Several companies developing proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitors fell on Friday on Twitter reaction to FDA concerns about neurocognitive adverse events for the class. The agency's concerns were disclosed in SEC filings from both Sanofi (Euronext:SAN; NYSE:SNY) and Regeneron Pharmaceuticals Inc. (NASDAQ:REGN), which are partnered for anti-PCSK9 mAb alirocumab (REGN727). FDA declined to provide details, including the compound or compounds that triggered the agency's concern and whether the events occurred for anti-PCSK9 monotherapy or in combination with statins. The labels for statins contain warning language about the risk of neurocognitive adverse events.
Sanofi said the partners are not aware of a neurocognitive signal for alirocumab, which is in the Phase III ODYSSEY program. Regulatory applications are slated for next year. Regeneron was off $10.34 to $328.11 on Friday, but ended the week off $4.39.
Amgen Inc. (NASDAQ:AMGN) was off $1.93 to $122.26 on Friday. The company said it also has been in communication with FDA about its evolocumab (AMG 145) but has not seen a signal. Amgen plans to submit applications for the human mAb against PCSK9 this year. Pfizer Inc. (NYSE:PFE) said it also is not aware of a neurocognitive safety signal for its anti-PCSK9 mAb bococizumab, which is in Phase III testing.
Aegerion Pharmaceuticals Inc. (NASDAQ:AEGR) gained $5.32 (11%) to $55.53 on Friday. The company's Juxtapid lomitapide is approved in the U.S., Canada and in the EU as Lojuxta to reduce LDL-C in adults with homozygous familial hypercholesterolemia (hoFH).
LSP-Life Sciences Partners closed its Health Economics Fund (HEF) at EUR 112 million ($154.3 million), above its EUR 100 million ($137.8 million) target. LSP plans to invest EUR 5-EUR 12 million ($6.9-$16.5 million) in 10-12 private healthcare companies in Europe and North America, with a focus on companies that have products close to or on the market that produces demonstrable savings to the healthcare system. The fund will focus on diagnostics, medtech and compliance-focused companies. LSP and Dutch healthcare insurer Achmea launched the fund in 2012. Achmea is the founding investor of LSP.
LSP's first investment from the fund was in medical device company IlluminOss Medical Inc. (East Providence, R.I.), which has CE Mark approval for its IlluminOss Photodynamic Bone Stabilization System to treat bone fracture.
Drug delivery play Flamel Technologies S.A. (NASDAQ:FLML) raised $105.3 million through the sale of 10.8 million ADSs at $9.75 in a follow-on underwritten by JMP Securities; SunTrust; Ladenburg Thalmann; Roth Capital Partners; and Summer Street Research Partners. Each ADS represents one share. Flamel proposed the offering late Thursday, when its share price was $10.71.
The company's drug delivery technologies allow for the controlled release of oral drugs, oral liquid drugs, injectable biologics and small molecules, as well as tamper-resistant formulations. Flamel markets Bloxiverz neostigmine methylsulfate in the U.S. to reverse the effects of non-depolarizing neuromuscular blocking agents after surgery.
On Friday, Flamel was down $0.61 to $10.10.
Aquinox Pharmaceuticals Inc. (NASDAQ:AQXP) raised $46.2 million in an IPO through the sale of a bumped-up number of shares at the midpoint of its proposed price range. The company sold 4.2 million shares at $11, which values the company at $110.5 million. Last month, Aquinox said it planned to sell 3.7 million shares at $10-$12. Jefferies; Cowen; and Canaccord are underwriters. Aquinox was up $0.95 to $11.95 on its first day of trading Friday.
Aquinox's AQX-1125 is in the Phase II FLAGSHIP trial to treat chronic obstructive pulmonary disease (COPD), with data expected in 1Q15; and in the Phase II LEADERSHIP trial to treat bladder pain syndrome/interstitial cystitis (BPS/IC), with data expected by year end. AQX-1125 is a SH2-containing inositol 5'-phosphatase (SHIP) agonist. SHIP is an enzyme that regulates the phosphoinositide 3-kinase (PI3K) pathway specifically in blood cells.
