BioShares Biotechnology Clinical Trials (BBC): $23.50, +$0.53, +27.6% YTD

BioShares Biotechnology Products (BBP): $38.29, +$0.92, +16.9% YTD

 

 

MARKET COMMENTARY

 

As concerns over Donald Trump’s setback on healthcare reform waned, U.S. stock index futures inched higher. Wholesale inventory, CaseShiller index for house prices and consumer confidence data are on the radar. European shares rose, buoyed by strong earnings and deals across the region, and Asian stocks closed higher. The dollar steadied on talks of more U.S. interest rate hikes this year, while gold slipped. Oil prices rose on supply disruption in Libya and the latest comments from officials suggesting OPEC could extend its deal cutting global production.

 

 

MARKET HIGHLIGHTS

 

Rockwell Medical received notice from the State Intellectual Property Office of the People’s Republic of China (SIPO) that patent claims relating to ESA Sparing, Compositions and Use for Triferic have been accepted for grant in China. Triferic is the only FDA approved therapy indicated to replace iron and maintain hemoglobin in hemodialysis patients who suffer from anemia. The claims allowed in the patent application, entitled “Methods of Treating Iron Deficiency with Soluble Ferric Pyrophosphate” include composition of matter and use of soluble ferric pyrophosphate to reduce ESA usage using hemodialysis solution with the specific dose. The patent provides protection through February 2034.

 

DelMar Pharmaceuticals will present three abstracts at the American Association of Cancer Research (AACR) Annual Meeting. The abstracts are based on research conducted with DelMar’s lead anti-cancer product candidate, VAL-083 (dianhydrogalactitol), a "first-in-class" small-molecule DNA-targeting agent. The AACR Annual Meeting will be held April 1-5, 2017 in Washington, D.C.

 

Catalyst Biosciences announced that the Korean Ministry of Food and Drug Safety (MFDS) approved the Investigational New Drug Application (IND) for CB 2679d/ISU304. Hemophilia is a serious bleeding disorder that results in spontaneous bleeding episodes as well as substantially prolonged bleeding times upon injury. 

 

BioTime announced the successful defense of two key patents from challenge before the European Patent Office (EPO) Opposition division. A large pharmaceutical company and an anonymous filer challenged the EPO’s previous grant of two European patents (EP2554661 and EP2147094), which cover the proprietary directed differentiation methods to produce pluripotent stem cell-derived cell replacement therapies being developed to treat retinal degenerative diseases such as age-related macular degeneration (“AMD”). BioTime addressed the challenges at the EPO headquarters in Munich, Germany during public oral hearings, which took place March 16 -17, 2017.

 

Aradigm Corporation announced financial results for the fourth quarter and full year ended December 31, 2016. The net loss for the year ended December 31, 2016 was $32.9 million, or $2.23 per share, compared with a net loss of $17.2 million, or $1.17 per share, in 2015. As of December 31, 2016, the Company’s cash and cash equivalents totaled $22.6 million.

 

Marrone Bio Innovations and Evogene Ltd announced that MBI will advance certain novel bacteria and Evogene-identified related proteins into MBI’s bioinsecticide product development pipeline under their previously announced multi-year collaboration for the discovery and development of novel insect control solutions.

 

OncoSec Medical will present preclinical results from recent studies describing the latest developments of its gene delivery platform, at a poster session at the upcoming American Association of Cancer Research (AACR) Annual Meeting, in Washington, D.C., on April 1- 5, 2017.

 

Kiadis Pharma N.V. announced that the Pediatric Committee (PDCO) of the European Medicines Agency (EMA) has accepted the Company’s Pediatric Investigation Plan (PIP) for ATIR101™ for the adjunctive treatment in hematopoietic stem cell transplantation (HSCT) for a malignant disease. In addition, the PDCO has agreed that the Company may defer conducting the studies defined in the PIP until after it files a Marketing Authorization Application (MAA) in Europe for the use of ATIR101™ for the treatment of blood cancers.

 

Lipocine will present a company overview at the 16th Annual Needham Healthcare Conference on Tuesday, April 4, 2017 at 9:20 a.m. ET.

