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BioShares Biotechnology Products (BBP): $38.40, +$0.35

 

 

MARKET COMMENTARY

 

U.S. stock index futures slipped ahead of growth data which will shed light on the health of the economy. Jobless claims data is on the radar. European shares seesawed as investors mulled implications of Britain formally beginning the process of leaving the European Union, while Asian stocks ended lower. The dollar rose against a basket of currencies, while gold dipped. Oil prices fell as ballooning U.S. inventories foreshadowed the impact of supply disruptions in Libya.

 

 

MARKET HIGHLIGHTS

 

Mateon Therapeutics announced 2016 financial results.  For the year ended December 31, 2016, Mateon reported a net loss of $13.7 million, similar to the net loss for the year ended December 31, 2015.  At December 31, 2016, Mateon had cash and short-term investments of $12.0 million.

 

VBI Vaccines announced the appointment of Avi Mazaltov as the Global Head of Manufacturing and General Manager of the SciVac manufacturing site in Rehovot, Israel.  In this new role, Mr. Mazaltov will define and manage VBI’s manufacturing activities, including SciVac’s manufacturing and commercialization operations in Israel.

 

Vermillion reported fourth quarter and full year ended December 31, 2016 financial results.  For the full year of 2016, net loss was $15.0 million or $(0.29) per share as compared to a net loss of $19.1 million or $(0.41) per share in 2015.  As of December 31, 2016, cash and equivalents totaled $5.2 million.

 

ASPiRA Labs, a Vermillion company announced it has signed an in-network, contracted agreement with TriCare South for ASPiRA’s U.S. FDA cleared, Centers for Medicare and Medicaid Services (CMS) covered, American College of Obstetricians and Gynecologists (ACOG) Level B recommended ovarian cancer risk assessment test, OVA1® (Multivariate Index Assay or MIA). TriCare South serves about 2.5 million beneficiaries in the states of Alabama, Arkansas, Florida, Georgia, Louisiana, Mississippi, Oklahoma, South Carolina, Tennessee, Texas (excluding El Paso) and Fort Campbell, Kentucky.

 

Ignyta announced data demonstrating the successful treatment with entrectinib – Ignyta’s investigational, orally available, CNS-active tyrosine kinase inhibitor targeting tumors that harbor TRK, ROS1 or ALK fusions – of a patient with a primary brain tumor harboring an NTRK1 fusion. The study, exploring genetic alterations associated with glioneuronal tumors, was led by researchers at Massachusetts General Hospital and was published in Precision Oncology. Researchers discovered novel oncogenic fusions involving members of the NTRK gene family in three out of 26 patients evaluated, and reported that in a patient with a BCAN-NTRK1 fusion, treatment with entrectinib resulted in a 60 percent regression in tumor size and the resolution of clinical symptoms that was maintained for 11 months on treatment. Entrectinib is currently being studied in a registration-enabling Phase 2 clinical trial known as STARTRK-2.

 

Aeglea BioTherapeutics announced that it will deliver a poster presentation, “Reducing systemic arginine with arginase (AEB1102) therapy does not suppress the immune response induced by anti-PD-1 and anti-PD-L1, and exerts an additive anti-tumor and synergistic survival benefit”at the 2017 American Association for Cancer Research (AACR) Annual Meeting taking place April 1 – 5 in Washington, DC.

 

BrainStorm Cell Therapeutics announced financial results for the year ended December 31, 2016.  Net loss for the year ended December 31, 2016, was $5.0 million, or ($0.27) per share, compared with a net loss of $8.5 million, or ($0.46) per share, for the year ended December 31, 2015.  As of December 31, 2016, BrainStorm had approximately $10.0 million in cash, cash equivalents and short-term deposits, compared with approximately $16.0 million at December 31, 2015.

 

Achaogen will be presenting at Needham & Company’s 16th Annual Healthcare Conference at 10:40 a.m. Eastern Time on Tuesday, April 4th.

