FDA approved Orkambi ivacaftor/lumacaftor from Vertex Pharmaceuticals Inc. (NASDAQ:VRTX) to treat cystic fibrosis patients aged 12 and older homozygous for the F508 mutation of the CF transmembrane conductance regulator (CFTR) gene. Chief Commercial Officer Stuart Arbuckle said on a conference call that Vertex will launch Orkambi "within days" at an annual wholesale acquisition cost (WAC) of $259,000.
In May, FDA's Pulmonary-Allergy Drugs Advisory Committee voted 12-1 to support approval of Orkambi. The therapy combines lumacaftor (VX-809), a small molecule CFTR corrector, and Kalydeco ivacaftor, a small molecule potentiator of CFTR (see BioCentury Extra, May 12).
According to Vertex, about 8,500 U.S. patients aged 12 and older are homozygous for the F508 mutation, representing 28% of the 30,000 patients with CF in the U.S. Arbuckle said Vertex estimates that 35-40% of patients with the mutation will be covered by Medicaid.
Orkambi has breakthrough therapy and Orphan Drug status in the U.S. An MAA for Orkambi is under EMA review.
Arbuckle said Vertex expects data next year from a U.S. Phase III trial of Orkambi in CF patients aged 6-11 who are homozygous for the F508 mutation.
Kalydeco is approved in the U.S. to treat CF patients aged six and older with 10 other mutations.
Vertex gained $5.07 to $131.26 on Thursday.
Biogen Inc. (NASDAQ:BIIB) and Applied Genetic Technologies Corp. (NASDAQ:AGTC) partnered to develop and commercialize gene therapies for ophthalmic diseases. AGTC will receive $124 million up front and is eligible for $1.1 billion in milestones.
Biogen will gain exclusive, worldwide rights to rAAV2tYF-CB-hRS1 to treat X-linked retinoschisis (XLRS) and a preclinical candidate to treat X-linked retinitis pigmentosa (XLRP). Biogen has no other ophthalmic candidates.
AGTC expects data this year from a Phase I/II study of rAAV2tYF-CB-hRS1, a recombinant adeno-associated virus (AAV) vector delivering retinoschisin. On a conference call Thursday, AGTC President and CEO Susan Washer said the company plans to file an IND for the XLRP candidate in 2H16.
Biogen will also get options to license discovery-stage programs from AGTC for two undisclosed ophthalmic indications and one undisclosed non-opthalmic indication, once clinical candidates are selected.
AGTC will have options to share development costs and profits for the XLRS and XLRP programs after Phase I/II data become available, and to obtain co-promotion rights in the U.S. for the second candidate to receive FDA approval, if both are approved. AGTC is responsible for leading development of the XLRS program through approval and the XLRP program through first human trials.
AGTC will receive $94 million in licensing fees and R&D funding, and Biogen will make a $30 million equity investment through the purchase of 1.4 million shares at $20.63 -- a 27% premium to AGTC's Wednesday close of $16.26 -- for an 8.1% stake in the company. AGTC is also eligible for $472.5 million in milestones from the XLRS and XLRP programs, $592.5 million in milestones from the discovery programs, and royalties. The parties expect the deal to close this quarter.
In January, Biogen began a collaboration with Fondazione Telethon (Milan, Italy) and Ospedale San Raffaele (Milan, Italy) to develop gene therapies to treat hemophilia A and B (see BioCentury Extra, Jan. 29).
AGTC climbed $2.77 (17%) to $19.03 on the day; Biogen lost $1.13 to $404.28.
The U.S. Court of Appeals for the Federal Circuit ruled that two patents covering Angiomax bivalirudin from The Medicines Co. (NASDAQ:MDCO) are invalid. The Medicines Co. had filed suit against Hospira Inc. (NYSE:HSP) in August 2010 after Hospira submitted two ANDAs seeking to market its generic bivalirudin before the two patents, U.S. Patents Nos. 7,582,727 and 7,598,343, expire in 2028.
Last year, the U.S. District Court for the District of Delaware found that the patents were valid, but ruled that Hospira's generic version of the small molecule direct thrombin inhibitor (DTI) did not infringe on them. The Medicines Co. appealed the claim construction and non-infringement rulings, and Hospira appealed the validity ruling.
The Medicines Co. climbed $2.94 (10%) to $31.28 on Thursday. Spokesperson Bob Laverty declined to comment on the stock move. Hospira did not respond to inquiries.
Data published Thursday in The Lancet Respiratory Medicine from a Phase II study of pGM169/GL67A to treat cystic fibrosis showed that the inhalable gene therapy led to a significant benefit in lung function. The U.K. Cystic Fibrosis Gene Therapy Consortium conducted the trial, which was funded by a partnership between the U.K.'s Medical Research Council and National Institute for Health Research.
