As FDA digests comments from last week's next-generation sequencing (NGS) workshop, Elizabeth Mansfield, the deputy office director for personalized medicine at the agency's Center for Devices and Radiological Health (CDRH), told BioCentury it could take a year or two to acquire the expertise and develop the tools required to set up a regulatory pathway for NGS tests.
The workshop included representatives from industry, academia, government and patient advocacy groups who discussed FDA's approach to developing performance, method and material standards that laboratories could use to demonstrate analytical performance. Stakeholders also discussed the development of criteria for curated, third-party genetic databases that would include information on genetic variants and their link to disease to establish the clinical validity of NGS tests (see BioCentury Extra, Feb. 18).
Mansfield noted that a challenge FDA will face as it moves forward with NGS regulation is ensuring that the tools and standards developed can be easily implemented by laboratories. She added that panelists were concerned with how FDA would then assess compliance with established standards. Public comments on the NGS workshop are open until March 20.
The Obama administration's FY16 budget includes a request for $9.7 million for FDA to develop NGS standards and curated genetic databases as part of the broader Precision Medicine Initiative.
Mansfield said FDA will continue to conduct premarket review of applications for NGS tests in the meantime. The MiSeqDx genome sequencing system from Illumina Inc. (NASDAQ:ILMN) was approved under FDA's de novo pathway in 2013, which the agency established for new types of devices that are lower risk and may not require a PMA but would not be cleared via the 510(k) pathway. MiSeqDx is still the only high throughput next-generation genomic sequencer approved by the agency for broad clinical use (see BioCentury Extra, Nov. 19, 2013).
The Sandoz unit of Novartis AG (NYSE:NVS; SIX:NOVN) filed an opposition brief to a motion by Amgen Inc. (NASDAQ:AMGN) seeking a preliminary injunction that would prevent Sandoz from marketing Zarxio (EP2006), a biosimilar of Amgen's Neupogen filgrastim methionyl human G-CSF.
Zarxio's earliest potential launch date is March 8, but Sandoz has agreed not to launch the biosimilar in the U.S. until the earlier of April 10 or a court ruling in favor of Sandoz. In the latter scenario, Sandoz would provide Amgen five days' notice before launching Zarxio.
Amgen is seeking a preliminary injunction as well as a partial judgment stating that Sandoz violated the Biologics Price Competition and Innovation Act of 2009 (BPCIA) when it refused to provide its BLA and notified Amgen prematurely of its plans to launch Zarxio (see BioCentury Extra, Feb. 10).
In its opposition, Sandoz argued that Amgen's motion fails to meet standards for granting a preliminary injunction. First, Sandoz alleges Amgen's interpretation of the patent resolution protocol laid out by the BPCIA would extend its exclusivity period from 12 years to 12.5 years, against "Congress's intent." Next, Sandoz claims Amgen cannot show irreparable harm since Amgen allegedly refused Sandoz's offers to provide its BLA under "confidentiality protections." Third, Sandoz argues that the "balance of equities" is in its favor, since an injunction would threaten its "years of effort and tens of millions of dollars" while a denial of the injunction "would not impose any undue hardship on Amgen." Lastly, Sandoz said Amgen's request for an injunction does not serve the public interest of access to discounted biosimilars.
A cascade of litigation between the companies started after FDA accepted in July Sandoz's BLA for Zarxio, the first disclosed BLA the agency had accepted for a biosimilar candidate. Amgen followed with a lawsuit and Citizen's Petition alleging that Sandoz refused to comply with BPCIA when it filed the BLA for Zarxio (see BioCentury Extra, Nov. 3, 2014).
In January, an FDA advisory committee voted 14-0 to recommend approval of Zarxio (see BioCentury Extra, Jan. 7).
AstraZeneca plc (LSE:AZN; NYSE:AZN) said it will spin out its anti-infectives unit into a subsidiary company based in Waltham, Mass. AZ will invest $40 million in the subsidiary and expects it to be fully operational in early May.
The as-yet-unnamed subsidiary will have 20-30 staff, including employees from AZ's Innovative Medicines and Early Development (iMED) unit. Manos Perros, head of infection in the iMED unit, will lead the subsidiary's formation and staffing.
The subsidiary will focus exclusively on developing early-stage antibiotics, including AZD0914, a benzisoxazole DNA gyrase inhibitor against Neisseria gonorrhoeae that is in Phase II testing. The compound has Qualified Infectious Disease Product (QIDP) and Fast Track designations to treat uncomplicated gonorrhea. AZ said the decision does not affect marketed products, late-stage small molecule infection programs or the biologic anti-infectives porfolio in the MedImmune LLC unit.
AstraZeneca said it plans to focus on its core therapeutic areas: oncology; cardiovascular and metabolic diseases; and respiratory, inflammation and autoimmune diseases.
On Wednesday, FDA approved AZ's Avycaz ceftazidime/avibactam to treat complicated urinary tract infections (cUTIs) and in combination with metronidazole to treat intra-abdominal infections (cIAIs) in patients who have few or no alternative treatment options. Actavis plc (NYSE:ACT) has rights to commercialize the drug in North America; AZ has rights elsewhere (see BioCentury Extra, Feb. 25).