 

Albireo Pharma will provide a corporate overview at the 1st Annual HCWainwright & Co NASH Investor Conference on Monday, April 3rd @ 11amET, and at the 16th Annual Needham Healthcare Conference on Tuesday, April 4th @ 3pmET.

 

VBL Therapeutics will provide a corporate overview at the H.C. Wainwright 1st Annual NASH Investor Conference on April 3 @ 8:20amET.

 

RXi Pharmaceuticals announced that it was granted a patent from the Japan Patent Office (JPO) for the composition of matter of sd-rxRNAs targeting connective tissue growth factor (CTGF) for the treatment or prevention of fibrotic disorders, including but not limited to skin fibrosis and proliferative retinopathy (Japanese Patent #: 6060071).  This patent includes the Company’s lead clinical candidate RXI-109, an sd-rxRNA therapeutic compound, which is currently being evaluated in Phase II clinical trials. The patent, once issued, will be set to expire in 2031.

 

Cara Therapeutics announced positive top-line results from Part A of its Phase II/III trial showing that I.V. CR845 met both primary and secondary endpoints for efficacy (reduced itching and improved quality of life, respectively) in patients with uremic pruritus (UP) with statistical significance. UP is an intractable and debilitating systemic itch condition with a high prevalence in patients with chronic kidney disease (CKD), for which there are no approved therapies in the United States.

 

Calithera Biosciences announced that it has achieved pharmacokinetic and pharmacodynamic goals for CB-1158 which, under its agreement with Incyte, entitles the Company to receive a $12 million payment from Incyte.

 

TESARO announced that the FDA has approved ZEJULA (niraparib), an oral, once-daily poly(ADP-ribose) polymerase (PARP) inhibitor, for the maintenance treatment of women with recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in a complete or partial response (CR or PR) to platinum-based chemotherapy. ZEJULA is the first PARP inhibitor to be approved by the FDA that does not require BRCA mutation or other biomarker testing. TESARO anticipates launching ZEJULA in the U.S. in late April. Following the news, SunTrust analyst Peter Lawson increased his price target to $235 from $219; Wedbush analyst David Nierengarten increased his price target to $174 from $164; Bank of America analyst Tazeen Ahmad increased her price target to $179 from $165.

 

TESARO announced a substantial expansion of its niraparib clinical development program. Following the landmark results of the Phase III NOVA trial of niraparib, a comprehensive portfolio review, and the FDA approval of ZEJULA (niraparib) for patients with recurrent ovarian cancer, TESARO is implementing its plans to initiate registration strategies in the settings of metastatic ovarian, breast and lung cancers.

 

Cempra announced that the company has withdrawn its MAA seeking EMA approval of oral capsule and intravenous formulations of solithromycin for the treatment of community-acquired pneumonia in adults.

 

AstraZeneca announced that the FDA has accepted the company’s NDA for LYNPARZA (olaparib) tablets (300mg twice daily) for use in platinum-sensitive, relapsed ovarian cancer patients in the maintenance setting. The FDA has also granted priority review status with a PDUFA set for third quarter 2017. The NDA submission includes the LYNPARZA Phase III SOLO-2 trial data, which showed a reduced risk of disease progression by 70 percent, compared with placebo in germline BRCA-mutated patients. SOLO-2 trial results were presented on March 14(th) at the Society of Gynecologic Oncology Annual Meeting on Women’s Cancer.

 

AcelRx Pharmaceuticals announced that the EMA has notified the company that the ARX-04 (sufentanil sublingual tablet, 30 mcg) MAA has passed validation, and that the scientific review of the MAA is underway. The MAA for ARX-04 (known as DSUVIA in the U.S.) was filed in early March 2017 for the treatment of patients with moderate-to-severe acute pain in a medically supervised setting. AcelRx expects an opinion on the MAA from the CHMP in the first half of 2018.