Recro Pharma Inc. (NASDAQ:REPH) raised $30 million in an IPO through the sale of a bumped-up number of shares below its proposed price range on Friday. The company sold 3.8 million shares at $8, which values the company at $57.2 million. Last month, Recro said it planned to sell 2.5 million shares at $10-$12. Aegis Capital and Brean Capital are underwriters. Recro was up $0.30 to $8.30 on its first day of trading on Friday.
This half, Recro plans to start a Phase IIb trial of lead product, Dex-IN, to treat postoperative pain. Dex-IN is an intranasal formulation of dexmedetomidine, an adrenergic receptor alpha 2 (ADRA2) agonist. Recro has exclusive rights outside of Europe, Turkey and the Commonwealth of Independent States from Orion Corp. (HSE:ORNAV; HSE:ORNBV) to commercialize dexmedetomidine, except when administered by injection or IV.
Akebia Therapeutics Inc. (Cambridge, Mass.) amended its planned IPO on NASDAQ and is aiming to sell 4.9 million shares at $14-$17. At the $15.50 midpoint, the company would raise $76 million and be valued at $283.4 million. Akebia filed to raise up to $75 million in the offering last month. Morgan Stanley; Credit Suisse; UBS; and Nomura are underwriters.
Akebia's AKB-6548 is in a Phase IIb trial to treat anemia in patients with chronic kidney disease (CKD) who are not dependent on dialysis. Data are expected in 4Q14. The compound is an oral hypoxia-inducible factor prolyl hydroxylase (HIF-PH; EGLN) inhibitor.
Quotient Ltd. (Penicuik, U.K.) filed to raise up to $75 million in an IPO on NASDAQ underwritten by UBS; Baird; and Cowen. Quotient markets reagents for characterizing blood and serological disease screening for the transfusion diagnostics market. The company is also developing MosaiQ, a fully automated high throughput instrument for blood grouping and serological disease screening for which the company plans to start validation studies by year end. Quotient had $15.1 million in revenues for the nine months ended Dec. 31, 2013.
Verona Pharma plc (LSE:VRP) proposed to raise up to L14 million ($23.4 million) through the sale of 637.3 million shares at 2.2p in a placing and open offer. The price is a 36% discount to Verona's close of 3.45p on Thursday, before the company proposed the offering. In the open offer, shareholders are eligible to purchase a share for every eight shares held. Verona's lead program is RPL554, a long-acting phosphodiesterase-3 (PDE-3) and PDE-4 inhibitor in Phase II testing for chronic obstructive pulmonary disease (COPD) and asthma.
On Friday, Verona was off 0.40p (12%) to 3.05p.
Incyte Corp. (NASDAQ:INCY) said twice-daily oral ruxolitinib met the primary endpoint in the Phase III RESPONSE trial to treat polycythemia vera (PV). Ruxolitinib improved the proportion of patients achieving a response -- defined as both the absence of phlebotomy and a 35% or greater reduction in spleen volume -- at week 32 vs. investigators' choice of best available therapy. The open-label trial enrolled 222 PV patients resistant to or intolerant of hydroxyurea. Incyte and partner Novartis AG (NYSE:NVS; SIX:NOVN) have an SPA from FDA for the trial.
This half, the partners plans to submit an sNDA to FDA for ruxolitinib for PV, with regulatory applications in the EU and Japan also slated for this year. The oral Janus kinase-1 (JAK-1) and JAK-2 inhibitor is already approved in the U.S. as Jakafi for intermediate or high-risk myelofibrosis (MF), including primary MF and post PV/essential thrombocythemia myelofibrosis (PPV-MF/PET-MF). The drug is approved in the EU as Jakavi for disease-related splenomegaly or symptoms in adults with chronic idiopathic MF, PPV-MF or PET-MF. Novartis has exclusive, ex-U.S. rights to ruxolitinib from Incyte under a 2009 deal.
