Amicus Therapeutics Inc. (NASDAQ:FOLD) shed $7.36 (53%) to $6.39 on Friday, losing all of its 2015 gains, after the company said it no longer planned to submit an NDA to FDA by YE15 for Amigal migalastat to treat Fabry's disease.
Last month, Amicus said it was on track to submit an NDA in 4Q15 requesting accelerated approval. On a conference call Friday, Chairman and CEO John Crowley said FDA is requesting data analyses that will take several months to complete.
Amicus had planned to submit data from the Phase III FACETS (Study 011) trial as the sole basis for accelerated approval, supplemented by data from the Phase III ATTRACT (Study 012) trial. However, Amicus said FDA has asked the company to integrate data from the two trials and conduct further analyses, which Amicus intends to complete by 1H16.
The company has not disclosed a new NDA timeline. If Amicus continues to pursue accelerated approval of Amigal, Crowley said it will likely be required to conduct a Phase IV gastrointestinal study in Fabry's disease patients.
Crowley said FDA also is open to considering Amigal for full approval, though the requirements needed to pursue the full approval pathway are "subject to further discussion."
The small molecule that enhances alpha galactosidase A activity is under accelerated assessment in the EU, where it is known as Galafold. Crowley said the European market represents about 70% of the commercial opportunity for the candidate (see BioCentury Extra, June 26).
FDA granted accelerated approval to Keytruda pembrolizumab from Merck & Co. Inc. (NYSE:MRK) to treat advanced squamous and non-squamous non-small cell lung cancer (NSCLC) in previously treated patients, though the label is limited to patients with PD-L1-positive tumors.
The approval was based on data from patients in the Phase Ib KEYNOTE-001 trial who demonstrated PD-L1 expression in at least 50% of tumor cells.
Among the first 394 patients treated in KEYNOTE-001 who had varying levels of PD-L1 expression, Keytruda led to an overall response rate (ORR) of 18%. Among those who expressed PD-L1 expression in at least 50% of tumor cells, the ORR was 41%.
Competing PD-1 inhibitor Opdivo nivolumab from Bristol-Myers Squibb Co. (NYSE:BMY) is approved to treat metastatic squamous NSCLC in patients who have progressed on or after platinum-based chemotherapy, regardless of PD-L1 expression levels.
Opdivo is under Priority Review to treat non-squamous NSCLC in previously treated patients; the PDUFA date is Jan. 2. In the Phase II CheckMate -057 trial of Opdivo in non-squamous NSCLC patients, PD-L1 status appeared to correlate with benefit. Opdivo did not show a benefit over docetaxel in patients with little or no PD-L1 expression (see BioCentury Extra, June 1).
Both drugs are also approved to treat melanoma. FDA approved Keytruda last year for melanoma patients previously treated with BMS's Yervoy ipilimumab. Opdivo is approved in the U.S. in combination with Yervoy to treat BRAF V600 wild-type unresectable or metastatic melanoma, and as a monotherapy to treat advanced melanoma in patients previously treated with Yervoy (see BioCentury Extra, Oct. 1).
In parallel with Friday's Keytruda approval, FDA granted premarket approval to the PD-L1 IHC 22C3 pharmDx test, a companion diagnostic from the Dako A/S subsidiary of Agilent Technologies Inc. (NYSE:A).
Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) said EMA accepted for review an MAA for Ace-ER (UX001) to treat GNE myopathy, a muscle-wasting disorder also known as hereditary inclusion body myopathy.
The company is seeking conditional approval for Ace-ER based on Phase II data. It expects data in early 2017 from an ongoing Phase III trial of the extended-release oral formulation of aceneuramic acid. The Phase III trial is designed to satisfy EMA's requirement for a confirmatory trial and to serve as the basis for an NDA submission. President and CEO Emil Kakkis declined to give a timeline for the submission.
Ace-ER has Orphan Drug designation in the U.S. and EU for HIBM. Ultragenyx has exclusive rights to the compound outside of Japan and other Asian territories from Nobelpharma Co. Ltd. (Tokyo, Japan).
Ultragenyx gained $5.71 to $104.69 on Friday.
The Sandoz unit of Novartis AG (NYSE:NVS;SIX:NOVN) said FDA accepted for filing a BLA for biosimilar etanercept (GP2015). The company is pursing approval in all indications approved for reference product Enbrel etanercept from Amgen Inc. (NASDAQ:AMGN). Novartis spokesperson Eric Althoff declined to disclose an FDA user action date for the tumor necrosis factor (TNF) alpha inhibitor.
Enbrel is approved to treat rheumatoid arthritis, polyarticular juvenile idiopathic arthritis, psoriatic arthritis, ankylosing spondylitis and plaque psoriasis. Althoff would not name the indications in which Sandoz had conducted clinical trials of the biosimilar, but said "psoriasis is the most sensitive indication for the evaluation of biosimilarity."
