Opko Health Inc. stands to receive a $90 million milestone payment from partner Pfizer Inc. on the U.S. FDA approval of once-weekly human growth hormone analogue Ngenla (somatrogon) for pediatric patients with growth hormone deficiency. Pfizer expects to make the drug available to patients starting in August 2023.
Disc Medicine Inc.’s positive phase II data from an ongoing, open-label trial called Beacon with oral bitopertin in erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP) whetted investor appetite for the results of the other mid-stage Disc experiment known as Aurora with the compound, due early next year.
After long years of endeavor, Scarlet Therapeutics Ltd. believes it is poised to realize the potential of red blood cells (RBCs) as drug delivery vehicles. The company recently closed a seed round and is now laying the ground for its first clinical trials in which cultured and genetically engineered RBCs will be used to deliver enzyme replacement therapies in the treatment of rare inherited forms of two metabolic disorders.
Word from Immix Biopharma Inc. of updated data due with NXC-201 brought to the forefront an ongoing push by drug developers to come up with a treatment for AL amyloidosis. Immix has the only CAR T therapy in the works for the disease, and the principal investigator in the Nexicart-1 phase Ib/IIa effort is slated to speak May 19 during the annual meeting of the American Society of Gene & Cell Therapy.
The most ambitious objective of any treatment is to eradicate the disease, acting on its origin to cure it instead of treating its symptoms. This is the purpose of the gene therapy against type 2 diabetes (T2D) and obesity that Fractyl Health Inc. is developing. Scientists from the Lexington, Mass.-based company have designed a strategy based on glucagon-like peptide-1 (GLP-1) to transform pancreatic cells and reverse the disease.
With PTC Therapeutics Inc.’s positive results in phase III with sepiapterin for pediatric and adult patients with phenylketonuria (PKU), attention turned to the would-be showdown with Kuvan (sapropterin dihydrochloride), the drug from Biomarin Pharmaceuticals Inc. that was approved in December 2017.
On the heels of the marketing OK in Europe, Protalix Biotherapeutics Inc. and the Chiesi Group’s global rare diseases unit scored approval of Elfabrio (pegunigalsidase alfa-iwxj) from the U.S. FDA for adults with Fabry disease.
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 mid-stage prospect for Pompe disease (PD) – this one from Maze Therapeutics Inc. – caught the eye of Sanofi SA, and the pair signed a potential $750 million deal to move along the oral glycogen synthase (GYS1) inhibitor MZE-001, a substrate reduction therapy.
Eli Lilly and Co. is selling worldwide rights to its hypoglycemia nasal treatment Baqsimi to Amphastar Pharmaceuticals Inc. in a deal potentially worth over $1 billion. Under the agreement, Amphastar, of Rancho Cucamonga, Calif., will foot a $500 million cash payment on closing, and will pay Indianapolis-based Lilly an additional $125 million on the one year anniversary of closing. Lilly is also eligible to receive milestone payments of up to $450 million, based on annual sales of $175 million and $200 million, and on total net sales over five years of $950 million.
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