A drugmaker focused on RNA interference and another firm developing drugs for a range of rare diseases have formed a partnership worth up to $1 billion focused on diseases of the eye and central nervous system.
Tarrytown, New York-based Regeneron Pharmaceuticals and Cambridge, Massachusetts-based Alnylam Pharmaceuticals announced the partnership Monday, which will be focused on discovery, development and commercialization of RNAi-based drugs. The partnership includes an $800 million upfront cash and equity investment in Alnylam, and up to $200 million in near-term milestones.
In a note to investors, Cowen analyst Ritu Baral wrote that the deal makes strategic sense given that it builds on Regeneron’s experience in ophthalmology and CNS diseases and ability to identify novel genetic targets with Alnylam’s gene-silencing toolkit. Nevertheless, Alnylam and Regeneron’s shares fell more than 3 percent Monday morning and were respectively down 1.03 percent and 3.02 percent in afternoon trading.
The two companies have an existing partnership, announced last year, to develop drugs for nonalcoholic steatohepatitis, also known as NASH.
Under the new partnership, both companies will work to discover RNAi drugs for eye and CNS diseases, while Regeneron will lead development and commercialization for all programs targeting eye diseases, potentially entitling Alnylam to milestone and royalty payments. Meanwhile, for CNS programs, the companies will jointly advance leadership and also alternate, with the company leading a project retaining global responsibility for development and commercialization.
The Food and Drug Administration approved Alnylam’s Onpattro for peripheral nerve disease caused by hereditary transthyretin-mediated amyloidosis, or hAATR, in August 2018 marking the first-ever FDA approval of an siRNA drug.
A Deep-dive Into Specialty Pharma
A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.
In addition to eye and CNS diseases, the companies will also work together on some programs focused on liver diseases. These include a plan to combine Regeneron’s drug REGN3918 (pozelimab) with Alnylam’s cemdirsan, which Regeneron will lead. Pozelimab has completed a Phase I study among healthy volunteers, while cemdirsan is in a Phase II study recruiting patients with immunoglobulin A nephropathy.
Both pozelimab and cemdirsan target complement protein C5, which is the same target as Alexion Pharmaceuticals’ drug Soliris (eculizumab) and its successor, Ultomiris (ravulizumab-cwvz). Soliris is approved for paroxysmal nocturnal hemoglobinuria, or PNH, and atypical hemolytic uremic syndrome, or aHUS, while Ultomiris is only approved for PNH. Amgen is developing a biosimilar to Soliris, ABP 959, with a Phase III trial comparing it to Soliris expected to start recruiting patients at the end of this month. The study is expected to enroll 40 PNH patients.
Cemdirsan has also completed a Phase I/II trial in PNH and was the subject of a Phase II study in aHUS, but the latter study was withdrawn early due to lack of enrollment.
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