INZ-701

BioMarin’s enzyme replacement therapy for a rare genetic disorder called ENPP1 deficiency delivered mixed results in a late-stage study. Patients with the condition don’t produce enough of the ENPP1 enzyme, which generates plasma inorganic pyrophosphate (PPi). That molecule regulates bone formation and prevents calcification of blood vessels and soft tissue. BioMarin’s therapy, called BMN 401, showed it can offer a statistically significant rise in plasma PPi compared with conventional therapy after a year’s treatment. But the increase in PPi did not translate to clinical improvements. BMN 401 did not succeed on the Phase 3 trial’s second co-primary endpoint, in which pediatric radiologists tracked structural bone changes over time using what’s called the Radiographic Global Impression of Change (RGI-C) score. There were “no positive trends” in the trial’s secondary endpoints, BioMarin said. These measures included the Rickets Severity Score and a measure evaluating patients’ body length and weight. “We are actively evaluating these data to determine the appropriate next steps,” BioMarin’s chief R&D officer Greg Friberg said in a Monday press release. The data are a setback to BioMarin, which took on the therapy when it bought Inozyme Pharma for about $270 million last year. At that time, BioMarin’s business chief James Sabry described the purchase as a “perfect fit” with the company’s background in enzyme therapies. In a Monday note, Leerink analysts said they “have questions about [the therapy’s] regulatory path forward.” TD Cowen analysts agreed, noting that the “FDA wanted to see at least trends” in the clinical endpoints. Nonetheless, they wrote that the results are “a minor disappointment” considering the therapy was only forecast to reach $170 million in revenue in 2030. BioMarin’s stock $BMRN dipped 4% at market close Monday. The Phase 3 trial, named ENERGY 3 , recruited 27 patients aged 1 to 12. More data will be reported at a future medical meeting. BioMarin also has another late-stage study called ENERGY 2 , in patients up to the age of 1. This study will also measure PPi plasma changes but with overall survival as another primary endpoint. While ENERGY 3 has a focus on bone issues, ENERGY 2 is recruiting patients with issues like vascular calcification. The 12-patient trial has a primary completion date in 2028.

TrumpRx welcomes 600 generic medicines BioMarin's $270M Inozyme bet faltersChinese firms Full-Life, Accro raise fresh capitalHansa out-licenses Idefirix in key ex-US marketsTrumpRx welcomes 600 generic medicines More than 600 generic products were added to the Trump administration's direct-to-consumer drug purchasing portal on Monday. The TrumpRx site initially launched in February with cash-pay coupons for about 40 brand-name drugs, many of which have cheaper generic alternatives.Now, the government-run website appears to segment branded products under a section labelled "Presidential deals," while the generic drugs — including cholesterol treatment atorvastatin, the blood thinner clopidogrel, blood pressure medication lisinopril and diabetes drug metformin — are listed under a "Standard prices" tab. By clicking on a generic product, patients can compare out-of-pocket costs at their local pharmacy with delivery options from private pharmacy companies like Mark Cuban's Cost Plus Drugs, Amazon Pharmacy and GoodRx. -Elizabeth EatonBioMarin's $270M Inozyme bet faltersA pivotal Phase III study of BioMarin Pharmaceutical's enzyme replacement therapy BMN 401 in children aged 1-12 with the rare genetic condition ENPP1 deficiency met only one of two co-primary endpoints. "We are actively evaluating these data to determine the appropriate next steps," said Greg Friberg, the firm's chief R&D officer.The ENERGY 3 trial showed that treatment with BMN 401 resulted in statistically significant increases in plasma inorganic pyrophosphate through week 52 compared to conventional therapy. However, the other main goal of improvement in Radiographic Global Impression of Change scores was missed, while there were no positive trends for secondary endpoints, including Rickets Severity Score and growth Z-score.BioMarin gained BMN 401 via last year's $270-million purchase of Inozyme Pharma, with the therapy at the centre of the deal.-Matt DennisChinese firms Full-Life, Accro raise fresh capitalTwo Chinese companies, radiotherapeutics firm Full-Life Technologies and clinical-stage biotech Accro Bioscience, respectively closed a $110-million series D and a $50-million series C.Full-Life will deploy the fresh funds to advance [225Ac]-FL-020, a radioligand therapy for prostate cancer, and [225Ac]-FL-261, a candidate targeting multiple solid tumours. The proceeds will also support 225Ac manufacturing at the company's Belgian facility to secure isotope supply ahead of clinical trials.The latest financing — led by Vivo Capital and joined by SK Biopharmaceuticals, Chengwei Capital, HSG, Junson Capital, Yunion, Plaisance, Sky9 Capital and TSG Capital — brings Full-Life's total funding since its 2021 inception to nearly $350 million. Meanwhile, Accro Bioscience's fundraise will help fund a Phase IIb trial of AC-101, a selective RIPK2 inhibitor targeting the NOD signalling pathway in moderate-to-severe ulcerative colitis. AC-101 previously cleared a Phase Ib/IIa proof-of-concept trial in Chinese patients. Accro's C round was fronted by OrbiMed, with participation from TCG Crossover, LAV, Cenova Capital, and existing backers Shenzhen Capital Group and Oriza Holdings.-Pavan KamatHansa out-licenses Idefirix in key ex-US marketsHansa Biopharma granted SERB Pharmaceuticals an exclusive licence to develop and commercialise Idefirix (imlifidase) across the EU, UK, Switzerland, Norway, Liechtenstein, Iceland, and Middle East and North Africa (MENA) regions in a deal worth up to €115 million ($134 million).Idefirix is conditionally approved in the EU for highly sensitised kidney transplant recipients; in the US, where it's supported by a separate pivotal trial, the desensitisation therapy is currently under FDA review with a target action date of December 19.Hansa will receive €110 million upfront and a further €5 million upon EMA acceptance of the full approval filing for Idefirix. SERB will take charge of an ongoing paediatric trial and long-term follow-up studies.-Pavan Kamat