The study's primary endpoint was relative percent change after 12 months in predicted forced expiratory volume in one second (FEV1) in patients receiving monthly doses of pGM169/GL67A vs. placebo. The primary analysis was per protocol; in that 116-patient population, the treatment group showed a significant improvement of 3.7% (95% CI: 0.1, 7.3, p=0.046) vs. placebo, meeting the endpoint. The treatment group had a relative FEV1 change of -0.4% (95% CI: -2.8, 2.1), while the placebo group had -4% (95% CI: -6.6, -1.4).
Among 121 evaluable patients in the study's intent-to-treat population, the treatment group showed an improvement of 3.6% (95% CI 0.2, 7.0, p=0.039) vs. placebo.
Imperial Innovations Group plc (LSE:IVO), which holds the patent portfolio for the therapy, said it would seek a commercialization partner to further develop the therapy. University of Oxford Professor Stephen Hyde, an author of the study, told BioCentury the consortium hopes to begin a Phase III study as soon as next year, if a partner can be found.
The therapy, which comprises plasmid DNA carrying the CF transmembrane conductance regulator (CFTR) gene complexed with a cationic liposome, has Orphan Drug designation in the U.S. and EU.
The consortium includes scientists and clinicians from Imperial College London, University of Oxford and University of Edinburgh, the Royal Brompton & Harefield NHS Foundation Trust and NHS Lothian.
Diagnostics company Natera Inc. (NASDAQ:NTRA) gained $4.74 (26%) to $22.74 in its first day of trading Thursday after raising $180 million through the sale of 10 million shares at $18 in an offering underwritten by Morgan Stanley; Cowen; Piper Jaffray; Baird; and Wedbush Pacgrow. The IPO price valued the company at $871.7 million.
In an SEC filing on Wednesday, Natera said it hoped to sell 10 million shares at $17-$18. The company had said last month it aimed to sell 6.3 million shares at $15-$17. A sale of 6.3 million shares at $16 would have raised $100 million and valued the company at $714.8 million.
Natera markets its Panorama test, a non-invasive prenatal multiplex assay that detects fetal trisomies 13, 18 and 21 and sex chromosome abnormalities in maternal blood cell-free DNA (cfDNA). The company raised $55.5 million in a series F round in April (see BioCentury Extra, April 6).
Mousera Inc. (San Mateo, Calif.) disclosed that it raised $20 million in an extension of its series A round. Data Collective led the financing; fellow new investors Dolby Family Ventures and Ame Cloud Ventures also participated along with existing investor Founders Fund.
Last fall, Mousera raised $8.8 million in the first close of the A round. Lux Capital led the first close, and Founders Fund participated.
Mousera Chairman and CEO Timothy Roberts told BioCentury the company is building a technology platform intended to speed up the preclinical drug discovery and development process. He said Mousera is working with undisclosed biotech and pharma partners "to screen drugs, test them and give them back with insights that are more predictive of what will happen in human clinical trials."
A new version of the 21st Century Cures Act released Thursday would provide $8.8 billion over five years for an NIH Innovation Fund, a decrease of $1.2 billion from previous versions of the bill.
The cut follows intensive negotiations and lobbying as the U.S. House Energy & Commerce Committee has scrambled to find budget offsets to pay for the legislation. The committee had voted to change the timing of payments to insurance companies that run Medicare Advantage drug benefit programs, saving the federal government $5-$7 billion over five years. Insurance industry lobbyists persuaded House Republican leaders to withdraw the provision.
Committee staff circulated a proposal Wednesday to offset about $1.2 billion from the cost of the bill by increasing reimbursement for biosimilars in the Medicare Part B program. Under the Affordable Care Act, a physician who administers a biosimilar to a Medicare patient receives a payment equal to 6% of the average sales price (ASP) of the reference product. The reimbursement rate was linked to the reference product so that physicians would not be dissuaded from prescribing lower priced biosimilars. The new proposal would have increased reimbursement for biosimilars to 8% of the reference product's ASP, creating a strong incentive for physicians to select biosimilars.
The biosimilars pay-for proposal provoked an explosive reaction from BIO and PhRMA, including threats to oppose the legislation, according to biopharma industry CEOs and lobbyists. Industry prevailed, and the committee, unable to obtain consensus for an alternative budget offset, decided to reduce the size of the NIH Innovation fund.
The new version also includes a change to the way average manufacturers' price (AMP) is calculated. AMP is used to determine rebates paid by manufacturers on brand drugs sold to Medicaid. The bill redefines AMP to exclude authorized generics from AMP calculations. A summary posted by the House Rules Committee says the policy "would have the effect of increasing the AMP of brand drugs and thus increasing the rebates drug manufacturers would owe to the states and federal government."
In addition, the new version omits a section granting the NIH director power to require that grant recipients share scientific data.
The House is expected to pass the 21st Century Cures Act next week. The Senate Health, Education, Labor and Pensions (HELP) Committee is working on a separate biomedical innovation bill. If the Senate bill is passed, the House and Senate will attempt to agree on a reconciled bill.
BioCentury Extra will not publish Friday, July 3, in observance of the Independence Day holiday in the U.S. BioCentury Extra will resume publishing on Monday, July 6.
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