 

CymaBay Therapeutics announced that management will provide a corporate overview at the H.C. Wainwright & Co. 1st Annual NASH Investor Conference on Monday, April 3rd @ 11:40amET, and the BioCentury Future Leaders in the Biotech Industry Conference on Friday, April 7th @ 1:30pmET.

 

TRACON Pharmaceuticals will present at the 16th Annual Needham Healthcare Conference at 2:20 pm EDT on Wednesday, April 5.

 

Moberg Pharma AB announced today that, following a successful test launch in Japan, Moberg Pharma’s local partner CMIC Group will initiate a national roll-out of Zanmira® Nail (Kerasal Nail®) in several major cities during the second quarter.

 

Progenics Pharmaceuticals announced that the Company’s registrational Phase IIb trial of its novel radiotherapeutic candidate, AZEDRA (iobenguane I 131) Injection, has achieved its primary endpoint. The open-label, multi-center study was conducted under a SPA agreement with the FDA. The trial was designed to evaluate the efficacy and safety of AZEDRA in patients with malignant and/or recurrent pheochromocytoma or paraganglioma, which are rare neuroendocrine tumors. There are currently no approved therapeutics in the U.S. for the treatment of malignant and/or recurrent pheochromocytoma or paraganglioma. AZEDRA has received Breakthrough Therapy, Orphan Drug and Fast Track designations from the FDA.

 

Omeros announced additional positive data from the company’s Phase II clinical trial of OMS721 for the treatment of serious kidney disorders, which frequently lead to end-stage renal disease and dialysis. OMS721 is Omeros’ lead human monoclonal antibody targeting mannan-binding lectin-associated serine protease-2 (MASP-2), the effector enzyme of the complement system’s lectin pathway. The clinical trial data include results from additional study patients treated with OMS721 as well as longer-term follow-up on earlier enrolled patients. The new data corroborate and expand on trial results reported in the fourth quarter of 2016. Based on the uniformly positive data in the fully enrolled cohort of OMS721-treated patients with immunoglobulin A nephropathy (IgAN), Omeros recently met with the FDA to discuss the Phase III development program.

 

Karyopharm Therapeutics announced that the FDA Division of Hematology Products has lifted the partial clinical hold placed on the clinical trials for selinexor (KPT-330), enabling patient enrollment and dosing of new patients in the Company’s clinical trials of selinexor in hematological malignancies. The partial clinical hold was not the result of any patient death or any change in the safety profile of selinexor.  Enrollment may now resume in all selinexor studies in hematologic malignancies, including the STORM study in refractory multiple myeloma, the SADAL study in relapsed/refractory diffuse large B-cell lymphoma (DLBCL), and the STOMP study of selinexor and backbone therapies in multiple myeloma.  In addition, Investigator Sponsored Trials in hematologic malignancies with selinexor may resume accruing patients.

 

Amgen announced that the FDA has accepted for priority review the sBLA for BLINCYTO (blinatumomab) to include overall survival (OS) data from the Phase III TOWER study. The application also includes new data supporting the treatment of patients with Philadelphia chromosome-positive (Ph+) relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL). The application aims to expand BLINCYTO’s indication for the treatment of all patients with relapsed or refractory B-cell precursor ALL and supports the conversion of BLINCYTO’s accelerated approval to full approval.

 

Novartis announced that the FDA has accepted the company’s BLA filing and granted priority review for CTL019 (tisagenlecleucel-T), an investigational chimeric antigen receptor T cell (CAR-T) therapy, in relapsed and refractory (r/r) pediatric and young adult patients with B-cell acute lymphoblastic leukemia (ALL). This is the first BLA submission by Novartis for a CAR-T. The priority review designation is expected to shorten the anticipated review time by the FDA.

