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BioCentury Extra
As published Thursday, January 19, 2017 6:52 PM PST


  • Collins to remain NIH director

    NIH Director Francis Collins will be "held over" by the Trump administration, spokesperson Renate Myles told BioCentury.

    On Wednesday, Myles said Collins would resign his post if he were not asked to stay by noon on Jan. 20 (see BioCentury Extra, Jan. 18).

    The agency named Collins director in August 2009.

  • BMS cuts expectations for Opdivo/Yervoy combo

    Bristol-Myers Squibb Co. (NYSE:BMY) said that it no longer plans to seek accelerated approval of Opdivo nivolumab in combination with Yervoy ipilimumab as a first-line treatment for lung cancer. The company said it based its decision on a review of available data.

    BMS has said it expects data next year from the Phase III CheckMate -227 study, which is evaluating the Opdivo/Yervoy combo, Opdivo as monotherapy, and Opdivo plus chemotherapy to treat non-small cell lung cancer (NSCLC). The company told BioCentury in August 2016 that an interim analysis is built into the study's design.

    In August, BMS's NSCLC program suffered a setback when Opdivo monotherapy missed the primary endpoint of the Phase III CheckMate -026 study. The drug failed to improve progression-free survival (PFS) vs. investigator's choice of chemotherapy in the first-line NSCLC setting (see BioCentury Extra, Aug. 5, 2016 and BioCentury, Aug. 22, 2016).

    When BMS presented CheckMate -026 data in October, it said first-line NSCLC may require combination therapies to confer benefit over chemotherapy. Opdivo led to a median PFS of 4.4 months vs. 5.9 months for chemotherapy in CheckMate -026 (see BioCentury Extra, Oct. 10, 2016).

    Opdivo is a mAb that inhibits PD-1. Yervoy is a CTLA-4 (CD152) inhibitor. Opdivo is approved to treat second-line NSCLC, with no restrictions on PD-L1 status, as well as melanoma, renal cell carcinoma and classical Hodgkin's lymphoma.

    Last week, Merck & Co. Inc. (NYSE:MRK) said FDA accepted and granted Priority Review to a BLA for competing PD-1 mAb Keytruda pembrolizumab. Merck is seeking an expanded label in first-line NSCLC that would not restrict use to PD-L1-expressing patients. In the first-line setting, Keytruda's current label restricts its use to patients with at least 50% PD-L1 expression (see BioCentury Extra, Jan. 11).

    BMS lost $0.58 to $55.49 on Thursday. It announced the update after market close, and dropped $2.89 to $52.60 in after-hours trading. Merck shed $0.84 to $60.33 during market hours, and gained $2.67 to $63 after-hours.

  • FDA approves Synergy's constipation drug

    FDA approved once-daily oral Trulance plecanatide from Synergy Pharmaceuticals Inc. (NASDAQ:SGYP) to treat chronic idiopathic constipation (CIC) in adults. Spokesperson Gem Hopkins told BioCentury that Synergy plans to launch the drug this quarter, and would disclose the price at launch.

    The agency said patients treated with Trulance may experience severe diarrhea, the drug's most common and serious side effect. FDA also stated Trulance should not be used in patients with known or suspected mechanical gastrointestinal obstructions. Trulance is a uroguanylin analog and guanylate cyclase C (GCC; GUCY2C) agonist.

    In December, Synergy said Trulance met the primary endpoint in the second of two Phase III trials to treat irritable bowel syndrome with constipation (IBS-C). Hopkins said the company plans to submit an sNDA this quarter for Trulance in that indication (see BioCentury Extra, Dec. 23, 2016).

    Ironwood Pharmaceuticals Inc. (NASDAQ:IRWD) and Allergan plc (NYSE:AGN) market GCC agonist Linzess linaclotide for both indications. Linzess' label carries a severe diarrhea warning.

    Synergy lost $0.12 (1.9%) to $6.41 on Thursday, while Ironwood slipped $0.44 to $14.76. FDA announced the approval after market hours.

