BioShares Biotechnology Clinical Trials (BBC): $20.60, +$0.05, +11.9% YTD

BioShares Biotechnology Products (BBP): $36.94, +$0.22, +12.8% YTD

 

 

MARKET COMMENTARY

 

U.S. stock index futures were little changed on the first trading day of the week, as investors await U.S. President Donald Trump’s speech on Tuesday for clues on his plans for the economy. Durable goods, building permits and pending home sales data occupy the day’s economic calendar. European shares rose helped by some well-received earning updates, while Asian equities fell. The dollar edged up against the yen but struggled to gain traction elsewhere. Oil prices rose as speculators raised U.S. crude net long positions to record high. Gold held steady near 3-1/2 month highs.

 

 

MARKET HIGHLIGHTS

 

La Jolla Pharmaceutical announced positive top-line results from the ATHOS-3 (Angiotensin II for the Treatment of High-Output Shock) Phase III study of LJPC-501 (angiotensin II) in patients with catecholamine resistant hypotension (CRH). The analysis of the primary efficacy endpoint, defined as the percentage of patients achieving a pre-specified target blood pressure response, was highly statistically significant: 23% of the 158 placebo-treated patients had a blood pressure response compared to 70% of the 163 LJPC-501-treated patients (p<0.00001). In addition, a trend toward longer survival was observed: 22% reduction in mortality risk through day 28 [hazard ratio=0.78 (0.57-1.07), p=0.12] for LJPC-501-treated patients. Throughout the study, safety outcomes were followed by an independent Data Safety Monitoring Board (DSMB). The DSMB recommended that the study continue as originally planned. In this critically ill patient population: 92% of placebo-treated patients compared to 87% of LJPC-501-treated patients experienced at least one adverse event, and 22% of placebo-treated patients compared to 14% of LJPC-501-treated patients discontinued treatment due to an adverse event. In collaboration with the investigators, La Jolla plans to present and publish detailed results from the ATHOS-3 study later this year.

 

OncoSec Medical received Fast Track designation from the FDA for its ImmunoPulse IL-12, a potentially first-in-class, Intratumoral anti-cancer gene therapy that expresses interleukin-12 (IL-12) for the treatment of metastatic melanoma, following progression on pembrolizumab or nivolumab.

 

Rexahn Pharmaceuticals announced financial results for the year ended December 31, 2016.  Rexahn’s net loss was $9.3 million, or $0.04 per share, for the year ended December 31, 2016, compared to a net loss of $14.4 million, or $0.08 per share, for the year ended December 31, 2015.  Rexahn’s cash and investments totaled approximately $20.3 million as of December 31, 2016, compared to approximately $23.4 million as of December 31, 2015.

 

Aimmune Therapeutics announced that new clinical data on AR101 will be presented during poster sessions on Monday, March 6, at the 2017 American Academy of Allergy, Asthma & Immunology (AAAAI) Annual Meeting taking place March 3–6, 2017, in Atlanta, Georgia. The presentations will report data from the Phase 2 ARC001 study (Abstract 803) and from the screening population of the PALISADE Phase 3 study (Abstracts L20 and 807).

 

Advaxis and SELLAS Life Sciences Group announced that Advaxis has granted SELLAS a license to develop a novel cancer immunotherapy agent using Advaxis’ proprietary Lm-based antigen delivery technology with SELLAS’ patented WT1 targeted heteroclitic peptide antigen mixture (galinpepimut-S).

 

Foamix Pharmaceuticals will present a corporate overview at the Cowen & Company 37th Annual Healthcare Conference on Monday, March 6th @ 3:20pmET.

 

AMAG Pharmaceuticals together with Palatin Technologies announced that additional results from the Phase 3 RECONNECT Studies (301, 302) of Rekynda™ (bremelanotide) were presented at the International Society for the Study of Women’s Sexual Health (ISSWSH) Annual Meeting in Atlanta, Ga. Rekynda is an investigational product being developed as a potential on-demand treatment for pre-menopausal women diagnosed with hypoactive sexual desire disorder (HSDD).

 

Evogene announced today its financial results for the fourth quarter and year ended December 31, 2016.  Net loss for the fourth quarter of 2016 was $6.6 million compared with a net loss of $5.5 million for the same period in 2015. Net loss for the year ended December 31, 2016, was $19.6 million compared with a net loss of $17.2 million for the same period in 2015.  As of December 31, 2016, Evogene had $88.2 million in cash, short-term bank deposits and marketable securities, representing a net cash usage of $12.5 million for the year. Assuming regular course of business and no new revenue sources, such as additional collaborations, the Company estimates that its net cash usage for full year 2017 will be in the range of $16 to $18 million.

