JNJ-74765340

💰 MeiraGTx buys back eye gene therapy from J&J for just $25M: The gene therapy, known as bota-vec, failed a Phase 3 study for X-linked retinitis pigmentosa (XLRP) last year. But physicians and patient advocates believed the data showed the gene therapy still had promise, and urged J&J to at least apply to regulators for approval. However, J&J did not do so. The big pharma had bought a suite of therapies including bota-vec from MeiraGTx for $100 million upfront in 2019. Now, MeiraGTx will be taking bota-vec back for $25 million. The London and New York-based biotech “intends to immediately pursue global regulatory filings for approval of bota-vec,” it said Thursday morning. MeiraGTx also announced a $100 million public offering that’s in part meant to fund the launch of bota-vec. — Lei Lei Wu 💵 STORM Therapeutics hauls in $56M Series C : The cancer-focused biotech will put the cash towards an asset called STC-15, a pill that blocks the RNA-modifying enzyme METTL3. The drug recently entered a Phase 2 study in selected sarcomas. Investors in the round included M Ventures, Pfizer Ventures and Taiho Ventures. — Elizabeth Cairns 💰 Adlai Nortye nabs $150M private placement: The biotech is developing a number of immuno-oncology programs and recently started human studies for a pan-RAS inhibitor. The RAS area is hot after Revolution Medicines’ Phase 3 pancreatic cancer trial succeeded earlier this week. — Max Gelman ⌚ FDA delays approval decision for Savara’s lung disease drug : The agency pushed the PDUFA date for molgramostim, Savara’s therapy for the rare condition autoimmune pulmonary alveolar proteinosis, back three months to Nov. 22. Savara recently submitted more information at the FDA’s request, and the FDA needs more time to assess it. — Elizabeth Cairns 🍎 Another twist in Apple Tree Partners’ bankruptcy case: A US bankruptcy judge will allow the VC firm’s bankruptcy case to proceed, rejecting arguments from its main funder Rigmora Holdings that the fund and its general partner were not in financial distress. But the judge also lifted a stay on certain aspects of parallel proceedings in the Cayman Islands, where Rigmora was seeking to wind up the partnership. A Cayman judge will now hear arguments that Rigmora lost confidence in Apple Tree to manage the fund, and about whether the fund “lost its substratum.” It isn’t immediately clear what impact the decision will eventually have on Apple Tree’s portfolio companies, many of which are in dire shape. But for now, both sides are claiming the decision as a victory. — Nicole DeFeudis 🤝 Aligos Therapeutics secures China license for hepatitis B treatment: Aligos will get $25 million upfront from Xiamen Amoytop Biotech, in exchange for a license to the chronic hepatitis B therapy pevifoscorvir sodium. Amoytop’s license includes China, Taiwan, Hong Kong and Macau. Aligos is eligible for another $420 million in milestone payments. Interim Phase 2 data are expected in the second half of this year, with topline results likely in 2027. — Max Gelman Longevity biotech Nula debuts with up to $30 million in funding: That includes a previously announced award of up to $20 million from ARPA-H. The biotech, which raised another $10 million from Apollo Health Ventures, is developing small molecule drugs targeted at the nuclear envelope and will first be focusing on metabolic dysfunction. The startup is led by Christopher Shepard, who is also a partner at Apollo. — Lei Lei Wu

MeiraGTx will get back full global rights to its experimental gene therapy for a rare inherited blindness disorder from Johnson & Johnson, marking a reversal of a multi-year collaboration that had placed the late-stage programme under the latter's control. Shares in MeiraGTx were down about 19% on Thursday.The deal relates to botaretigene sparoparvovec (bota-vec; AAV-RPGR), a gene therapy for X-linked retinitis pigmentosa (XLRP). The disorder, which affects mostly males, is driven primarily by mutations in the RPGR and RP2 genes that cause progressive vision loss and often leads to total blindness in early adulthood.Under the asset purchase agreement announced Thursday, MeiraGTx will pay J&J $25 million in cash upfront, alongside a future one-time regulatory and commercial milestone tied to US approval and sales performance, plus a high double-digit royalty on global net sales starting in mid-2029.The transaction follows a longer partnership history between the companies. J&J initially entered the programme through a 2019 collaboration with MeiraGTx, paying $100 million upfront to develop gene therapies for inherited retinal diseases. J&J took full control of bota-vec in late 2023 through a deal valued at about $130 million in upfront and near-term milestones, with potentially another $285 million tied to first commercial sales of the gene therapy in the US and EU.  MeiraGTx said it intends to move quickly toward global regulatory submissions, supported by late-stage data from the Phase III LUMEOS trial. The study failed to meet its primary endpoint of vision-guided mobility assessment (VMA) at 52 weeks, but MeiraGTx said the VMA data were "directionally supportive with treated subjects 2.4x more likely to respond than untreated subjects." The company also touted improvements across multiple secondary measures, including low-luminance visual acuity and retinal sensitivity.Regulatory readiness"We are intimately familiar with AAV-RPGR, having collaborated with J&J during the development of the programme from Phase I onward," said CEO Alexandria Forbes. "Importantly, from a regulatory CMC (chemistry, manufacturing and controls) perspective, as the commercial manufacturer of the product, we have completed PPQ (process performance qualification) with the CMC datasets for filing with global regulators," with plans to submit in the US, Europe and Japan "as soon as possible."Separately, MeiraGTx priced a public offering of about $100 million, selling roughly 11.1 million ordinary shares at $9 each. It said proceeds, together with existing cash, are expected to fund operating and capital needs – including potential commercial launches of both bota-vec and the company's radiation-induced xerostomia candidate AAV-hAQP1 – into the second half of 2028.Data from the pivotal AQUAx 2 study of AAV-hAQP1 to treat grade 2/3 radiation-induced xerostomia are expected in the second quarter of 2027.