Zafgen Inc. (Cambridge, Mass.) filed to raise up to $86.3 million in an IPO on NASDAQ underwritten by Leerink; Cowen; Canaccord; and JMP Securities. Zafgen is developing beloranib, a subcutaneous formulation of a methionine aminopeptidase 2 (MetAP2) inhibitor. This year, Zafgen plans to start Phase III testing to treat Prader-Willi syndrome, a severe form of genetic obesity with a prevalence of about 5,000-7,000 people in the U.S. Next half, Zafgen plans to start a Phase IIb trial in severely obese patients.
Zafgen has exclusive, worldwide rights to develop and commercialize beloranib outside of Korea from Chong Kun Dang Pharmaceutical Corp. (KSE:001630). On Thursday, Zafgen drew down $7.5 million of a new, $20 million senior credit facility with Oxford Finance and MidCap Financial.
FDA's Bob Rappaport said the agency's review team has been "unable to conclude that there is a safety benefit" for MoxDuo IR morphine/oxycodone from QRxPharma Ltd. (ASX:QRX; OTCQX:QRXPY), which is under review to treat moderate to severe acute pain. The comments from Rappaport, who is director of the Division of Anesthesia and Analgesia and Addiction Drug Products at FDA's Center for Drug Evaluation and Research, came in briefing documents released ahead of Tuesday's meeting of the Anesthetic and Analgesic Drug Products Advisory Committee to discuss the immediate-release combination of oxycodone and morphine.
Rappaport noted that FDA has never approved a combination product comprising two drugs from the same pharmacologic class, which he said is "contrary to the purpose" of the combination rule, which aims to "assure that the combination provides some benefit to patients that could not be obtained by prescribing the individual components alone." QRxPharma is arguing that MoxDuo IR has a clear overall safety benefit, including a respiratory safety benefit, compared with either oxycodone or morphine.
The PDUFA date for MoxDuo IR is May 25. FDA issued a complete response letter for the product in August. The Australian and U.S. markets were closed on Friday (see BioCentury Extra, Aug. 28, 2013).
FDA's Oncologic Drugs Advisory Committee will meet on June 25 to discuss an NDA from AstraZeneca plc (LSE:AZN; NYSE:AZN) for olaparib as maintenance treatment of platinum-sensitive relapsed ovarian cancer. The pharma said it submitted the NDA this year, but declined to disclose when it did so or the PDUFA date. The poly(ADP-ribose) polymerase (PARP) inhibitor is also under review in Europe.
The NDA covers use of olaparib in patients with germline breast cancer early onset (BRCA) mutation as detected by an FDA-approved test. Earlier this month, Myriad Genetics Inc. (NASDAQ:MYGN) submitted to FDA the first of four modules of a PMA for BRACAnalysis test as a companion diagnostic for olaparib.
Myriad markets BRACAnalysis to assess a woman's risk of developing breast or ovarian cancer based on detecting genetic mutations in the BRCA1 and BRCA2 genes.
The American College of Cardiology and American Heart Association are the latest physician groups to integrate value assessments for treatments into clinical practice guidelines and performance measures. In a joint statement published in Circulation and the Journal of the American College of Cardiology, the groups cited the "need for more explicit and transparent assessment" of value and proposed using quality-adjusted life year (QALY) measures to sort treatments into "level of value" categories. The groups -- which currently only explicitly consider clinical efficacy and outcomes -- did not provide specifics or a time frame for implementing the change.
According to a June analysis published in JAMA Internal Medicine, 17 of 30 U.S. physician groups already explicitly account for costs and value when drafting clinical guidelines based on the methodological statements for their clinical guidance documents. Four groups implicitly integrate costs and three intentionally exclude costs, according to the analysis.
Last month, American Society of Clinical Oncology CMO Richard Schilsky told BioCentury the organization plans to release a scorecard this year that determines the value of drugs based on benefit, toxicity and cost (see BioCentury, March 24).
In an article published in the New England Journal of Medicine, researchers at NIH's National Cancer Institute made several proposals on how to fund comparative effectiveness trials for expensive new cancer drugs vs. cheaper drugs. The researchers noted that high prices protect market share because they preclude "comparative effectiveness trials that would seek to establish equally effective but cheaper alternatives."
For instance, the researchers estimated that the purchase price for prostate cancer drug Zytiga abiraterone acetate from Johnson & Johnson (NYSE:JNJ) for a single non-inferiority trial comparing Zytiga with the generic ketoconazole could be almost $70 million. The researchers identified ketoconazole as a logical comparator because the drug has a similar mechanism of action to Zytiga and was used off-label for prostate cancer prior to Zytiga's approval. Both drugs inhibit cytochrome P450 17 alpha-hydroxylase/C17, 20 lyase (CYP17).
To fund comparative effectiveness trials, the NCI researchers suggested collaborations among stakeholders like NCI's Cancer Therapy Evaluation Program, oncologic cooperative groups and payers. The researchers said companies also could provide their drugs free of cost to investigators, noting that, "if these drugs were true advances, companies could benefit from formal demonstration of that fact." Finally, the NCI researchers said comparative effectiveness trials could use study designs that allow a patient's insurance to cover the cost of the newer drug so that only the comparator has to be purchased.
The U.S. Department of Veteran Affairs recommended regimens containing Sovaldi sofosbuvir from Gilead Sciences Inc. (NASDAQ:GILD) and Olysio simeprevir from Johnson & Johnson (NYSE:JNJ) for most HCV patients, but said it would be "reasonable" for doctors to defer treatment for some patients until newer therapies are available. According to new treatment guidelines released by the VA, physicians should take into account stage of liver fibrosis and whether a patient has cirrhosis or hepatocellular carcinoma (HCC) when deciding whether to wait for new interferon-free and ribavirin-free regimens, which the VA said are expected to be safer and less complex to administer and are expected to be approved in the next 12-24 months.
The guidelines break out recommended treatment regimens by HCV genotype, whether patients are treatment-naïve or treatment-experienced, cirrhotic or non-cirrhotic and interferon-eligible. The guidelines note that the VA will "use the optimal drug treatments available, after analysis of efficacy, safety, and costs." In FY13, the VA provided health insurance to 8.9 million people.
Private payers are currently covering Sovaldi and Olysio, but told BioCentury that as they develop formal coverage policies, they would consider restricting access to advanced patients with cirrhosis or liver disease (see BioCentury, March 24).
Mapi-Pharma Ltd. (Ness Ziona, Israel) amended its IPO on NASDAQ and now plans to sell 2.9 million shares at $13-$15. At the $14 midpoint, the company would raise $40 million and be valued at $215 million. Aegis Capital is underwriter. Mapi-Pharma filed to raise up to $46 million in the offering last month.
Mapi-Pharma develops generics and complex APIs. The company also develops life cycle management products that integrate off-patent APIs with its formulations to create a final combination expected to be patentable.
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