 

BrainStorm Cell Therapeutics announced today the appointments of June S. Almenoff, M.D., Ph.D., FACP, and Arturo O. Araya, M.A., M.B.A. to its Board of Directors.

 

Addex Therapeutics announced today the publication of a study demonstrating the anti-epileptic effects of ADX71149, a metabotropic glutamate receptor subtype 2 (mGlu2) positive allosteric modulator (PAM), given alone or in combination with the globally marketed antiseizure drug levetiracetam (LEV) in a preclinical model of epilepsy. These data, published in Epilepsia, the peer-reviewed journal of the international league against epilepsy (http://onlinelibrary.wiley.com/doi/10.1111/epi.13659/abstract), support the potential utility of ADX71149 to address treatment-resistant epilepsy.

 

Achaogen announced the addition of Ms. Janet Dorling as Chief Commercial Officer effective today. Ms. Dorling will report to Blake Wise, Achaogen’s President and Chief Operating Officer.

 

Pieris Pharmaceuticals announced that it has granted ASKA Pharmaceutical an exclusive option to license development and commercial rights to Pieris’ anemia drug, PRS-080, in Japan and certain other Asian markets following completion of a multi-dose Phase 2a study to be conducted by Pieris in dialysis-dependent anemia patients.

 

BioCryst Pharmaceuticals announced results from an interim analysis of its Phase II APeX-1 trial in hereditary angioedema (HAE). APeX-1 is a dose ranging trial designed to evaluate the efficacy, safety, tolerability, pharmacokinetics and pharmacodynamics of orally administered once daily (QD) BCX7353 for 28 days, as a preventative treatment to reduce the frequency of attacks in HAE patients. In other company news, BioCryst Pharmaceuticals announced financial results for the fourth quarter and full year ended December 31, 2016.

 

Sangamo Therapeutics announced that the FDA has granted rare pediatric disease designation for SB-318, the Company’s in vivo genome editing product candidate for the treatment of Mucopolysaccharidosis Type I (MPS I), a lysosomal storage disorder.

 

AcelRx Pharmaceuticals announced the FDA has accepted the company’s NDA under section 505(b)(2) for DSUVIA (formerly ARX-04) (sufentanil sublingual tablet, 30 mcg) for the treatment of patients with moderate-to-severe acute pain in a medically supervised setting. The acceptance of the NDA indicates that the FDA has deemed the application sufficiently complete to allow a substantive review. The FDA has also set a PDUFA date of October 12, 2017. The PDUFA date is the target date set by the FDA to communicate its decision on the NDA.

 

Cellectar Biosciences provided an update of its Phase I clinical study of CLR 131 in patients with relapsed or refractory multiple myeloma following the previously announced successful completion of its third cohort and initiation of Cohort 4 at a 31.25 mCi/m(2) dose.

 

Inovio Pharmaceuticals announced that its SynCon WT1 cancer immunotherapy was capable of breaking immune tolerance and inducing neo-antigen-like T cell responses to cause tumor regression in pre-clinical studies. Breaking tolerance has been a major challenge for developing a potent cancer therapy; researchers have tried many other methods and been unsuccessful for decades. Notably, the WT1 antigen is over-expressed in multiple cancer types but not found in most normal tissue, giving it potential to be used as part of a universal cancer vaccine against multiple tumor types.

 

Horizon Pharma reported 4Q16 non-GAAP EPS of $0.64, which compares to $0.64 for the same period a year ago. The company beat the non-GAAP EPS mean estimate of $0.51. Total revenue for 4Q16 was $310.3 million, which compares to $244.5 million for the same period a year ago. This compares to a mean estimate of $309.6 million.

 

Mesoblast provided a strategic update, financial results for the half-year period ended December 31, 2016 and operational highlights. At December 31, 2016, the Group had cash reserves of $55.6 million after adjusting for the committed capital of $21.7 million from the equity purchase agreement with Mallinckrodt Pharmaceuticals announced on December 23, 2016, the proceeds of which were subsequently received on January 6, 2017. During the half-year FY2017, the Company has executed its planned operational streamlining and reprioritization of projects to successfully absorb the incremental costs of the MPC-150-IM program in advanced chronic heart failure (CHF). In line with this objective, operating cash outflows for the halfyear FY2017 excluding MPC-150-IM for CHF were reduced by 22% ($11.5 million) compared with the half-year period ended December 31, 2015 (half–year FY2016). After absorbing the incremental R&D costs associated with the CHF program, total operating cash outflows were $47.3 million, still a reduction of 9% ($4.6 million) compared with the half-year FY2016.