 

Upsher-Smith Laboratories announced that it has received FDA final approval of two sNDAs for Qudexy XR (topiramate) Extended-Release Capsules for use as prophylaxis of migraine headache in adults and adolescents 12 years of age and older. Topiramate is the molecule that neurologists prescribe most frequently for the prevention of migraines, and the American Academy of Neurology and the American Headache Society have given topiramate a "top tier" rating for migraine prevention in adults. A study designed to address post-marketing requirements in the United States is planned to evaluate the efficacy and safety of Qudexy XR for the prophylaxis of migraine in pediatric patients ages 6-11. Upsher-Smith was granted tentative approval of its sNDA in April 2016, but was not eligible to receive final approval until after the innovator drug’s exclusivity for the adolescent population expired in March 2017.

 

Agenus announced that it is reorganizing its business and operations to sharpen its focus on clinical development of its two checkpoint inhibitor antibodies and vaccine program. Agenus plans to close its Basel site and consolidate key functions to its Cambridge, UK and Lexington, MA facilities, and phase out approximately 50 positions across the organization. Additionally, Robert Stein, M.D., Ph.D., President of R&D, will retire to become a senior R&D advisor exclusive to Agenus.

 

Bristol-Myers Squibb and Foundation Medicine announced a collaboration that leverages Foundation Medicine’s comprehensive genomic profiling and molecular information solutions to identify predictive biomarkers such as Tumor Mutational Burden (TMB) and Microsatellite Instability (MSI) in patients enrolled across clinical trials investigating Bristol-Myers Squibb’s cancer immunotherapies. Biomarkers can be used to characterize a tumor and the tumor microenvironment, which may reveal immune-related mechanisms predictive of how a patient may respond to immunotherapy.

 

Anthera Pharmaceuticals announced the findings from the Extension Period of the Phase III SOLUTION, non-inferiority clinical study in cystic fibrosis patients with exocrine pancreatic insufficiency (EPI). The Extension Period was designed as an observational analysis of the long-term effects of Sollpura and Pancreaze as it pertains to weight, height, BMI, and safety.

 

Advanced Accelerator Applications S.A. announced a 10-year exclusive supply agreement for lutetium 177 with the University of Missouri Research Reactor (MURR). Through this agreement, MURR will supply AAA with GMP-quality lutetium 177 Chloride, the precursor for production of investigational product lutetium Lu 177 dotatate (Lutathera and other Lu 177-based therapeutics in development.

 

Invitae announced a major expansion of its genetic testing services with the introduction of exome sequencing and interpretation services, bringing the company’s available test menu to more than 20,000 genes.

 

Valeant Pharmaceuticals International‘s wholly owned subsidiary, Bausch Lomb,announced the launch of Bausch + Lomb ULTRA for Astigmatism contact lenses, an innovative monthly silicone hydrogel lens that combines MoistureSeal technology, which helps to maintain 95% of lens moisture for a full 16 hours, and OpticAlign design, engineered for stability to promote a successful first fit.

 

Halozyme Therapeutics announced it has been informed by SWOG, an independent network of researchers that design and conduct cancer clinical trials, that the SWOG Phase Ib/II trial evaluating PEGPH20 plus modified FOLFIRINOX chemotherapy versus modified FOLFIRINOX alone in patients with previously untreated metastatic pancreas cancer has been temporarily closed to enrollment.

 

Shire announced that its partner in Japan, Shionogi & Co., has received the approval of the Japanese Ministry of Health, Labor and Welfare to manufacture and market INTUNIV. INTUNIV is a new, once-daily non-stimulant indicated for the treatment of ADHD.

 

Mundipharma announced that mundesine, a Purine-nucleoside phosphorylase (PNP) inhibitor, has been approved for the treatment of relapsed/refractory PTCL by the Ministry of Health, Labor and Welfare in Japan. The Minister’s decision follows successful clinical trials and makes Japan the first in the world to make mundesine available for treatment.

 

Egalet announced that one of the company’s specialty sales forces has begun promotion of ARYMO ER (morphine sulfate) extended-release tablets for oral use only –CII. Egalet executives will commemorate the product’s official launch by ringing the Nasdaq Stock Market closing bell.