On Friday, AZ shed $0.11 to $68.90 in New York and gained 4.50p to 4,469.50p in London.
WuXi PharmaTech Inc. (NYSE:WX) said FDA granted breakthrough designation to ibalizumab (TMB-355) from its partner Taimed Biologics Inc. (GreTai:4147) to treat HIV.
TaiMed has completed a Phase IIb trial of IV ibalizumab and a Phase I trial of a subcutaneous formulation. The companies did not respond to inquiries about future development plans.
TaiMed has exclusive, worldwide rights to ibalizumab from the Genentech Inc. unit of Roche (SIX:ROG; OTCQX:RHBBY), which gained the compound through its 2007 acquisition of Tanox Inc.
Last year, TaiMed and WuXi said the human mAb against CD4 became the first biologic manufactured in China to receive FDA clearance for U.S. use under an IND (see BioCentury Extra, May 5, 2014).
The designation was announced after market close on Friday. WuXi lost $0.59 to $39.95 in New York; the GreTai exchange was closed.
Amarin Corp. plc (NASDAQ:AMRN) granted Eddingpharm Inc. (Shanghai, China) exclusive rights to hypertriglyceridemia drug Vascepa icosapent ethyl in Greater China.
Amarin will receive $15 million up front and is eligible for $154 million in regulatory and sales milestones, plus tiered double-digit royalties. Eddingpharm will develop and commercialize Vascepa in mainland China, Hong Kong, Macao and Taiwan. Amarin will supply the drug.
Amarin VP of Finance Michael Farrell said Eddingpharm will likely need to run a clinical trial in China before seeking its approval there.
Vascepa, a >96% pure ethyl ester of eicosapentaenoic acid (ethyl-EPA), is approved in the U.S. as an adjunct to diet to reduce triglyceride levels in adults with severe hypertriglyceridemia, defined as triglyceride levels of greater than or equal to 500 mg/dL.
Ablynx N.V. (Euronext:ABLX) said during its 4Q14 earnings call that partner Merck Serono S.A. is likely to terminate a 2013 deal to develop Nanobodies against cancer, immunology and neurology targets. Ablynx did not respond to inquiries and Merck Serono declined to discuss the deal.
Ablynx said it has received EUR 11.5 million ($13.1 million) of the EUR 25 million ($28.4 million) in research funding it had expected to receive under the four-year deal, and that it thinks Merck Serono may not provide the remaining funding.
The companies have struck four deals since 2008 to discover and develop Nanobodies against various targets. Nanobodies are therapeutic proteins based on single-domain antibody fragments. Merck Serono plans to complete an ongoing Phase I trial of trivalent Nanobody ALX-0761 targeting IL-17A and IL-17F to treat moderate to severe psoriasis; that compound was covered by the partners' 2008 deal. Last year, Ablynx opted out of co-developing oncology Nanobody ALX-0751.
Ablynx's shares were unchanged Friday after falling EUR 1.44 (13%) to EUR 9.96 on Thursday's announcement.
Chiasma Inc. (Jerusalem, Israel) closed an oversubscribed $70 million series E round led by an undisclosed crossover fund. New investors Rock Springs Capital and Sofinnova Ventures participated, as did existing investors MPM Capital; F2 Capital; 7 Med Health Ventures; Abingworth; and ARCH Venture Partners.
The company intends to submit an NDA to FDA next quarter for octreotide acetate to treat acromegaly. Chiasma designed the oral somatostatin mimetic using its Transient Permeability Enhancer (TPE) technology, with which it creates oral formulations of injectable drugs. The compound has Orphan Drug designation from FDA and EMA. Chiasma regained worldwide rights to the candidate from Roche (SIX:ROG; OTCQX:RHHBY) last year (see BioCentury Extra, Jan. 5).
Cardio3 BioSciences S.A. (Euronext:CARD) raised EUR 31.7 million ($36 million) through the sale of 713,380 shares at EUR 44.50 in a private placement with undisclosed institutional investors in the U.S. and Europe. UBS was the bookrunner and placement agents were Petercam and Bryan Garnier. Cardio3 gained EUR 0.44 to EUR 46.10 on Friday.
Cardio3's C-Cure is in the Phase III CHART-1 trial to treat chronic heart failure (CHF) secondary to ischemic cardiomyopathy, with data expected in by YE15. C-Cure is a cellular therapy based on autologous stem cells differentiated into cardiac precursors called cardiopoietic cells.
In January, the company gained three autologous chimeric antigen receptor (CAR) T therapies via its acquisition of Oncyte, the oncology division of Celdara Medical LLC (Lebanon, N.H.). It expects data from a Phase I trial of CM-CS1, the lead candidate from the deal, in mid-2016.
PureTech Ventures (Boston, Mass.) named former Sanofi (Euronext:SAN; NYSE:SNY) CEO Christopher Viehbacher to its board.
PureTech CEO and Managing Partner Daphne Zohar said the board is involved in choosing thematic areas for investment and making investment decisions.
PureTech, which has raised at least $105 million since October 2014, typically provides seed funding to new companies that are set up as operating subsidiaries, then seeks outside investors as the newcos mature (see BioCentury Extra, Jan. 9, 2015).
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