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The latest Biotech News and Views |
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| Good morning! After watching her son stand from his wheelchair years into ALS, Mitze Klingenberg joined other patients urging the FDA to approve NurOwn - a stem cell therapy they say has prolonged lives, restored function, and offers hope for ALS patients. Despite results showing a 90% five-year survival rate, the FDA rejected the therapy twice. Patients are now collecting their own data in the hope that the drug will be approved. |
| Enjoy today's read! |
| —Joachim E. |
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| SNAPSHOT |
| Otsuka spends on $613M Swedish autoimmune therapy |
 Photo: Selina (Unsplash) |
| Otsuka Pharmaceutical is paying $33 million upfront and offering up to $580 million in milestones for global rights to Sweden-based Cantargia's IL1RAP-targeting autoimmune antibody program. |
| Why it matters: The deal expands Otsuka's autoimmune R&D pipeline and marks a major investment in next-gen immunology treatments. Cantargia's stock surged over 200% on the news. |
| Backstory: Cantargia's lead candidate, CAN10, is in phase 1 trials for hidradenitis suppurativa (a skin condition also known as Verneuil's disease) and is also being evaluated for systemic sclerosis. Otsuka gains rights not only to CAN10 but also to a similar preclinical "backup antibody," plus exclusive negotiation rights on future antibodies targeting IL1RAP, a key immune modulator in the IL-1 family. |
| Big picture: This move aligns with Otsuka's broader autoimmune push, following recent deals and leveraging platforms from subsidiaries Visterra and Jnana Therapeutics. The IL1RAP platform could unlock treatments across multiple diseases. |
| The trial in detail: Otsuka's recent autoimmune moves include a $600M+ deal for Harbour BioMed's bispecific T-cell engager. |
| Yes, but: Otsuka's decision to prioritise autoimmune diseases may prove to be a judicious one, as other segments of its portfolio are facing challenges. Just ahead of Friday's advisory committee vote, FDA staff raised concerns over conflicting efficacy data for brexpiprazole combined with sertraline in treating PTSD, leaving its approval uncertain. |
| What's next: The drug was acquired as part of the $3.5 billion purchase of Avanir Pharmaceuticals in late 2014. |
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| SNIPPETS |
| What's happening in biotech today? |
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| �� Deal revival: Repare Therapeutics has secured a deal with Debiopharm to advance its paused PKMYT1 inhibitor, lunresertib, with Debiopharm acquiring the rights for $10 million upfront, plus up to $262 million in potential milestone payments and royalties. The agreement builds on an existing collaboration exploring lunresertib in combination with Debiopharm's WEE1 inhibitor, Debio 0123, for treating difficult cancers. Debiopharm will now lead the ongoing study, citing promising early clinical data. Meanwhile, Repare is focusing on two phase 1 trials for its PLK4 inhibitor RP-1664 and Polθ ATPase inhibitor RP-3467, with clinical readouts expected in the second half of 2025. |
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| �� FDA tensions: FDA vaccine chief Vinay Prasad has once again overridden internal reviewers by approving only limited use of Moderna's COVID-19 vaccine Spikevax for children aged 6 months to 11 years who are at increased risk, citing insufficient evidence of benefit for healthy kids. This marks the third time since May that Prasad has narrowed COVID vaccine approvals against staff recommendations, reflecting his longstanding skepticism toward pediatric COVID vaccination and his broader push, alongside FDA Commissioner Martin Makary, for stricter regulatory standards. |
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| ❌ Rare disease flop: AstraZeneca's experimental drug anselamimab failed to meet its primary goals in two Phase 3 trials for late-stage AL amyloidosis, a rare disorder marked by toxic protein deposits that damage organs. The trials showed no significant reduction in overall mortality or cardiovascular hospitalizations in the general patient population. However, the company reported a "highly clinically meaningful improvement" in a prespecified subgroup. The setback comes amid broader challenges in AL amyloidosis drug development and affects AstraZeneca's rare disease strategy following its acquisition of Alexion Pharmaceuticals. |
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| �� Eye relief: Okyo Pharma's urcosimod (OK-101) has shown promising results in a Phase 2 trial for neuropathic corneal pain (NCP), achieving a statistically significant average pain reduction of 5.5 points on a 10-point scale in the 0.05% dosage group. The placebo group saw a 2.75-point reduction, yielding a strong treatment effect size. With no current FDA-approved treatments for NCP, Okyo plans further development and FDA discussions. |
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| SNAP AGAIN |
| Sarepta slashes workforce and scales back Duchenne gene therapy |
 Photo: Tyler Lastovich (Pexels) |
| Sarepta Therapeutics is laying off about 500 employees and halting several research programs after safety concerns and deaths linked to its Duchenne muscular dystrophy gene therapy, Elevidys. |
| Why it matters: The cuts reflect a sharp pivot by Sarepta to stabilize finances and preserve its future, following an 80% stock drop and regulatory challenges. The fate of Elevidys, a first-of-its-kind therapy, is now uncertain, impacting patients and the broader gene therapy field. |
| Backstory: Elevidys was the first gene therapy approved in the U.S. for Duchenne muscular dystrophy in 2023. Despite initial promise, recent patient deaths triggered FDA action and reduced the eligible patient pool. Sarepta halted sales to higher-risk non-ambulatory patients and paused confirmatory trials. The FDA also announced this week that a black box warning over liver failure risks will be added to Elevidys. |
| Big picture: Sarepta's future now depends on navigating FDA scrutiny, maintaining revenue from its other Duchenne drugs, and advancing new RNA-based therapies. The layoffs and research pause are part of a bid to save $400M annually and retain profitability amid growing uncertainty. Elevidys sales dropped from $375M in Q1 to $282M in Q2 and are expected to stabilize at around $500M per year. |
| What's next: The company intends to focus its resources on RNA drugs developed with Arrowhead for rare neurological disorders. |
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| SPEED READ |
| More news |
| The UK has approved Alnylam's Amvuttra for treating both wild-type and hereditary transthyretin amyloid cardiomyopathy by targeting the gene that drives disease progression |
| Ventus Therapeutics is laying off staff as part of a strategic shift to prioritize mid-stage trials, expand its AI-driven drug platform, and advance its immune-targeting pipeline. |
| VC firm Modi Ventures has closed an $88 million second fund to back AI, biology, and medtech startups |
| BriaCell Therapeutics raised $15 million through a public offering to fund operations and advance its cancer immunotherapy pipeline amid ongoing clinical development |
| FDA reviewers have raised doubts about GSK's bid to reapprove Blenrep for multiple myeloma, citing unresolved efficacy concerns, unique eye toxicities, and insufficient dosing optimization. |
| Kezar Life Sciences has regained FDA clearance to resume zetomipzomib trials in autoimmune hepatitis and is considering reviving its lupus nephritis program despite prior safety concerns |
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| Upcoming events |
| ���� Paris, 21-23 August 2025 – 11th International Conference on Bioengineering and Biosciences |
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| ���� Boston, 15-18 September 2025 – Biotech Week Boston |
| ���� Dubai, 23-25 September 2025 – ArabLab |
| ���� Basel, 30 September-2 October 2025 – Festival of Biologics |
| ���� Rome, 23-24 March 2026 – Global Longevity Federation 2026 |
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| Disclaimer: The opinions expressed here are the views of the writer and do not necessarily reflect the views and opinions of News Medical or AZoNetwork. |
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