Versartis Inc. (NASDAQ:VSAR) gained $4.30 (29%) to $18.92 on Monday after FDA lifted a partial clinical hold on the Phase III VELOCITY study of VRS-317, a long-acting recombinant human growth hormone (rhGH) to treat growth hormone deficiency (GHD). In May, FDA suspended enrollment of the trial and requested additional bioanalytical data from the company.
VELOCITY's primary endpoint is non-inferiority in mean height velocity among patients receiving semi-monthly VRS-317 vs. daily rhGH treatment in treatment-naive children. Versartis expects interim results by YE16 and top-line 12-month results on the primary endpoint by mid-2017.
Versartis has rights to develop VRS-317 from Amunix Operating Inc. (Mountain View, Calif.). The compound has Orphan Drug designation in the U.S. and EU to treat GHD.
Versartis' news did not negatively affect the stocks of competing GHD companies. Ascendis Pharma A/S (NASDAQ:ASND), which expects data this summer from a Phase II study of its TransCon PEG Growth Hormone, gained $0.17 to $18.61. Opko Health Inc. (NYSE:OPK) gained $0.57 to $16.82. In December, Opko granted Pfizer Inc. (NYSE:PFE) a license to hGH-CTP, a long-acting hGH analog in Phase III testing.
Catabasis Pharmaceuticals Inc. (NASDAQ:CATB) gained $1.24 (10%) to $13.64 Monday after FDA granted Fast Track designation to CAT-1004 to treat Duchenne muscular dystrophy. The company began a Phase I/II trial last month of the conjugate of salicylate and docosahexaenoic acid (DHA) that inhibits activated NF-kappa B (NF-kB).
Catabasis raised $60 million through the sale of 5 million shares at $12 in a June IPO (see BioCentury Extra, June 25).
Desano Pharma (Shanghai, China) will manufacture the active pharmaceutical ingredient (API) of HIV drug Tivicay dolutegravir from ViiV Healthcare Ltd. (Brentford, U.K.) for sale in China and other developing countries. ViiV spokesperson Sebastien Desprez declined to disclose financial terms or name the other countries.
Desprez told BioCentury the price of Tivicay in China will be lower than its price in the U.S., Europe and Australia, but declined to give a range. He said the wholesale acquisition cost (WAC) of Tivicay in the U.S. is about $45 per day.
The HIV integrase inhibitor is under review by the China Food and Drug Administration (CFDA). Tivicay is approved in 70 countries to treat HIV-1 infection.
GlaxoSmithKline plc (LSE:GSK; NYSE:GSK) is the largest stakeholder in ViiV, an HIV-focused joint venture with Pfizer Inc. (NYSE:PFE) and Shionogi & Co. Ltd. (Tokyo:4507).
GSK pledged last year to increase access to its products in China "through greater expansion of production and through price flexibility." The company apologized last September after a Chinese court found the GSK China Investment Co. Ltd. subsidiary of GSK guilty of bribing non-government personnel for commercial gain and fined the company RMB3 billion ($488 million) (see BioCentury Extra, Sept. 19, 2014).
CNS play Zogenix Inc. (NASDAQ:ZGNX) hired Gail Farfel as EVP and chief development officer and Thierry Darcis as EVP and general manager of Europe. Farfel was chief clinical and regulatory officer at Marinus Pharmaceuticals Inc. (NASDAQ:MRNS). Darcis was general manager of Europe at NPS Pharmaceuticals Inc., which Shire plc (LSE:SHP; NASDAQ:SHPG) acquired.
Dermatology company Revance Therapeutics Inc. (NASDAQ:RVNC) said Jacob Waugh stepped down as CSO.
Ophthalmology play Acucela Inc. (Tokyo:4589) named Roger Girard chief strategy officer, effective Sept.1. Girard was CFO and co-founder of CareCap LLC (Bellevue, Wash.).
Pulmonary company Moerae Matrix Inc. (Morristown, N.J.) named Bill Bradford CMO. He was SVP of clinical science and biometrics at InterMune Inc., which Roche (SIX:ROG; OTCQX:RHHBY) acquired.
Genomics company 23andMe Inc. (Mountain View, Calif.) has raised $79.1 million of a planned $150 million round of funding, according to an SEC filing. The company declined to comment.
In March, 23andMe said it would launch a therapeutics group to identify novel drug targets mined from the company's database of customer DNA sequences (see BioCentury Extra, March 12).
In February, FDA approved the company's Bloom syndrome carrier test, the first direct-to-consumer genetic test the agency has approved (see BioCentury Extra, February 20).
Healthcare investment firm Essex Woodlands has raised a total of $360.5 million toward its planned $750 million Essex Woodlands Fund IX, according to an SEC filing. Essex Woodlands said last year it had raised $221.5 million toward the fund (see BioCentury Extra, July 3, 2014).
The firm closed its Fund VIII in 2009 at $900 million. Essex Woodlands invests in pharmaceuticals, medical devices, and healthcare services and IT. It targets companies with $20-$200 million in revenues that are EBITDA-positive or are soon to be EBITDA-positive.
Frazier Healthcare hired Tadataka Yamada as a venture partner on its life sciences team and senior advisor to its growth buyout team. Yamada was CMO and CSO at Takeda Pharmaceutical Co. Ltd. (Tokyo:4502).
FDA issued draft guidance describing conditions for use of plasma fibrinogen as a prognostic biomarker to enrich clinical trials of therapies to treat chronic obstructive pulmonary disease (COPD). The guidance discusses use of the biomarker to select patients at increased risk for exacerbations or all-cause mortality, and describes conditions under which the biomarker is qualified through the Center for Drug Evaluation and Research (CDER) Biomarker Qualification Program.
The guidance says sponsors may use plasma fibrinogen under qualifying conditions in IND, NDA and BLA submissions, and that FDA will consider its use outside of those conditions on a case-by-case basis.
FDA did not determine an optimal enrichment threshold for baseline plasma fibrinogen levels, and said sponsors should propose a threshold to FDA while developing trial protocols.
Comments on the guidance are due Sept. 5.
The Chronic Obstructive Pulmonary Disease Biomarkers Qualification Consortium (CBQC) submitted a biomarker qualification package to FDA in 2013 for plasma fibrinogen. According to CBQC, the submission was the first for clinical biomarker qualification in the U.S. (see BioCentury Extra, Sept. 10, 2013).
Last year, FDA published final guidance outlining a process for qualifying drug development tools including biomarkers that can be used routinely in IND, NDA and BLA submissions (see BioCentury Extra, Jan. 9, 2014).
Rates of chronic diseases are on the rise in China, according to a 10-year study by the nation's National Health and Family Planning Commission (NHFPC).
NHFPC said 86.6% of deaths in China in 2012 were due to chronic diseases. The commission said that in 2012, 25.2% of adults in China had hypertension and 9.7% had diabetes. Additionally, 30.1% were overweight and 11.9% were obese. All four rates were higher than in 2002.
The commission also evaluated lifestyle factors that contribute to disease. It said 28.1% of Chinese older than 15 were smokers, 9.3% of people aged 18 and older were excessive drinkers, and only 18.7% exercised regularly.
NHFPC said the Chinese government will take steps to prevent chronic disease, improve nutrition, and promote health education.
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