 

InVivo Therapeutics Holdings announced that the patient enrolled in January in the INSPIRE study of the Neuro-Spinal Scaffold has improved from a complete AIS A spinal cord injury (SCI) to an incomplete AIS B SCI in the time between the one-month and the two-month evaluations. This is the seventh out of the 11 patients (63.6% conversion rate) with at least one month of follow-up to have had an AIS grade improvement. Two additional patients are early in follow-up and have not yet had their one-month visit. The AIS conversion rate observed in the INSPIRE study to date is considerably higher than published rates observed in a range of SCI natural history databases that are all below 25%.

 

Kadmon Holdings announced preclinical data demonstrating the importance of the Rho-associated coiled-coil kinase (ROCK) signaling pathway in the pathogenesis of pulmonary fibrosis, supporting the therapeutic potential of ROCK inhibition to treat the disease. The data will be presented today as a poster at the 2017 Keystone Symposia on Injury, Inflammation and Fibrosis, taking place in Snowbird, UT.

 

AstraZeneca announced new results data which evaluated the cost-effectiveness of SYNAGIS (palivizumab) for respiratory syncytial virus (RSV) in preterm infants 29-34 weeks gestational age compared to those who did not receive SYNAGIS. These results, derived from age-specific information on the incidence and cost of RSV hospitalization and cost of SYNAGIS, demonstrated that SYNAGIS may reduce overall costs in infants born at 29-32 weeks gestational age who are <3 months of age over a one-year period. These data were presented as a poster at the Academy of Managed Care Pharmacy (AMCP) Annual Meeting 2017 in Denver, CO.

 

Ligand Pharmaceuticals announced that CNA Development LLC, an affiliate of Janssen Pharmaceuticals, Inc. has filed an IND for an antibody discovered using Ligand’s OmniAb technology. This IND filing results in a $1 million milestone payment to Ligand.

 

Fibrocell Science announced that it received notice from The NASDAQ Stock Market LLC (NASDAQ) on March 27, 2017 indicating that the Company has regained compliance with the minimum bid price requirement under NASDAQ Listing Rule 5550(a)(2) for continued listing on The NASDAQ Capital Market. Accordingly, Fibrocell is in compliance with all applicable listing standards and its common stock will continue to be listed on The NASDAQ Capital Market and NASDAQ considers the matter closed.

 

CRISPR Therapeutics reported that the European Patent Office (EPO) has announced its intention to grant a patent broadly covering CRISPR’s in-licensed gene editing technology. The claims are directed to the CRISPR/Cas9 single-guide gene editing system for uses in both non-cellular and cellular settings, including in cells from vertebrate animals such as human or mammalian cells – as well as composition claims for use in any setting, including claims for use in a method of therapeutic treatment of a patient.

 

Icagen announced a collaboration with Bayer to develop new X-ray fluorescence assays using the proprietary XRpro platform.  These assays are designed to help Bayer to progress various early discovery programs in the area of transporter targets.

 

Ampio Pharmaceuticals announced that it entered into a Waiver and Consent Letter Agreement with CVI Investments, amending the terms of warrants previously issued to CVI on September 1, 2016.

 

AbbVie and M2Gen announced that AbbVie has joined the Oncology Research Information Exchange Network (ORIEN) Avatar Research Program. Launched in April of 2016, the ORIEN Avatar Research Program fosters collaboration among key stakeholders in cancer research with the shared goal of discovering and developing novel therapies in clinical trials.

 

Sofinnova Partners has appointed Antoine Papiernik as Chairman. Papiernik succeeds Denis Lucquin who remains a Managing Partner. The appointment starts a new chapter in Sofinnova Partners’ growth strategy.

 

ERS Genomics announced that the European Patent Office intends to grant Dr. Emmanuelle Charpentier, ERS Genomics’ co-founder, together with the University of California and University of Vienna, a patent with very broad claims covering the use of CRISPR-Cas9 technology for gene editing. The claims intended for grant by the EPO are directed to the widely-used single-guide CRISPR-Cas9 gene editing system and cover uses in both cellular and non-cellular settings, including use in bacteria, plants, animals, and cells from vertebrate animals such as humans.

 

epliCel Life Sciences reported compelling safety and clinical data from its phase I/II tendon repair study investigating the use of RepliCel’s type 1 collagen-expressing, hair follicle-derived fibroblasts (RCT-01) as a treatment for Achilles tendinosis.

