BioShares Biotechnology Clinical Trials (BBC): $23.05, -$0.14, +25.2% YTD

BioShares Biotechnology Products (BBP): $37.17, -$0.58, +13.5% YTD

 

 

MARKET COMMENTARY

 

U.S. stock index futures fell as investors appeared concerned over President Donald Trump’s ability to deliver on his policy plans. Data on international trade and factory orders is due later in the day. Weakness in the autos sector weighed on the European shares even as gains in oil-related stocks and miners provided some support. Asian stock markets recorded a mixed trading day. The dollar edged up against a basket of major currencies but lost ground against the safe-haven Japanese yen. Oil prices recovered from recent losses to trade higher and gold rose.

 

 

MARKET HIGHLIGHTS

 

Onconova Therapeutics announced promising pre-clinical data for first-in-class dual inhibitor of CDK4/6 + ARK5, as well as a Type 1 novel inhibitor of FLT3 and Src pathways as a novel strategy for Acute Myeloid Leukemia (AML) therapy at the 2017 AACR Annual Meeting. The Company presented its findings in two poster presentations on April 3, 2017.

 

BioTime Co-Chief Executive Officer Michael D. West, Ph.D., is delivering a keynote presentation at the GTC Bio Stem Cell Summit, on April 6 at the Hyatt Regency in Boston, Massachusetts.

 

BIOPHYTIS announced that it has gathered funding to conduct its phase 2b SARA-OBS/SARA-INT clinical study aimed at testing its leading product, Sarconeos, for the treatment of sarcopenia. First, a capital increase subscribed by several private investors among which Bracknor Fund, and the management, of an amount of €3.7 million was completed by issuing 1,310,431 new shares at a price of €2.85 per share. Secondly, a line of funding that could reach €15 million was set up with Bracknor Fund in the form of 1,500 note warrants for Bonds Redeemable in Cash or New or Existing Shares (ORNANE), at a par value of €10,000 each, combined with Share Subscription Warrants (BSAs), together referred to as ORNANEBSA.

 

Sigmoid Pharma Limited has entered into an agreement with Dr. Falk Pharma GmbH to advance Sigmoid’s SmPill® cyclosporine franchise for ulcerative colitis, graft versus host disease and other gastrointestinal diseases.  The terms of the agreement include an upfront equity investment in Sigmoid, development funding, milestone payments as well as tiered double-digit royalties for the future development and commercialization of CyCol®, AlloCol® and related products in Europe. Sigmoid retains the rights to these programs outside of Europe and Canada. This collaboration with Dr. Falk in Europe will serve as an important catalyst to Sigmoid advancing its pipeline to patients in territories including the United States and Japan.

 

Paratek Pharmaceuticals announced positive top-line results from a global, pivotal Phase 3 clinical study comparing its once-daily oral and IV, broad spectrum investigational antibiotic, omadacycline, to moxifloxacin in the treatment of patients with community-acquired bacterial pneumonia (CABP).  This study represents the second positive Phase 3 registration study of omadacycline, which will be used to support marketing applications to the United States Food and Drug Administration (FDA) and the European Medicines Agency (EMA).  The global, pivotal Phase 3 clinical study known as OPTIC (Omadacycline for Pneumonia Treatment in the Community), compared the safety and efficacy of once-daily, IV-to-oral omadacycline to IV-to-oral moxifloxacin for treating adults with CABP.  In the study, 774 patients were randomized.  Omadacycline met the FDA-specified primary endpoint of statistical non-inferiority (NI) in the intent-to-treat (ITT) population (10% NI margin, 95% confidence interval) compared to moxifloxacin at the early clinical response (ECR) 72-120 hours after initiation of therapy. The ECR rates for the omadacycline and moxifloxacin treatment arms were 81.1 % and 82.7%, respectively.

 

VBL Therapeutics announced today the presentation of new data on MOSPD2, a novel potential target in oncology. VBL’s study, entitled "MOSPD2, a Newly Characterized Protein, Promotes Breast Cancer Metastasis" by Mendel et al., will be presented today at the American Association of Cancer research (AACR) conference in Washington, DC. The study observed from clinical biopsies that MOSPD2 is prevalent in invasive human breast cancer tissue and that levels of MOSPD2 correlate to breast cancer invasiveness. It was further observed that a knockdown of MOSPD2 in a human breast cancer cell line using CRISPR technology led to blockade of EGF signaling and significant reduction of breast cancer cell migration in vitro and metastasis in a mouse model.

 

Santhera Pharmaceuticals announced that a large majority of shareholders approved all proposals by the Board of Directors at the Annual Shareholders` Meeting (ASM) today. Participating shareholders represented a total of 3,097,267 shares or 49.3% of the share capital with voting power.

