Sutro Biopharma Inc. (South San Francisco, Calif.) raised $26 million in a series D round led by existing investors Alta Partners; Amgen Ventures; Celgene Corp. (NASDAQ:CELG); Lilly Ventures; Skyline Ventures; and SV Life Sciences. Sutro is using its cell-free protein synthesis technology to generate bispecific antibodies and site-specific antibody-drug conjugates (ADCs) with unnatural amino acids. The company's internal pipeline includes undisclosed preclinical ADC programs. Sutro said it plans to advance a bispecific antibody for immunology or cancer indications into the clinic in the next 12-18 months.
Last year, Sutro partnered with Celgene to develop ADCs and bispecific antibodies for two undisclosed targets in a deal Sutro said could be worth more than $500 million, excluding royalties (see BioCentury Extra, Dec. 18, 2012).
CytRx Corp. (NASDAQ:CYTR) jumped $1.63 (68%) to $4.02 on Wednesday after reporting that first-line treatment with IV aldoxorubicin (INNO-206) met the primary endpoint of improving progression-free survival (PFS) vs. doxorubicin in a Phase IIb trial to treat soft tissue sarcoma (STS). Aldoxorubicin improved median PFS as assessed by investigators (8.4 vs. 4.7 months, p=0.0002) and by blinded central laboratory review (5.7 vs. 2.8 months, p=0.018) vs. doxorubicin. The open-label, international trial enrolled 123 patients with metastatic, locally advanced or unresectable STS. Overall survival (OS) data, a secondary endpoint, are expected in 3Q14.
Next quarter, CytRx plans to start a Phase III trial with aldoxorubicin as second-line treatment of STS. The company has an SPA from FDA for the trial. CytRx said it is "questionable" at the moment as to whether it will pursue a first-line indication for STS after an NDA submission for second-line use. Aldoxorubicin is a 6-maleimidocaproyl hydrazone prodrug of doxorubicin.
ImmunoCellular Therapeutics Ltd. (NYSE-M:IMUC) tumbled $1.48 (54%) to $1.24 in early after-hours trading after reporting data late Wednesday from a Phase II trial evaluating the company's ICT-107 to treat newly diagnosed glioblastoma multiforme (GBM). In the 124-patient trial, ICT-107 given following resection and chemoradiation missed the primary endpoint of improving overall survival (OS) vs. placebo following resection and chemoradiation in both the intent-to-treat (ITT) population (n=124, p=0.58) and the per protocol (PP) population (n=117, p=0.4).
ImmunoCellular said ICT-107 did meet the secondary endpoint of improving progression-free survival (PFS) vs. placebo. Median PFS was two months longer in patients who received ICT-107 compared to placebo in the ITT population (p=0.014) and three months longer in the PP population (p=0.0074). The company said it plans to request an end-of-Phase II meeting with FDA to discuss next steps, including a possible Phase III trial. ICT-107 is an intradermal tumor associated antigen-pulsed dendritic cell-based therapeutic vaccine.
ImmunoCellular was off $0.05 to $2.72 on Wednesday.
Prothena Corp. plc (NASDAQ:PRTA) and Roche (SIX:ROG; OTCQX:RHHBY) partnered to co-develop and co-commercialize mAbs targeting alpha synuclein (SNCA) including Prothena's PRX002, which is in preclinical development for Parkinson's disease and other synuclein-related diseases. Prothena will receive $45 million in an undisclosed upfront payment and a near-term milestone payment tied to the start of a Phase I trial for PRX002; the trial is slated to start next half. Roche and Prothena will share U.S. costs and profits 70/30, and Prothena has an option to co-promote PRX002 in the U.S. Roche will be responsible for development and commercialization of PRX002 outside the U.S. Prothena is eligible for up to $555 million in milestones, including $175 million in ex-U.S. commercial milestones, plus double-digit royalties on ex-U.S. sales. Additionally, Prothena and Roche will work to optimize early stage antibodies targeting SNCA, including using Roche's brain shuttle technology.
