BioShares Biotechnology Clinical Trials (BBC): $20.73, +$0.03, -28.6% YTD

BioShares Biotechnology Products (BBP): $34.68, -$0.17, +12.5% YTD

 

 

MARKET COMMENTARY

 

U.S. stock index futures were higher, as crude prices surged on optimism that oil producers would arrive at an agreement to limit output. ADP national employment report, pending home sales numbers, PCE price index and consumer spending data are on the economic calendar. European shares rose, staying on track to end the month in positive territory and Asian shares also closed higher. The dollar edged up against a basket of currencies, while gold dropped.

 

 

MARKET HIGHLIGHTS

 

BIOPHYTIS announced the successful completion of its SARA-PK clinical study for Sarconeos, lead drug candidate for the treatment of sarcopenia. The results of the study, conducted in young and elderly healthy volunteers, confirmed Sarconeos’ appropriate safety profile, with no serious adverse events associated with the administration of Sarconeos.

 

TxCell SA announced that it has completed adapting its organization to deliver results from its new strategic priorities.  TxCell has appointed François Meyer, Executive Chairman of TxCell, to head its new research group.  The research group encompasses three units: Cell Engineering, Pharmacology and Process Development. The group currently comprises 31 employees, including 10 PhD and 9 engineers.  Arnaud Foussat, PhD, formerly CSO, is now SVP, Corporate Development and Head of External Collaborations & Alliance Management.  Miguel Forte, EVP, COO, will be leaving the company.

 

Genticel announced that a determining milestone in its partnership with Serum Institute of India Pvt. Ltd. (Serum Institute) has been successfully completed.  As per the agreement signed in February 2015 with Genticel, Serum Institute has been evaluating the benefits of using GTL003 in new and improved multivalent vaccines and has obtained excellent preclinical results.  In preclinical in vivo experimentation, Genticel’s proprietary reengineered adenylate cyclase, GTL003, fulfilled the predetermined objectives.  This was the last preclinical milestone of the agreement, corresponding to a $1.2 million milestone payment.

 

Innate Pharma announced preliminary safety results from the dose-ranging part of a Phase I/II trial investigating monalizumab as a single agent in patients with advanced gynecologic malignancies.  The data were presented today as a poster at the EORTC-NCI-AACR Molecular Targets and Cancer Therapeutics Symposium (Munich, Germany) 10:15 a.m. – 5:00 p.m. CET.  In this dose-ranging part of the study, 18 patients with advanced, heavily pretreated ovarian cancer were randomized to receive three dose levels of monalizumab (1, 4 and 10mg/kg, every two weeks – six patients at each dose level).

The data showed that monalizumab was well tolerated in this patient population, with no dose-limiting toxicities observed.  No major differences in terms of safety were observed across the different dose levels.  The cohort expansion part of this trial (up to 98 patients) is ongoing at the recommended Phase II dose (10 mg/kg) in patients with platinum sensitive ovarian cancer, platinum-resistant ovarian cancer, epithelial endometrial cancer and squamous cell carcinoma of the cervix.

 

TRACON Pharmaceuticals announced the closing of its underwritten public offering of 3,018,750 shares of its common stock, which includes 393,750 shares of common stock issued upon the exercise in full of the option to purchase additional shares granted to the underwriters, at a public offering price of $5.75 per share. The total gross proceeds to TRACON from the offering, before deducting the underwriting discounts and commissions and other offering expenses, are $17.4 million.

 

Susan G. Komen and Heron Therapeutics, Inc. today announced that the two organizations have joined forces to encourage supporters to take action in the fight against breast cancer. Through their new partnership, Susan G. Komen and Heron Therapeutics strive to raise awareness of the importance of supportive care in breast cancer treatment, with the goal of allowing patients to complete their treatment regimens and thereby increase survival rates.

