Rare disease and neurodegeneration pipelines drew fresh capital this week, with three separate deals seeing companies acquire, expand, and license their way into Niemann-Pick disease, difficult-to-drug protein targets, and Parkinson's disease.

"The flurry of activity underscores a dynamic playing out across the industry. As we head into the $500 billion patent cliff between 2026 and 2032, large pharma companies will become increasingly reliant on deal-making and sourcing externally to rejuvenate and continue to grow,” Dan Chancellor, vice president of thought leadership at Norstella recently told Pharmaceutical Executive.

Shionogi to acquire IntraBio

Shionogi & Co., Ltd.'s board of directors have approved an agreement for Shionogi to acquire IntraBio Inc., a biopharmaceutical company developing and commercializing therapies for neurodegenerative diseases.

Under terms of the agreement, IntraBio is expected to become a wholly owned subsidiary of Shionogi Inc., Shionogi's New Jersey-based subsidiary.¹ Shionogi Inc. will also acquire all outstanding shares of IntraBio for an upfront consideration of $2.0 billion payable to IntraBio shareholders.¹

The proposed transaction is subject to customary closing conditions.¹

As part of the deal, Aqneursa (levacetylleucine) and IntraBio's rare disease capabilities are expected to join Shionogi's portfolio.¹ Aqneursa was approved by FDA back in September 2024 for neurological manifestations of Niemann-Pick disease Type C in adults and pediatric patients, and by the European Medicines Agency in January 2026. FDA recently approved it on September 18, 2026 for the treatment of Ataxia in adults and pediatric patients diagnosed with Ataxia-Telangiectasia and is currently under review by the EMA for adult and pediatric A-T patients.¹

"The planned acquisition of IntraBio actively demonstrates Shionogi's solid commitment to building a leading global rare disease business," said Isao Teshirogi, Ph.D., chief executive officer of Shionogi. "Shionogi has a strong record of delivering innovative medicines for challenging infectious diseases, and we are applying that same focus and determination to rare diseases. Bringing Aqneursa to Shionogi after our acquisition of Radicava will deepen our commitment to rare disease communities, expand our capabilities and strengthen our portfolio as we advance future innovation for patients with significant unmet needs."

Gate Bioscience expands collaboration with Eli Lilly

Gate Bioscience, a biotechnology company developing molecular gates, announced an expansion of its collaboration and license agreement with Eli Lilly and Company to discover, develop and commercialize molecular gate therapeutics.

The expanded collaboration adds one target, with the opportunity to add a second target, to the companies' existing work.²

Under the multi-year research collaboration, Gate is set to receive an upfront payment and a subsequent payment upon selection of the second target, plus research funding to support its discovery efforts.² Gate is also eligible for payments upon achievement of certain preclinical, clinical and commercial milestones, as well as tiered royalties on global net sales, with the total potential value of the agreement exceeding $870 million, contingent on milestone achievement.²

Gate is expected to lead discovery efforts under the agreement, applying its Molecular Gate Discovery Platform to selected targets while continuing to advance its own wholly owned portfolio of molecular gate medicines toward the clinic, while Eli Lilly will be responsible for later-stage preclinical development, clinical development, manufacturing and commercialization of medicines arising from the collaboration.²

The deal also includes an exclusive, worldwide license to research, develop, commercialize, and otherwise exploit products arising from the collaboration for the selected targets for Eli Lilly.²

"Our existing collaboration with Lilly has shown that our discovery engine can systematically deliver selective molecular gates against difficult-to-drug proteins," said Jordi Mata-Fink, Ph.D., co-founder and chief executive officer of Gate Bioscience. "Expanding into an additional therapeutic area reflects Lilly's confidence in that engine and in the team behind it. Lilly's expertise has accelerated our progress, and the collaboration strengthens the wholly owned portfolio of molecular gate medicines we continue to build alongside it."

Alector licenses Al050 to Genentech

Alector, Inc. announced an exclusive global licensing agreement with Genentech granting Genentech worldwide rights to develop and commercialize Alector's Al050 program, its brain-penetrant engineered glucocerebrosidase ERT.³

Under the agreement, Alector will receive an upfront payment of $100 million and will be eligible for additional payments of up to $1.17 billion upon the achievement of development, regulatory and commercial milestones, plus tiered royalties on net sales.³ Genentech will also be responsible for development, regulatory, manufacturing and commercialization across all indications, while Alector retains ownership of its Alector Brain Carrier technology and full rights to apply the platform across its own wholly owned pipeline.³

Al050 is designed to address GCase deficiency, a driver of neurodegeneration in Parkinson's disease that is most pronounced in, but not limited to, carriers of GBA1 mutations. It combines an Alector-engineered GCase, optimized for greater enzymatic activity and a longer half-life, with Alector's ABC, designed to transport therapeutics across the blood-brain barrier.³ Delivered into the brain, the enzyme is intended to break down glucosylsphingosine and glucosylceramide, lipids that accumulate in neurons and other brain cells upon GCase deficiency and contribute to neurodegeneration, with the aim of reducing cellular dysfunction and slowing disease progression.³

"This partnership extends our cash runway into 2029, providing resources to advance our fully active, subcutaneously delivered, brain-enabled anti-Aβ antibody to multi-cohort clinical data in Alzheimer's patients, and move our subcutaneously delivered, brain-enabled Tau siRNA and alpha-synuclein siRNA programs through IND-enabling studies and to IND filings," said Arnon Rosenthal, Ph.D., chief executive officer of Alector.

Sources

1. Shionogi Continues Rare Disease Expansion With Planned Acquisition of IntraBio Inc. Shionogi Inc. October 5, 2026, https://www.businesswire.com/news/home/20261005854293/en/Shionogi-Continues-Rare-Disease-Expansion-With-Planned-Acquisition-of-IntraBio-Inc.
2. Gate Bioscience Expands Collaboration and License Agreement with Lilly to Target Difficult-to-Drug Proteins Gate Bioscience October 5, 2026, https://www.globenewswire.com/news-release/2026/10/05/3374492/0/en/gate-bioscience-expands-collaboration-and-license-agreement-with-lilly-to-target-difficult-to-drug-proteins.html
3. Alector Enters Global License Agreement with Genentech for Brain Penetrant GCase Enzyme Replacement Therapy for Parkinson’s Disease and Other Neurodegenerative Diseases Alector Inc. October 5, 2026, https://www.globenewswire.com/news-release/2026/10/05/3374409/0/en/alector-enters-global-license-agreement-with-genentech-for-brain-penetrant-gcase-enzyme-replacement-therapy-for-parkinson-s-disease-and-other-neurodegenerative-diseases.html