The U.S. Supreme Court granted a petition from the Sandoz unit of Novartis AG (NYSE:NVS; SIX:NOVN) to review a lower court's interpretation of the Biologics Price Competition and Innovation Act (BPCIA) that effectively delays the launch of a biosimilar until at least 180 days after FDA approval.
Language in the BPCIA requires the sponsor of a biosimilar to give the reference product's sponsor 180 days notice before the date of first commercial marketing of the biosimilar. The U.S. Court of Appeals for the Federal Circuit (CAFC) ruled in July 2015 that the notice can be given only after FDA has approved the biosimilar. The ruling delayed the launch of Sandoz's Zarxio filgrastim-sndz, a biosimilar of Neupogen filgrastim from Amgen Inc. (NASDAQ:AMGN) (see BioCentury Extra, July 21, 2015).
Last month, acting U.S. Solicitor General Ian Gershengorn wrote an amicus brief recommending the Supreme Court review the CAFC's decision. He said the six-month delay undermines procedures laid out in the BPCIA to encourage early litigation of patent claims (see BioCentury Extra, Dec. 8, 2016).
The Supreme Court denied a similar petition from Apotex Inc. (Toronto, Ontario) last month (see BioCentury Extra, Dec. 12, 2016).
Rare diseases company Swedish Orphan Biovitrum AB (SSE:SOBI) said CEO Geoffrey McDonough will leave the company on July 1. The company is searching for a replacement. The company also named Armin Reininger SVP and head of global medical and scientific affairs. He was head of medical affairs EMEA for hemophilia and head of medical affairs global for hematology at Baxalta Inc., which Shire plc (LSE:SHP; NASDAQ:SHPG) acquired.
Antifungal company Amplyx Pharmaceuticals Inc. (San Diego, Calif.) promoted Ciara Kennedy to president and CEO from COO and promoted Karen Shaw to CSO from VP of biology.
Bicyclic peptide company Bicycle Therapeutics Ltd. (Cambridge, U.K.) named Nicholas Keen CSO. He was head of oncology research in the U.S. at Novartis AG (NYSE:NVS; SIX:NOVN).
T cell therapy company Adaptimmune Therapeutics plc (NASDAQ:ADAP) named Mark Dudley SVP of global bio-process and development. He was director of new cell products for cell and gene therapy at Novartis.
Antibody company MorphoSys AG (Xetra:MOR; Pink:MPSYY) named Malte Peters chief development officer, effective March 1. He will succeed Arndt Schottelius. He was global head of clinical development of biopharmaceuticals at Novartis' Sandoz unit.
Rare genetic diseases company Therachon AG (Basel, Switzerland) named Luca Santarelli CEO and director. He was SVP and global head of neuroscience, ophthalmology and rare diseases at Roche (SIX:ROG; OTCQX:RHHBY).
Oncology, autoimmune and nephrology company 3SBio Inc. (HKSE:1530) named Zhenping Zhu president of R&D and CSO. He was EVP of global biopharmaceuticals at Kadmon Holdings Inc. (NYSE:KDMN).
Complement-mediated disease company True North Therapeutics Inc. (South San Francisco, Calif.) named Gary Patou CMO. He will continue to serve as head of clinical for Chiasma Inc. (NASDAQ:CHMA) on a limited basis. True North also promoted Adam Rosenthal to CBO from VP of corporate development.
Dermatology company Dermira Inc. (NASDAQ:DERM) named Lori Lyons-Williams chief commercial officer. She was VP of sales and marketing at Allergan plc (NYSE:AGN).
Antibody company AnaptysBio Inc. (San Diego, Calif.) named Dominic Piscitelli CFO. He was VP of finance, strategy and investor relations at Medivation Inc., which Pfizer Inc. (NYSE:PFE) acquired.
Immunotherapy company Adicet Bio Inc. (Menlo Park, Calif.) named Anne Altmeyer CBO. She was VP of negotiations, global business development and licensing at Baxalta.
Newly released data showed that Spinraza nusinersen from Biogen Inc. (NASDAQ:BIIB) met a second primary endpoint of the Phase III ENDEAR study to treat spinal muscular atrophy. The therapy significantly reduced risk of death or permanent ventilation at an end of study analysis.
FDA approved Spinraza last month to treat SMA. It is the first drug approved in the indication (see BioCentury Extra, Dec. 23, 2016).
Biogen halted ENDEAR in August 2016 after an interim analysis showed Spinraza met another primary endpoint, improving the proportion of responders to motor milestones. The company had added the interim analysis to the study last year based on input from regulatory agencies (see BioCentury Extra, Aug. 1, 2016).
On Friday, Biogen said ENDEAR's end of study analysis showed that 39% of Spinraza-treated patients died or required permanent ventilation, vs. 68% of untreated infants (p<0.01). The analysis included 78 patients who had a final study visit after the interim analysis was completed. The 121-patient trial compared Spinraza vs. sham treatment in patients with infantile-onset SMA.