 

Rockwell Medical filed a preliminary proxy statement with the Securities and Exchange Commission for the Company’s 2017 Annual Meeting of Shareholders, highlighting the nomination of David Domzalski to the Company’s Board of Directors, disclosing the appointment of a Lead Independent Director to the Company’s Board of Directors and describing corporate governance enhancements.

 

Galena Biopharma announced a poster was presented on the Company’s NeuVax (nelipepimut-S) investigator-sponsored Phase II clinical trial (IST) in high-risk, HER2 3+ patients at the American Association for Cancer Research (AACR) Annual Meeting 2017 in Washington, DC.  The Phase II trial is a multi-center, prospective, randomized, single-blinded, placebo-controlled trial combining NeuVax and trastuzumab in the adjuvant setting to prevent recurrence in HER2-positive (HER2 3+) breast cancer patients.

 

ContraVir Pharmaceuticals provided new insights into the mechanism of action (MOA) of its hepatitis B virus (HBV)-optimized cyclophilin inhibitor CRV431. New studies show that CRV431 potently blocks the interaction between hepatitis B surface antigen (HBsAg), a key HBV protein, and cyclophilin A, an important cellular protein. This new information could explain previous findings that CRV431 reduces HBsAg in many experimental systems. High levels of HBsAg in HBV-infected patients is a predictor for progression of disease, including liver fibrosis, cirrhosis, and cancer.

 

TherapeuticsMD announced that data on TX-001HR, an investigational bio-identical hormone therapy combination of 17ß-estradiol and progesterone in a single, oral softgel, for the treatment of moderate to severe vasomotor symptoms (VMS) due to menopause, was presented at ENDO 2017, the annual meeting of the Endocrine Society in Orlando, Florida. The positive results of the pivotal Phase III Replenish Trial data were featured in an oral and a poster presentation.

 

RedHill Biopharma announced that the FDA has granted YELIVA (ABC294640) Orphan Drug designation for the treatment of cholangiocarcinoma.

 

Teva announced that the FDA has approved AUSTEDO (deutetrabenazine) tablets for the treatment of chorea associated with Huntington’s disease (HD). Previously referred to by the developmental name SD-809, AUSTEDO is the first deuterated product approved by the FDA and only the second product approved in HD. The product was previously granted Orphan Drug Designation by the FDA.

 

uniQure N.V. announced the online publication in Gene Therapy of data demonstrating widespread transduction in the CNS following direct injection of uniQure’s AAV5 vector in a large animal model. uniQure has exclusive, worldwide rights to AAV5 for use in therapeutic products delivered to the brain or liver.

 

Boston Children’s Hospital, the pediatric teaching hospital of Harvard University, and Mesoblast announced that the FDA has cleared the commencement of a 24-patient trial combining Mesoblast’s proprietary allogeneic mesenchymal precursor cells (MPCs) with corrective heart surgery in children under the age of 5 with hypoplastic left heart syndrome (HLHS). The trial is sponsored and funded by the Boston Children’s Hospital with support from Bulens and Capozzi Foundation and the Ethan Lindberg Foundation.

 

Aclaris Therapeutics announced that Columbia University has received a Notice of Allowance from the USPTO for two patent applications covering methods related to the use and administration of baricitinib (LY3009104) and decernotinib (VX-509), respectively, for the treatment of hair loss disorders and for inducing hair growth. These newly allowed patent applications are owned by The Trustees of Columbia University in the City of New York and exclusively licensed to Aclaris Therapeutics, Inc. These patent applications are the latest U.S. applications to be allowed in connection with Aclaris’ janus kinase inhibitor (JAK) drug development program for hair loss disorders. A separate Columbia patent covering the use of ruxolitinib for the treatment of hair loss disorders and for inducing hair growth was issued in the U.S. in December 2015.

 

Immunomedics announced that its lead antibody-drug conjugate (ADC), sacituzumab govitecan (IMMU-132), with a proprietary SN-38-delivery platform, has the potential to provide clinical benefit both to chemosensitive solid tumors with low Trop-2 expression, as well as to chemoresistant tumors with high Trop-2 expression.

 

Benitec Biopharma announced that the initial pre-clinical efficacy results of the OPMD program have been published in Nature Communications, an open access scientific journal published by the Nature Publishing Group.  OPMD, a rare progressive muscle-wasting disease caused by mutation in the poly(A)-binding protein nuclear 1 (PABPN1) gene, is characterised by eyelid drooping, swallowing difficulties, and proximal limb weakness.

 

Bellicum Pharmaceuticals announced two presentations of preclinical results on the Company’s proprietary dual-switch technology for use in CAR-T and TCR product candidates at the 2017 American Association for Cancer Research (AACR) Annual Meeting in Washington, D.C.