Last year, Elan Corp. plc (NYSE:ELN) spun out its discovery and early stage programs, including PRX002 (NEOD002), into Prothena (originally Neotope Biosciences plc). Perrigo Co. (NASDAQ:PRGO; Tel Aviv:PRGO) is acquiring Elan.
Prothena, which announced the deal after market close on Wednesday, was off $0.77 to $27.39 on the day.
FDA's Endocrinologic and Metabolic Drugs Advisory Committee voted 11-1 that the Amylin Pharmaceuticals Inc. subsidiary of Bristol-Myers Squibb Co. (NYSE:BMY) provided substantial evidence that the benefits of Myalept metreleptin exceed the risks in patients with generalized lipodystrophy. However, the panel voted 10-2 that the benefits of metreleptin did not exceed the risks in patients with metabolic disorders associated with partial lipodystrophy. Bristol-Myers has proposed a REMS for metreleptin that would include a physician and pharmacy certification component, as well as a prescriber education program and a metreleptin safety registry. Metreleptin is under Priority Review for the indications, with a Feb. 24 PDUFA date.
Last year, Bristol-Myers folded Amylin's diabetes portfolio, including metreleptin, into its diabetes deal with AstraZeneca plc (LSE:AZN; NYSE:AZN). Amylin has rights to the recombinant form of human leptin from Amgen Inc. (NASDAQ:AMGN) (see BioCentury, July 9, 2012).
The Genzyme Corp. subsidiary of Sanofi (Euronext:SAN; NYSE:SNY) said FDA accepted and granted Priority Review to an NDA for Cerdelga eliglustat to treat Type I Gaucher's disease. The company declined to disclose the PDUFA date. The product -- an oral ceramide analog that inhibits glucosylceramide synthase (GCS) -- is also under review in Europe.
Genzyme already markets Cerezyme imiglucerase for Type I Gaucher's disease. Cerezyme is a recombinant glucocerebrosidase is administered as an IV infusion.
Orexigen Therapeutics Inc. (NASDAQ:OREX) resubmitted an NDA to FDA for Contrave naltrexone/bupropion to treat obesity. The resubmission is based on an interim analysis in the Phase III Light Study, which showed that Contrave met FDA's pre-specified criteria for ruling out excess cardiovascular risk. The Light Study is evaluating CV outcomes with Contrave, which FDA requested in a 2011 complete response letter. In October, Orexigen submitted an MAA to EMA for the obesity candidate, which is partnered in North America with Takeda Pharmaceutical Co. Ltd. (Tokyo:4502) (see BioCentury Extra, Nov. 25).
On Wednesday, Orexigen was off $0.27 to $5.56.
FDA accepted for review a resubmitted NDA from QRxPharma Ltd. (ASX:QRX; OTCQX:QRXPY) for MoxDuo IR morphine/oxycodone to treat moderate to severe acute pain. The PDUFA date is May 25. The company said FDA will schedule an advisory committee meeting to discuss the NDA, which contains an audit of data from the company's Phase III Study 022 trial. In August, FDA issued a complete response letter for the product after QRxPharma submitted a revised analysis following discovery of an error in the time points at which some respiratory data in Study 022 were collected. MoxDuo IR is an immediate-release combination of oxycodone and morphine (see BioCentury Extra, Aug. 28).
QRxPharma was off A$0.02 to A$0.61 on Wednesday. In the U.S., the stock was unchanged at $2.84.
GlaxoSmithKline plc (LSE:GSK; NYSE:GSK) said it will invest about L200 million ($327.4 million) for manufacturing in the U.K. The pharma said it will build new manufacturing facilities at its Ware site as well as a new bulk sterile building and filing line at its Worthing site for the pharma's antibiotic Augmentin, a combination of amoxicillin and clavulanate, a beta lactamase (LACTB) inhibitor. GSK said it also will build a new center to develop practical manufacturing applications from emerging science and technologies. The pharma said it expects the investment to create 250 new jobs.
