BioShares Biotechnology Clinical Trials (BBC): $19.15, +$0.09, -34.1% YTD

BioShares Biotechnology Products (BBP): $34.09, +$0.13, +10.6% YTD

 

 

MARKET COMMENTARY

 

U.S. stocks looked set to open little changed on Monday as investors awaited a speech by Federal Reserve Chair Janet Yellen, less than a week after the Fed raised interest rates for only the second time since the financial crisis.  U.S. stocks fell on Friday, weighed by a more than 4 percent drop in Oracle ORCL.O shares and news that a Chinese Navy warship seized a U.S. underwater drone.  Still, the Dow posted its sixth straight week of gains, its longest streak in a year. The blue-chip index remains less than 1 percent away from 20,000, a level it has never breached.  U.S. stocks have been on a tear since the Nov. 8 presidential election, with the S&P rising 5.7 percent on bets that President-elect Donald Trump’s expected deregulation and infrastructure spending will boost the economy.  However, there are some concerns that the rally may run out of steam as policy will take time to be implemented and will likely change as it makes its way through Congress.

 

 

MARKET HIGHLIGHTS

 

Innate Pharma SA announced key leadership team and Board changes, including the appointment of Mondher Mahjoubi as Chairman of Innate Pharma’s Executive Board, succeeding Hervé Brailly who becomes Chairman of the Supervisory Board. The leadership team and Board changes will take effect on December 30th, 2016.

 

Threshold Pharmaceuticals has entered into a collaboration with the National Cancer Institute (NCI), part of the National Institutes of Health (NIH), to study TH-3424, the company’s new drug candidate for the treatment of cancer. The collaboration will explore the effects of TH-3424 against T-cell acute lymphoblastic leukemia (T-ALL) xenograft cell lines with high AKR1C3 expression. The studies will be conducted through the NCI-funded Pediatric Preclinical Testing Consortium (PPTC). Under this collaboration, Threshold will supply TH-3424, and the NCI will fund the studies that will be conducted at the PPTC leukemia research program led by Professor Richard Lock of Children’s Cancer Institute (Sydney, Australia).

 

Catalyst Biosciences announced it has secured all rights to the manufacturing process for marzeptacog alfa (activated) (formerly known as CB 813d) from Wyeth LLC, a wholly-owned subsidiary of Pfizer. Marzeptacog alfa (activated) is a next-generation Factor VIIa product that was designed to allow for the effective, long-term, subcutaneous prophylaxis in hemophilia patients with inhibitors. Catalyst has successfully completed an intravenous Phase 1 clinical trial of marzeptacog alfa (activated) in patients with severe hemophilia A and B with and without inhibitors and has demonstrated the feasibility of subcutaneous dosing in preclinical models.

 

BrainStorm Cell Therapeutics recently completed a successful End-of-Phase 2 Meeting with the FDA. Brainstorm has reached general agreement with the FDA to proceed to a Phase 3 trial. Importantly, the FDA has accepted the key elements of the Phase 3 program to support a Biologic License Application (BLA) for NurOwn in ALS. The planned Phase 3 clinical trial will be a randomized, double-blind, placebo-controlled multi-dose trial that will be conducted at multiple sites in the US and in Israel. The trial is expected to begin enrolling patients in Q2/17.  Brainstorm plans to submit an application in Israel that will allow patient access to NurOwn as a treatment that has been granted Hospital Exemption. This recently approved pathway would permit Brainstorm to partner with a medical center in Israel and be allowed to treat patients with NurOwn for a fee.

 

MediciNova announced that the external Data and Safety Monitoring board (DSMB) for the ongoing Phase 2B clinical trial of MN-166 (ibudilast) in progressive multiple sclerosis (progressive MS) has reviewed the results of the interim efficacy analysis and made the recommendation to the NIH National Institute of Neurological Diseases and Stroke (NINDS) that the trial should continue as planned and this recommendation was accepted by NINDS.

 

Spring Bank Pharmaceuticals and Arbutus Biopharma Corporation announced an agreement to perform collaborative preclinical studies in chronic Hepatitis B virus (HBV) involving the co-administration of Spring Bank’s SB 9200, an orally-available selective immune-modulator, and Arbutus Biopharma’s AB-423, a capsid assembly inhibitor.  SB 9200 and AB-423 are each investigational compounds currently under development by the respective companies for the treatment of chronic HBV.

