Astrazeneca plc is acquiring rare diseases specialist Amolyt Pharma SA in a $1.05 billion deal, taking ownership of eneboparatide (AZP-3601), which is in phase III development for the treatment of hypoparathyroidism. Of the total, $800 million will be paid up front, with the balance of $250 million contingent upon achievement of a regulatory milestone.
Aeterna Zentaris Inc. has offered an update on its development pipeline programs. Macimorelin acetate (AEZS-130), a ghrelin agonist approved and commercialized as a test for adult growth hormone deficiency, is in preclinical development for amyotrophic lateral sclerosis (ALS). The company has now successfully developed an alternative formulation suitable for use in ALS.
It’s not often that getting an NDA rejected by the U.S. FDA is cause for a company’s stock to rise. But in the case of Ascendis Pharma A/S, the agency’s anticipated complete response letter (CRL) for Transcon PTH (palopegteriparatide) offered some much-needed clarity and a potentially optimistic time frame for an NDA resubmission seeking approval of the once-daily hormone replacement therapy for hypoparathyroidism, news that sent the company’s shares (NASDAQ:ASND) up 24%, or $16.78, to close May 1 at $86.74.
A frustrating lack of detail left analysts with little to do but speculate on the odds for a 2023 U.S. FDA approval of Ascendis Pharma A/S’s Transcon PTH (palopegteriparatide), a parathyroid hormone prodrug for hypoparathyroidism, after the company disclosed a letter from the agency citing unspecified deficiencies that preclude further discussions about labeling and postmarketing requirements.
Phase III data are due any day from Ascendis Pharma A/S, and Wall Street’s thoughts have turned to hypoparathyroidism (HPT), an indication fraught with questions in recent years.
Shares of Israeli drug delivery specialist Entera Bio Ltd. (NASDAQ:ENTX) led the U.S. market March 11, in both percentage gain and volume, climbing 152.5% to $4.04 after a report that its oral parathyroid hormone candidate, EB-613, significantly boosted levels of P1NP, a biomarker indicating new bone formation vs. placebo at three months in an ongoing phase II osteoporosis study.
Shares of Entera Bio Ltd. (NASDAQ:ENTX), a lightly traded Israeli drug delivery specialist, fell 27% on May 21 after the company said an ongoing phase II trial of its oral parathyroid hormone (PTH) candidate in osteoporosis patients found the two lowest doses tested demonstrated "suboptimal increases" in P1NP, an important biomarker of bone formation.