Investors added $1 billion in market cap to Avanir Pharmaceuticals Inc. (NASDAQ:AVNR) on Monday after the company's AVP-923 dextromethorphan/quinidine met the primary endpoint vs. placebo of reducing agitation in a Phase II trial to treat patients with Alzheimer's disease (AD).
In a 220-patient study, 30/10 mg oral dextromethorphan/quinidine twice daily reduced scores on the agitation/aggression subscale of the Neuropsychiatric Inventory (NPI) after 10 weeks (p=0.00008). There are currently no FDA-approved treatments for the indication.
AVP-923 also improved most secondary endpoints vs placebo, including NPI total score (p=0.014), clinical global impression of change-agitation (p=0.0003), patient global impression of change (p=0.001) and strain on caregivers (p=<0.05). Data will be presented at the American Neurological Association's meeting in Baltimore on Oct. 12-14.
The company plans to meet with FDA to discuss pivotal trial designs, as well as the possibility of an SPA.
AVP-923 combines the NMDA receptor antagonist dextromethorphan with quinidine sulfate, a cytochrome P450 2D6 enzyme inhibitor.
Avanir markets the compound in a different dosage as Nuedexta in the U.S. for pseudobulbar affect (PBA), which is characterized by involuntary episodes of laughing and/or crying. It is dosed at 20/10 mg once daily for seven days and then twice daily as maintenance therapy thereafter. The compound also is in Phase II testing to treat Parkinson's disease levodopa-induced dyskinesia (PD-LID).
Avanir closed up $5.75 (85%) to $12.49 on Monday to a market cap of $2.1 billion.
NPS Pharmaceuticals Inc. (NASDAQ:NPSP) lost $5.42 (17%) to $27.29 on Monday after FDA's Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC) recommended approval of Natpara for hypoparathyroidism by an 8-5 vote Friday. Trading of NPS was halted on Friday.
In briefing documents released ahead of the meeting, FDA reviewers concluded that once-daily subcutaneous injections of Natpara reduced the need for calcium and vitamin D supplements. However, reviewers raised concerns about both hypocalcemia and hypercalcemia associated with Natpara's use, and noted that osteosarcoma rates in Natpara-treated rats were similar to those seen with Forteo teriparatide, a PTH fragment from Eli Lilly and Co. (NYSE:LLY) approved for osteoporosis that has a black-box warning about osteosarcoma risk and is not indicated for use beyond two years (see BioCentury Extra, Sept. 10).
On NPS's conference call to discuss the panel results, one analyst asked whether the company expected Natpara's label to contain a black box related to osteosarcoma and a two-year cap on its use. EVP and CMO Roger Garceau said the black box issue is in FDA's hands but did say that "we clearly and I think the panel members clearly stated that this is lifelong therapy."
Gilead Sciences Inc. (NASDAQ:GILD) signed non-exclusive licensing agreements allowing seven Indian generics companies to manufacture its chronic HCV drugs sofosbuvir and sofosbuvir/ledipasvir for distribution in 91 developing countries.
Licensees will be able to set their own prices for the generics and pay a 7% royalty to Gilead. The agreements allow the generics companies to manufacture other combination therapies including sofosbuvir or ledipasvir.
Licensees include the Mylan Laboratories Ltd. subsidiary of Mylan Inc. (NASDAQ:MYL); Cipla Ltd. (Mumbai, India); Hetero Drugs Ltd. (Hyderabad, India); Ranbaxy Laboratories Ltd. (NSE:RANBAXY; BSE:500359); Cadila Pharmaceuticals Ltd. (Ahmedabad, India); Sequent Scientific Ltd. (Karnataka, India); and Strides Arcolab Ltd. (BSE:532531; NSE:STAR).
In August, Gilead said it would make its branded version of sofobuvir, Sovaldi, available in India and Egypt for about $900 for a 12-week course, which the company said is its standard low-income public pricing structure for the drug (see BioCentury Extra, August 7).
Gilead closed down $2.67 to $100.99 on Monday.
The Drug Controller General of India (DCGI) approved manufacturing and marketing of autoimmune therapy Infimab infliximab from Epirus Biopharmaceuticals Inc. (NASDAQ:EPRS).
Infimab, formerly BOW015, is India's first approved biosimilar of Remicade from Johnson & Johnson (NYSE:JNJ) and Merck & Co. Inc. (NYSE:MRK). The approval is Epirus' first anywhere.
J&J and Merck market Remicade worldwide to treat inflammatory diseases including rheumatoid arthritis (RA), Crohn's Disease, ankylosing spondylitis, ulcerative colitis, psoriatic arthritis and psoriasis.
In January, Epirus granted Ranbaxy Laboratories Ltd. (NSE:RANBAXY; BSE:500359) commercialization rights to Infimab in India and other Southeast Asian and North African markets. Epirus and Ranbaxy expect to launch Infimab by 1Q15.
Epirus also partnered with Orygen Biotecnologica Ltd. last October to develop and commercialize the drug candidate in Brazil (see BioCentury, April 21).
Epirus plans to begin an additional Phase III trial of Infimab in Europe in early 2015.
Epirus was off $0.12 to $8.66 on Monday.
