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BioCentury Extra
As published Thursday, October 08, 2015 6:29 PM PST

  • Biopharma CEOs discuss TPP with Obama

    The Pharmaceutical Research and Manufacturers of America (PhRMA) issued a statement acknowledging Thursday's meeting between President Obama and biopharma CEOs to discuss IP protections in the Trans-Pacific Partnership. The meeting included Chairman and CEO Kenneth Frazier of Merck & Co. Inc. (NYSE:MRK) and CEO George Scangos of Biogen Inc. (NASDAQ:BIIB) (see BioCentury Extra, Oct. 7).

    "Several representatives of our member companies met with the President today to discuss the Trans-Pacific Partnership (TPP) and its implications for patients," the statement said. "We emphasized that strong intellectual property protection is necessary for the discovery and development of new treatments and therapies for the world's patients and are disappointed that the TPP, which, by failing to secure 12 years of data protection for biologic medicines, will compromise the next wave of innovation and disrupt the development of new, critically-needed medicines."

  • Octapharma licenses Glycotope's coagulation factors

    Glycotope GmbH (Berlin, Germany) granted Octapharma AG (Lachen, Switzerland) exclusive, worldwide rights to develop preclinical human blood coagulation factors based on Glycotope's GlycoExpress platform.

    Glycotope's technology consists of glycoengineered human suspension cell lines which are used to produce complex proteins with human and tailored glycosylation patterns.

    Glycotope will receive an initial EUR 80 million ($90 million) payment and is eligible for milestones and royalties. Octapharma will take a minority equity stake in Glycotope. The companies did not respond to inquiries regarding the deal's financial terms.

    Octapharma's hemophilia portfolio already includes Nuwiq simoctocog alfa. FDA approved a BLA last month for the B-domain-deleted recombinant Factor VIII (rFVIII) to treat adults and children with hemophilia A. The product is produced in a genetically modified human embryonic kidney cell line. The European Commission first approved the therapy in August 2014.

    Glycotope said the deal will allow it to focus on its oncology pipeline, which includes two Phase IIb candidates: PankoMab-GEX, a humanized IgG1 antibody targeting glycoepitope and mucin 1 (MUC1; CD227), for maintenance treatment of ovarian carcinoma; and CetuGEX, a human version of cetuximab, a chimeric mAb against EGFR, to treat squamous cell carcinoma of the head and neck (SCCHN).

  • Neurocrine gains on Phase III dyskinesia data

    Neurocrine Biosciences Inc. (NASDAQ:NBIX) gained $4.67 (12%) to $43.60 after its NBI-98854 met the primary endpoint in the Phase III Kinect 3 trial to treat tardive dyskinesia. Daily 80 mg doses of NBI-98854 significantly reduced involuntary movement at week six, reducing scores from baseline on the Abnormal Involuntary Movement Scale (AIMS) by a least squares mean of 3.1 points more than placebo in the intent-to-treat population (p<0.0001).

    Next year, the company plans to submit an NDA to FDA for the selective vesicular monoamine transporter 2 (VMAT2; SCL18A2) inhibitor.

    Frequency of adverse events was similar between study arms. Neurocrine said it would release full results from the study in an upcoming publication. It also is running a one-year open-label safety study, Kinect 4, to support the NDA submission.

    In June, Teva Pharmaceutical Industries Ltd. (NYSE:TEVA) said its VMAT2 inhibitor Austedo deutetrabenazine (SD-809) significantly reduced AIMS from baseline after 12 weeks of treatment in the Phase II/III ARM-TD trial. Austedo improved AIMS by 3 points vs. 1.6 points for placebo (p=0.0188) (see BioCentury Extra, June 16).

    Teva obtained the candidate via its acquisition of Auspex Pharmaceutics Inc., which was completed in May (see BioCentury, April 6).

    Teva gained $0.69 to $59.24 Thursday.

  • BioCryst sinks on HAE updates

    BioCryst Pharmaceuticals Inc. (NASDAQ:BCRX) lost $2.12 (18%) to $9.75 on Thursday after slowing its development timeline for hereditary angioedema therapy avoralstat (BCX4161) and disclosing discussions with FDA that may delay its NDA submission for the candidate by one year. The company also reported data from a Phase I trial of BCX7353 for HAE.

