Partnering for pulmonary disease

Rare disease-focused companies link up to identify, develop therapeutics

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CAMBRIDGE, Mass.—Acceleron Pharma and Fulcrum Therapeutics have a collaboration and license agreement underway for the new year as they join forces to identify small molecules against a pulmonary disease target. Acceleron is focused on the discovery and development of therapeutics within the TGF-beta superfamily for the treatment of rare diseases, and Fulcrum shares that focus on orphan diseases, though its niche of choice is genetically defined rare diseases.
“This collaboration brings together Fulcrum’s skill in identifying drug targets based on modulation of genetic pathways associated with disease and Acceleron’s deep expertise in TGF-beta superfamily signaling in an effort to generate potentially disease-modifying therapeutics,” Habib Dable, CEO of Acceleron Pharma, stated in a press release. “With this agreement, along with the advancement of the Acceleron-discovered assets sotatercept—in Phase 2 trials in pulmonary arterial hypertension—and ACE-1334, we underscore our growing commitment to the development of novel therapies for patients with pulmonary diseases of high unmet medical need.”
Per the terms of the agreement, Acceleron gains access to Fulcrum’s proprietary product engine and target identification platform to identify small molecules that control the expression of target genes implicated in pathways associated with a pulmonary disease of interest. In return, Acceleron will make a one-time, upfront payment of $10 million to Fulcrum, as well as reimbursement for relevant R&D costs. Fulcrum also stands to receive research, development and commercialization milestone payments of up to $295 million for the first product commercialized and up to a maximum of $143.5 million in additional milestone payments for any subsequent products. In addition, Fulcrum is also eligible for tiered royalty payments in the mid-single-digit to low double-digit range on net sales.
The two companies will work together to identify therapeutic targets and small-molecule drug candidates, and Acceleron will assume responsibility for all development and commercialization activities for therapeutics identified under this agreement.
“We are very pleased to partner with Acceleron on this important research initiative,” remarked Dr. Robert J. Gould, CEO of Fulcrum Therapeutics. “This collaboration builds on and extends the proven potential of our platform to identify therapies that can address the root cause of diseases, including our progress with losmapimod, currently in a Phase 2 clinical trial for FSHD and extensive preclinical and early-stage research targeting other genetically defined diseases. This new opportunity to screen and identify pulmonary disease-specific therapies is another reflection of the broad potential applications of the Fulcrum platform in gene modulation.”
Bryan Stuart, chief operating officer of Fulcrum, tells DDNews that this is the first time Fulcrum and Acceleron have worked together, and that their business relationship sprang up from a shared interest in a different rare disease.
“Our focus is around gene regulation in genetically defined rare diseases, and our lead program is for FSHD—facioscapulohumeral dystrophy, which is a very progressive and debilitating form of muscular dystrophy,” says Stuart. “Through our product engine and our approach, we identified what is now our lead candidate and in Phase 2 trials for FSHD, and Acceleron previously had a program in FSHD. Through that mutual interest, we got to know Acceleron, and their understanding of our approach led to their interest in working with us on a different disease.”
Fulcrum’s proprietary product engine identifies drug targets that can modulate gene expression in order to treat rare diseases by directly targeting the source of gene mis-expression. The company’s approach combines disease modeling, scalable cell biology with custom robotics, characterization in patient-derived models and profiling of therapeutic candidates against a variety of patient-derived tissue-relevant cells.
On its end, Acceleron is advancing two pipeline candidates in pulmonary areas at present. Sotatercept is in two Phase 2 trials—the PULSAR trial and the SPECTRA exploratory trial—in pulmonary arterial hypertension, and ACE-1334 is in Phase 1 development in an undisclosed pulmonary disease.
“As Fulcrum looks at different diseases and therapeutic areas, we plan to both develop and commercialize therapies ourselves, and at the same time, there are also other diseases where we feel it’s more appropriate to work with a partner who would commercialize. So we want to be very mindful of which areas we plan on building up a development and commercial presence. While we haven’t disclosed the disease per se, we believe this is significant disease where we feel that with Acceleron’s expertise, in terms of development in this space, this has the potential to address a meaningful unmet need in the market,” Stuart concludes.

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