The new funding is in addition to the L530 million ($867.5 million) that GSK announced last year it would invest in U.K. manufacturing (see BioCentury, March 22, 2012).
Crealta Pharmaceuticals LLC (Lake Forest, Ill.) will acquire the pharmaceutical portfolio of Savient Pharmaceuticals Inc. (OTCQB:SVNTQ), including gout drug Krystexxa pegloticase, for about $120.4 million. The deal is the first for Crealta, which debuted in August to acquire specialty pharmaceutical companies and products that are already approved or marketed, as well as late-stage development assets. Crealta is a partnership between Chairman and CEO Ed Fiorentino and private equity firm GTCR. Savient filed for Chapter 11 bankruptcy in the U.S. Bankruptcy Court for the District of Delaware in October. Krystexxa is approved in the U.S. and EU for chronic gout.
Sprout Pharmaceuticals Inc. (Raleigh, N.C.) is appealing a complete response letter from FDA for flibanserin to treat hypoactive sexual desire disorder (HSDD). Rhe company received the complete response letter for the resubmitted NDA in September. Sprout said it expects a decision on the appeal in 1Q14.
In 2011, Sprout acquired flibanserin from Boehringer Ingelheim GmbH (Ingelheim, Germany), which had discontinued development of the serotonin (5-HT1A) receptor agonist and 5-HT2A receptor after FDA issued a complete response letter in 2010 (see BioCentury Extra, Oct. 8, 2010).
CymaBay Therapeutics Inc. (Hayward, Calif.) has raised $35.2 million since September, which the company said should provide enough runway to get to a Phase IIb readout in gout for lead compound arhalofenate (MBX-102). The company raised $30.2 million through the sale of 6 million shares at $5 in private placements that closed on Sept. 30 and Oct. 31. Investors also received warrants. National Securities Corp. was placement agent for both offerings. Also in September, CymaBay drew down $5 million of a $10 million loan from Oxford Finance and Silicon Valley.
In October, CymaBay (formerly Metabolex Inc.) became a publicly reporting company via the Form 10 pathway. Arhalofenate, an uricosuric agent, has completed three Phase II gout trials. The company declined to say when it plans to start the Phase IIb trial, but said it expects data from the trial in 1H15. CymaBay is going public via the Form 10 pathway, but declined to provide a timeline on when it plans to list on the OTC Bulletin Board.
The Senate and House subcommittees responsible for appropriating FDA and NIH funding are not yet scheduled to discuss appropriations for the agencies for the remainder of FY14. However, the House and Senate Conference Committee on the Budget reached an agreement, H.J. Res. 59, on top-line spending levels for the remainder of FY14 as well as FY15 that bumped FY14 discretionary spending by about $45 billion from post-sequester levels and will "turn off" the next round of planned sequestration cuts.
The full House is scheduled to vote on the top-line budget deal on Thursday, followed by the Senate. Once the Bipartisan Budget Act is signed into law, the Congressional Appropriations Committees will then work to allocate the budget before Jan. 15, when the current continuing resolution funding the federal government expires. The government has been operating at post-sequestration FY13 levels since October, when President Obama signed the continuing resolution ending a federal government shutdown (see BioCentury Extra, Oct. 17).
Steven Grossman, deputy executive director at the Alliance for a Stronger FDA, noted that the agency can "reasonably expect to get more money" in the rest of FY14 than the $2.39 billion it received post-sequester in FY13, though he cautioned that the House and Senate Appropriations committees do not have to appropriate the same funding for FDA the committees had set earlier this year. The Senate Appropriations Committee passed a FY14 budget bill that would have allocated about $2.56 billion in base appropriations, or taxpayer funding, for FDA in FY14, while the House had allocated $2.49 billion in base appropriations in FY14 to the agency.