    The company said it will report data in early 2016 from its Phase IIb OPuS-2 trial of avoralstat to prevent HAE; it had expected to do so by YE15.

    BioCryst also said FDA has not yet agreed to defer its requirement of a two-year rat carcinogenicity study of avoralstat. The company plans to begin the study in early 2016, and said such a study typically is required at the time of a regulatory submission. BioCryst had intended to submit an NDA for avoralstat in 2017, but said that without a deferral a submission would occur in 2018. The company plans to meet with the agency to discuss the deferral after OPuS-2 is completed.

    EMA has agreed to allow BioCryst to defer submission of its carcinogenicity results until after its MAA submission for avoralstat, a selective inhibitor of plasma kallikrein that subsequently suppresses bradykinin production.

    Also on Thursday, the company said BCX7353 demonstrated a dose-dependent effect on kallikrein inhibition (p<0.0001) and did not lead to any dose-limiting toxicities or serious adverse events (AEs) in a Phase I trial. BioCryst plans to begin a Phase II trial by early 2016 of the second-generation kallikrein inhibitor to prevent attacks in patients with HAE.

    However, two subjects discontinued the Phase I study due to moderate gastrointestinal AEs and one subject developed a delayed-type hypersensitivity rash after seven days on treatment. The rash was resolved with oral and topical steroids.

    Rival HAE candidate DX-2930 from Dyax Corp. (NASDAQ:DYAX) did not lead to any serious AEs, dose-limiting toxicities or discontinuations due to AEs while significantly reducing HAE attacks in a previous Phase Ib trial. The human mAb against plasma kallikrein, which is expected to enter Phase III testing by YE15, has Fast Track, breakthrough therapy and Orphan Drug designations from FDA to prevent HAE attacks (see BioCentury Extra, March 31).

  • Roche presents detailed ocrelizumab data

    Roche (SIX:ROG; OTCQX:RHHBY) announced data from two Phase III studies of ocrelizumab (RG1594) showing superiority to Rebif interferon beta-1a from Merck KGaA (Xetra:MRK) to treat relapsing multiple sclerosis. The company also announced data from a third Phase III study showing ocrelizumab reduced the risk of disease progression in patients with primary progressive MS (PPMS).

    Roche had already announced top-line results from the three studies. Data are to be presented at this week's European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) in Barcelona (see BioCentury Extra, June 30 and Sept. 28).

    In the identical OPERA I and OPERA II studies to treat relapsing MS, ocrelizumab reduced the annualized relapse rate (ARR) by 46% and 47%, respectively, compared to Rebif over a two-year period (p<0.0001 for both), meeting the study's primary endpoint.

    The compound also met secondary endpoints in both trials. It delayed confirmed disability progression (CDP) by about 40% sustained for 12 and 24 weeks vs. Rebif in prespecified pooled analyses of both studies (p=0.0006 and p=0.0025, respectively), as measured by the Expanded Disability Status Scale (EDSS). Ocrelizumab also led to significant reductions in acute MS-related inflammation and brain injury and reduced the emergence of more chronic or growing areas of MS-related brain injury compared with Rebif.

    In the Phase III ORATORIO study to treat PPMS, ocrelizumab significantly reduced the risk of progression of clinical disability sustained for at least 12 weeks as measured by EDSS by 24% vs. placebo (p=0.0321), meeting the primary endpoint. The compound also met secondary endpoints. It significantly reduced the risk of progression for at least 24 weeks by 25% vs. placebo (p=0.0365) and reduced timed 25-foot walk times over 120 weeks by 29% (p=0.0404). It also significantly decreased the volume of chronic inflammatory brain lesions (p<0.0001) and the rate of whole brain volume loss vs. placebo (p=0.0206).

    Roche plans to submit global regulatory applications for ocrelizumab to treat both PPMS and relapsing MS in early 2016.

    Biogen Inc. (NASDAQ:BIIB) is eligible for tiered royalties on sales of the second-generation humanized mAb against CD20 under a deal the parties amended in 2010.