 

Fibrocell Science named John Maslowski CEO and Douglas Swirsky has been appointed Chairman; to succeed David Pernock effective immediately.  Maslowski joined Fibrocell in 2005 and most recently served as the company’s SVP of Scientific Affairs with oversight of research and development, clinical and regulatory

 

Orexigen announced that its wholly owned subsidiary Orexigen Therapeutics Ireland Ltd. and Biologix FZCO, have executed a commercialization and distributorship agreement in the Middle East for Contrave (naltrexone HCl / bupropion HCl prolonged release) monotherapy for weight management in overweight or obese adult patients.  This agreement covers ten countries in the Middle East: Saudi Arabia, the United Arab Emirates, Kuwait, Oman, Qatar, Bahrain, Lebanon, Jordan, Iraq and Iran.  Under the terms of the agreement, Biologix will be responsible for obtaining regulatory approvals and local product registrations in each of the ten countries and for all commercialization activities.  Orexigen will supply Contrave to Biologix at an agreed transfer price.  Biologix expects Contrave to be available for patients in some countries starting in Q3 of 2017.

 

Portola Pharmaceuticals has signed a $50M loan agreement with Bristol-Myers Squibb Company and Pfizer Inc. that provides additional funding toward development and clinical studies of AndexXa (andexanet alfa).  Under the terms of the agreement, Bristol-Myers Squibb and Pfizer will each loan Portola $25M.  The principal and interest will be repaid primarily through royalties on AndexXa commercial sales.  No shares, warrants, options or other equity components were or will be issued in connection with the loan. The non-secured loan does not involve any transfer of patent ownership or licenses.  Portola previously entered into two separate clinical collaboration agreements with Bristol-Myers Squibb and Pfizer to support Phase 2 and registrational studies of andexanet alfa in the United States and Europe.  Bristol-Myers Squibb and Pfizer also have a collaboration agreement with Portola to develop and commercialize andexanet alfa in Japan.

 

Tetraphase Pharmaceuticals to submit MAA to the EMA for IV eravacycline for the treatment of cIAI in the second half of 2017.  The MAA submission will be supported by data from IGNITE1, the company’s successfully completed phase 3 clinical trial which evaluated the efficacy and safety of twice-daily IV eravacycline for the treatment of cIAI.  In addition to IGNITE1, eravacycline is currently being evaluated in IGNITE4, a phase 3 clinical trial assessing the efficacy and safety of twice-daily IV eravacycline compared to meropenem in cIAI.  As previously stated, Tetraphase expects to report top-line data from IGNITE4 during Q4 of 2017. Assuming a successful outcome from IGNITE4, these data, along with data from IGNITE1, will support an NDA filing to the FDA for IV eravacycline in cIAI.

 

RedHill Biopharma announced that the first patients have been screened and a first patient has been dosed in the Phase Ib/II clinical study evaluating YELIVA (ABC294640) in patients with refractory or relapsed multiple myeloma.  The open-label, dose escalation Phase Ib/II study is being conducted at Duke University Medical Center and is expected to enroll up to 77 patients with refractory or relapsed multiple myeloma who have previously been treated with proteasome inhibitors and immunomodulatory drugs.  The study is supported by a $2M grant from the National Cancer Institute (NCI) Small Business Innovation Research Program (SBIR) awarded to Apogee Biotechnology Corp. (Apogee), in conjunction with Duke University, with additional support from RedHill.

 

AbbVie has submitted a New Drug Application (NDA) to the FDA for the company’s investigational, pan-genotypic regimen of glecaprevir/pibrentasvir (G/P), being evaluated for the treatment of chronic hepatitis C virus (HCV).  AbbVie is on track to submit a Marketing Authorization Application for G/P in the European Union in early 2017.

 

Eagle Pharmaceuticals announced that David Pernock will join Eagle as President and Chief Commercial Officer, effective January 2017. Mr. Pernock brings vast experience in pharmaceutical and biotechnology leadership to the Company. He will be responsible for all commercial strategy and execution for Eagle’s growing product portfolio, including the launch of expanded indications for Ryanodex® for Exertional Heat Stroke (“EHS”) and drug induced hyperthermia, if approved by the Food and Drug Administration (“FDA”). Mr. Pernock will report to Scott Tarriff, Eagle’s Chief Executive Officer.  David Pernock has been a member of Eagle’s Board of Directors since April 2015. Mr. Pernock will relinquish his Eagle Board position effective January 2017, in connection with his new role as President and Chief Commercial Officer.

 

Loxo Oncology announced a comprehensive program update for larotrectinib (LOXO-101) and its pipeline drug candidates, LOXO-292 and LOXO-195.  Loxo Oncology’s larotrectinib program is currently ~85% enrolled to goal, and the company plans to complete enrollment for the primary efficacy analysis in early 2017.  The efficacy and safety database sizes for larotrectinib will be within precedents set by prior targeted therapy drug approvals in oncology.  The company expects to be in a position to report top-line data for the NDA dataset in H2 of 2017 and expects to submit a NDA in late 2017 or early 2018 and a European MAA in 2018.