 

Arrowhead Pharmaceuticals announced a strategic redeployment of resources to support the development of RNAi therapeutics that utilize the company’s new proprietary subcutaneous (subQ) and extra-hepatic delivery systems. Arrowhead will discontinue development of clinical stage drug candidates ARC-520, ARC-521, and ARC-AAT, which utilize the DPC(iv), or EX1, delivery vehicle. Following the news, Chardan analyst Madhu Kumar downgraded the stock to “neutral” from “buy” and decreased his price target to $2 from $8; William Blair analyst Katherine Xu downgraded the stock to “market perform” from “outperform” and decreased her price target to $2 from $12; Cantor Fitzgerald analyst Elemer Piros downgraded to “hold” from “buy” and decreased his price target to $1 from $15; Jefferies analyst Eun Yang decreased her price target to $2 from $5; Piper Jaffray analyst Edward Tenthoff downgraded the stock to “neutral” from “overweight” and decreased his price target to $2 from $10; RBC analyst Michael Yee decreased his price target to $2 from $12.

 

Celsion announced that following a review of data from its multinational, randomized pivotal Phase III clinical study of ThermoDox in combination with optimized radiofrequency ablation (RFA) for primary liver cancer (the OPTIMA Study), the study’s Data Monitoring Committee (DMC) has unanimously recommended that the trial continue enrollment. With approximately 40% of patients currently enrolled in the trial, Celsion expects to complete patient enrollment in this 550 patient trial by early 2018.

 

Dow Jones reported that Novo Nordisk said its new insulin Tresiba carries about the same risk of serious heart problems as Sanofi‘s Lantus but offers significantly lower risk of dangerously low blood sugar in people with Type-2 diabetes. Denmark-based Novo Nordisk said its trial of more than 7,500 patients found that those on Tresiba were 27% less likely to suffer an episode of severe hypoglycemia, a dangerous condition that occurs when insulin removes too much sugar from the blood, than those on Lantus. It added that for episodes of severe hypoglycemia during sleep, Tresiba reduced the risk by 54% compared with Lantus. Patients on Tresiba and Lantus were at equal risk of having serious heart problems such as heart attack and stroke.

 

Pfizer announced that the pivotal REFLECTIONS B3271002 study, a comparative safety and efficacy study of PF-05280014 versus Herceptin (trastuzumab), met its primary endpoint. PF-05280014 is being developed by Pfizer as a potential biosimilar to Herceptin.

 

Ultragenyx Pharmaceutical announced positive 78-week data from the Phase II study of UX007 (triheptanoin) in patients with long-chain fatty acid oxidation disorder (LC-FAOD). The frequency and duration of major medical events were reduced significantly during treatment with UX007, and patients demonstrated improved exercise tolerance and quality of life during the study.

 

Agenus announced that the first patient has been dosed in a Phase I/II clinical trial of the anti-OX40 agonist antibody INCAGN1949. The trial is being conducted by, and in collaboration with, Incyte.

 

Global Blood Therapeutics announced that the European Commission, acting on a positive recommendation from the Committee for Orphan Medicinal Products (COMP) of the EMA, has designated GBT440 as an orphan medicinal product for the treatment of sickle cell disease (SCD).

 

H. Lundbeck A/S (Lundbeck) and Otsuka Pharmaceutical Co., Ltd. announced the FDA has determined that the sNDA for the expanded labeling of Abilify Maintena for the maintenance treatment of Bipolar I disorder in adult patients is sufficiently complete to permit a substantive review and is considered filed. Under the PDUFA, the FDA has set a target date of 28 July 2017, to complete its review.

 

Protalix BioTherapeutics announced that the first patient has been enrolled in its Phase II clinical trial of OPRX-106 for the treatment of ulcerative colitis. OPRX-106 is a plant cell-expressed recombinant human tumor necrosis factor receptor II fused to an IgG1 Fc domain (TNFRII-Fc).

 

Ablynx announced that it has achieved an initial discovery milestone with a multi-specific Nanobody construct as part of its collaboration with Novo Nordisk, triggering a €1 million milestone payment to Ablynx. Under the terms of the agreement with Novo Nordisk, signed on 25 November 2015, Ablynx received an upfront license fee of €5 million and will receive up to €4 million in research funding during the initial three year research term of the collaboration. In addition, Ablynx is eligible to receive potential development, regulatory and commercial milestone payments of up to €182 million plus tiered royalties on the annual net sales of any products resulting from the collaboration. Novo Nordisk is responsible for the development, manufacturing and commercialization of any products resulting from this agreement.