 

Jazz Pharmaceuticals announced that it has entered into license agreements withNippon Shinyaku for Defitelio (defibrotide sodium) and Vyxeos (cytarabine and daunorubicin liposome injection), or CPX-351, in Japan. Under the terms of the agreements, Nippon Shinyaku will receive exclusive rights to develop and commercialize Defitelio and Vyxeos in Japan in return for an upfront payment to Jazz Pharmaceuticals and subsequent payments based on the successful achievement of certain regulatory and commercial milestones. Jazz Pharmaceuticals will manufacture and supply Defitelio and Vyxeos to Nippon Shinyaku, and will receive revenue based on a percentage of product sales in Japan. Financial terms of the agreement have not been disclosed.

 

Lannett announced that the company voluntarily made a $25 million payment against its existing revolving credit facility.

 

Spectrum Pharmaceuticals announced that the University of Texas MD Anderson Cancer Center initiated a Phase II trial of poziotinib in NSCLC patients with EGFR exon 20 insertion mutations. This Phase II trial will evaluate Objective Response Rate (ORR) as the primary endpoint and is expected to yield preliminary results before year-end.

 

FibroGen announced that clinical results from two Phase II studies in China of roxadustat for the treatment of anemia in patients with chronic kidney disease (CKD) were published in the journal Nephrology Dialysis Transplantation. In these studies, in dialysis-dependent, and in non-dialysis-dependent CKD patients, roxadustat corrected and maintained hemoglobin levels regardless of the patients’ baseline iron repletion status, and levels of C-reactive protein (CRP), a marker of inflammation, consistent with what was seen in previously published results from the roxadustat global program. The publication, entitled “Phase 2 studies of oral hypoxia-inducible factor prolyl hydroxylase inhibitor FG-4592 for treatment of anemia in China,” is now available online at Nephrol Dial Transplant 2017 gfx011.

 

Takeda Pharmaceutical Company announced that data from a 6-month interim analysis of the ongoing DEN-204 trial of its live-attenuated tetravalent dengue vaccine candidate, TAK-003 (also referred to as TDV), have been published in The Lancet Infectious Diseases.

 

Akcea Therapeutics, a subsidiary of Ionis Pharmaceuticals announced the initiation of a Phase IIb dose-ranging study of AKCEA-APO(a)-L(Rx) in patients with hyperlipoproteinemia(a) and established cardiovascular disease. The goal of the study is to determine the dose level and frequency for use of AKCEA-APO(a)-L(Rx) in a planned Phase III cardiovascular outcome study.

 

AbbVie announced that the FDA approved the inclusion of moderate to severe fingernail psoriasis data in the HUMIRA (adalimumab) prescribing information for patients with moderate to severe chronic plaque psoriasis. HUMIRA is now the first-and-only biologic treatment with data on fingernail psoriasis in its U.S. prescribing information.

 

Vanda Pharmaceuticals announced that it has entered into a license agreement with UC San Francisco (UCSF), under which Vanda will acquire an exclusive worldwide license from UCSF to develop and commercialize a portfolio of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) activators and inhibitors.

 

Alzheon announced the results from studies of ALZ-801 and its active molecule, tramiprosate. The new clinical data and analyses support Alzheon’s study design for the pivotal clinical program for ALZ-801, a first-in-class, oral amyloid-targeted medicine, in a genetically-defined population of patients at the Mild stage of Alzheimer’s disease, with ongoing amyloid accumulation. In addition, the company presented clinical data with pharmacokinetic and pharmacodynamics analyses that support the dose selection of ALZ-801 for the pivotal study expected to initiate in 2017.

 

Aura Biosciences announced that it has enrolled and dosed the first patient in its Phase Ib clinical trial of light-activated AU-011, an investigational, first-in-class targeted therapy in development for the treatment of ocular melanoma. Aura additionally announced that the FDA has granted AU-011 fast track designation for the treatment of primary ocular melanoma, also known as uveal or choroidal melanoma. This designation enables Aura to have more frequent interactions with the FDA throughout AU-011’s drug development process, as well as priority review of the NDA. 

