Chiesi Ltd.

Roche devastated the Angelman syndrome community in 2023, when it ended clinical development of rugonersen, a treatment candidate for the rare neurodevelopmental condition. But after losing a potential acquirer for the drug last year, the Swiss pharma giant has found new life for the drug. Oak Hill Bio is picking up rugonersen and aims to dose the first patient in a Phase 3 trial in the first quarter of 2026, the small biotech exclusively told Endpoints News . Oak Hill and Roche declined to disclose terms of the deal. “This is a positive step forward for patients living with Angelman syndrome and their families,” a Roche spokesperson said in an emailed statement. Angelman leads to severe developmental delays, leaving many unable to speak. The rare condition is thought to impact about 500,000 people worldwide, and is the result of a mutation in a gene inherited from a person’s mother. With rugonersen, an antisense oligonucleotide, Roche hoped to boost the paternal version of the gene, called UBE3A, in the central nervous system. But the pharma company stopped development of the treatment in early 2023 because it “did not meet Roche’s internal criteria.” At least 10 other Angelman treatment candidates have been stopped in recent years. Roche continues to develop a different type of drug, a small molecule called alogabat, for the condition. “When we looked at the data,” Oak Hill CEO Josh Distler said in an interview, “we concluded very rapidly that there was really promising data and it was desperately needed.” The Cambridge, MA-based biotech will be at least a few quarters behind Ionis and Ultragenyx in the development of an antisense oligonucleotide for Angelman. Ultragenyx dosed the first patient with its candidate, called GTX-102, in December, and Ionis plans to start its own Phase 3 for its candidate ION582 in May, according to the study entry in the federal trials database. “We benefit from a couple companies that have gone before us. We’ve been able to look at what they’ve done, and there’s some advantages of not being the first mover,” Distler said. The company is working with clinical investigators and patient groups and will share its Phase 3 design later this year, Distler said. Late-stage development may be a tall task for Oak Hill, which has said little about its funding to date. The company emerged in 2022 with rare disease drug candidates from Takeda . The company is backed by a family office, but Distler declined to disclose the investor’s name. Oak Hill is also working off of money it’s receiving as part of a license and development agreement with Chiesi for an experimental drug for complications of extremely premature birth. The partners didn’t disclose terms of that January 2024 deal.

Welcome to Endpoints News’ manufacturing briefs, where we bring you essential news on new builds, collaborations, recalls and more. Chiesi is investing €430 million ($358 million) over the next five years to refurbish an old building in Milan, Italy, to become a factory for making sterile inhalers and other biologics, according to a Wednesday press release. Vaccine manufacturer Bharat Biotech is building a 50,000-square-foot cell and gene therapy site in Genome Valley in Hyderabad, India. The facility will be the country’s first “integrated” CGT site, the company said on Mar. 20. Aspen Biopharma Labs was placed on import alert on Jan. 15 after an inspection of its site in Telangana, India, resulted in an FDA warning letter published Tuesday. The inspection uncovered four issues, including that the factory was in a state of disrepair. Currax Pharmaceuticals has obtained FDA approval for a second manufacturing site for its obesity drug Contrave to meet demand for weight loss drugs, the company said March 20. Redwire Corporation has secured a contract with NASA to use its tech to aid drug development on the International Space Station, according to a Tuesday press release. The tech, dubbed the “Pharmaceutical In-space Laboratory,” will be used in four studies investigating the manufacture of crystals for pharmaceutical production. Emzor Pharmaceutical is planning to finish the construction of its $230 million active pharmaceutical ingredient plant by the fourth quarter of this year, according to reports. Operations are to begin by the end of the year. The factory was originally slated to be completed in the first quarter of 2024.

AstraZeneca and Alteogen's subcutaneous drug delivery deal, Taiho's acquisition of an ADC biotech and WuXi AppTec's 2025 growth projection made our news this week. AstraZeneca has tapped Korea's Alteogen to develop subcutaneous cancer drugs. Taiho Pharmaceutical is paying $400 million to acquire antibody-drug conjugate expert Araris Biotech. WuXi AppTec expects sales to grow 10% to 15% in 2025 as the fate of the BIOSECURE Act remains uncertain. And more.1. AstraZeneca signs Alteogen deal worth up to $1.35B for subcutaneous cancer drugs despite Merck-Halozyme patent dramaAstraZeneca has enlisted Alteogen and its hyaluronidase technology, ALT-B4, to advance subcutaneous formulations of “several oncology assets.” In its filings to the Korea Exchange, Alteogen disclosed two separate agreements together potentially worth up to $1.35 billion. The deal comes as Halozyme is engaged in a patent dispute with Merck & Co. on its subcutaneous formulation of Keytruda, which also uses ALT-B4.2. Otsuka's Taiho pays $400M for Swiss partner to 'turbocharge' 3 ADCs to clinicTaiho Pharmaceutical is paying $400 million upfront and committing up to $740 million in milestones to acquire Swiss antibody-drug conjugate specialist Araris Biotech. In 2023, the Otsuka unit inked a licensing deal to use Araris’ AralinQ platform to develop ADCs. Araris has three preclinical programs expected to enter the clinic over the course of 2025 and 2026.3. WuXi AppTec offers rosy 2025 guidance despite threat of US-China trade tensionsWuXi AppTec expects to grow sales by 10% to 15% in 2025. The rosy projection comes as the fate of the BIOSECURE Act remains uncertain. The company’s revenue slipped about 3% to 39.24 billion Chinese yuan ($5.4 billion) last year. Nevertheless, U.S. sales, which made up the bulk of the CRDMO’s business, still grew nearly 8% despite the draft bill’s threats of forced industry decoupling.4. Curevo rounds up $110M to challenge GSK’s Shringrix, nabs Moncef Slaoui to lead boardCurevo Vaccines, founded and invested in by Korea’s GC Biopharma, has raised $110 million in series B led by new investor Medicxi. OrbiMed, Sanofi Ventures and HBM Healthcare Investments also joined as new backers of the U.S. biotech. The money will be used on a phase 2 extension program for amezosvatein, a potential competitor to GSK’s blockbuster shingles vaccine Shingrix.5. Elevation drops sole clinical-stage ADC over poor phase 1 data, lays off 70% of staffElevation Oncology has decided to ditch its sole clinical asset, a Claudin18.2 antibody-drug conjugate coded EO-3021, which it licensed from China’s CSPC Pharma for $27 million upfront in 2022. Elevation unveiled lackluster tumor response data from a small group of stomach patients last year but pressed on by focusing on a subgroup. Updated data only showed a 22% response rate among those biomarker-enriched patients.6. AstraZeneca offers early look at Datroway-Tagrisso combo in EGFR lung cancer as phase 3 trial rolls onAstraZeneca found that a combination of its Daiichi Sankyo-partnered Datroway and Tagrisso induced a 36% response rate in a small group of patients with EGFR-mutated non-small cell lung cancer (NSCLC) who had progressed on first-line Tagrisso, according to results from the phase 2 Orchard trial. The two companies in October launched a phase 3 trial to test Datroway either alone or together with Tagrisso in post-Tagrisso NSCLC.Other News of Note:7. Dr. Reddy’s, Sun Pharma and Zydus issue US recalls, citing failed specs and labeling issues8. Chiesi bails on TiumBio’s respiratory drug deal (Korea Biomedical Review)9. Klick buys Ward6 Singapore’s operations to fuel Asian expansion10. Japan’s RegCell moves to US with $45.8M to advance Treg platform (release)