On Thursday, Reps. Phil Gingrey (D-Ga.) and Gene Green (D-Texas) are expected to introduce legislation in the U.S. House of Representatives that is anticipated to include provisions to create a limited population approval pathway for antibiotics and to recommend HHS update breakpoints for antibiotics based on pharmacokinetic and pharmacodynamic data. The sponsors declined to disclose details of the bill prior to its introduction.
The Infectious Diseases Society of America first proposed a limited population antibacterial drug (LPAD) approval pathway, which would allow for approval for antibiotics targeting serious or life-threatening infections based on clinical trials with smaller numbers of patients than trials for more widely used antibiotics (see BioCentury Extra, July 13).
A breakpoint is defined as the concentration of a drug at which a given pathogen is susceptible or resistant to a particular antibiotic when used at the approved dose. Breakpoints are included on a drug's label and are generally based on preclinical and clinical data that are available at the time of approval. In October, FDA's Anti-Infective Drugs Advisory committee recommended using new PK/PD modeling that incorporates microbiologic data and some clinical data to set new susceptibility and resistance breakpoints for older antibiotics (see BioCentury, Nov. 11).
Life Technologies Corp. (NASDAQ:LIFE) and NIH's National Center for Advancing Translational Sciences (NCATS) made data from the company's Silencer Select small interfering RNA library available to researchers for free via NIH's PubChem database. The library contains 65,000 siRNA sequences targeting more than 20,000 human genes. NCATS is also releasing data on the effects of siRNA molecules on biological functions. Life Tech is being acquired by Thermo Fisher Scientific Inc. (NYSE:TMO).
According to an HHS report, 258,497 people selected a qualified health plan in the healthcare exchanges in November, bringing the total number to 364,682 since the exchanges opened on Oct. 1. The total number enrolled includes both people who have and have not paid a premium. Among those who have selected a plan through the end of November, 227,478 (62%) have enrolled through a state-run exchange and 137,204 (38%) through the federal exchange. Additionally, HHS said 1.9 million people who have filled out applications have been determined to be eligible for exchange plans but have not yet selected a plan.
Thirteen states and the District of Columbia are running their own exchanges, while the federal government is facilitating or running the exchanges in the remaining states. In May, the Congressional Budget office (CBO) estimated that 7 million people would be enrolled in insurance exchanges by the end of 2014.
On the Dec. 15 edition of BioCentury This Week television, Judith Feder, a professor at the Georgetown Public Policy Institute, the American Enterprise Institute's Joe Antos, and Paul Keckley, a healthcare analyst and former executive director of the Deloitte Center for Health Solutions, will discuss the latest numbers and what challenges lie ahead for Obamacare. The program will be broadcast at 8:30 a.m. in Washington, D.C., on WUSA, channel 9. It will be available at www.BioCenturytv.com beginning at 9 a.m., and also will be broadcast on selected PBS stations.
The Cancer Prevention and Research Institute of Texas released a request for applications for grant awards, the first request since Texas Gov. Rick Perry and state officials lifted a moratorium on the agency. CPRIT agreed to the moratorium in December 2012 at the request of state officials after the institute came under fire following public complaints by former CSO Alfred Gilman that CPRIT's management was interfering in the scientific review process (see BioCentury Extra, Oct. 31).
Last month, CPRIT named Wayne Roberts CEO. Roberts had been serving as interim executive director of the institute since December 2012. The institute also hired David Reisman as chief compliance officer. Reisman, previously executive director of the Texas Ethics Commission, will oversee compliance strategy and planning for CPRIT's grant award process. Additionally, CPRIT appointed new members to its scientific review council, including new Chairman Richard Kolodner. The council oversees the grant peer review process for CPRIT. Kolodner is a professor at the University of California, San Diego, and head of academic affairs at the Ludwig Institute for Cancer Research.