Shares of Milestone Pharmaceuticals Inc. (NASDAQ:MIST) were trading midday at $7.96, up $4.62, or 138%, after the company provided an update on etripamil, a short-acting calcium channel blocker self-administered via nasal spray for patients with paroxysmal supraventricular tachycardia (PSVT). The FDA has agreed with the company’s plan to modify the ongoing study called Node-301B, renamed Rapid, including a primary endpoint of time to conversion within 30 minutes. The Rapid effort, along with the finished Node-301 study – which will also now use a 30-minute endpoint as well – should fulfill efficacy requirements for an NDA, Milestone said. Afflicting about 2 million people in the U.S., PSVT is characterized by a rapid heart rate, with intermittent episodes of SVT that start and stop suddenly and without warning. Symptoms include palpitations, sweating, chest pressure or pain, shortness of breath, sudden onset of fatigue, lightheadedness or dizziness, fainting and anxiety. The company also disclosed that it raised $25 million by way of a private placement with existing shareholder RTW Investments LP.

FDA lifts Cymabay’s clinical holds while Genfit struggles

The FDA lifted the clinical holds it placed on seladelpar from Cymabay Therapeutics Inc., of Newark, Calif., last November for INDs in nonalcoholic steatohepatitis (NASH), primary biliary cholangitis and primary sclerosing cholangitis. Cymabay stopped all studies of seladelpar, a PPAR-delta agonist, when atypical histologic findings created doubts about the phase II NASH study and the FDA then placed the INDs on hold. The FDA lifted the restrictions when independent investigators found no clinical, biochemical or histological evidence of seladelpar-related liver injury in the study. Meanwhile, after several weeks of reviewing data, Genfit SA decided to discontinue its phase III NASH and fibrosis study of lead candidate elafibranor, which failed to meet its predefined primary surrogate efficacy endpoint. Lifting the clinical holds lifted Cymabay shares (NASDAQ:CBAY) at midday Thursday, as they rose 28%, while Genfit stock (NASDAQ:GNFT) bumped upward about 2%.

Advocacy groups push for place at PDUFA negotiating table

The negotiating table for PDUFA VII could become a lot more crowded if consumer advocacy groups have their way. In the past, they – along with patients, health care providers, industry and the scientific/academic community – have been invited to the public meetings that precede the actual industry-FDA negotiations of reauthorization of the five-year user fee agreements. But “we’re not at the table for PDUFA, and that’s very unfortunate,” Diana Zuckerman, president of the National Center for Health Research, said today at the opening public meeting for the next round of PDUFA. Being at public meetings is a lot different than being at the negotiating table, she added. However, consumer advocacy groups, some of which are politicized, are often at odds with the patient voice that all stakeholders agree is vital to drug development.

BIO Asia-Taiwan 2020: COVID-19 highlights supply chain debate among industry challenges

In the shadow of COVID-19, experts at the BIO Asia-Taiwan conference on Wednesday warned of present and future challenges for the biotech industry. Changes in manufacturing logistics and financial distress will continue to cause concern for the industry. Supply chains remained a focus of discussion, as experts believe those are moving toward localization due to restrictions on the global level. Joseph Damond, executive vice president of international affairs at the Biotechnology Innovation Organization, warned of the unintended consequences of moving supply chains and manufacturing locally.

From cells to cortices, ways to improve neurological disease classification

Cancer treatment has been transformed, at its root, by a transformational change in how it is classified. These days, which organ a tumor arises in is often less important than its molecular drivers, which can be sensitive either to specific targeted treatments, or increase the chance that a tumor will respond to immunotherapy. Those successes have not escaped the notice of researchers in other areas of biomedicine, and diseases including heart failure, asthma and polycystic ovarian syndrome are being looked at with an eye to subdividing them in ways that brings diagnostics into the molecular era.

Newco news: G2GBIO seeks further funds after $9.53 million series B financing

HONG KONG – Daejeon, South Korea-based biotechnology company G2GBIO Inc has raised ₩11.4 billion (US$9.53 million) from a series B financing round, with the funds to be used on clinical trials for a sustained-release Alzheimer’s treatment as well as nonclinical trials for diabetes and sustained-release postoperative pain treatments. Investors include Korea Investment Partners, Quad Investment Management, BNK Venture Capital Co. Ltd, Industrial Bank of Korea, Seoul Investment Partners, ARK Impact Asset Management and Emford Equity Partners. “Although it was not easy to obtain financing at the beginning of the COVID-19 outbreak, we had as many IR meetings as possible, and kept our focus on our technology’s core value to investors,” G2GBIO’s managing director for business development, Soojung Lee, told BioWorld.

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Also in the news

Acelrx, Acurx, ADC, Adial, Allovir, Amag, Ampio, Annexon, Aravive, Arthex, Aurinia, Biontech, Brainstorm, Clarity, Corcept, Cymabay, Dynavax, Elsalys, G1, Genfit, Hansoh, Humanetics, Incyte, Inotrem, Inozyme, Intravacc, Kaleido, Medigen, Memed, Milestone, Nabriva, Nervgen, Neumentum, Norgine, Noxopharm, Nurix, Onconova, Ovid, Quadriga, Scopus, Synaffix, Taiho, United, Vita