In 2013, Sandoz sought a declaratory judgment of non-infringement against two of Amgen's Enbrel patents, which are set to expire in 2028 and 2029. A district court ruled against Sandoz, finding that the company could not seek a judgment before it submitted an application to FDA; a federal appeals court dismissed an appeal on procedural grounds. Althoff declined to comment on legal issues (see BioCentury Extra, Dec. 8, 2014).
FDA first approved Enbrel in 1998. The drug is a recombinant p75 tumor necrosis factor (TNF) receptor linked to the Fc portion of Human IgG1 (TNFr:Fc).
XenoPort Inc. (NASDAQ:XNPT) gained $0.84 (24%) to $4.32 after the company said it is replacing its CEO and plans to partner XP23829 and other pipeline candidates to focus on commercializing Horizant gabapentin enacarbil.
EVP and COO Vincent Angotti succeeds Ronald Barrett, who retired as CEO and director. Angotti has led Horizant's commercialization. The company will also cut about 25 jobs, including positions related to the XP23829 program.
Horizant is approved in the U.S. to treat restless legs syndrome (RLS) and to manage postherpetic neuralgia. NIH's National Institute on Alcohol Abuse and Alcoholism (NIAAA) is conducting a Phase II trial of Horizant to treat alcohol use disorder.
XenoPort regained U.S. commercialization rights for Horizant from former partner GlaxoSmithKline plc (LSE:GSK; NYSE:GSK) and re-launched the drug in the U.S. in 2013. The company reported $20.2 million in U.S. Horizant sales in 2014.
Astellas Pharma Inc. (Tokyo:4503) markets the transported prodrug of gabapentin in Japan as Regnite.
XenoPort remains 36% below its close of $6.73 on Sept. 14, before the company reported data from a Phase II trial of XP23829 to treat moderate to severe chronic plaque-type psoriasis. While two doses of XP23829 met the primary endpoint, the high incidence of adverse events (AEs) in the trial sent shares tumbling. XP23829 is an oral prodrug of monomethyl fumarate (MMF) that induces and activates the nuclear factor (erythroid-derived 2)-like 2 (NFE2L2; NRF2) pathway (see BioCentury Extra, Sept. 15).
Bayer AG (Xetra:BAYN) promoted Habib Dable to president of its U.S. pharmaceuticals division, Bayer HealthCare Pharmaceuticals. Dable was global head of specialty medicine for Bayer HealthCare Phamaceuticals.
Molecular diagnostics play Myriad Genetics Inc. (NASDAQ:MYGN) named Jan Schluechter chief commercial officer of its Myriad Genetics GmbH international subsidiary, effective Oct. 15. Schluechter was global head of key account management at Novartis AG (NYSE:NVS; SIX:NOVN).
Cancer company Mission Therapeutics Ltd. (Cambridge, U.K.) named David Luther CFO. He was VP of finance for commercial and supply chain operations in U.K., Ireland and Italy at Spotless Group S.A.S. (Neuilly-sur-Seine, France).
Microbiome play Synthetic Biologics Inc. (NYSE-M:SYN) named Raymond Stapleton SVP of manufacturing. Stapleton was executive director of global vaccine technology and engineering at Merck & Co. Inc. (NYSE:MRK).
Generics and biosimilars company Alvogen Inc. (Pinebrook, N.J.) named Graham Baker CFO. Baker was VP of finance for international business at AstraZeneca plc (LSE:AZN; NYSE:AZN).
Oncology play Varian Medical Systems Inc. (NYSE:VAR) named Rafael Torres SVP of business development and strategy. Torres was global head of healthcare at GE Ventures and GE Equity.
Advanced Accelerator Applications S.A. (Saint Genis Pouilly, France) filed to raise up to $75 million in an IPO of ADSs on NASDAQ underwritten by Citigroup; Jefferies; Canaccord; and JMP Securities. In February, the company withdrew a planned IPO in which it sought to raise $75 million.
In June, Advanced Accelerator raised EUR 23 million ($26 million) in a crossover round (see BioCentury, July 13).
The company expects to submit regulatory applications to FDA and EMA by 1Q16 for Lutathera (177Lu-DOTA0-Tyr3-octreotate) to treat midgut neuroendocrine tumors. FDA already has granted Priority Review to an NDA for the company's Somakit, a companion PET diagnostic for Lutathera.
Last month, Advanced Accelerator said the radiolabeled somatostatin analog that targets somatostatin receptors met the primary endpoint in the Phase III NETTER-1 study, significantly improving progression-free survival (PFS) compared with a double dose of Sandostatin LAR Depot octreotide acetate. Lutathera has Orphan Drug designation from FDA and EMA and Fast Track designation from FDA to treat neuroendocrine tumors. Somakit also has Orphan Drug designation from FDA and EMA.
Karen Midthun will retire at YE15 as director of FDA's Center for Biologics Evaluation and Research, FDA spokesperson Tara Goodin told BioCentury. The agency is seeking a replacement and will collect applications through Oct. 21, according to a job posting.
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