  • CytomX cuts price, raises $80M in IPO

    CytomX Therapeutics Inc. (NASDAQ:CTMX) gained $0.90 to $12.90 in its first day of trading Thursday after it raised $80 million through the sale of 6.7 million shares at $12 in an IPO underwritten by BofA Merrill Lynch; Jefferies; Cowen; and Oppenheimer. The price valued the company at $419.1 million.

    CytomX filed in August to raise up to $100 million. Last week, it said it hoped to sell 6.7 million shares at $14-$16. At $15, it would have raised $100 million and been valued at $523.9 million.

    The company develops Probodies, which consist of an antibody, a peptide "mask" that blocks the antibody's active site, and an endogenous protease substrate that links the two and is cleaved in the presence of the appropriate protease to reveal an active antibody. CytomX plans to submit an IND to FDA or a similar regulatory filing to a foreign agency for CX-072 in 2H16. The compound is a Probody that inhibits PD-L1.

    CytomX is at least the fifth life sciences listing since Sept. 30 to price below its proposed range (see BioCentury Extra, Oct. 7).

  • TauRx raises $135M in equity financing

    TauRx Pharmaceuticals Ltd. (Singapore) said it received the final tranche of a $135 million equity financing that began in March. Executive Chairman Claude Wischik declined to name its investors, but said most were new investors.

    TauRx expects data next year from two Phase III trials of lead candidate LMTX to treat Alzheimer's disease and one to treat frontotemporal dementia. The second generation tau aggregation inhibitor is a modified form of rember methylthioninium chloride (methylene blue).

    Wischik told BioCentury the company has additional protein aggregation inhibitors that are Phase I-ready for neurodegenerative diseases including Parkinson's disease.

    Wischik said the company has received more than $400 million since its inception in 2002.

  • Vivus rises on Icahn debt news

    Vivus Inc. (NASDAQ:VVUS) climbed $0.18 (10%) to $2 on Thursday after the Carl Icahn's Icahn Enterprises L.P. (NASDAQ:IEP) said it acquired $170.2 million of Vivus debt from existing holders, representing 68% of the outstanding 4.5% notes due 2020. Each noteholder will receive $680 in cash per $1,000 principal amount of notes, plus accrued and unpaid interest.

    In 2013, Vivus raised $220 million through the sale of the notes, which convert into stock at $14.86. At the time, the conversion was a 9% premium to Vivus' stock price (see BioCentury Extra, Sept. 9).

  • CTTI publishes best practices for patient engagement

    Public-private partnership Clinical Trials Transformation Initiative published a set of best practices for industry, academia and patient groups to engage with patients.

    The document includes recommendations for all stakeholders; a separate set of recommendations for research sponsors including industry and academia; and a third set of recommendations for patient groups.

    The document recommends that industry sponsors match patient expertise to the specific needs and phases of R&D programs; ensure patients are "essential partners" throughout the R&D process; establish guiding principles and clear lines of communication to collaborate with patients; measure the impact of patient engagement; establish ongoing relationships with patient groups; build awareness of patient needs within companies; and create road maps to engage with patients throughout the R&D process.

    BioCentury made many of the same recommendations for industry, along with several others, in its 23rd annual Back to School essay (see BioCentury, Sept. 7).

  • Senators urge CMS to delay biosimilar reimbursement policy

    Sens. Pat Roberts (R-Kan.) and Tom Carper (D-Del.) led a bipartisan group of 20 U.S. senators who sent a letter to Acting CMS Administrator Andrew Slavitt urging the agency to withhold its biosimilar reimbursement policy until FDA has finalized regulations regarding biosimilars.

    CMS proposed a rule in July under which all biosimilars for a given reference product covered under Medicare Part B would be issued the same Healthcare Common Procedure Coding System (HCPCS) code and would therefore be reimbursed at the same rate. CMS expects to issue a final rule by Nov. 1 (see BioCentury Extra, July 10).

    Biosimilar manufacturers and members of the U.S. House of Representatives have urged CMS to drop the proposal and provide biosimilars with unique HCPCS codes and unique payment rates. Sponsors who wish to price biosimilars differently from one another would prefer to have unique codes (see BioCentury Extra, Aug. 5)

    In August, FDA issued draft guidance and a proposed rule on biosimilar naming. The agency plans to release guidance on biosimilar interchangeability this year (see BioCentury Extra, Aug. 27).

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