LifeSci Logo

BioShares Biotechnology Clinical Trials (BBC): $20.70, -$0.12, +12.4% YTD

BioShares Biotechnology Products (BBP): $37.08, -$0.03, +13.2% YTD

 

 

MARKET COMMENTARY

 

U.S. stock index futures were trading sideways and the dollar fell as investors turned cautious ahead of Janet Yellen’s testimony later in the day that may give clues on the Federal Reserve’s interest rate strategy. The market will also focus on the quarterly results from top companies including American International Group, Devon Energy and Express Scripts. Producer prices data is on the economic calendar. European stocks were little changed and Asian markets declined with Japan’s Nikkei Index sliding as Toshiba weighed on investor sentiment. Oil rose helped by OPEC-led output cuts and gold prices edged up.

 

MARKET HIGHLIGHTS

 

Vermillion announced today that investors, including several Vermillion directors and members of management as well as certain significant stockholders, have agreed to purchase approximately $5.6 million of unregistered shares of Vermillion’s common stock and warrants to purchase unregistered shares of Vermillion’s common stock in a private placement. Under the terms of the private placement, Vermillion has agreed to sell an aggregate of 3,747,125 shares of its common stock at the price of $1.40 per share, the closing price quoted on NASDAQ on February 10, 2017. In addition, the investors will receive warrants to purchase up to an aggregate of 2,810,338 shares of Vermillion’s common stock at an exercise price of $1.80 per share. The warrants become exercisable six months after the closing of the private placement and expire five years from the date of issuance or, if earlier, five business days after Vermillion delivers notice that the closing price per share of its common stock exceeded the exercise price for 20 consecutive trading days during the exercise period.

 

Catalyst Biosciences provided an update on its clinical development plans for its next-generation Factor VIIa and Factor IX candidates and reiterated its intention to commence two clinical trials in individuals with hemophilia B in 2017.  The Company plans to initiate a subcutaneous (SQ) efficacy trial of marzeptacog alfa (activated), a next-generation Factor VIIa, in individuals with hemophilia B with inhibitors in 2017. In addition, Catalyst and its collaboration partner, ISU Abxis, plan to initiate a SQ Phase 1/2 proof-of-concept clinical trial of CB 2679d/ISU304, a next-generation coagulation Factor IX, in individuals with hemophilia B in the second quarter of 2017.

 

Evogene and ICL Innovation announced today they have entered a multi-year research and development collaboration for the discovery of novel crop enhancers for the improvement of nutrient use efficiency in selected crops.  Under the terms of the agreement, Evogene will utilize its computational discovery platform to identify the potential of certain compounds to improve nutrient use efficiency. Successful candidates identified from the collaboration may be integrated into ICL’s product pipeline for further development and formulation. Expected commercialization of products derived from the joint efforts may occur as early as five years from initiation of the research and development activities.

 

Aurinia Pharmaceuticals announced the results of a supportive Phase I safety, pharmacokinetic (PK) and pharmacodynamic (PD) study in healthy Japanese patients which supports further development of voclosporin in this patient population.  Based on evaluations comparing the Japanese ethno-bridging data vs. previous PK and PD studies in non-Japanese patients, voclosporin demonstrated no statistically significant differences in exposure with respect to Area Under the Curve (AUC) measurements. Furthermore, the PK parameters in Japanese patients were generally consistent with previously evaluated PK parameters in non-Japanese volunteers. There were no unusual or unexpected safety signals in the study.

 

Ohr Pharmaceutical announced a strategic business update and reported results for its first quarter ended December 31, 2016. The Company remains in active ongoing discussions with potential pharmaceutical partners.  In parallel with these ongoing business discussions, for strategic reasons, Ohr has paused enrollment in the first Phase 3 clinical trial of its lead drug candidate squalamine lactate ophthalmic solution, 0.2% (“Squalamine”), for the treatment of neovascular age-related macular degeneration (wet AMD). The enrollment pause is not related to any safety issue. Currently, there are more than 200 subjects enrolled in the study. The study remains double-masked and no interim efficacy or futility analysis has been performed.  Subjects currently enrolled in the study continue to undergo scheduled visits and assessments as well as receive assigned study treatments as per the current protocol.  For the quarter ended December 31, 2016, the Company reported a net loss of approximately $7.0 million, or ($0.21) per share, compared to a net loss of approximately $6.1 million, or ($0.20) per share in the same period of 2015.  At December 31, 2016, the Company had cash and cash equivalents of approximately $13.5 million. This compares to cash and equivalents of approximately $12.5 million at September 30, 2016.