 

Nabriva Therapeutics announced the terms of its rights offering for up to 588,127 common shares, including common shares represented by ADSs, to its existing common shareholders and ADS holders. The record date of the rights offering is November 29, 2016. Holders of common shares on the record date are entitled to exercise statutory rights to subscribe for new common shares to maintain their proportionate ownership interest in Nabriva in accordance with the relevant provisions of the Austrian Stock Corporation Act. Each ADS represents one tenth (1/10) of a common share.

 

Galapagos announced a share capital increase arising from warrant exercises. Galapagos issued 86,250 new ordinary shares on 28 November 2016, for a total capital increase (including issuance premium) of €771,337.50. Pursuant to the warrant exercise program of Galapagos’ Executive Committee, Executive Committee members automatically are committed to exercise a minimum number of warrants, subject to certain conditions. In accordance with the rules of this program, CEO Onno van de Stolpe exercised 15,000 warrants. Two other Executive Committee members exercised an aggregate number of 65,000 warrants.

 

Cerecor announced top-line clinical results from its major depressive disorder (MDD) Phase II clinical trial (Clin301-203) of adjunctive treatment of CERC-301, an oral, NR2B specific, NMDA receptor antagonist. Overall, the trial failed to demonstrate efficacy on the primary endpoint for mean improvement in Bech-6, a subset of the Hamilton Depression Scale (HDRS-17), averaged over days 2 and 4 post dose. However, the study showed signals for the CERC-301 20 mg dose group at Day 2, at pre-specified secondary endpoints, indicating a potentially clinically-meaningful effect, though not statistically significant, on the Bech-6 and HDRS-17.

 

Neurocrine Biosciences announced that the Psychopharmacologic Drugs Advisory Committee of the FDA will review data included in the NDA of INGREZZA (valbenazine) for the treatment of tardive dyskinesia on February 16, 2017. The FDA has granted Priority Review status to the INGREZZA NDA with a PDUFA target action date of April 11, 2017.

 

OncoQuest announced positive interim results from the Phase IIb clinical study of its lead product, oregovomab, in patients with advanced epithelial ovarian, adnexal or peritoneal carcinoma (QPT-ORE-002). This study is being conducted at 13 centers in Italy and the U.S, and is a randomized controlled study to compare the effectiveness of first-line chemotherapy (carboplatin and paclitaxel) versus chemoimmunotherapy (carboplatin-paclitaxel and oregovomab) in this patient population according to an optimized schedule of vaccination identified in a prior Phase II study assessing the schedule of these combinations.

 

Cellectar Biosciences announced the closing of its underwritten public offering of 1.6 million shares of its common stock and 68 shares of its preferred stock, which includes the previously announced $8 million offering and the underwriter’s full exercise of their $1.2 million over-allotment option.

 

Omeros announced that an international consortium of complement experts from Italy, United Kingdom, Germany, Spain and Poland was awarded EUR1.3 million in grant funding to study the benefits of inhibiting mannan-binding lectin-associated serine protease-2 (MASP-2) and the lectin pathway in traumatic brain injury (TBI). Omeros’ OMS721 is a human monoclonal antibody that inhibits MASP-2, which is the effector enzyme of the lectin pathway of the complement system. Omeros controls the worldwide rights to MASP-2 and all therapeutics targeting MASP-2.

 

Helix BioPharma announced that after reviewing safety data from the Phase I/II study of L-DOS47 in non-squamous non-small cell lung cancer (LDOS002), the FDA has accepted an accelerated escalation scheme for L-DOS47 dosing in the U.S. Phase I study (LDOS001) up to 12ug/kg in combination with pemetrexed/carboplatin.

 

Trovagene announced it has entered into a long-term Supply and Distribution Agreement with Boreal Genomics, merging their respective technologies to co-develop urine and blood ctDNA assay kits.

 

Merck announced that it has entered into a set of agreements with Evotec AG, whereby Evotec AG will provide screening services for Merck’s collection of genetic reagents such as CRISPR and shRNA libraries. Combining access to these libraries with Evotec’s screening expertise offers an accelerated pathway to explore and identify new drug targets.

 

Aytu BioScience announced the summary of findings that demonstrated significant clinical improvements in erectile function and mood in hypogonadal men treated with Natesto nasal testosterone gel (NTG). Natesto, Aytu’s FDA-approved nasal testosterone gel, was launched in late July 2016.