 

Enanta Pharmaceuticals announced that the CHMP of the EMA has granted a positive opinion for an eight-week treatment regimen of AbbVie‘s VIEKIRAX (ombitasvir/paritaprevir/ritonavir tablets) + EXVIERA(R) (dasabuvir tablets) as an option for previously untreated adult patients with genotype 1b (GT1b) chronic HCV infection and minimal to moderate fibrosis.

 

Antares Pharma announced that the NDA for QuickShot Testosterone (QST), a drug-device combination product for the delivery of testosterone enanthate using a subcutaneous auto injector, has been accepted for standard review by the FDA. QST was developed to treat adult men with low testosterone associated with a diagnosed condition known as hypogonadism.

 

Merck announced the first Phase III study results for V212, the company’s investigational inactivated varicella zoster virus vaccine (VZV) for the prevention of herpes zoster or HZ, also known as shingles, in immunocompromised patients. This was a double-blind, randomized, placebo-controlled, multi-center trial to study safety, tolerability, efficacy and immunogenicity of inactivated VZV Vaccine in Recipients of Autologous Hematopoietic Stem Cell Transplants (auto-HSCT). In the trial, V212 met its primary endpoint and reduced the incidence of confirmed HZ cases by an estimated 64 percent (95% CI, 0.48, 0.75) in recipients of auto-HSCT. These results were presented, as an oral presentation, at the combined annual meetings of the Center for International Blood & Marrow Transplant Research (CIBMTR) and the American Society for Blood and Marrow Transplantation (ASBMT) during a “Best Abstracts” session in Orlando, Florida.

 

Reuters reported that Pfizer said it received subpoenas from the U.S. Attorney’s Office for the District of Massachusetts related to the drugmaker’s support for organizations that provide financial help to Medicare patients. In a regulatory filing, Pfizer said it received subpoenas in December 2015 and July 2016 related to groups that help cover patient co-payments for prescription drugs. Pfizer said it has been "providing information to the government in response to these subpoenas."

 

argenx announced the extension of its strategic partnership with Shire to advance the discovery and development of novel human therapeutic antibodies for diverse rare and unmet diseases for a further year until May 30, 2018.

 

CTI BioPharma announced the appointment of Adam Craig, M.D., Ph.D., as President and Chief Executive Officer (CEO) and member of the Board of Directors effective March 20, 2017. Dr. Craig succeeds Richard Love, interim President and CEO who will continue to serve on the company’s Board of Directors.

 

Valeant Pharmaceuticals’ wholly owned subsidiary, Bausch + Lomb, and Nicox S.A. announced the resubmission of a NDA to the FDA seeking approval for latanoprostene bunod ophthalmic solution, 0.024%. Latanoprostene bunod is an intraocular pressure (IOP) lowering single-agent eye drop dosed once daily, for patients with open angle glaucoma (OAG) or ocular hypertension (OHT). The data submitted in the NDA support latanoprostene bunod as the first nitric-oxide donating prostaglandin F2α analog for ophthalmic use. Latanoprostene bunod was licensed by Nicox to Bausch + Lomb.

 

TESARO announced that the EMA CHMP has rendered a positive opinion for the Company’s MAA for VARUBY (oral rolapitant tablets) for the prevention of delayed nausea and vomiting associated with highly and moderately emetogenic cancer chemotherapy in adults.  

 

AbbVie announced that the EC for Medicinal Products for Human Use (CHMP) of the EMA has granted a positive opinion for a shorter, eight-week treatment of VIEKIRAX (ombitasvir/paritaprevir/ritonavir tablets) + EXVIERA (dasabuvir tablets) as an option for previously untreated adult patients with genotype 1b (GT1b) chronic hepatitis C virus (HCV) and minimal to moderate fibrosis*.  VIEKIRAX + EXVIERA is currently approved in the EU for use as a 12-week treatment for GT1b chronic HCV-infected patients without cirrhosis or with compensated cirrhosis.