 

Moberg Pharma reported financial results for the year ended December 31, 2016.  Net sales for the year were SEK 334.3 million.  The company reported a net profit after tax of SEK 32.7 million.   Earnings per share were SEK 2.25.

 

Quintiles reported 4Q16 non-GAAP EPS of $1.09, which compares to $0.90 for the same period a year ago. The company beat the non-GAAP EPS mean estimate of $1.05. Total revenue for 4Q16 was $1.95 billion, which compares to $1.13 billion for the same period a year ago. This compares to a mean estimate of $1.97 billion.

 

Incyte reported 4Q16 GAAP EPS of $0.05, which compares to $0.29 for the same period a year ago. The company missed the GAAP EPS mean estimate of $0.13. Total revenue for 4Q16 was $326.5 million, which compares to $243.9 million for the same period a year ago. This compares to a mean estimate of $324.4 million.

 

Charles River Labs reported 4Q16 non-GAAP EPS of $1.21, which compares to $1.00 for the same period a year ago. The company beat the non-GAAP EPS mean estimate of $1.11. Total revenue for 4Q16 was $466.8 million, which compares to $353.9 million for the same period a year ago. This compares to a mean estimate of $441.6 million.

 

Aviragen Therapeutics announced top-line data from its Phase IIb SPIRITUS trial, a multi-center, randomized, double-blind, placebo-controlled, dose-ranging study of vapendavir in moderate to severe asthmatics with a rhinovirus (RV) infection. Vapendavir did not demonstrate a statistically significant reduction in the asthma control questionnaire-6 (ACQ-6) at day 14, the primary endpoint, for either the 264 mg or 528 mg cohorts compared to placebo.

 

Axsome Therapeutics received Fast Track designation from the FDA for AXS-05 for treatment resistant depression (TRD). AXS-05 is currently being evaluated in the STRIDE-1 study, a Phase III, randomized, double-blind, active-controlled trial to assess the efficacy and safety of AXS-05 in the treatment of TRD.

 

Catalyst Biosciences provided an update on its clinical development plans for its next-generation Factor VIIa and Factor IX candidates and reiterated its intention to commence two clinical trials in individuals with hemophilia B in 2017. The Company plans to initiate a subcutaneous (SQ) efficacy trial of marzeptacog alfa (activated), a next-generation Factor VIIa, in individuals with hemophilia B with inhibitors in 2017. In addition, Catalyst and its collaboration partner, ISU Abxis, plan to initiate a SQ Phase I/II proof-of-concept clinical trial of CB 2679d/ISU304, a next-generation coagulation Factor IX, in individuals with hemophilia B in the second quarter of 2017.

 

Marinus Pharmaceuticals announced the publication of preclinical data in the January issue of Neuropharmacology, showing that its positive allosteric modulator of GABAA, ganaxolone ameliorates many of the behavioral abnormalities in a  mouse model of adult Angelman syndrome (AS). Evidence from experimental models suggests that AS may be associated with deficient extrasynaptic GABAA receptor function. AS is a rare neurogenetic disorder characterized by severe developmental delay, motor impairments and epilepsy.

 

Incyte and Agenus announced that the companies have amended the License, Development and Commercialization Agreement that was originally entered into January 9, 2015. The amended agreement converts the ongoing GITR and OX40 antibody programs from co-funded development and profit-sharing arrangements to royalty-bearing programs, with Incyte now responsible for funding and conducting global development and commercialization. Should candidates from either of these two programs be approved, Agenus would now become eligible to receive 15 percent royalties on global net sales of each approved product.

 

Janssen Sciences Ireland announced positive results from the full data read out for two Phase III studies evaluating the safety and efficacy of switching virologically suppressed patients from a three- or four-drug antiretroviral regimen to the two-drug regimen of dolutegravir (ViiV Healthcare) and rilpivirine (Janssen). Full results were presented at the annual Conference on Retroviruses and Opportunistic Infections (CROI) in Seattle, WA.

 

Amgen announced the submission of a sBLA to the FDA for BLINCYTO (blinatumomab) to include overall survival (OS) data from the Phase III TOWER study, supporting the conversion of BLINCYTO’s accelerated approval to full approval. The sBLA 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 broaden BLINCYTO’s indication for the treatment of patients with relapsed or refractory B-cell precursor ALL.

 

Progenics Pharmaceuticals announced that enrollment of an investigator initiated Phase I clinical trial with 131-I-MIP-1095 (1095) has begun at MSK.  1095 is one of Progenics’ PSMA-targeted small molecule radiopharmaceutical candidates being developed for the treatment of metastatic prostate cancer.