 

Adaptimmune Therapeutics and Bellicum Pharmaceuticals have entered into a staged collaboration to evaluate, develop, and commercialize next-generation T-cell therapies.  Under the agreement, the companies will evaluate Bellicum’s GoTCR technology (inducible MyD88/CD40 co-stimulation, or iMC) with Adaptimmune’s affinity-optimized SPEAR T-cells for the potential to create enhanced TCR product candidates.  Depending on results from the preclinical proof-of-concept phase, the companies expect to progress to a two-target co-development and co-commercialization phase.

 

Novocure’s announced that the Japanese Ministry of Health, Labour and Welfare has approved Optune (NovoTTF-100A) – Novocure’s Tumor Treating Fields delivery system – in the treatment of adult patients with supra-tentorial glioblastoma (GBM) following maximal safe surgical resection and radiation therapy.  Novocure will prepare to submit an application for public reimbursement of Optune for newly diagnosed GBM in Japan.

 

Cepheid announced that it has received clearance from the FDA for Xpert MRSA NxG, the next generation methicillin-resistant Staphylococcus aureus (MRSA) infection control test from the leader in healthcare-associated infection (HAI) testing.  Xpert MRSA NxG is an accurate, on-demand, molecular test that delivers actionable results in about an hour.

 

Editas Medicine enters license agreements related to CRISPR technologies.  Under the terms of the combined licenses for Cpf1, advanced forms of Cas9, and additional Cas9-based genome editing technologies from the Broad Institute, Harvard University, MIT, Wageningen University, the University of Iowa, and the University of Tokyo, Editas Medicine will make total upfront cash payments of $6.25M and issue a promissory note totaling $10M that can be settled in stock or cash over a predefined period.  In the future, Editas Medicine may make additional payments, in cash or stock upon reaching goals and targets related to research and development, commercialization, and market capitalization, and will pay royalties on products based on these technologies.

 

Ionis reports phase 3 COMPASS study of Volanesorsen meets primary endpoint.  For the primary endpoint of the study, volanesorsen-treated patients (n=75) achieved a statistically significant (p<0.0001) mean reduction in triglycerides of 71.2% from baseline after 13 weeks of treatment, compared with a mean reduction of 0.9% in placebo-treated patients (n=38).  This represented a mean absolute reduction of 869 mg/dl in treated patients.  The treatment effect observed was sustained through the end of the 26 week treatment period.  In addition, 82% of patients treated with volanesorsen, including three of the FCS patients, achieved triglyceride levels less than 500 mg/dl after 13 weeks of treatment, compared to 14% of placebo-treated patients (p<0.0001).  The most common adverse event in the volanesorsen-treated group of patients was injection site reactions (ISRs), which were mostly mild.  In this study with patients who are largely asymptomatic and, unlike FCS patients, do not need to manage the daily burden and symptoms of their disease, 13% of treated patients discontinued due to ISRs and 7% of treated patients discontinued treatment for other non-serious adverse events.  There were no deaths in the study.  There were no serious platelet events in the study.  There was one potentially related SAE on the drug-treated arm. This was a report of serum sickness that occurred two weeks after the last study dose and resolved without treatment, and after thorough investigation the sponsor determined that the case was not likely caused by the drug.

 

Merck, known as MSD outside the United States and Canada, announced that KEYTRUDA (pembrolizumab), the company’s anti-PD-1 therapy, has been approved in Japan for the treatment of certain patients with PD-L1-positive unresectable advanced/recurrent non-small cell lung cancer (NSCLC) in the first- and second-line treatment settings at a fixed dose of 200 mg every three weeks.  MSD will manufacture and market KEYTRUDA in Japan and will promote it with Taiho Pharmaceutical Co., Ltd.

 

Cerus provided an update on the timeline for the INTERCEPT Blood System for Red Blood Cells (RBCs) in Europe.  The target timing for CE Mark submission has been extended, and the company’s European red cell study in chronic anemia patients has completed enrollment.  Due to the need for additional time to complete quality control tests on the Chemistry, Manufacturing, and Control (CMC) registration lots required for regulatory submission, the company now plans to provide an update on new submission timing on its 4Q16 earnings call in early March.

