Royalty Pharma (New York, N.Y.) paid $3.3 billion to acquire the Cystic Fibrosis Foundation's rights to royalties from cystic fibrosis (CF) treatments developed by Vertex Pharmaceuticals Inc. (NASDAQ:VRTX). The deal includes royalties from Kalydeco ivacaftor, lumacaftor (VX-809), VX-661 and second generation correctors.
The foundation has committed $150 million to Vertex's research efforts since the company started its CF research program in 2000 as part of a deal with CFF.
The deal covers royalties ranging from single digit to sub-teen percentages of net sales for CF drugs from Vertex, according to Royalty Pharma spokesperson Alexander Perfall.
Kalydeco, a small molecule CFTR potentiator, is approved in the U.S. and EU to treat CF patients with one of nine mutations in the CFTR gene. Last month, an FDA advisory committee voted 13-2 to recommend approval of Vertex's sNDA for Kalydeco to add patients with the R117H mutation in CFTR (see BioCentury Extra, Oct. 21).
Vertex also submitted an NDA to FDA and an MAA to EMA this month for the combination of 250 mg of Kalydeco and 400 mg of lumacaftor to treat CF in patients with two copies of the F508del mutation in the CFTR gene. The combination has breakthrough therapy designation in the U.S. Lumacaftor is a small molecule CFTR corrector (see BioCentury Extra, Nov. 5).
VX-661, another small molecule CFTR corrector, is in Phase IIb testing in combination with Kalydeco to treat adults with two copies of the F508del mutation.
Knight Therapeutics Inc. (TSX:GUD) sold a Priority Review voucher to Gilead Sciences Inc. (NASDAQ:GILD) for $125 million. Knight obtained the voucher under FDA's Tropical Disease Priority Review voucher program after the agency approved Knight's Impavido miltefosine to treat cutaneous, mucosal and visceral leishmaniasis in March (see BioCentury Extra, March 19).
Gilead spokesperson Nathan Kaiser said the company will make a decision on where to apply the voucher based on an upcoming pipeline review.
The price tag for Knight's voucher was almost 2x that of a previous voucher. In July, BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) sold a voucher to Regeneron Pharmaceuticals Inc. (NASDAQ:REGN) and partner Sanofi (Euronext:SAN; NYSE:SNY) for $67.5 million. Regeneron and Sanofi plan to use the voucher to obtain Priority Review for their BLA for alirocumab, a human mAb targeting proprotein convertase subtilisin/kexin type 9 (PCSK9), to treat primary hypercholesterolemia (see BioCentury, Aug. 4).
Also on Wednesday, the U.S. Senate's Health, Education, Labor and Pensions (HELP) Committee passed a bill revising the terms for using and transferring a Priority Review voucher obtained under the tropical disease program. The bill seeks to shorten to 90 days from one year the advance notification time a company must provide FDA before redeeming a voucher, and to allow a voucher to be transferred from sponsor to sponsor an unlimited number of times, rather than only once. Both revisions would bring the tropical disease voucher program in line with the agency's pediatric disease voucher program.
In addition, the bill added Ebola to the list of diseases whose treatments are eligible for tropical disease vouchers.
Knight gained C$0.49 to C$6.53 on Wednesday.
Lee's Pharmaceutical Holdings Ltd. (HKSE:0950) received rights from Ikaria Inc. (Hampton, N.J.) to INOmax in China, Hong Kong, Macau and Taiwan. The inhaled nitric oxide to treat hypoxic respiratory failure associated with pulmonary hypertension in term and near-term infants is Ikaria's only marketed product. Neither company responded to inquiries seeking details on the financial terms of the deal.
Agios Pharmaceuticals Inc. (NASDAQ:AGIO) rose $12.16 (15%) to $95.97 after it said four of 14 evaluable patients had complete remissions in a Phase I trial of AG-120 to treat advanced hematologic malignancies including acute myelogenous leukemia (AML). The trial enrolled patients with an isocitrate dehydrogenase I (IDH1) mutation who had failed at least one prior therapy, and included four dose levels.
Seven patients achieved objective responses, including two marrow complete remissions and one partial remission. Agios said early evidence showed durability of remission from 15 days to five months among the four complete remissions. It added that the maximum tolerated dose has not been achieved.
Investigators presented the data at the EORTC-NCI-AACR Symposium on Molecular Targets and Cancer Therapeutics in Barcelona.
Agios plans to initiate multiple expansion cohorts of the trial in 1H15.
In February, Agios exercised its option under a 2010 deal with Celgene Corp. (NASDAQ:CELG) to retain U.S. development and commercialization rights to AG-120. Celgene holds an option to license ex-U.S. rights after Phase I testing is completed.
AG-120, an inhibitor of mutated IDH1, is also in Phase I testing to treat solid tumors that have an IDH1 mutation.
Bind Therapeutics Inc. (NASDAQ:BIND) lost $2.15 (20%) to $8.40 on Wednesday, giving back most of its recent gains, after reporting no new partial responses from seven additional patients in its Phase II trial of BIND-014 to treat stage III/IV non-small cell lung cancer (NSCLC).
The shares had risen $1.11 to $8.94 on Oct. 30 after the company released an abstract in advance of the EORTC-NCI-AACR Symposium on Molecular Targets and Cancer Therapeutics in Barcelona, Spain. In the abstract, Bind said five (15%) of 33 patients who were evaluable at the time had PRs. Among eight patients who tested positive for the K-Ras (KRAS) mutation, two (25%) had PRs (see BioCentury Extra, Oct. 30).
With 40 patients now in the dataset, including one new patient with the KRAS mutation, Bind said the number of PRs has not increased, lowering the PR rate to 12.5% overall and 22.2% for the subpopulation with KRAS mutations.
