Welcome to the latest edition of our weekly EuroBiotech Report. We start this week with Nightstar Therapeutics, which reported early clinical data on its gene therapy treatment for X-linked retinitis pigmentosa. Elsewhere, Minoryx raised €21.3 million ($24.9 million) to expand its CNS R&D activities. Israel's Entera Bio named Arthur Santora, M.D., Ph.D., as its CMO. The day on which Novartis will need to decide whether to pay $150 million to license a hyperlipoproteinemia candidate edged closer. And more. — Nick Taylor
1. Nightstar posts early-phase data on retinal gene therapy
Nightstar Therapeutics has presented early clinical data on its gene therapy treatment for X-linked retinitis pigmentosa. Around half of the patients in higher-doses cohorts experienced vision improvements, encouraging Nightstar to move NSR-RPGR into the larger phase 2 portion of the trial.
2. Roche-backed Minoryx raises €21M to expand CNS R&D
Minoryx has raised €21.3 million in series B funding. The round sets Minoryx up to expand clinical development of a neurodegenerative disease drug that has attracted the interest of organizations including Roche Venture Fund.
3. Former Merck clinical research VP Arthur Santora joins Entera Bio as CMO
Former Merck clinical research leader Arthur Santora, M.D., Ph.D., is coming out of retirement to serve as the new chief medical officer of Entera Bio, to help the company develop its oral hormone-based treatments for osteoporosis and hypoparathyroidism.
4. With positive phase 2 data, Akcea, Ionis could pocket $150M from Novartis
Akcea Therapeutics reported topline data showing that all doses of its hyperlipoproteinemia candidate lowered lipoprotein(a) levels in a phase 2 study involving 286 patients with cardiovascular disease. If all goes well at the end-of-phase-2 meeting with the FDA, its partner Novartis could license the drug, triggering a $150 million payout for Akcea and Ionis.