Biogen has rights to Spinraza from Ionis Pharmaceuticals Inc. (NASDAQ:IONS).
AveXis Inc. (NASDAQ:AVXS) is developing AVXS-101, a gene therapy to treat SMA that may offer durable effects from a single IV treatment. An interim analysis of a Phase I study of the therapy last fall showed that all 12 patients were still alive. CEO Sean Nolan told BioCentury last month that there was no diminution of AVXS-101's therapeutic effects after two years of follow-up (see BioCentury, Jan. 2).
Spinraza is an antisense oligonucleotide that modulates splicing of survival of motor neuron 2 centromeric (SMN2) mRNA. AVXS-101 uses NAV rAAV9 vector technology from RegenxBio Inc. (NASDAQ:RGNX) to deliver the SMN gene.
Biogen shed $3.02 to $284.75 on Friday. AveXis slipped $0.76 to $52.61.
Biotech stocks made a partial recovery late in the week from losses incurred after President-elect Donald Trump blasted the biopharma industry during a press conference Wednesday. Trump said pharma companies are "getting away with murder" and called for "bidding" on drug prices (see BioCentury Extra, Jan. 11).
The BioCentury 100 index closed Friday at 5,146.16, for a two-day gain of 1.5% from Wednesday's close of 5,067.68 and putting the BC100 about halfway back to Tuesday's close of 5,221.34.
The NASDAQ Biotechnology Index (NBI) closed Friday at 2,931.56, regaining almost 2% from its intraday bottom at 2,875.26 on Thursday. It had traded above 2,980 just prior to Trump's press conference. The iShares NASDAQ Biotechnology ETF (IBB) and the NYSE Arca Biotechnology Index (BTK) followed similar patterns, recovering about half of their losses.
Qiming Venture Partners has set a $125 million target for its planned Qiming U.S. Healthcare Fund L.P., according to an SEC filing.
The firm closed its fourth renminbi fund at RMB1.5 billion ($221.4 million) in November 2016. It closed its fifth U.S. dollar fund at $648 million in January 2016. Both invest in Chinese companies (see BioCentury, Nov. 21, 2016).
In a discussion paper addressing regulation of laboratory-developed tests, FDA said it is continuing to evaluate comments and proposals from stakeholders to determine an appropriate way to regulate laboratory-developed tests, and is seeking a "complementary approach" that would garner support from the broadest array of stakeholders. The agency said a prospective oversight framework focused on new and higher risk LDTs would best serve public health while advancing laboratory medicine.
FDA proposed a framework in 2014 that would phase in FDA regulation of LDTs over a nine-year period and enforce premarket review requirements based on risks associated with LDTs. CMS, which currently oversees LDT regulation under the Clinical Laboratory Improvement Amendments (CLIA), would continue to oversee laboratory operations (see BioCentury, Aug. 11, 2014).
In its discussion document, FDA acknowledged that agency oversight of LDTs was opposed by the laboratory community. Based on its feedback, the agency proposed possible alternatives to its 2014 guidance proposing the phased approach, including an oversight system that would exempt existing LDTs, along with low risk tests and those for rare diseases, and provide additional time before implementing FDA oversight. It also discussed phasing in premarket review over four years rather than nine.
In November 2016, FDA said it will not finalize its 2014 draft guidance, and would instead work to develop a new oversight policy "with stakeholders, our new Administration, and Congress to get our approach right" (see BioCentury Extra, Nov. 18, 2016).
Congressional Republicans, including Rep. Michael Burgess (R-Texas), have opposed the proposed FDA oversight. Burgess challenged the agency's statutory jurisdiction to regulate LDTs in a 2014 hearing of the House Energy and Commerce Subcommittee on Health. Burgess is now chairman of the subcommittee (see BioCentury Extra, Sept. 9, 2014).
In draft guidance released Friday, FDA clarified the use of reference standards for bioequivalency studies in generic drug applications. The guidance said FDA will take measures to speed review of generic applications and ensure availability of reference standards.
The document clarified the definitions of reference standards and reference listed drugs. It specified that the reference standard is the compound selected by FDA that must be used in bioequivalency studies, while the reference listed drug is the previously approved drug that the generic duplicates. FDA said the two are ordinarily the same, but may not be in cases where the listed drug is no longer marketed.
FDA said that to ensure generic manufacturers have access to reference standards, it will consider selecting a new reference standard if available quantities of the existing reference standard are "so limited" that an applicant is unable to obtain a sufficient amount for bioequivalence testing. To speed availability of generics, the agency said it will now approve generic applications that use withdrawn reference listed drugs, provided they were not withdrawn for safety or efficacy reasons, without waiting for the Federal Register to publish a determination concerning the withdrawal.