 

InVivo Therapeutics Holdings announced that it has received approval from the Toronto Western Hospital’s Research Ethics Board to enroll patients as part of its cervical spinal cord injury study.

 

CBT Pharmaceuticals announced that the first patient has been enrolled in the genolimzumab (CBT-501) Phase I study, the company’s first clinical trial. The trial is designed to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of genolimzumab in patients with advanced solid tumors, recurrent or refractory to standard of care therapies. The trial also aims to determine any dose limiting toxicities or biologically relevant dose and establish a recommended Phase II dose for future clinical studies. Once the dose and schedule is established, two or more select tumors will be evaluated for preliminary efficacy, and secondarily, progression free survival.

 

Cellectar Biosciences announced the initiation of a Phase II clinical study of its lead phospholipid drug conjugate (PDC) CLR 131 in patients with multiple myeloma and other hematologic malignancies.

 

Sun BioPharma, provided top-line, interim data from the Phase I dose-escalation phase of its clinical study and financial results for the year ended December 31, 2016.

 

Anavex Life Sciences announced the filing of an order of dismissal of the appeal in the Cortina v. Anavex Life Sciences Corp. et. al. lawsuit, without payment of any fees or costs by the Company.  The withdrawal represents a full and final conclusion of the lawsuit.

 

Exelixis announced that it has repaid all amounts outstanding under its term loan with Silicon Valley Bank initiated in 2010 and which was due for repayment on May 31, 2017. The $80.1 million payment included $80.0 million in principal and approximately $60,000 in interest outstanding. Exelixis also plans to eliminate another source of indebtedness later this year by retiring the Deerfield Notes, a series of Convertible Secured Notes issued to entities associated with Deerfield Management Company due July 1, 2018.

 

Tonix Pharmaceuticals Holding announced the pricing of an underwritten public offering of 1,800,000 shares of its common stock at a public offering price of $4.45 per share. The gross proceeds to Tonix from this offering are expected to be $8,010,000. Tonix intends to use the net proceeds from this offering to support the continued development of TNX-102 SL for the treatment of PTSD, including the HONOR study in military-related PTSD, to further develop other pipeline programs, for working capital and other general corporate purposes, and possibly acquisitions of other companies, products or technologies, though no such acquisitions are currently contemplated. Tonix has granted the underwriters a 45-day (or as otherwise specified in the underwriting agreement) option to purchase up to 270,000 additional shares of common stock at the public offering price, less the underwriting discounts and commissions. The offering is expected to close on or about April 4, 2017. Aegis Capitalis acting as the sole book-running manager for the offering.

 

Bellicum Pharmaceuticals announced the closing, on March 29, 2017, of its previously announced underwritten public offering of 5,750,000 shares of its common stock, including 750,000 shares sold pursuant to the underwriters’ full exercise of their option to purchase additional shares, at a public offering price of $12.00 per share. The aggregate offering size was approximately $69.0 million, before deducting the underwriting discounts and commissions and other offering expenses. Citigroup and Jefferies acted as lead book-running managers for the offering. Guggenheim SecuritiesLadenburg ThalmannRaymond James and SunTrust Robinson Humphrey acted as co-managers.

 

Cara Therapeutics announced that it has commenced an underwritten public offering of $80 million of shares of its common stock. In addition, Cara Therapeutics expects to grant the underwriters a 30-day option to purchase up to $12 million of additional shares of its common stock on the same terms and conditions. The proceeds of the offering are expected to be used to fund the company’s clinical and research development activities, including the completion of the Phase III program for I.V. CR845 in uremic pruritus, two Phase III trials of I.V. CR845 in acute pain and a Phase IIb trial of oral CR845 in osteoarthritis pain, as well as for working capital and general corporate purposes. Piper Jaffray & Co. and Stifel are acting as joint book-running managers for the proposed offering.