  • GSK names Miels president of global pharmaceuticals

    GlaxoSmithKline plc (LSE:GSK; NYSE:GSK) named Luke Miels president of global pharmaceuticals. Miels will succeed Abbas Hussain, who will leave the company later this year. Hussain has held the position since October 2014.

    Miels is EVP of AZ's European business. GSK said his start date "will be announced in due course."

    GSK is also undergoing a CEO transition. Emma Walmsley is to succeed Sir Andrew Witty when he retires on March 31 (see BioCentury Extra, Sept. 20, 2016).

  • FDA approves Imbruvica in marginal zone lymphoma

    FDA granted accelerated approval to Imbruvica ibrutinib from AbbVie Inc. (NYSE:ABBV) and Johnson & Johnson (NYSE:JNJ) to treat relapsed or refractory marginal zone lymphoma (MZL), a type of non-Hodgkin's lymphoma (NHL). Specifically, the drug is approved to treat patients who require systemic therapy and have received at least one prior anti-CD20-based therapy. The drug is the first to be approved in the U.S. for the indication.

    AbbVie said FDA based the approval on data from the Phase II PCYC-1121 trial, in which Imbruvica led to an overall response rate (ORR) of 46% in relapsed or refractory MZL patients.

    Imbruvica is already approved in the U.S. to treat four other types of NHL: chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL), including in patients with the 17p deletion; mantle cell lymphoma (MCL) in a second-line setting; and Waldenstrom's macroglobulinemia.

    J&J holds ex-U.S. rights to the drug from AbbVie. The companies share rights in the U.S.

    Imbruvica is a Bruton's tyrosine kinase (Btk) inhibitor that covalently binds to cysteine residue 481.

  • FDA again reviewing Shire's triple-bead ADHD therapy

    Shire plc (LSE:SHP; NASDAQ:SHPG) said FDA accepted for review a resubmitted NDA for SHP465 to treat ADHD. Its PDUFA date is June 20.

    Shire hopes to launch SHP465 next half. It consists of three-component, extended release, single-entity mixed amphetamine salts (MAS).

    Shire first submitted the NDA in 2006 to treat adult ADHD. To support a resubmission, FDA requested additional pediatric data (see BioCentury Extra, April 7, 2015).

    In April 2016, SHP465 met the primary endpoint of improving ADHD symptoms among children and adolescents in a Phase III study (see BioCentury Extra, April 4, 2016).

    On Thursday, Shire shed 20p to 4,483.50p in London and lost $2.30 to $164.11 on NASDAQ.

  • Aduro using Stanford tech in next-generation LADDs

    Aduro Biotech Inc. (NASDAQ:ADRO) said it gained an exclusive license to neoantigen identification technology from Stanford University. Aduro plans to use the technology to engineer personalized Listeria-based immunotherapies encoding multiple neoantigens, with a Phase I trial to treat GI cancers planned for this year.

    Aduro said the deal gives it rights to bioinformatics algorithms and computational workflows for neoantigen identification and selection. It plans to incorporate antigens identified on patient tumors into its live-attenuated double-deleted (LADD) Listeria immunotherapy platform. The company said the personalized LADD (pLADD) therapies are designed to produce a general immune response through the presence of Listeria, as well as a tumor-specific response through neoantigen expression.

    Aduro lost $0.40 to $11 on Thursday.

  • Gastric cancer data show Opdivo's survival benefit

    Bristol-Myers Squibb Co. (NYSE:BMY) said data from a Phase III study of Opdivo nivolumab to treat gastric cancer showed that the PD-1 inhibitor led to median overall survival (OS) of 5.32 months vs. 4.14 months for placebo (p<0.0001). The trial, ONO-4538-12, evaluated Opdivo in patients with unresectable advanced or recurrent gastric cancer refractory to or intolerant of standard therapy.

    BMS and partner Ono Pharmaceutical Co. Ltd. (Tokyo:4528) had said in November that Opdivo met the trial's primary endpoint measuring OS, and that Opdivo was the first immuno-oncology candidate to show a survival benefit in gastric cancer (see BioCentury Extra, Nov. 10, 2016).

