US – The US Food and Drug Administration (FDA) has recognized the potential of US-based biotech company Disc Medicine’s DISC-3405 in treating the rare blood cancer, polycythemia vera (PV), by granting it an orphan drug designation (ODD). 

Disc Medicine obtained exclusive rights to develop and market DISC-3405 in the US and other territories through a partnership with Mabwell Therapeutics in a licensing agreement worth US$412.5 million announced in January 2023. 

In October 2023, DISC initiated a Phase I clinical trial (NCT06050915) for DISC-3405, previously referred to as MWTX-003. Data from this ongoing study is anticipated in the first half of 2024. 

 The orphan drug designation follows a prior fast track designation granted by the FDA. 

The Phase I study involves 64 patients, divided into groups receiving either single or multiple doses of DISC-3405, or a placebo.  

The trial includes a single ascending dose (SAD) phase, with two sentinel subjects – one receiving DISC-3405 and the other placebo. Subsequent cohorts for multiple ascending doses (MAD) are enrolled after a safety observation period, eliminating the need for sentinels. 

Polycythemia vera (PV), categorized under myeloproliferative neoplasms, leads to excessive red blood cell production. DISC-3405, a humanized monoclonal antibody, targets TMPRSS6, a protein involved in regulating hepcidin production. 

Also read:

1: Gilead gets hold of innovative liver disease drug with US$4.3B acquisition of CymaBay

2: Hemogenyx receives FDA trail approval for therapy designed to combat acute myeloid leukaemia

 Hepcidin controls iron absorption, thereby limiting red blood cell production, addressing the overproduction associated with PV. 

John Quisel, CEO of Disc, emphasized that designation was very important to the company, and underscored that it will have groundbreaking potential in addressing PV, a disease with limited treatment options. 

Notably, pharmaceutical company Takeda recently entered into a global license and partnership agreement with Protagonist Therapeutics for the development and marketing of rusfertide, an investigational hepcidin mimetic peptide for PV treatment. 

 Protagonist stands to receive $300 million as an upfront payment from Takeda as part of this collaboration.

For all the latest healthcare industry news from Africa and the World, subscribe to our NEWSLETTER, and YouTube Channel, follow us on Twitter and LinkedIn, and like us on Facebook.