 

Eli Lilly and Incyte announced that the EC has granted marketing authorization for Olumiant (baricitinib) 4 mg and 2 mg film-coated tablets in Europe for the treatment of moderate-to-severe active rheumatoid arthritis (RA) in adult patients who have responded inadequately to, or who are intolerant to, one or more disease-modifying antirheumatic drugs (DMARDs). This is the first regulatory approval around the world for Olumiant — the first JAK inhibitor approved to treat RA in the EU — which may be used as monotherapy or in combination with methotrexate. In clinical studies, Olumiant has demonstrated significant improvement in the signs and symptoms of RA compared to standard of care therapies such as methotrexate and Humira* (adalimumab) with background methotrexate.

 

Reuters reported that Marathon Pharmaceuticals said it was "pausing" the launch of its Duchenne muscular dystrophy drug after U.S. lawmakers questioned why the company priced it at $89,000 a year when patients had been able to import it for as little as $1,000. In a statement posted on the patient advocacy website Cure Duchenne, Marathon’s chief executive, Jeffrey Aronin, said the company was pausing the launch amid "concerns about how the pricing and reimbursement details will affect individual patients and caregivers." The company said it would maintain its "expanded access program" under which 800 patients currently receive the drug for nothing. It said patients currently importing the drug from overseas would continue to be able to do so.

 

Gilead Sciences announced data from a Phase II study evaluating the efficacy, safety and tolerability of a combination of bictegravir (75 mg) (BIC) and emtricitabine/tenofovir alafenamide (200/25 mg) (FTC/TAF) versus dolutegravir (50 mg) (DTG) and emtricitabine/tenofovir alafenamide (200/25 mg) (FTC/TAF) in treatment naïve, HIV-1 infected adults. Results found that the BIC+FTC/TAF and DTG+FTC/TAF regimens both demonstrated high virologic response rates at Week 24 and Week 48. Bictegravir is a novel investigational once-daily integrase strand transfer inhibitor (INSTI) that is currently in Phase III trials as part of a single tablet regimen in combination with FTC/TAF for the treatment of HIV (bictegravir 50 mg/emtricitabine 200 mg/tenofovir alafenamide 25 mg). The data are being presented in an oral session (Session O-4) at the 2017 Conference on Retroviruses and Opportunistic Infections (CROI) in Seattle.

 

Vifor Pharma, a company of the Galenica Group, and ChemoCentryx, announced today that Vifor Pharma has gained rights to commercialize avacopan in Asia, including Japan and the Middle-East. ChemoCentryx retains all rights in the United States and China. Avacopan is an orally-administered inhibitor of the complement 5a receptor (C5aR) and is currently in Phase III development for rare renal diseases.

 

AMAG Pharmaceuticals and Endoceutics announced that they have entered into an exclusive license agreement that provides AMAG with U.S. commercial rights to Intrarosa (prasterone). Intrarosa is the only FDA-approved, locally administered, daily, non-estrogen product for the treatment of moderate-to-severe dyspareunia (pain during intercourse), a common symptom of vulvar and vaginal atrophy (VVA), due to menopause. Unlike conventional pharmacological estrogen-containing medications, Intrarosa does not carry a boxed safety warning in its label.

 

REGENXBIO announced the IND is active for the planned multi-center, open-label, multiple-cohort, dose-escalation Phase I clinical trial of RGX-314 for the treatment of wet age-related macular degeneration (wet AMD).

 

Ionis Pharmaceuticals announced the advancement of IONIS-FXIRx in clinical development under an existing exclusive license agreement with Bayer. Under this agreement, Ionis will also initiate development of IONIS-FXI-LRx, which uses Ionis’ proprietary LIgand Conjugated Antisense, or LICA, technology.  In conjunction with the decision to advance these programs, Ionis will receive a $75 million payment from Bayer. 

 

Viking Therapeutics announced an agreement with PoC Capital to fund Viking’s initial clinical development of VK2809 in glycogen storage disease type Ia (GSD Ia).  Viking plans to file an IND application for VK2809 for the treatment of GSD Ia and initiate a human proof-of-concept study in the second half of 2017.

 

Aequus Pharmaceuticals announced that it has completed the treatment phase of its multi-dose Proof of Concept clinical trial evaluating the bioavailability and safety of its once-weekly transdermal patch for aripiprazole, AQS1301. Aripiprazole is an atypical antipsychotic and the active ingredient in Abilify, a leading medication in the US used for the treatment of a number of psychiatric disorders including bipolar I disorder, schizophrenia, major depressive disorder and irritability associated with autistic disorder. Aequus’ novel once-weekly formulation is designed to provide patients with an easy to use and convenient long-acting alternative to the once a day oral pill. The Company expects to report topline results from this multi-dose study in the first quarter of 2017.