GlaxoSmithKline plc (LSE:GSK; NYSE:GSK) exercised its option to license drug candidates discovered as part of a 2010 deal with Five Prime Therapeutics Inc. (NASDAQ:FPRX). GSK took exclusive rights to an undisclosed muscle disease program, discovered using the biotech's library of human extracellular proteins and target screening and discovery technologies.
GSK will have exclusive rights to develop and commercialize the programs globally, at its own cost. Five Prime will receive a $1.5 million payment triggered by the exercise and is entitled to up to $122.5 million in milestones as well as single digit royalties. It received $15 million upfront as well as research funding from the pharma.
Five Prime has ongoing deals with UCB Group (Euronext:UCB) and Bristol-Myers Squibb Co. (NYSE:BMY), as well as a second option deal with GSK to identify targets relevant to refractory asthma and chronic obstructive pulmonary disease (COPD) (see BioCentury Extra, April 17, 2012).
Five Prime closed down $0.29 to $11.51 on Monday. The news was announced after market closing.
FDA approved HyQvia from Baxter International Inc. (NYSE:BAX) and Halozyme Therapeutics Inc. (NASDAQ:HALO) to treat adults with primary immunodeficiency (PI).
HyQvia is a subcutaneous formulation of Gammagard, an IgG antibodies plasma-based therapy from Baxter, and recombinant human PH20 hyaluronidase (rHuPH20) from Halozyme. The two companies partnered in 2007.
Baxter said HyQvia gives PI patients the option of monthly self-administration or administration by a professional, while current subcutaneous immunoglobulin treatments require weekly or bi-weekly treatment with multiple infusion sites per treatment.
Baxter said it plans to launch HyQvia in the coming weeks, but declined to discuss the drug's price.
Baxter said Baxalta, the biopharmaceutical company it will create next year when Baxter splits in two, will hold its Hyqvia rights.
Halozyme was down $0.41 to $8.99 on Monday, while Baxter was off $0.15 to $74.55.
Scholar Rock LLC (Cambridge, Mass.) raised $20 million in a series A round led by new investor ARCH Venture Partners. Founding investors Polaris Partners and Timothy Springer joined new investors EcoR1 Capital and The Kraft Group in the round.
Scholar Rock is developing biologics that modulate the activation of growth factors in disease microenvironments. The company is investigating preclinical programs to treat fibrosis, musculoskeletal diseases and autoimmune disorders.
Scholar Rock also hired Elan Ezickson as its first COO and head of corporate development. Ezickson formerly was EVP and COO of Aveo Pharmaceuticals Inc. (NASDAQ:AVEO).
In January, the Boston Innovation Center of Johnson & Johnson (NYSE:JNJ) and J&J's Janssen Biotech Inc. unit partnered with Scholar Rock to discover and develop niche activators targeting transforming growth factor (TGF) beta 1 (TGFB1) for autoimmune diseases and cancer (see BioCentury, Jan. 13).
Civitas Therapeutics Inc. (Chelsea, Mass.) and EyeGate Pharmaceuticals Inc. (Waltham, Mass.) each set terms for their proposed IPOs on NASDAQ.
Civitas plans to sell 5 million shares at $14-$16. At $15, Civitas would raise $75 million and be valued at $363.2 million. The company filed to raise up to $86.3 million last month. Underwriters are J.P. Morgan; BofA Merrill Lynch; Cowen; and Oppenheimer. Civitas plans to start Phase III testing of CVT-301 to treat motor fluctuations associated with Parkinson's disease (PD) early next year, with a potential NDA submission by the end of 2016. CVT-301 is an inhaled formulation of levodopa delivered via Civitas' Arcus inhalation technology.
EyeGate plans to sell 1.9 million shares at $12-$14. At $13, EyeGate would raise $25 million and be valued at $91.7 million. The company filed to raise up to $28.8 million in July. Aegis is underwriting the offering. This half, EyeGate plans to start a second pivotal trial of EGP-437, a dexamethasone phosphate ophthalmic solution delivered by the EyeGate II Ocular Drug Delivery System, to treat non-infectious anterior segment uveitis. The company expects to complete the trial in 1H16.
NeuroDerm Ltd. (Rehovot, Israel) filed to raise up to $65 million in an IPO on NASDAQ underwritten by Jefferies; Cowen; Oppenheimer; and Roth Capital Partners.
NeuroDerm's lead program is ND0612L, a low-dose liquid formulation of levodopa and carbidopa administered by a subcutaneous belt pump device that is in an Israeli Phase IIa trial to treat moderate Parkinson's disease (PD). The company also plans to begin testing a high-dose version for severe PD. The company has submitted an IND application to FDA for both versions.
Janet Woodcock, director of FDA's Center for Drug Evaluation and Research, announced in an internal memo that the agency hired Grail Sipes as director of CDER's Office of Regulatory Policy (ORP), effective Sept. 22.
Prior to joining CDER in 2011 as senior regulatory counsel in ORP, Sipes was a partner with Covington & Burling LLP and a member of the firm's food and drug practice and life sciences industry groups.
Acting ORP director Nancy Hayes will return to her prior role as director of ORP's division of Regulatory Policy I.
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