Dino Mustafić

January 7, 2019 Off

Dutch ProQR Therapeutics OKd by FDA to take sepofarsen (QR-110) into the next phase of development

A Dutch maker of of transformative RNA medicines for the treatment of severe genetic rare diseases, ProQR Therapeutics, has got the U.S. Food and Drug Administration (FDA) tips for a Phase 2/3 pivotal trial Illuminate for sepofarsen, in patients with Leber’s congenital amaurosis 10 (LCA10) due to the p.Cys998X mutation in the CEP290 gene, the leading genetic cause of childhood blindness.

January 7, 2019 Off

Galapagos kicks off clinical testing of its investigational drug in Toledo program

By Dino Mustafić

Galapagos has discovered GLPG3312, a first generation compound against a novel and undisclosed inflammation target class, and it started Phase 1 trial.

January 7, 2019 Off

Basilea posts revenue €118.37M, 31% up from last year

By Dino Mustafić

Basilea Pharmaceutica posted preliminary revenue for the financial year 2018, which is expected to increase by 31% over 2017 to about CHF133 million (€118.37M), from the last year’s CHF101.5 million (€90.3M).

January 7, 2019 Off

Axsome Therapeutics’ AXS-05 nvestigational drug meets endpoint in Phase 2 trial in major depressive disorder

By Dino Mustafić

Axsome Therapeutics’ AXS-05, a novel, oral, investigational NMDA receptor antagonist with multimodal activity, met the prespecified primary endpoint and significantly improved symptoms of depression in the ASCEND Phase 2 trial in major depressive disorder (MDD), the company said Monday.

January 7, 2019 Off

Adverum Biotechnologies has sufficient cash to fund operations at least through the first half of 2020, to advance Novel Gene Therapy ADVM-022 for Wet AMD in OPTIC Phase 1 clinical trial

By Dino Mustafić

Adverum Biotechnologies, a California-based ophthalmology and rare diseases focused biotech company, expects to provide an update on enrollment from the OPTIC phase 1 clinical trial in the first half of 2019, and interim data on the three cohorts from the OPTIC phase 1 clinical trial by the first quarter of 2020, it said in an outlook for 2019.

January 7, 2019 Off

Stelexis Therapeutics closes $43 million Series A financing

By Dino Mustafić

New York-based cancer therapeutics company, Stelexis Therapeutics has closed a $43 million Series A financing to expand its proprietary platform to discover and selectively target pre-cancerous stem cells.

January 7, 2019 Off

AstraZeneca pays $15 million to its partner Sosei Group, as it hits milestone in development of cancer candidate

By Dino Mustafić

AstraZeneca has reached a clinical development milestone with immuno-oncology candidate AZD4635, thus triggering a $15 million payment to its partner, Sosei Group.

January 7, 2019 Off

4D pharma to test its diverse cancer settings Live Biotherapeutic oncology pipeline with University of Texas MD Anderson Cancer Center

By Dino Mustafić

4D pharma and The University of Texas MD Anderson Cancer Center will together work on testing 4D’s Live Biotherapeutic oncology pipeline across a range of cancer settings, the company said on Monday in a press release

January 7, 2019 Off

Orexo’s nasal spray satisfied in test, turns to FDA for consultation

By Dino Mustafić

Orexo AB’s nasal spray formulations for opioid overdose reversal has been compared to Narcan in a study, in which it showed similar or better results, the company said in a press release on Monday.

January 7, 2019 Off

Sanofi to pay Regeneron $462 million as companies restructure immuno-oncology agreement for more independence in each research

By Dino Mustafić

Regeneron Pharmaceuticals and Sanofi restructure immuno-oncology agreement from 2015, where Regeneron retains full rights to all its other investigational immunooncology programsto, while Sanofi is able to independently chase own immuno-oncology programs.