 

Amgen announced the submission of a sBLA to the FDA and an application for a variation to the marketing authorization to the EMA for XGEVA (denosumab). The submissions to regulatory authorities seek to expand the currently approved XGEVA indication for the prevention of skeletal-related events (SREs) in solid tumors to include patients with multiple myeloma. The applications include new data from the pivotal Phase III head-to-head ‘482 study, the largest international multiple myeloma trial ever conducted.

 

Nektar Therapeutics announced five preclinical data presentations for its immuno-oncology programs made at the American Association for Cancer Research (AACR) Annual Meeting 2017.  The presentations featured new preclinical data on NKTR-214, the Company’s immuno-stimulatory CD122-biased agonist, as well as on NKTR-255, the Company’s IL-15 therapeutic candidate.

 

NANOBIOTIX announced the expansion and acceleration of its clinical development activities. These include: Acceleration of the head and neck cancer program. Phase I/II data will be presented at ASCO in June; Expansion of Nanobiotix’s Immuno-Oncology program into patients focused on the objective of turning cold tumors into hot tumors. Nanobiotix will present the first clinical data from this program mid-year.

 

Galapagos announced three new Phase II Proof-of-Concept studies investigating filgotinib in Sjögren’s syndrome, ankylosing spondylitis, and psoriatic arthritis. The Sjögren’s study is being led by filgotinib collaboration partner Gilead Sciences; the ankylosing spondylitis and psoriatic arthritis studies by Galapagos.

 

Karyopharm Therapeutics announced that Justin Renz, Executive Vice President, Chief Financial Officer and Treasurer, has stepped down in order to pursue other opportunities, effective. Mr. Renz will continue to serve the Company in an advisory capacity in order to ensure a smooth transition.

 

NantKwest and Viracta Therapeutics announced the initial closing of a Series B Preferred financing round of up to $18.4 million with NantKwest as the lead investor. Joining NantKwest as a new investor in Viracta is Wicklow Capital. Viracta’s founding investors, Latterell Venture Partners and Forward Ventures also participated in the round.

 

AMAG Pharmaceuticals announced the closing of the licensing agreement withEndoceutics for the U.S. commercial rights to Intrarosa (prasterone). Intrarosa is the only FDA-approved, locally administered, non-estrogen steroid hormone for the treatment of moderate-to-severe dyspareunia (pain during intercourse), a common symptom of vulvar and vaginal atrophy (VVA), due to menopause. Unlike all other FDA-approved medications for the treatment of VVA symptoms, Intrarosa does not carry a boxed safety warning in its label. The agreement was announced on February 14, 2017, and its closing broadens AMAG’s presence in women’s health.

 

Syros Pharmaceuticals announced that new preclinical data on SY-1425, its first-in-class selective retinoic acid receptor alpha (RARα) agonist currently in a Phase II clinical trial in genomically defined subsets of patients with acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS), were presented at the American Association of Cancer Research (AACR) Annual Meeting in Washington, D.C.

 

Peregrine Pharmaceuticals announced the presentation of results of a new analysis of the Phase III SUNRISE trial of bavituximab in patients with previously treated locally advanced or metastatic non-squamous non-small cell lung cancer (NSCLC).  Data demonstrated that for patients in the study’s bavituximab plus docetaxel treatment arm who received subsequent immunotherapy, the median overall survival (mOS) was not reached, while mOS was 13.0 months for patients in the study’s placebo plus docetaxel arm who received subsequent immunotherapy [HR = 0.43; p=0.005].  These are the first clinical results reported supporting the hypothesis that bavituximab may modulate the tumor microenvironment to enhance the anti-tumor activity of immunotherapy agents. Data were presented by Peregrine scientists at the 2017 Annual Meeting of the American Association for Cancer Research (AACR), which is being held April 1-5, 2017 in Washington, D.C.

 

RepliCel Life Sciences reported statistically and clinically significant positive data from the interim analysis of its phase I study evaluating RCS-01 for the treatment of aging and sun-damaged skin.

 

Provectus Biopharmaceuticals announced the entry into definitive agreements related to the previously announced Definitive Financing Commitment Term Sheet (the "Definitive Financing") it entered into on March 19 with a group of the Company’s stockholders referred in the Definitive Financing as the "PRH Group."

 

AbbVie announced a sNDA was accepted for review by the FDA for ibrutinib (IMBRUVICA) in chronic graft-versus-host-disease (cGVHD) after failure of one or more lines of systemic therapy. cGVHD is a severe, potentially life-threatening consequence of stem cell or bone marrow transplant. If approved by the U.S. FDA, ibrutinib will be the first therapy specifically approved to treat this condition. IMBRUVICA is jointly developed and commercialized by Pharmacyclics, an AbbVie company, and Janssen Biotech.