 

Omeros announced presentation of a case report describing resolution of hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA) in a dialysis- and transfusion-dependent adolescent girl who was treated with OMS721 under a compassionate-use protocol. The presentation “Resolution of acute kidney injury secondary to TA-TMA by the anti-MASP-2 monoclonal antibody OMS721 in a pediatric HSCT recipient” occurred at the 43rd Annual Meeting of the European Society for Blood and Marrow Transplantation in Marseille, France on Monday, March 27, 2017. Marco Zecca, M.D., Director of Pediatric Oncology at the Fondazione IRCCS Policlinico San Matteo, presented the data. OMS721 is Omeros’ lead human monoclonal antibody targeting mannan-binding lectin-associated serine protease-2 (MASP-2), the effector enzyme of the lectin pathway of the complement system.

 

AVEO Pharmaceuticals announced that it has priced an underwritten public offering of 30,000,000 shares of common stock at a price to the public of $0.50 per share, before underwriting discounts, which would result in aggregate gross proceeds of approximately $15 million. All of the shares in the offering are to be sold by AVEO. AVEO has also granted the underwriter a 30-day option to purchase up to an additional 4,500,000 shares of common stock on the same terms and conditions. Closing of the offering is expected to occur on or about March 31, 2017, subject to customary closing conditions. Piper Jaffray & Co. is acting as the sole bookrunner for the offering.

 

The Parker Institute for Cancer Immunotherapy, Bristol-Myers Squibb and the Cancer Research Institute announced a multi-year clinical research collaboration to coordinate and rapidly initiate clinical Immuno-Oncology (I-O) studies across the Parker Institute and CRI networks. Bristol-Myers Squibb will work closely with leading Parker Institute and CRI scientists and researchers, soliciting clinical research proposals from their networks and coordinating multi-site collaboration clinical studies to pursue some of the most difficult questions in cancer research. It will provide scientists with an ecosystem of advanced translational tools, precision immunotherapy and cutting-edge bioanalytical expertise to maximize learning and ensure the generation of high quality data to inform future development.

 

Millendo Therapeutics announced the initiation of a Phase II clinical trial evaluating the safety and efficacy of a novel oral drug candidate, ATR-101, in patients with endogenous Cushing’s syndrome (CS), a rare endocrine disease characterized by increased cortisol production from the adrenal glands. ATR-101, an adrenal-selective small molecule inhibitor of ACAT1, is also being studied in an ongoing Phase II clinical trial in patients with classic congenital adrenal hyperplasia (CAH) and a Phase I clinical trial in patients with adrenocortical carcinoma (ACC).

 

Akebia Therapeutics announced the publication of positive results from a Phase IIa study of vadadustat, a once-daily oral HIF stabilizer in development for the treatment of anemia related to chronic kidney disease (CKD). The study demonstrated that vadadustat increased hemoglobin levels in a dose-dependent manner and improved iron mobilization in non-dialysis CKD patients when compared to placebo. The peer-reviewed paper, titled “Clinical Trial of Vadadustat in Patients with Anemia Secondary to Stage 3 or 4 Chronic Kidney Disease,” was published online by the American Journal of Nephrology. Vadadustat is currently being evaluated in a global Phase III clinical program in non-dialysis and dialysis patients with anemia related to CKD.

 

CytoSorbents announced the issuance today of U.S. Patent No. 9,604,196 entitled, "Size Selective Hemocompatible Polymer System".

 

Compugen announced the selection of COM902 as the lead clinical antibody candidate for its CGEN-15137/TIGIT T cell checkpoint inhibitor program in immuno-oncology. COM902 follows COM701 into the Company’s preclinical development pipeline. COM701 is the Company’s lead therapeutic antibody targeting PVRIG, for which IND is anticipated later this year. As previously disclosed, PVRIG and TIGIT represent two distinct but complementary arms of the same biological pathway, and inhibition of the two results in increased activation of tumor infiltrating lymphocytes (TILs). This provides a strong clinical rationale for the combination of COM701 and COM902, in addition to monotherapy use, as immunotherapies to treat various cancer types.