Incyte was off $1.25 to $64.36 on Friday.
NewLink Genetics Corp. (NASDAQ:NLNK) fell $6.11 (16%) to $31.60 on Friday after an independent DSMC said the Phase III IMPRESS trial evaluating NewLink's HyperAcute Pancreas algenpantucel-L for surgically resected pancreatic cancer did not meet the predefined statistical threshold to stop the trial early. The DSMC recommended continuing the trial based on a planned interim analysis following 222 patient deaths; a second interim analysis is scheduled when there are 333 patient deaths. NewLink has an SPA from FDA for the open-label trial, which is evaluating chemotherapy with and without HyperAcute Pancreas. The primary endpoint is overall survival (OS).
HyperAcute is an allogeneic cancer vaccine containing irradiated pancreatic tumor cells modified to express alpha 1,3- galactosyltransferase. The product is also in the Phase III PILLAR trial for borderline resectable or locally advanced unresectable pancreatic cancer.
Takeda Pharmaceutical Co. Ltd. (Tokyo:4502) submitted an NDA to Japan's Ministry of Health, Labor and Welfare (MHLW) for trelagliptin (SYR-472) to treat Type II diabetes. The once-weekly oral dipeptidyl peptidase-4 (DPP-4) inhibitor is in Phase II testing in the U.S. and EU.
MolMed S.p.A. (Milan:MLM) submitted an MAA to EMA seeking conditional approval of TK as adjunctive treatment for high-risk leukemia patients undergoing hematopoietic stem cell transplantation (HSCT). MolMed said TK -- which comprises donor lymphocytes engineered to express the human herpes simplex thymidine kinase suicide gene -- allows patients to undergo HSCT from a mismatched donor without post-transplant immunosuppression. The company is seeking conditional approval based on data from the Phase I/II TK007 trial, which showed TK reduced transplant-related mortality and increased overall survival in patients with a mismatched HSCT donor, as well as initial data from the ongoing Phase III TK008 trial.
MolMed was up EUR 0.04 to EUR 0.74 on Friday.
Rep. Renee Ellmers (R-N.C.) introduced in the U.S. House of Representatives a bill that would prevent HHS from implementing changes to Medicare Part D that are contained in a proposed rule issued by CMS in January. The proposed changes include eliminating two of the six protected classes for which plans must provide "substantially all" approved drugs and limiting the number of plans a given insurance company can offer in a single region. The bill, H.R. 4160, has been referred to the House Energy and Commerce and Ways and Means Committees. Comments on CMS's proposed rule are due Friday.
Last month, patient groups told BioCentury the proposed changes to protected drug classes would reduce access to necessary medications for transplant and mental illness. Healthcare policy specialists and consultants said they are worried the proposed changes would reduce competition and eliminate preferred pharmacy networks and the discounts they provide (see BioCentury, Feb. 17).
Legislators have continued to pressure CMS Administrator Marilyn Tavenner and HHS Secretary Kathleen Sebelius to withdraw or not implement the proposed changes, as have organizations including patient groups, health insurers and the Biotechnology Industry Organization (BIO) and Pharmaceutical Research and Manufacturers of America (PhRMA). Some legislators and patient groups support a number of the proposed changes, however, including the proposed expansion of preferred pharmacy networks (see BioCentury Extra, Feb. 26).
Douglas Hoey, CEO of the National Community Pharmacists Association; Chris Holt, director of health care policy at the American Action Forum; Edith Rosato, CEO of the Academy of Managed Care Pharmacy; and Andrew Sperling, director of federal legislative advocacy at the National Alliance on Mental Illness will discuss the proposed Part D changes on the March 9 edition of BioCentury This Week television. The program will be broadcast at 8:30 a.m. in Washington, D.C., on WUSA, channel 9. It will be available at www.BioCenturytv.com beginning at 9 a.m., and also will be broadcast on selected PBS stations.