 

Xenetic Biosciences announced that it has appointed James F. Parslow, MBA, CPA as Chief Financial Officer, effective April 3, 2017

 

Flexion Therapeutics announced the U.S. Patent and Trademark Office has issued two new patents covering the company’s lead investigational product candidate Zilretta™(FX006). The new patents further strengthen the existing patent estate surrounding Zilretta. The patents cover the injectable formulation comprised of controlled or sustained-release microparticles that contain triamcinolone acetonide in a poly lactic-co-glycolic acid co-polymer (PLGA) matrix.

 

Aytu BioScience announced that it presented two posters demonstrating the safety advantages of Natesto testosterone nasal gel, including the one-year hematologic safety and tolerability among men with seasonal allergies, in men with hypogonadism.

 

Medidata and Karyopharm Therapeutics announced an expanded partnership to bring novel machine-learning technology and targeted risk-based monitoring (RBM) techniques to clinical oncology trials.

 

Kite Pharma highlighted a publication in the latest issue of Nature Methods that describes pre-clinical findings demonstrating that a novel serum-free, 3-dimensional cell culture technology, known as the Artificial Thymic Organoid (ATO) cell culture system, recapitulates T-cell differentiation. The findings support the potential of ATO technology to generate off-the-shelf engineered T cells to treat cancer and other diseases. The research was led by Gay M. Crooks, M.D. at the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at the University of California, Los Angeles (UCLA). Kite holds an exclusive license to the ATO cell culture technology from UCLA.

 

Evoke Pharma announced that the Company recently completed a positive Type A meeting with the FDA to finalize the design of the pivotal comparative exposure pharmacokinetic (PK) trial and to reach agreement on additional aspects of the Chemistry, Manufacturing & Controls (CMC) section of the NDA for Gimoti, the Company’s patented nasal delivery formulation of metoclopramide for the relief of symptoms associated with acute and recurrent diabetic gastroparesis in adult women.

 

NewLink Genetics reported interim results from NLG2103, a Phase II study evaluating its IDO pathway inhibitor, indoximod, in combination with checkpoint inhibitors for the treatment of patients with advanced melanoma.

 

Diffusion Pharmaceuticals announced that a key patent has been issued by the USPTO on March 28, 2017 for its lead drug candidate trans sodium crocetinate (TSC).

 

Chembio Diagnostics is collaborating with FIND to develop a POC test that can identify multiple life-threatening acute febrile illnesses common in the Asia Pacific region. Over the next twelve months, Chembio and FIND will endeavor to develop a simple, rapid, and cost-effective test based on Chembio’s proprietary DPP technology platform to detect multiple diseases simultaneously.

 

Kadmon Holdings announced that on March 31, 2017, it submitted its second ANDA to the FDA for KD034, the Company’s trientine hydrochloride formulation for the treatment of Wilson’s disease, a rare genetic liver disorder, for patients who are intolerant of penicillamine. The Company also submitted an ANDA for a bottled form of trientine hydrochloride in December 2016.

 

Depomed announced that, in accordance with its stated plans and the terms of the secured debt facility with Deerfield and Pharmakon Advisors, LP, the company prepaid $100 million of principal amount of its $475 million secured indebtedness. In addition, Depomed paid a prepayment fee of $4 million. Under the terms of the 7-year loan agreement, which matures in 2022, Depomed intends to refinance the remainder of the $375 million secured indebtedness in 2017.

 

 

ANALYST RECOMMENDATIONS

 

Jefferies analyst Biren Amin assumed coverage of Progenics with a “buy” rating and increased his price target to $14 from $13, citing Azedra’s recent positive Phase III in rare PGG/PCC indications positions Azedra to be approved by FDA in early 2018.

 

Chardan analyst Madhu Kumar upgraded Arbutus to “buy” from “neutral” and increased his price target to $6 from $3, citing HBV represents a significant market in infectious diseases, multiple RNAi drugs (including ARB-1467) have demonstrated clinical proof of concept for hepatitis B surface antigen (HBsAg) suppression, and preclinical data from HBV combination therapies has looked promising.

 

Rodman & Renshaw analyst Joseph Pantginis initiated coverage of BeyondSpring with a “buy” rating and $43 price target, citing BeyondSpring’s lead asset, Plinabulin, is in pivotal studies for both the treatment of neutropenia (abnormally low neutrophils) associated with chemotherapy and non-small cell lung cancer (NSCLC).

 

Aegis analyst Difei Yang increased her price target of Nektar Therapeutics to $27 from $24, citing a valuation methodology update for NKTR-181 and NKTR-214.

 

FBR suspended coverage of the following companies, due to a reallocation of resources: Achillion; Aduro; Alnylam; Arena; Asterias; BioCryst; Curis; Genocea; Trevena; and Zafgen.