 

Nuvo Pharmaceuticals announced that it has entered into an exclusive license agreement with Sayre Therapeutics PVT to distribute, market and sell Pennsaid 2% in India, Sri Lanka, Bangladesh and Nepal (the Territory).  Nuvo has received an upfront payment and is eligible to receive milestone payments and a double-digit royalty on net sales.  Nuvo will supply Pennsaid 2% to Sayre on an exclusive basis from its manufacturing facility in Varennes, Québec.

 

Orexigen Therapeutics announced that its wholly owned subsidiary, Orexigen Therapeutics Ireland, and Bruno Farmaceutici S.p.A. have executed a distributorship agreement for Mysimba (naltrexone HCl / bupropion HCl prolonged release) in Italy. Mysimba is approved by the EMA for the management of weight in adult patients (≥18 years) with an initial Body Mass Index (BMI) of ≥ 30 kg/m2 (obese), or ≥ 27 kg/m2 to < 30 kg/m2 (overweight) in the presence of one or more weight-related co-morbidities (e.g., type 2 diabetes, dyslipidaemia, or controlled hypertension).

 

Therapix Biosciences announced the closing of its public offering in the U.S of 2,000,000 ADSs, each ADS representing 40 ordinary shares of the Company, at a price of $6.00 per ADS. In addition, Therapix has granted the underwriters a 45-day over-allotment option to purchase up to 300,000 additional ADSs at the public offering price. The gross proceeds to Therapix from this offering were $12,000,000, prior to deducting underwriting discounts and offering expenses. Therapix plans to use the net proceeds from this offering to advance the formulation and clinical development efforts for its two lead product candidates, including Phase II clinical trials, and for working capital and other general corporate purposes. Laidlaw & Company is acting as sole book running manager for the offering.

 

Calithera Biosciences announced the closing of its previously announced public offering of 7,854,500 shares of common stock, including 1,024,500 shares sold pursuant to the underwriters’ exercise in full of their option to purchase additional shares. Gross proceeds from the offering at a public offering price of $10.25 per share, before underwriting discounts and commissions and offering expenses, were approximately $80.5 million. Leerink Partners acted as sole book-running manager for the offering. Wells Fargo Securities acted as lead manager and JMP Securities acted as co-manager for the offering.

 

Perrigo Company announced it has completed the divestiture of its rights to the royalty stream from global net sales of the multiple sclerosis drug Tysabri (natalizumab) to RPI Finance Trust, an affiliate of Royalty Pharma ("RPI"). The transaction comprises a total consideration of $2.2 billion in cash and up to $650 million in royalties earned if global net sales of Tysabri meet specific thresholds in 2018 and 2020.

 

Mylan N.V. announced the appointment of Daniel M. Gallagher as chief legal officer. He will be joining the company on April 17, 2017.

 

 

ANALYST RECOMMENDATIONS

 

HC Wainwright analyst Shaunak Deepak increased his price target of Calithera to $14 from $10, citing the addition of CB-1158 to the valuation on the strength of the partnership.

 

Aegis analyst Difei Yang initiated coverage of Novocure with a “buy” rating and $14 price target, citing Novocure has a ground breaking technology named Tumor Treating Fields (TTFields) which has been approved for use in treating glioblastoma (GBM) patients based on well controlled clinical trials that an impressive 4-5 months in overall survival benefits was demonstrated.

 

Maxim analyst Lauren Chung initiated coverage of VistaGen Therapeutics with a “buy” rating and $4 price target, citing VistaGen’s AV-101, is part of an emerging class of antidepressant drugs targeting the NMDA receptor for adjunctive treatment of MDD and has the potential for rapid relief of depressive symptoms within days (vs. months).

 

Chardan analyst Madhu Kumar initiated coverage of the following companies: CRISPR Therapeutics with a “buy” rating and $23.50 price target; Editas with a “buy” rating and $65 price target; Intellia Therapeutics with a “buy” rating and $19 price target; ToolGen with a “buy” rating and KRW162,500 price target.