Bind spokesperson Gina Nugent said BIND-014 met the trial's primary objective of showing anti-tumor activity as measured by a 10% overall response rate in its once-every-three-weeks dosing arm. No patients achieved a complete response. Bind plans to conduct an additional Phase II study of BIND-014 in patients with KRAS mutations.
The compound is a polymeric nanoparticle containing docetaxel that concentrates in the neovasculature surrounding tumors and targets prostate-specific membrane antigen (PSMA; FOLH1; GCPII).
Receptos Inc. (NASDAQ:RCPT) gained $4.97 to $107.95 Wednesday after it raised $360 million through the sale of 3.6 million shares at $100 in a follow-on underwritten by Credit Suisse; Leerink; Evercore; BMO; Wedbush PacGrow; and Nomura. Receptos proposed to raise $325 million after market close on Monday when the shares finished at $104.22.
In December 2013, the company began the Phase III portion of the ongoing RADIANCE trial of RPC1063 to treat relapsing multiple sclerosis (MS). Receptos plans to begin a second Phase III study to treat MS in 1H15. Both trials have an SPA from FDA. The company also plans to start a Phase III study of the compound to treat ulcerative colitis (UC) and a Phase II trial to treat Crohn's disease next year.
Receptos shares have gained $40.21 (59%) from $67.74 on Oct. 27. After market close that day, the company said RPC1063, a selective sphingosine 1-phosphate receptor (S1PR1; S1P1; EDG1) modulator, met the primary endpoint in the Phase II TOUCHSTONE trial to treat UC (see BioCentury Extra, Oct. 27).
In a regulatory filing, Receptos said FDA has indicated that it could consider TOUCHSTONE as a Phase III trial if its results are "statistically and clinically persuasive." If so, TOUCHSTONE would be one of two induction Phase III studies required for approval in UC, along with a maintenance study.
Receptos has raised $682.6 million in three follow-ons this year. The company raised $117.4 million in January and $205.2 million in June.
The follow-on is the largest of 2014 for biotechs, topping a $282.5 million offering by InterMune Inc. (NASDAQ:ITMN) in March.
Baird Capital closed its fourth venture fund, BVP IV, at $185 million, exceeding its target of $150 million. The firm invests in technology and healthcare companies, with an emphasis on medical devices and diagnostics.
The firm already has made five investments from the new fund. It led the $30.3 million series B round of Integrated Diagnostics Inc. (Seattle, Wash.) in April, and participated in the $21 million series B round of NeuMoDx Molecular Inc. (Ann Arbor, Mich.) in March.
N30 Pharmaceuticals Inc. (Boulder, Colo.) raised $30 million in a mezzanine financing. New investors included Wellington; RA Capital; Jennison; Rock Springs; and Sabby. Existing investor Deerfield also participated. Cowen served as the placement agent.
N30's N91115, an oral s-nitrosoglutathione reductase inhibitor, is in Phase I testing to treat cystic fibrosis (CF) patients with the F508del cystic fibrosis transmembrane conductance regulator (CFTR) mutation.
Amicus Therapeutics Inc. (NASDAQ:FOLD) raised $90 million to commercialize its Amigal migalastat to treat Fabry's disease after pricing a follow-on of 13.9 million shares at $6.50 per share on Wednesday.
Amicus intends to meet with European regulators by year end and with FDA in early 2015 to discuss a regulatory path for the compound.
The company's shares gained $0.93 (20%) to $5.50 on Aug. 20 after Amicus said that the small molecule that enhances alpha galactosidase A activity met the primary endpoints in the Phase III ATTRACT (Study 012) trial (see BioCentury Extra, Aug. 20).
The stock jumped $1.01 (17.8%) to $6.70 on Monday after the company reported at the American Society of Nephrology's Kidney Week that Amigal met secondary endpoints in ATTRACT.
Amicus was down $0.22 to $6.53 on Wednesday. Underwriters on the follow-on are JPMorgan; Cowen; Leerink; and Janney Montgomery Scott.
HHS and NIH on Wednesday proposed new rules governing the transparency of clinical trials that could more than double the number of submissions of study results to ClinicalTrials.gov.
The agencies issued a notice of proposed rulemaking that would require sponsors to report summary results from all trials regardless of whether the compound or device has been approved. Currently, sponsors need only report data from trials of approved drugs and devices.
The proposed rule includes "interventional studies of drugs, biological products, and devices that are regulated by the FDA" but which "exclude Phase I studies of drugs and biological products and feasibility studies of devices."
NIH is also seeking comments on a separate draft policy that would extend the proposed rule to all NIH-funded clinical trials regardless of phase.
Most of the new data would come from industry-sponsored trials. In a press briefing, ClinicalTrials.gov director Deborah Zarin said she expects the number of submissions to rise to 200-250 a week from the current 100 weekly submissions if the policies are adopted. Of this, Zarin said NIH-funded trials would likely contribute only 12 new submissions per week.
The agencies outline the proposed changes in a summary posted online.
Comments are due Feb. 19, 2015.
Rep. Frank Pallone (D-N.J.) defeated Rep.Anna Eshoo (D-Calif.) in a 100-90 vote to replace Rep. Henry Waxman (D-Calif.) as the ranking Democrat of the House Energy & Commerce Committee. E&C has oversight authority for FDA, NIH, CMS and the U.S. Patent and Trademark Office (PTO).
Pallone's public positions on biopharma issues largely reflect those of Waxman. Pallone sided with Waxman on biosimilars when the latter initially argued that innovative biologics did not need protection from biosimilar competition. After Waxman and Pallone later called for six years of exclusivity, Eshoo argued for 12 years and successfully incorporated her biosimilars legislation into the Affordable Care Act in 2010 (see BioCentury, May 5).
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