The ultra-rare disease creatine transporter deficiency (CTD) wreaks havoc on its patient population of young boys. The x-linked autism spectrum disorder prevents the transport of creatine – a nitrogenous organic acid that serves as fuel for the body’s cells – into the brain, causing patients to suffer seizures, speech delay, behavioral abnormalities and severe cognitive impairment.
Shares of Raptor Pharmaceutical Corp. (NASDAQ:RPTP) hit a 52-week high of $17.69 Thursday after the company reported encouraging top-line results from a planned 18-month analysis of an ongoing, three-year phase II/III trial of RP103 (delayed-release cysteamine) in Huntington’s disease (HD).
Based on its name, Longevity Biotech Inc. might be mistaken for a company seeking a prescription for aging. In a way, that’s true, admitted Scott Shandler, president and CEO, since the company is building a platform designed to help patients to live longer and healthier lives. But the name also is a bit of a double entendre, alluding to the company’s technical ability to improve the half-life of molecules of interest.
Versartis Inc., which holds worldwide rights to the long-acting recombinant human growth hormone (rHGH) VRS-317, last year suggested it could potentially move the treatment for growth hormone deficiency (GHD) all the way to approval and marketing on its own.
Signaling the RNAi space remains a hot ticket, Arrowhead Research Corp. priced an underwritten offering of 5.5 million shares of common stock at $18.95 apiece, seeking to raise $104.225 million. The Pasadena, Calif.-based company granted the underwriters a 30-day option to purchase up to an additional 825,000 shares, potentially increasing the deal size by $15.6 million.
In the eight years since Berg Pharma LLC was co-founded by billionaire businessman, venture capitalist and real estate mogul Carl Berg, scientist Niven R. Narain, who serves as the company’s president and chief technology officer, and Berg LLC managing director and investor Mitch Gray, the company has quietly amassed an enormous portfolio of early stage drug candidates and diagnostics across multiple indications, with the largest concentration in cancer.
Investors raised their wallets Thursday for Concert Pharmaceuticals Inc., which priced its initial public offering (IPO) at $14 – the top of its proposed range – after previously upsizing from 5 million to 6 million shares. The stock swelled above the offering price as trading commenced on the Nasdaq Global Market under the ticker “CNCE,” hitting $16.24 before settling back to close at $14.18 for a gain of 18 cents on the day.
The acquisition of Cadence Pharmaceuticals Inc. by Mallinckrodt plc for $14 per share in cash, or approximately $1.3 billion on a fully diluted basis, demonstrated that a single asset can still beat the drums when it comes to deal terms.
Frank Yu wasn’t the most heralded participant at last month’s J.P. Morgan Healthcare (JPM) Conference in San Francisco, the giant partnering confab that draws biotechs, medtechs, pharmas and investors from around the world. Instead, Yu, founder and CEO of the $500 million Hong Kong private equity fund Themes Investment Partners and of the investment firm Ally Bridge Group, preferred to stay under the radar while assessing the pulse of the life sciences industry.