    In ONO-4538-12, Opdivo reduced the risk of death by 37% (HR:0.63, p<0.0001). BMS said 12-month OS rates were 26.6% for Opdivo vs. 10.9% for placebo. The objective response rate was 11.2% for Opdivo and zero for placebo, and median response duration was 9.53 months.

    BMS did not respond to inquiries about its regulatory plans for Opdivo in the indication.

    Pfizer Inc. (NYSE:PFE) and Merck KGaA (Xetra:MRK) are conducting a Phase III trial of PD-L1 mAb avelumab as a first-line treatment for gastric cancer. According to ClinicalTrials.gov, final data collection in the study is due to occur around November 2018. The candidate is also under FDA and EMA review to treat Merkel cell carcinoma (MCC).

    BMS presented the Opdivo results Thursday at the Gastrointestinal Cancers Symposium in San Francisco. Ono conducted the study.

  • Stanford team reports live imaging of CAR T cells

    In a paper published online by Science Translational Medicine on Wednesday, researchers reported the first PET-based imaging of chimeric antigen receptor T cells in a cohort of seven glioma patients in a clinical trial. Stanford University radiology professor Sanjiv Gambhir led the work in collaboration with researchers from Sangamo BioSciences Inc. (NASDAQ:SGMO) and the City of Hope (Duarte, Calif.).

    The PET-based technology may provide an early glimpse of response to CAR T treatment weeks to months before clinical readouts are available through conventional imaging. The technology lets clinicians follow and quantify T cells homing to tumor sites and can be used to track the cellular therapy longitudinally.

    The imaging could help prevent "pseudoprogression," a phenomenon that complicates early evaluations of tumor size in immuno-oncology trials by standard imaging methods because infiltration of therapeutic lymphocytes increases the size of tumors (see BioCentury, October 24, 2016).

    The engineered T cells studied in Wednesday's paper express the IL-13 zeta-kine CAR, which targets interleukin-13 (IL-13) receptor alpha 2 (IL-13RA2; IL-13R; CD213A2) on tumors, and a thymidine kinase reporter gene, which makes a protein that "traps" a PET-detectable radiolabeled probe.

    The researchers looked at 10 tumor foci in the seven patients. At clinically confirmed tumor sites in all patients, there was a significant increase in PET scan activity (p=0.014) after the T cell infusions. A doubling of total activity was seen for several patients. The paper did not estimate the number of tumor infiltrating T cells.

    Ganbhir told BioCentury the technology is not applicable to diffuse blood cancers such as leukemias, but has been used to monitor cardiac stem cell therapy and has been incorporated into a virus to track the pathogen during infection. Gambhir said several trials of the technology are under way in a diverse set of solid tumors.

    Gambhir said the next step is to engineer cells that are visualized only when the T cells engage tumor cells. He cited proof-of-concept animal studies that have validated the approach.

    CellCyte Genetics Corp. (OTCBB:CCYG) holds an exclusive license to the technology, Gambhir said.

  • Octimet raises EUR 11.3M in series A

    Cancer play Octimet Oncology N.V. (Beerse, Belgium) raised EUR 11.3 million ($12 million) in a series A financing led by V-Bio Ventures and Fund+. DROIA Oncology Ventures and OMNES Capital also participated.

    Octimet has licensed small molecule inhibitors of c-Met receptor tyrosine kinase (c-MET, MET, HGFR, c-Met proto-oncogene) from the Janssen Pharmaceutica N.V. unit of Johnson & Johnson (NYSE:JNJ). Its lead program, selective MET inhibitor OMO-1, has completed a Phase I trial in healthy volunteers. The company intends to study the candidate in patients with MET biomarkers, as monotherapy or in combination with targeted agents and standard of care to treat solid tumors.

    Octimet is based at J&J's JLINX incubator in Belgium. The pharma's Johnson & Johnson Innovation and Janssen Pharmaceutica units launched JLINX in March 2016 to provide start-ups with access to J&J's scientific, technical and business capabilities (see BioCentury Extra, March 16, 2016).

