A positive growth trajectory for three pharmaceutical startups

Based in Basel, Santhera has pipeline of drugs for neuromuscular and pulmonary diseases that currently lack treatment options. Their drug candidate AGAMREE® (vamorolone) is a first-in-class dissociative steroid with novel mode of action, which was investigated in a pivotal study in patients with Duchenne muscular dystrophy (DMD) as an alternative to standard corticosteroids. DMD is a rare inherited X-chromosome-linked disease, which almost exclusively affects males. Shortly after receiving approval for vamorolone as a treatment for DMD in the U.K., the company launched the drug in Germany and the U.S. earlier this year.The China National Medical Products Administration (NMPA) has accepted for priority review the new drug application (NDA) for vamorolone, submitted by Sperogenix Therapeutics, Santhera's specialized rare disease partner for China. This priority review for vamorolone was granted for patients aged 4 years and older which could, subject to a positive outcome, lead to approval by Q1 2025. Previously, the Center for Drug Evaluation (CDE) of the Chinese drug authority NMPA included vamorolone for the treatment of DMD in the Breakthrough Therapy Program, which addresses serious diseases lacking effective treatments and includes drugs offering clear clinical advantages over existing treatments. 

Evidence for clinical efficacy and safety of vamorolone was based on data from the positive pivotal VISION-DMD study, showing superiority of vamorolone compared with a placebo, and three open-label studies in which vamorolone was administered at doses between 2 and 6 mg/kg/day for a total treatment period of up to 30 months. The filing is further supported by two more studies, one of which included healthy adult Chinese volunteers. According to the license agreement between Santhera and Sperogenix, the latter holds exclusive development and commercialization rights to vamorolone in DMD and all other rare disease indications for China. Upon commercialization, Sperogenix will pay Santhera sales-based milestones and double-digit percentage royalties on net sales. 

Relief Therapeutics sells rights in U.S.
Relief Therapeutics, a biopharmaceutical company seeking to provide patients therapeutic relief from serious diseases, has sold U.S. rights over PKU GOLIKE®, a commercial ultra-rare disease medical formula tackling Phenylketonuria (PKU), to U.S. pharma company Eton Pharmaceuticals. PKU is a rare inherited disorder caused by a defect in the enzyme needed to break down phenylalanine, leading to a toxic buildup of phenylalanine when eating foods that contain proteins. Treatment of PKU requires patients to follow a strict diet that severely limits phenylalanine content and typically requires low protein foods supplemented by phenylalanine-free medical formulas. It is estimated that 8,000 PKU patients in the U.S. utilize medical formulas to manage their diet.

The transaction between Relief Therapeutics and Eton Pharmaceuticals is expected to be accretive to Eton’s 2024 earnings and the company expects peak sales of more than $10 million annually. Under the terms of the agreement, Relief will receive an upfront payment of $2.2 million and is eligible to receive an additional $2.0 million in sales milestones payments as well as mid-teens royalties on net sales. In the fourth quarter of 2023, PKU GOLIKE's annualized net sales exceeded $1 million in the U.S., continuing its growth trajectory since its launch in late 2022. According to Michelle Lock, interim CEO of Relief, this transition is fully aligned with the company's strategy of moving the GOLIKE products into partnership models in the U.S. and Europe. Moreover, Relief will continue to own PKU GOLIKE rights outside the United States. 

Basilea reports continued sale success
Basilea Pharmaceutica, a commercial-stage biopharmaceutical company committed to meeting the needs of patients with severe bacterial and fungal infections, announced that the strong sales performance of the antifungal Cresemba® (isavuconazole) by its license partner Pfizer exceeded the sales threshold in the Asia Pacific region and China, triggering a USD 1.25 million milestone payment. The license agreement between Basilea and Pfizer for Cresemba covers Europe (excluding the Nordic countries) as well as 16 countries in the Asia Pacific region and China. Cresemba is approved and marketed in more than 70 countries, including the United States, most EU member states as well as Iceland, Liechtenstein, the UK and Norway. According to the latest available market data, total global in-market sales of Cresemba in the twelve months between October 2022 and September 2023 amounted to USD 445 million, a 22 percent growth year-on-year. isavuconazole is approved for the treatment of adult patients with invasive aspergillosis and for adult patients with mucormycosis for whom amphotericin B is inappropriate.2 In China, the oral and intravenous formulations are approved for the treatment of adult patients with invasive aspergillosis and invasive mucormycosis. Isavuconazole is also approved in Japan.  

(Press releases / SR)