 

Blueprint Medicines announced the pricing of an underwritten public offering of 5,000,000 shares of its common stock at a public offering price of $40.00 per share, before underwriting discounts and commissions. In addition, Blueprint Medicines has granted the underwriters a 30-day option to purchase up to an additional 750,000 shares of its common stock at the public offering price, less underwriting discounts and commissions. Goldman SachsMorgan Stanley and Cowen and Company are acting as joint book-running managers for the offering. Raymond James is acting as co-manager for the offering.

 

Inovio Pharmaceuticals announced that its academic and industry collaborators received a multi-year $6.95 million grant from the NIH’s National Institute of Allergy and Infectious Diseases to develop a single or combination therapy using Inovio’s PENNVAX-GP with the goal of attaining long-term HIV remission in the absence of antiviral drugs.

 

Cellular Biomedicine Group announced the completion of its newly expanded 30,000 square foot facility in Huishan High Tech Park in Wuxi, China. 20,000 square feet of the Wuxi GMP facility will be dedicated to advanced stem cell culturing, centralized plasmid and viral vector production, cell banking and development of reagents.  By the end of 2017, the Company anticipates that the combination of this Wuxi site, the new Zhangjiang Shanghai and the Beijing GMP facilities will be capable of supporting simultaneous clinical trials for five different CAR-T and stem cell products, or capacity to treat up to 10,000 cancer and 10,000 Knee Osteoarthritis (KOA) patients per year.

 

Promentis Pharmaceuticals announced it has completed a third round of equity investment totaling $26,000,000. The Series C investment is led by OrbiMedF-PrimeCapital Partners (formerly Fidelity Biosciences) and Aisling Capital. Existing investors, including Black Pearl GmbH, the Golden Angel Network and individual investors, also participated in the Series C financing.

 

ACADIA Pharmaceuticals announced Michael J. Yang has joined ACADIA as Executive Vice President and Chief Commercial Officer. Mr. Yang succeeds Terrence O. Moore, who has served as ACADIA’s Chief Commercial Officer since 2013, and is now retiring.

 

Sarepta Therapeutics announced the addition of Kenneth Fischbeck, M.D., and Matthew Wood, M.D., Ph.D., to the Company’s Strategic and Scientific Advisory Board. Dr. Fischbeck and Dr. Wood join Dr. Beverly Davidson and Dr. Louis Kunkel, who were appointed to the board at the time of its formation in 2015.

 

 

ANALYST RECOMMENDATIONS

 

Cantor analyst Chiara Russo increased her price target of Paratek Pharmaceuticals to $32 from $28, citing a renewed confidence that PRTK’s omadacycline is correctly dosed for success in the IV/Oral Phase 3 in ABSSSI, that the heritage of tetracyclines should not present any safety concerns and that PK/PD and in vitro data all point to a positive read-out for pneumonia.

 

UBS analyst Martin Auster initiated coverage of the following companies: Alexion with a “buy” rating and $138 price target; Avexis with a “buy” rating and $95 price target; Spark Therapeutics with a “buy” rating and $70 price target; Alnylam with a “neutral” rating and $55 price target; BioMarin with a “neutral” rating and $92 price target; United Therapeutics with a “sell” rating and $112 price target; the analyst was also transitioned coverage of Esperion which he upgraded to “buy” from “neutral” and increased his price target to $52 from $23; Selecta the analyst maintained a “buy” rating and decreased his price target to $26 from $29.

 

SunTrust analyst Peter Lawson upgraded NewLink Genetics to “buy” from “hold” and increased his price target to $30 from $12, citing favorable risk/reward ahead of upcoming data.

 

HC Wainwright analyst Ed Arce upgraded Galectin Therapeutics to “buy” from “neutral” and increased his price target to $3.50 from $0.60, citing a more positive view of the therapeutic potential of the mechanism of galectin-3 inhibition.

 

Cowen analyst Tyler Van Buren increased his price target of Innoviva to $16 from $15, citing increased Breo estimates after Mylan announced that it received a CRL for its generic Advair program, which was first in line.