 

Bristol-Myers Squibb announced that Columbia University Medical Center and Peter MacCallum Cancer Centre (Peter Mac) have joined the International Immuno-Oncology Network (II-ON), a global peer-to-peer collaboration between Bristol-Myers Squibb and academia that aims to advance Immuno-Oncology (I-O) science and translational medicine to improve patient outcomes. Launched in 2012 by Bristol-Myers Squibb, the II-ON was one of the first networks to bring academia and industry together to further the scientific understanding of I-O, and has expanded from 10 to 15 sites including more than 250 investigators working on over 150 projects across 20 tumor types. The II-ON has generated cutting-edge I-O data that have informed the development of new I-O agents, yielded publications and produced some of the earliest findings on a variety of biomarkers and target identification and validation.

 

Exelixis and Bristol-Myers Squibb announced the companies have entered into a clinical development collaboration to evaluate CABOMETYX (cabozantinib), Exelixis’ small molecule inhibitor of receptor tyrosine kinases, with Opdivo (nivolumab), Bristol-Myers Squibb’s PD-1 immune checkpoint inhibitor, either alone or in combination with Yervoy (ipilimumab). The clinical development program, which will be co-funded by the companies, is expected to include a Phase III pivotal trial in first-line renal cell carcinoma, with additional trials planned in bladder cancer, hepatocellular carcinoma (HCC), and potentially other tumor types.

 

Valeant Pharmaceuticals International‘s wholly owned subsidiary, Salix, announced that it has rapidly scaled up its sales force by nearly 40 percent, effective immediately.

 

Recro Pharma announced completion of enrollment for its double-blind Phase III safety study evaluating the safety and tolerability of intravenous (IV) meloxicam (N1539) following major surgery.

 

Exelixis announced a new collaboration with Roche on a Phase Ib dose escalation study that will evaluate the safety and tolerability of cabozantinib, Exelixis’ tyrosine kinase inhibitor (TKI), in combination with atezolizumab, Roche’s anti-PD-L1 immunotherapy, in patients with locally advanced or metastatic solid tumors. Enrollment is scheduled to begin mid-year 2017; Exelixis will be the sponsor of the trial, and Roche will provide atezolizumab.

 

Johnson & Johnson announced it has completed the acquisition of Abbott Medical Optics, a wholly-owned subsidiary of Abbott. The all-cash $4.325 billion acquisition was originally announced Sept. 16, 2016, and includes ophthalmic products in three areas of patient care: cataract surgery, laser refractive surgery and consumer eye health.

 

Helix BioPharma announced that it has completed a license agreement with the National Research Council of Canada ("NRC") for the worldwide right to anti-CEACAM6 antibody 2A3 for oncology applications.

 

Amicus Therapeutics has commenced the commercial launch of the precision medicine Galafold in the UK following the final publication of reimbursement guidelines by the NICE Highly Specialized Technologies Evaluation Committee (EC). Galafold is reimbursed within the NHS in England as a first line therapy for long-term treatment of adults and adolescents aged 16 years and older with a confirmed diagnosis of Fabry disease (alpha-galactosidase A deficiency) and who have an amenable mutation.

 

Fate Therapeutics announced an expansion of its research collaboration with the Regents of the University of Minnesota (UMN) to initiate the clinical translation of a first-of-kind product candidate, an off-the-shelf targeted natural killer (NK) cell cancer immunotherapy derived from an engineered induced pluripotent stem cell (iPSC) line.

 

Aclaris Therapeutics announced it has submitted a NDA to the FDA for A-101 40% topical solution (A-101) as a treatment for seborrheic keratosis (SK). A-101, an investigational drug, is being developed by Aclaris as a non-invasive, in-office topical treatment for SK.

 

Clearside Biomedical announced that it has initiated a strategic realignment of its research and development resources from its pre-clinical development program for axitinib for the treatment of wet age-related macular degeneration ("wet AMD") toward its ongoing clinical development program for the treatment of diabetic macular edema ("DME") assessing Zuprata, its proprietary suspension formulation of the corticosteroid triamcinolone acetonide.

 

Lexaria Bioscience announced it has received a Notice of Acceptance from the Australian Patent Office that Lexaria’s Australian patent application 2015274698 has been accepted with a patent issuance date expected in June, 2017. This establishes Lexaria’s first successful acceptance for patent issuance outside of the USA, in what the Company expects will be a series of successful international patent awards.

 

Sunovion Pharmaceuticals announced that its Phase III clinical study evaluating Latuda (lurasidone HCl) in children and adolescents aged 10 to 17 years with depression associated with bipolar I disorder (bipolar depression) met its primary endpoint. LATUDA is currently indicated in the U.S. for the treatment of major depressive episodes associated with bipolar I disorder (bipolar depression) as monotherapy and as adjunctive therapy with lithium or valproate in adults and for the treatment of schizophrenia in adults and adolescents aged 13 to 17 years.

