Alexion's R&D Plans At Increased Pace With Rhythm Voucher
Follows Last Month's Acquisition Agreement With AstraZeneca
Executive Summary
After last month’s news of its proposed acquisition by AstraZeneca, Alexion Pharmaceuticals has bought a priority review voucher from Rhythm Pharmaceuticals, which would speed one of its development projects towards the marketplace.
Alexion Pharmaceuticals Inc.’s attractions for its likely acquirer AstraZeneca PLC now include a rare pediatric disease priority review voucher (PRV), which Alexion is buying from Rhythm Pharmaceuticals, Inc. for $100m. Rhythm announced the sale on 5 January, when its share price rose by 4%.
AstraZeneca announced in the middle of last month that it was offering $39bn to acquire Alexion, with the deal expected to close in the third quarter of 2021. The rare disease focus of Alexion, including its marketed products Soliris (eculizumab) and Ultomiris (ravulizumab), but also its research pipeline, were said to be of interest to AstraZeneca (see sidebar).
There are a growing number of projects in development at Alexion where the voucher might be used, including the potential Wilson disease therapy, ALXN1840, which is in Phase III, CAEL-101, which is Phase III for amyloidosis, and the Factor D product, ALXN2040, which is advancing towards Phase III studies in paroxysmal nocturnal hemoglobinuria, A bi-specific product, ALX 1720, is in early clinical studies for renal disease.
Alexion is also aiming to extend the indications for the new-generation complement C5 inhibitor, Ultomiris (ravulizumab-cwvz) to include myasthenia gravis, expected in 2022, and neuromyelitis optica spectrum disorder (NMOSD). Ultomiris is also in Phase III for amyotrophic lateral sclerosis (ALS), with top-line results expected in the second half of 2022. Clinical studies are underway to extend the use of the antidote, Andexxa (andexanet alfa).
Rhythm initially received the voucher from the US Food & Drug Administration for the development of Imcivree (setmelanotide) for a rare disease – it was approved in November 2020 for chronic weight management in adult and pediatric patients 6 years of age and older with obesity due to proopiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1) or leptin receptor (LEPR) deficiency confirmed by genetic testing. (Also see "Rhythm May Have Data To Add Indication To Imcivree’s Label" - Scrip, 22 Dec, 2020.)
The funds will be used by Rhythm for further product development: “The non-dilutive capital from the sale of our PRV provides an important source of additional funding to advance the continued development of setmelanotide as a precision medicine for people whose severe obesity and insatiable hunger may be caused by genetic variants associated with the melanocortin-4 (MCR4) receptor pathway,” said Rhythm chair, president and CEO, David Meeker.