Rare Diseases

Valencia – November 04, 2026 -- ARTHEx Biotech, a clinical-stage RNA therapeutics developer, has appointed Christian (Chris) Freitag, M.D., as Chief Medical Officer, adding more than 20 years of clinical development experience to a pipeline led by ATX-01 for myotonic dystrophy type 1 (DM1).

Freitag arrives from CMO roles at Azafaros and Dynacure

Freitag most recently served as Chief Medical Officer at Azafaros, which develops treatments for rare genetic disorders. Before that, he was CMO at Dynacure, where he was responsible for medical and regulatory strategy, including clinical development of the company's lead investigational therapy for centronuclear myopathies.

LOUISVILLE – October 28, 2026 -- US WorldMeds Ventures, LLC has acquired Orbus Therapeutics, Inc., adding a late-stage eflornithine program for recurrent Grade 3 astrocytoma. In a prespecified subset of the Phase 3 STELLAR study, patients with IDH-mutant disease reached median overall survival of 34.9 months, versus 23.5 months on lomustine alone.

US WorldMeds made an undisclosed upfront payment under a definitive merger agreement. Orbus is eligible for development, regulatory and sales-based milestone payments, plus tiered royalties on U.S. net sales. It is now a wholly owned subsidiary of US WorldMeds.

STELLAR missed its primary endpoint in the full study population

The 343-patient trial compared eflornithine plus lomustine with lomustine alone in recurrent Grade 3 astrocytoma, defined under the 2016 WHO classification. It showed no overall survival difference between arms in the intent-to-treat population, which was the primary endpoint.

Stockholm – October 19, 2026 -- Sobi will present seven accepted abstracts on Aspaveli/Empaveli (pegcetacoplan) at the American Society of Nephrology (ASN) Kidney Week 2026, running 21–25 October in Denver, Colorado, including two oral and five poster presentations.

Oral sessions report 76-week outcomes in two rare kidney diseases

The two oral presentations cover 76-week outcomes with Aspaveli/Empaveli in C3 glomerulopathy (C3G) and primary immune-complex membranoproliferative glomerulonephritis (IC-MPGN). According to Sobi, the data show maintained proteinuria reduction and stabilised eGFR. The package includes a paediatric subgroup analysis from the VALIANT study and its VALE extension.

Madrid – September 20, 2026 -- mAbxience and Sandoz have signed a licensing, development, manufacturing and commercialization agreement for a proposed emicizumab biosimilar targeting hemophilia A, a treatment referencing Roche's Hemlibra with an estimated global reference market of USD 5.7 billion.

mAbxience will develop and manufacture the biosimilar at GMP facilities in Spain and Argentina

Under the agreement, mAbxience is responsible for development and manufacturing through its facilities in the two countries. Sandoz will handle global commercialization.

Sandoz secures exclusive global rights excluding three South American markets

Sandoz holds exclusive commercialization rights worldwide, with the exception of Argentina, Uruguay and Paraguay. Financial terms of the deal were not disclosed.