22:59 , Jul 8, 2019 |  BC Extra  |  Company News

July 8 Company Quick Takes: Merck, Skyhawk in discovery deal; plus MorphoSys, BioMarin, Roche-Spark and Hanmi

Skyhawk, Merck in neurology, cancer deal  Skyhawk Therapeutics Inc. (Waltham, Mass.) and Merck & Co. Inc. (NYSE:MRK) parterned to discover, develop and commercialize small molecules capable of modulating RNA splicing discovered using Skyhawk’s SkySTAR platform...
01:58 , Jun 29, 2019 |  BioCentury  |  Finance

Biotech backs down in 2Q19

In a reversal from 1Q19, biotech stocks across all market cap tiers fell in the second quarter, leading to a total loss of $22.3 billion. However, the year-to-date change in market cap remains well in...
00:47 , Jun 29, 2019 |  BioCentury  |  Finance

All eyes on new modality launches, cancer and rare disease data in 3Q19

While the typical summer lull means fewer catalysts in the third quarter, investors will be keeping a close eye on launches of new modality drugs. Of special interest to multiple buysiders is the launch of...
21:57 , Jun 27, 2019 |  BC Innovations  |  Product Development

Gene therapy’s next frontier lies beyond rare, monogenic diseases

As in vivo gene therapies continue notching approvals for rare monogenic diseases, the next frontier for the modality will be in treating complex indications without obvious genetic drivers. At least seven companies have clinical or...
23:16 , Jun 21, 2019 |  BC Extra  |  Company News

June 21 Company Quick Takes: FDA rejects Daiichi's AML candidate; plus Sanofi, Novartis and more

Complete response letter for Daiichi's quizartinib  Daiichi Sankyo Co. Ltd. (Tokyo:4568) said FDA issued a complete response letter for quizartinib, its candidate to treat relapsed/refractory FLT3-ITD-positive acute myelogenous leukemia (AML). In May, an FDA advisory...
21:07 , Jun 13, 2019 |  BC Innovations  |  Product Development

Why BioMarin took gene therapy manufacturing into its own hands

As gene therapy companies fight for space at contract manufacturers, BioMarin has leveraged its expertise in biologics to design its own capabilities for manufacturing gene therapies and gain control of the process -- a move...
23:46 , Jun 4, 2019 |  BC Extra  |  Company News

June 4 Company Quick Takes: Lilly's Emgality gets expanded indication; plus Vimizim, Zerbaxa and more

Emgality gets U.S. approval for episodic cluster headaches  FDA approved Emgality galcanezumab-gnlm from Eli Lilly and Co. (NYSE:LLY) to treat episodic cluster headache in adults. The anti-CGRP mAb is already approved as a prophylaxis for...
00:05 , May 29, 2019 |  BC Extra  |  Clinical News

Longevity of hemophilia gene therapy in question as BioMarin seeks accelerated approval

As BioMarin reported interim Phase III data Tuesday that sets the company up for regulatory submissions, long-term Phase I/II data cast doubt on the durability of gene therapy valoctocogene roxaparvovec to treat severe hemophilia A....
12:00 , May 20, 2019 |  BioCentury  |  Emerging Company Profile

Reneo: Small molecules for rare mitochondrial diseases

With a $50 million series A led by NEA and a management team guided by veterans of Shire takeout Lumena, Reneo believes its small molecule could carve out a niche in the rare genetic mitochondrial...
02:23 , May 18, 2019 |  BioCentury  |  Product Development

AAVs on the brink: Charting the clinical landscape of in vivo gene therapies

Gene therapy is finally teetering on the edge of becoming a drug development mainstay, with at least nine AAV-based therapies in Phase III or II/III testing. An analysis of the AAV therapies in the clinic...