 

Cerus announced that additional options totaling $10,825,555 have been exercised under its contract with the Biomedical Advanced Research and Development Authority (BARDA).  The newly exercised options include funding for in vitro pathogen inactivation and red blood cell (RBC) function studies to support FDA licensure, as well as the manufacturing of clinical trial materials needed for a Phase III trial in the continental United States.  As part of the agreement, base period funding totaling $31M was previously allocated to support a clinical trial known as “RedeS” to assess the safety and efficacy of INTERCEPT RBCs compared to conventional RBCs in regions heavily impacted by the Zika virus epidemic including Puerto Rico, as well as activities related to in vitro studies to facilitate potential pivotal Phase III clinical trials in the continental U.S.  The base period funding also can be used to extend the RedeS trial to Florida which has now become epidemic for Zika

 

Kite Pharma announced several updates to its broad intellectual property portfolio relating to the use of chimeric antigen receptors (CARs) to harness the power of a patient’s immune cells.  These updates include a recent U.S. Patent and Trademark Office (USPTO) decision concerning one narrow patent related to CAR products containing a pre-specified CD28 costimulatory domain, and a recent favorable result in a challenge at the USPTO to one of Kite’s important CAR-T patents, U.S. Patent Number 6,319,494.

In August 2015, Kite preemptively filed a petition with the U.S. Patent and Trademark Office (USPTO) to institute an inter partes review (IPR) of U.S. Patent No. 7,446,190 owned by Sloan Kettering Institute for Cancer Research and licensed by Juno Therapeutics, Inc (JUNO).  The USPTO’s recent ruling in this matter did not revoke the patent. However, Kite continues to believe the patent to be invalid and plans to appeal the USPTO decision to the U.S. Court of Appeals for the Federal Circuit.  This patent does not have any counterpart patents outside of the United States.  The USPTO’s decision will have no impact on the timing of the rolling submission or review of the Biologics License Application for Kite’s lead product candidate, axicabtagene ciloleucel (KTE-C19).  Axicabtagene ciloleucel is currently being developed for the treatment of CD19 positive B cell malignancies, including non-Hodgkin lymphoma and acute lymphoblastic leukemia.  Separately, Kite recently defeated an anonymous challenge filed against U.S. Patent Number 6,319,494, developed by Kite’s SVP of Discovery Research, Margo Roberts, Ph.D. and colleagues. This patent, which covers methods for treating a viral disease or malignancy using modified T cells that contain single-chain variable fragment (scFv) binding elements and other key CAR-T features, impacts multiple competitor CAR-T cell product candidates under development.

 

uniQure BV approves appointment of Matthew Kapusta as CEO, effective immediately.  Kapusta has served as interim CEO since September 2016 and as CFO since January 2015.  Kapusta will continue to serve as an executive member of the uniQure board.

 

Pfizer announced the European Commission has approved an expanded indication for Nimenrix (meningococcal group A, C, W-135, and Y conjugate vaccine) for active immunization against invasive meningococcal disease (IMD) caused by Neisseria meningitidis serogroups A, C, W-135, and Y (MenACWY) in infants as early as six weeks of age.

 

ProMetic Life Sciences announced that it has initiated the rolling submission of its Biologics License Application for plasminogen with the U.S. FDA for treatment of patients with plasminogen congenital deficiency.  Plasminogen’s Fast Track designation allows rolling submissions of portions of the regulatory application to be submitted and reviewed by the FDA on an ongoing basis.  The BLA submission for plasminogen has commenced on schedule, and ProMetic is on target for an expected commercial launch of plasminogen in the USA in 2017.

 

AmpliPhi Biosciences reported final results from Phase 1 trial of AB-SA0 targeting Staphylococcus aureus infections, in patients with chronic rhinosinusitis.  AB-SA01 met the trial’s primary endpoints of safety and tolerability and all nine patients enrolled in the study experienced a reduction in the quantity of S. aureus infecting their sinuses, with some patients showing complete eradication of the bacterial infection.  Key findings from the study include:  Primary endpoints of safety and tolerability were met; All patients experienced a reduction in S. aureus bacterial load at the end of the study compared to baseline; Comparison of pre- and post-treatment endoscopic images showed symptomatic improvement, including reductions in mucosal edema, discharge and polyps; All enrolled patients completed the trial.  Detailed results from the Phase 1 trial of AB-SA01 in CRS patients will be presented by Dr. Mian Ooi, of the University of Adelaide and The Queen Elizabeth Hospital’s department of Otolaryngology, Head and Neck Surgery, at an upcoming medical conference in 2017.

 

CytRx names chief medical officer Daniel Levitt to newly created role of COO.  Going forward, Levitt will continue to serve as CytRx’s Chief Medical Officer, a role he has held in since joining CytRx in 2009.