  • Heron raises $150M in follow-on

    Heron Therapeutics Inc. (NASDAQ:HRTX) raised $150 million through the sale of 12.3 million shares at $12.20 in a follow-on underwritten by BofA Merrill Lynch, Cowen, Leerink, Cantor Fitzgerald, JMP Securities, Noble Capital Markets, LifeSci Capital, Aegis Capital and Lake Street Capital Markets. The price is a 4.7% discount to Heron's close of $12.80 on Wednesday, before it proposed the offering after market hours.

    Heron markets Sustol granisetron extended release (APF530) to prevent chemotherapy-induced nausea and vomiting (CINV). The product is a long-acting formulation of a serotonin (5-HT3) receptor antagonist formulated using Heron's Biochronomer polymer-based drug delivery technology.

    The company has submitted an NDA to FDA for Cinvanti (HTX-019), another candidate for CINV. It is a polysorbate 80-free formulation of aprepitant, which antagonizes the tachykinin receptor (TACR1; SPR; NK1R).

    Heron also hopes to start Phase III testing this year of HTX-011 to treat postoperative pain. HTX-011 is a long-acting formulation of the local anesthetic bupivacaine in a fixed-dose combination with the anti-inflammatory meloxicam formulated using Biochronomer.

    Heron gained $1.05 to $13.85 on Thursday.

  • Zynerba raises $50.4M in follow-on

    Zynerba Pharmaceuticals Inc. (NASDAQ:ZYNE) raised $50.4 million through the sale of 2.8 million shares at $18 in a follow-on underwritten by Jefferies, Piper Jaffray, Cantor Fitzgerald, Oppenheimer and Roth Capital Partners. The price is a 20% discount to Zynerba's closing price of $22.46 on Wednesday, before it proposed the offering after market hours. Zynerba lost $4.35 (19%) to $18.11.

    Zynerba's ZYN002 is in Phase II studies to treat refractory epilepsy patients with focal seizures, knee pain due to osteoarthritis (OA) and fragile X syndrome. The candidate is a synthetic cannabidiol (CBD) formulated as a transdermal gel.

    The company also hopes to start a Phase I trial this half of ZYN001 to treat fibromyalgia and peripheral neuropathic pain. ZYN001 is a prodrug of delta-9-tetrahydrocannabinol (THC) delivered via the skin with a patch.

  • FDA declines to discuss Kalavritinos report

    FDA declined to comment Thursday on a report that former lobbyist Jack Kalavritinos will take on a major role at the agency in the Trump administration. Kalavritinos is a member of the Trump transition team and was the director of government affairs at Covidien plc, which Medtronic plc (NYSE:MDT) acquired in 2015.

    Multiple sources who did not wish to be identified told BioCentury that Kalavritinos would have a role at FDA, but did not specify a title. Kalavritinos did not respond to inquiries.

    Kalavritinos was also director of HHS's Office of Intergovernmental Affairs and a White House liaison for the Department of Labor.

    FDA Commissioner Robert Califf's resignation will go into effect at noon on Jan. 20. The agency has not officially named an acting director to succeed him, but FDA regulations call for the deputy commissioner for foods and veterinary medicine, currently Stephen Ostroff, to hold the position until the U.S. Senate confirms a permanent successor.

  • FDA officially launches Oncology Center of Excellence

    FDA formally established the agency's Oncology Center of Excellence and named Richard Pazdur as its director. Pazdur had been acting director since the center was created in June 2016 to coordinate and streamline clinical reviews of cancer products among the agency's centers for drugs, biologics and medical devices (see BioCentury Extra, June 29, 2016).

    The agency established the center within its Office of Medical Products and Tobacco.

    In a statement Thursday, FDA Commissioner Robert Califf said oncology is now the first disease area for which reviews will be coordinated across the agency's three medical product centers.

  • NICE pauses appraisal cost recovery plan

    The U.K.'s NICE said it will delay its plan to recover appraisal costs of drugs, medical devices and diagnostics until the U.K. government finishes its life sciences strategy.

    NICE had planned to charge companies for appraisals so that the agency could be "more flexible and more responsive to changes in demand."

    Currently, appraisals are funded by a grant from the Department of Health.

    In a July 2015 triennial review, NICE's Cabinet Office proposed charging companies for the appraisals.


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