 

Achillion Pharmaceuticals announced the start of patient dosing in a global Phase IIb open-label OMEGA-1 study of JNJ-4178, a 3DAA combination of odalasvir, simeprevir, and AL-335, has been initiated by Janssen Research & Development, in treatment-naive and treatment-experienced patients with chronic hepatitis C virus infection (HCV) without cirrhosis.

 

Lannett Company announced that the FDA granted the company a 90-day extension to submit documentation concerning its Methylphenidate Hydrochloride (HCl) Extended-Release (ER) Tablets.  The new deadline for submitting the supporting documentation is March 20, 2017.

 

Leica Biosystems and Advanced Cell Diagnostics, a Bio-Techne brand, announced the availability of the Aperio RNA ISH Algorithm for use with ACD’s RNAscope ISH assays.

 

Axial Biotherapeutics announced the launch of the Company with a $19.15 million Series A round of financing. The Company is leveraging the groundbreaking work of scientific founder, Professor Sarkis Mazmanian, Ph.D., which uses the gut microbiome to help treat diseases and disorders of the central nervous system (CNS). Dr. Mazmanian, the Louis & Nelly Soux Professor of Microbiology, Division of Biology & Biological Engineering, California Institute of Technology (Caltech), is a leader in the field of interactions between the gut microbiome and the brain as it relates to neurological disorders. Dr. Mazmanian has published his groundbreaking research in journals that include Nature, Science and Cell. The Mazmanian Laboratory was among the first to demonstrate disease modifying effects in mouse models of autism spectrum disorders (ASD) via novel and proprietary microbiome interventions. Axial will leverage those findings to build a microbiome discovery platform targeting the gut-brain axis which provides the opportunity to generate a diverse pipeline of new therapies for patients with neurological diseases and disorders.

 

Sangamo BioSciences announced that its collaborative zinc finger nuclease (ZFN)-mediated genome editing program for hemoglobinopathies will transfer to Bioverativ.

 

Otonomy announced the appointment of Kathie M. Bishop, Ph.D., as chief scientific officer.



ANALYST RECOMMENDATIONS

 

Cantor Fitzgerald analyst Mara Goldstein initiated coverage of Advaxis with a “buy” rating and $19 price target, citing the company is pursuing a unique approach to immuno-oncology, with key points of differentiation from other approaches.

 

Ladenburg Thalmann analyst Wangzhi Li initiated coverage of Bellicum with a “buy” rating and $31 price target, citing both potential upside catalysts after ASH2016 and long-term appreciation potential of BLCM’s robust pipeline and platform.

 

JP Morgan analyst Cory Kasimov assumed coverage of Acorda Therapeutics with an “overweight” rating and $32 price target, citing two upcoming events have the potential to act as significant points of inflection, including Phase 3 data for CVT-301 and a decision in the pending Ampyra IP cases.

 

Goldman analyst Salveen Richter increased her price target of the following companies, citing AVXS-101 would be used heavily notwithstanding efficacy declines post several years and Spinraza would be used as a second-line/maintenance regimen after gene therapy: Avexis to $91 from $71; Ionis to $25 from $22; Biogen to $322 from $316.

 

Following the news that Arrowhead is discontinuing all clinical RNAi programs, Chardan analyst Madhu Kumar upgraded Arbutus to "buy" from "neutral" and increased his price target to $4 from $3, and increased his price target of Alnylam to $89 from $73. 

 

Aegis analyst Jason Wittes initiated coverage of Xtant Medical Holdings with a “buy” rating and $3.50 price target, citing the company is the result of a merger of two complementary businesses with sales and geographic synergies.

 

Following Mallinckrodt’s earnings, Mizuho analyst Irina Koffler decreased her price target to $85 from $87; Leerink analyst Jason Gerberry decreased his price target to $70 from $92.

 

Barclays analyst Jack Meehan revised his price target of the following companies: Charles River upgraded to “equal-weight” from “underweight” and increased his price target to $77 from $65; Parexel increased to $73 from $70; ICON increased to $82 from $75; Reinstated coverage of Quintiles with and “overweight” rating and increased his price tart to $90 from $88.