 

Sanofi Genzyme, the specialty care global business unit of Sanofi, announced the start of a Phase II trial of an investigational oral therapy for patients with Parkinson’s disease who carry a single copy of a gene mutation that is the most common genetic risk factor for the disease. The trial will assess the drug’s dynamics, efficacy and safety. This is the first industry-sponsored Phase II clinical trial in a genetically defined population of Parkinson’s disease.

 

Insmed announced that the USPTO issued U.S. Patent Number 9,566,234 for ARIKAYCE, the Company’s liposomal amikacin for inhalation. The claims of the patent relate in part to systems and methods for treating pulmonary infections, including nontuberculous mycobacteria (NTM) infections. The systems each include a pharmaceutical formulation containing an aqueous dispersion of liposomal complexed aminoglycoside, which can be amikacin sulfate, with a nebulizer. The patent, the seventh U.S. patent to issue to Insmed for ARIKAYCE in NTM, is expected to provide patent coverage for ARIKAYCE in NTM into January 2034, thereby extending previously existing patent coverage by five years and five months. Insmed is currently evaluating ARIKAYCE in a global Phase III trial in patients with NTM lung disease.

 

Propanc Health announced the successful completion of the low dose group for the GLP-Compliant 28-day repeat-dose toxicity study for its lead product, PRP, and received approval to proceed with the middle and high dose groups from the International Animal Care and Use Committee (IACUC) in Melbourne, Australia. PRP is a solution for once daily intravenous administration of pancreatic proenzymes trypsinogen and chymotrypsinogen.

 

Global Blood Therapeutics announced that a paper describing the discovery of GBT440 and its ability to bind to hemoglobin and prevent red blood cells from sickling was published online in ACS Medicinal Chemistry Letters, a peer-reviewed publication of the American Chemical Society. GBT440 is in Phase III development as a potentially disease-modifying therapy for sickle cell disease (SCD).

 

Aldeyra Therapeutics announced that it intends to offer and sell shares of its common stock in an underwritten public offering. Aldeyra also expects to grant the underwriters a 30-day option to purchase additional shares of common stock. All of the shares in the offering are to be sold by Aldeyra, with net proceeds to be used for the continued development of Aldeyra’s lead compound, ADX-102, and other product candidates, including further clinical testing of ADX-102, as well as for research and development activities, working capital and other general corporate purposes. Stifel and Canaccord Genuity are acting as joint book-running managers and Laidlaw & Company is acting as the lead manager for the offering.

 

Galena Biopharma announced the closing of its previously announced underwritten public offering of 17,000,000 shares of common stock and warrants to purchase 17,000,000 shares of common stock priced at $1.00 per share and accompanying warrant. The warrants are immediately exercisable with a strike price of $1.10 and will expire on the fifth anniversary of the date of issuance.  The shares of common stock and the warrants will be issued separately and will be separately transferable immediately upon issuance. The net proceeds to Galena are approximately $15.5 million, assuming no exercise of the warrants and after deducting underwriting discounts and commissions and estimated offering expenses payable by Galena. Canaccord Genuity acted as sole book-running manager for the offering.

 

Vernalis announced the achievement of a clinical milestone from its collaboration with Corvus Pharmaceuticals triggering a payment of $3 million to Vernalis. In February 2015, the Company licensed exclusive, worldwide rights to its adenosine receptor antagonist programme, CPI-444, for use in all therapeutic applications to Corvus, a US-based biotechnology company focused on developing novel immuno-oncology therapies.

 

Arena Pharmaceuticals announced the appointment of three new members to the company’s Board of Directors. Jayson Dallas, M.D., Oliver Fetzer, Ph.D., and Garry A. Neil, M.D. have joined as independent directors.

 

 

Analyst Recommendations

 

Mizuho analyst Irina Koffler downgraded Depomed to “neutral” from “buy” and decreased her price target to $17 from $24, citing a foreseeable deal is less likely, slowing sales trends, and recent sales force disruption.

 

Ladenburg Thalman analyst Kevin DeGeeter downgraded Aviragen Therapeutics to “neutral” from “buy,” and removed his price target, citing failure of Phase IIb SPRITUS trial.

 

Berenberg analyst Laura Sutcliffe decreased her price target of Gilead to $78 from $112, citing decreased cash flows from HCV adds to the pressure already on management to engage in earnings accretive M&A but the right opportunity seems to be elusive at the moment.

 

Chardan analyst Gbola Amusa upgraded Sage Therapeutics to “neutral” from “sell” and increased his price target to $60 from $18, citing encouraging top-line phase II results from its open label clinical trial of orally-administered SAGE-217 for major depressive disorder (MDD).