by Anne Wojcicki’s nonprofit TTAM Research Institute, edging out Regeneron in a bidding war that reignited debate over the future of consumer genomics.
BIOTECH/PHARMA NEWS 🧬
🔹 Sarepta Therapeutics is making deep cuts, laying off 500 employees (over a third of its workforce) as part of a major restructuring aimed at slashing $400M in annual costs. The company is narrowing its focus to higher-impact programs, particularly its siRNA platform, while stepping back from many gene therapy efforts. Despite the tough news for staff, investors reacted positively: Sarepta’s stock jumped ~40% and continued rising after hours. The company says the cost-saving measures are critical to meet upcoming financial obligations and support long-term growth.
🔹The Alphabet spinout SandboxAQ has entered into a partnership with iOncologi to develop an AI-driven mRNA vaccine for glioblastoma. The joint program aims to bring a lead candidate into the clinic within 18 months. The collaboration combines SandboxAQ’s AI platform, AQBioSim, with iOncologi’s precision immunotherapy expertise to address challenges in treating brain tumors protected by the blood-brain barrier and immune tolerance. The deal expands SandboxAQ’s focus in oncology and advances its goal of accelerating the drug discovery process through simulation-based AI tools. Financial details of the agreement were not disclosed.
🔹 After a last minute bid, 23andMe cofounder Anne Wojcicki’s nonprofit, TTAM Research Institute, has officially completed its $305M acquisition of the genetic testing company. TTAM’s offer topped Regeneron’s $256M bid. The decision to walk away from the Regeneron deal sparked backlash from some investors and stakeholders, who saw the biotech firm as a more strategic fit. As part of TTAM, 23andMe will continue to offer personalized DNA testing and research services. The nonprofit has also pledged to maintain strict privacy policies amid ongoing public concern over the potential misuse of personal genetic data following the company’s prior data breach and financial struggles.
🔹 MiNK Therapeutics surged over 500% after publishing a case study in Nature’s Oncogene, detailing a complete and lasting remission in a patient with metastatic, treatment-resistant testicular cancer who received its off-the-shelf iNKT cell therapy, agenT-797. The patient had previously failed multiple therapies including stem cell transplant and checkpoint inhibitors, yet showed no evidence of disease two years after a single infusion with no signs of serious side effects. While investor enthusiasm has driven the stock up sharply, analysts are tempering expectations as the company’s gastric cancer phase 2 trial continues and MiNK explores next-generation assets like CAR-iNKT therapies.
🔹 Capricor Therapeutics hit a major setback last Friday after the FDA rejected its biologics license application for Deramiocel, a cell therapy aimed at treating heart complications in Duchenne muscular dystrophy. Regulators cited insufficient evidence of efficacy and requested more clinical data. In response, Capricor plans to include results from its ongoing phase 3 HOPE-3 trial and resubmit later this year. While the company expressed surprise at the decision, it remains committed to the program, aiming to resolve regulatory concerns through additional data and a formal meeting with the agency. Shares plunged over 50% on the news.
🔹 Ultragenyx also received a Complete Response Letter from the FDA for its gene therapy UX111, intended to treat Sanfilippo syndrome type A, which is a fatal neurodegenerative disease affecting young children. While the agency acknowledged strong clinical and biomarker data, it flagged manufacturing-related issues uncovered during facility inspections. These concerns are reportedly fixable and unrelated to product quality, but they will delay potential approval into 2026. Ultragenyx plans to work closely with the FDA to resolve the observations and resubmit the application.
CLINICAL TRIAL UPDATES 📊
🔹 DiaMedica Therapeutics posted promising interim results from its phase 2 trial of DM199 for preeclampsia, sending shares modestly higher. The drug showed statistically significant reductions in both systolic and diastolic blood pressure, along with a key improvement in uterine artery blood flow, an encouraging sign for fetal health. Crucially, DM199 did not cross the placenta and was well tolerated, avoiding the safety pitfalls that have plagued other candidates in this space. With no approved treatments for preeclampsia and most interventions relying on early delivery, DM199 could represent a major step forward. The trial will now advance to expanded dosing cohorts and initiate a study arm focused on fetal growth restriction.
🔹 AstraZeneca reported disappointing results from its Phase 3 CARES study of anselamimab, a light chain–depleting antibody. The drug did not achieve statistical significance for the primary endpoint - a hierarchical combination of time to all-cause mortality (ACM) and frequency of cardiovascular hospitalizations (CVH) - in patients with AL amyloidosis. However, the company noted that a prespecified subgroup of patients experienced a clinically meaningful improvement, suggesting potential benefit in a more targeted population. AstraZeneca plans to continue evaluating the efficacy and safety of anselamimab and may advance development for a narrower AL amyloidosis patient group.
🔹 SELLAS Life Sciences reported strong results from its phase 2 trial of SLS009, a CDK9 inhibitor for relapsed/refractory acute myeloid leukemia (AML), exceeding all efficacy and survival benchmarks. The study showed a 44% response rate in patients with AML with myelodysplasia-related changes and 50% in high-risk molecular subtypes, far above the 20% target. Median overall survival reached nearly 9 months in some cohorts, well beyond the historical 2-4 months seen with current options. The treatment was well tolerated, with no dose-limiting toxicities. Following FDA guidance, SELLAS will move into an AML trial in newly diagnosed patients with enrollment set for early 2026.
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.
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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.
The Sarepta story is pretty fast moving-management has not acted creditably to say the least.