Neurimmune AG

Dive Brief:AstraZeneca is bolstering its rare disease division with the purchase of Amolyt Pharma, an endocrine disease specialist backed by Novo Holdings.Under the deal announced Thursday, AstraZeneca will pay Amolyt shareholders $800 million in cash up front, with the potential for another $250 million for reaching a specific regulatory milestone. The companies expect the transaction to close in the third quarter.For AstraZeneca, the jewel of the acquisition is eneboparatide, a drug in Phase 3 testing to treat patients with hypoparathyroidism. The disease, which mostly affects women, is caused by a deficiency in parathyroid production that lowers calcium in the blood and can lead to debilitating symptoms including kidney disease. It affects about 115,000 people in the U.S.Dive Insight:The $39 billion acquisition of Alexion three years ago instantly transformedAstraZeneca into a major player in rare diseases. Since then, the U.K. drugmaker has been selectively picking up additional assets.In September 2021, the company exercised an option to buy the rest of rare disease drugmaker Caelum Biosciences for $150 million. A few months later, AstraZeneca struck two deals in transthyretin amyloidosis, paying $30 million to Switzerlands Neurimmunefor rights to an experimental therapy and inking a development pact with Ionis Pharmaceuticals for a drug that won approval in late 2023.AstraZeneca is also using its foothold in rare diseases to make forays into genetic medicine, acquiring gene-editing specialist LogicBio Therapeutics and scooping up Pfizers early gene therapy work for as much as $1 billion. And last year, the company announced plans to work with Quell Therapeutics to develop cell-based treatments for autoimmune diseases.Amolyt has moved from a startup with preclinical assets to a Phase 3 company in less than five years. Its largest investor is Novo Holdings, the controlling shareholder of both Novo Nordisk and Novozymes. Owned by the Novo Nordisk Foundation, Novo Holdings co-led the Series A financing for Amolyt in 2019 and added to its investment with Series B and Series C funding rounds.In addition to eneboparatide, Amolyt has an experimental drug called AZP-3813 designed as a potential treatment for acromegaly, which occurs when the body makes too much growth hormone. '

Biogen has announced that it will be discontinuing the development and commercialisation of its Alzheimer's treatment, Aduhelm (aducanumab-avwa). The intravenous drug was approved by the US Food and Drug Administration (FDA) in June 2021 under the accelerated approval pathway, a decision that faced controversy as many argued that there was insufficient evidence of patient benefit. The phase 4 post-marketing confirmatory ENVISION study, which was a requirement of the FDA’s decision, will now be terminated, Biogen said. The drugmaker outlined that the decision is not related to any safety or efficacy concerns, but that it had “considered the time and investment required for the post-marketing confirmatory ENVISION study and the likely advancements in the field by the time of potential Aduhelm FDA traditional approval". The company said it has recorded a one-time charge of approximately $60m related to closing out costs for the programme in the fourth quarter of 2023 and that it will hand back the Aduhelm rights to Neurimmune, from which it had licensed the drug in 2007. Affecting more than 6.5 million people in the US, Alzheimer’s disease is an irreversible, progressive brain disorder that slowly destroys memory and thinking skills and, eventually, the ability to carry out simple tasks. Biogen said it will now focus its efforts on its Eisai-partnered Alzheimer’s drug Leqembi (lecanemab-irmb), as well as potential new treatment modalities. Leqembi was granted traditional approval by the FDA in July 2023 for use in patients with mild cognitive impairment or early-stage Alzheimer’s disease as a bi-weekly intravenous infusion. Eisai’s supplemental biologics application for approval under the traditional pathway was supported by data from the phase 3 confirmatory Clarity AD clinical trial, in which Leqembi reduced clinical decline by 27% on the global cognitive and functional scale compared with placebo at 18 months. Christopher Viehbacher, president and chief executive officer of Biogen, said: “We plan to further advance the launch of Leqembi, together with Eisai, and continue to bolster innovation with the development of the other assets in our pipeline. “When searching for new medicines, one breakthrough can be the foundation that triggers future medicines to be developed.”

Biogen is cutting ties with Aduhelm, the Alzheimer’s disease drug that won a landmark but controversial regulatory approval and then became a commercial flop. Development and commercialization of Aduhelm will be discontinued, Biogen announced Wednesday. The company is also terminating the post-marketing confirmatory study that was a condition of the therapy’s 2021 accelerated FDA approval. Aduhelm came from the labs of Swiss company Neurimmune, which licensed the drug to Biogen. Biogen has terminated that license, returning the drug’s rights to its developer. Biogen developed Aduhelm under a neuroscience partnership with Eisai. The deal made Biogen the leader on Aduhelm development with Eisai sharing in the drug’s commercialization. In 2022, the partners amended the pact to give Biogen sole responsibility for the drug. Eisai turned its full attention to Leqembi, a different antibody Alzheimer’s disease drug that went on to win accelerated FDA approval in early 2023 and then full FDA approval about six months later. Leqembi is part of the partnership with Biogen, but Eisai is the leader on this therapy’s development and commercialization. As the first new Alzheimer’s disease drug approved in decades, Aduhelm was projected to become a blockbuster seller. However, market uptake of the drug was weak. Some clinicians hesitated to prescribe the drug due to safety concerns. Insurance companies balked at covering the drug’s high price. A 50% price cut did little to move the needle on sales. Most of the patients eligible for Aduhelm are Medicare beneficiaries, but this market was radically narrowed by a 2022 Centers for Medicare and Medicaid Services decision to limit coverage of the drug only to beneficiaries enrolled in a clinical trial. Biogen also encountered hurdles overseas. The company withdrew an application seeking approval of Aduhelm in Europe after an advisory body to regulators there issued a negative opinion on the drug. With Aduhelm facing limited commercial opportunity, Biogen restructured its operations in 2022, moves that included eliminating its commercial infrastructure for the Alzheimer’s drug. The corporate shakeup led to job losses for many, including former CEO Michel Vounatsos. Under his successor, Chris Viehbacher, Biogen last year began a portfolio review that included seeking potential partners or external financing for Aduhelm. The company found no partners or financing for the drug. In the fourth quarter of last year, Biogen recorded a $60 million charge for costs related to closing out the Aduhelm program. The company said resources that previously supported the drug will now go toward Leqembi and the development of new Alzheimer’s drugs in other therapeutic modalities. Those programs include BIIB080, an antisense oligonucleotide licensed from Ionis Pharmaceuticals that targets the tau protein. Biogen is also taking another approach to tau with BIIB113, an oral small molecule. “As a pioneer in Alzheimer’s disease, Biogen is reprioritizing resources to build a leading franchise to address the multiple pathologies of the disease and patient needs,” Viehbacher said in a prepared statement. “We plan to further advance the launch of Leqembi, together with Eisai, and continue to bolster innovation with the development of the other assets in our pipeline.”