 

Syros Pharmaceuticals announced the discovery of a novel genomics based approach to stratify acute myeloid leukemia (AML) patients based on super-enhancer profiles. In their research, Syros scientists demonstrated a strong association between distinct super-enhancer profiles of six AML patient clusters and patients’ overall survival outcomes and response to certain therapies in development. These data were presented at the Cold Spring Harbor Systems Biology: Global Regulation of Gene Expression Conference.

 

RegeneRx Biopharmaceuticals announced that its licensee for RGN-137, GtreeBNT, received a positive response from the FDA for its Phase III clinical trial design for RGN-137 to treat epidermolysis bullosa ("EB"). RGN-137 is a dermal wound healing gel that incorporates Thymosin beta 4 ("Tβ4") as the active pharmaceutical ingredient. GtreeBNT is planning to enter into the Phase III trial in the U.S. during the third quarter of 2017. 

 

Matinas BioPharma Holdings announced that its common stock has been approved for listing on the NYSE MKT. The Company’s common stock is expected to begin trading on NYSE MKT under the symbol "MTNB" beginning on March 2, 2017. The Company’s common stock will continue to trade on the OTCQB until the close of the market on March 1, 2017.

 

Regeneron Pharmaceuticals and Sanofi announced that detailed results from the Phase III CHRONOS study will be presented as a late-breaking abstract at this year’s Annual Meeting of the American Academy of Dermatology (AAD). The CHRONOS study evaluated the use of investigational DUPIXENT for one year with topical corticosteroids versus topical corticosteroids alone for adults living with uncontrolled moderate-to-severe atopic dermatitis (AD). Additionally, interim safety and efficacy data will be presented from a Phase III long-term open-label study of DUPIXENT monotherapy for uncontrolled moderate-to-severe AD at the Annual Meeting of the American Academy of Asthma, Allergy, and Immunology (AAAAI).

 

Ampio Pharmaceuticals announced that the Office of Tissue and Advanced Therapies (OTAT), a branch of the Center for Biologics Evaluation and Research (CBER) of the FDA, has recently provided additional guidance for the path to approval of Ampion as a treatment for pain due to severe Osteoarthritis of the knee (OAK).

 

Ligand Pharmaceuticals announced the completion of enrollment in the Company’s Phase II clinical trial with its novel, small-molecule glucagon receptor antagonist LGD-6972 for the treatment of Type II diabetes mellitus (T2DM). This randomized, double-blind, placebo-controlled study is evaluating the safety and efficacy of LGD-6972 as an adjunct to diet and exercise in subjects with T2DM whose blood glucose levels are inadequately controlled with metformin. The Company expects to report topline results in September 2017.

 

 

ANALYST RECOMMENDATIONS

 

Needham analyst Serge Belanger initiated coverage of Heron Therapeutics with a “buy” rating and $28 price target, citing HRTX made the transition to a commercial co. in 4Q16 with the launch of Sustol for CINV.

 

Morgan Stanley analyst Matthew Harrison increased his price target of Ultragenyx to $86 from $80, citing upcoming data from the trihep seizure study in the near-term.

 

Evercore analyst Ross Muken upgraded Illumina to “buy” from “hold,” and increased his price target to $190 from $173, citing sequencing is about to enter a new era of utility and it is expected of NovaSeq to eventually usher it in.

 

Baird analyst Michael Ulz downgraded Tesaro to “neutral” from “outperform” and increased his price target to $182 from $127, citing valuation.

 

HC Wainwright analyst Ed Arce increased his price target of The Medicines Company to $60 from $57, citing increased POS for both inclisiran and Carbavance.

 

Ladenburg Thalman analyst Christopher James decreased his price target of Achillion to $9 from $10, citing model updates.

 

Avondale analyst Greg Bolan increased his price target of Allscripts to $15.50 from $15, citing model adjustments following earnings.

 

Roth analyst Michael Higgins upgraded Cempra to “buy” from “neutral,” citing Taksta’s potential for FDA approval in ABSSSI is likely.

 

Roth analyst Scott Henry resumed coverage of Ligand Pharmaceuticals with a “buy” rating and $118 price target, citing the combination of a strong base business, near-term new revenue generators, and a long-term pipeline.

 

Ladenburg Thalmann analyst Christopher James decreased his price target of Achillion to $9 from $10, citing model updates.