BioShares Biotechnology Clinical Trials (BBC): $21.70, -$0.38, +17.9% YTD

BioShares Biotechnology Products (BBP): $36.47, -$0.32, +11.4% YTD

 

 

MARKET COMMENTARY

 

Wall Street looked set to follow global stocks higher, after the market-favored candidate, Emmanuel Macron, won the first round of the French election, reducing the risk of another Brexit-like shock. The euro surged, while safe-haven assets including gold and the yen fell. Oil prices rose driven by expectations that OPEC will extend output cuts till the end of 2017. Alcoa, T-Mobile and Whirlpool are scheduled to report quarterly results after the closing bell.

 

 

MARKET HIGHLIGHTS

 

Immune Pharmaceuticals announced a major corporate restructuring with the objective of prioritizing and segregating its research and development efforts on a focused set of products in inflammatory disease and dermatology and strengthening its financial position. In line with this prioritization, the Company’s Board of Directors has authorized Dr. Daniel Teper to lead the Company’s oncology business within the Company’s Cytovia subsidiary and to pursue a possible spin-off of Cytovia into a separate, stand-alone company independent from Immune. Cytovia will focus on the development and commercialization of novel immuno-oncology and hematology therapeutics, led by Ceplene, an immunotherapy treatment in late stage development in combination with low dose interleukin 2 (IL-2) for the remission maintenance of patients with Acute Myeloid Leukemia; Azixa and crolibulin, two Phase II drug candidates with synergistic potential with immuno-oncology drugs; and a bispecific antibody platform to be supported by collaborative partnerships. Under the leadership of Dr. Teper, Cytovia will aim to grow into a global specialty biopharmaceutical company through these product candidates and the acquisition of additional late stage or commercial stage oncology products. Cytovia intends to raise sufficient capital to support R&D investment through product licensing and partnership transactions, government grants and issuance of debt and equity.

 

Immune Pharmaceuticals announced that on April 18, 2017 the Company has received written notice from the Listing Qualifications Department of The NASDAQ Stock Market LLC notifying that the Company does not comply with Nasdaq’s filing requirements for continued inclusion set forth in Listing Rule 5250(c)(1) because it had not yet filed its Form 10-K for the year ended December 31, 2016.  The Company is working to complete the 2016 Form 10-K and will file it as soon as practicable.

 

DURECT Corporation announced that clinical data on DUR-928 were presented at The International Liver CongressTM 2017 (the 52nd annual meeting of the European Association for the Study of the Liver (EASL)) on April 22 in Amsterdam. 

 

Lipocine announced the completion of enrollment for both its dosing validation ("DV") study and its dosing flexibility ("DF") study for LPCN 1021.  LPCN 1021 is an oral testosterone product candidate for testosterone replacement therapy in adult males for conditions associated with a deficiency of endogenous testosterone, also known as hypogonadism.

 

iCAD will release financial results for the three months ended March 31, 2017 after the close of the market, and host a conference call at 4:30pm Eastern Time, on Tuesday, May 2, 2017.

 

Eiger BioPharmaceuticals announced that an abstract describing prevalence of hepatitis delta virus (HDV) in the U.S. will be presented at Digestive Disease Week (DDW) in Chicago, Illinois, May 6 to 9, 2017.

 

Arrowhead Pharmaceuticals presented clinical data from a Phase 1a/1b study of ARC-AAT, the company’s prior generation investigational medicine that was being studied for the treatment of liver disease associated with alpha-1 antitrypsin deficiency (AATD), at The International Liver Congress™ 2017 (ILC), the annual meeting of the European Association for the Study of the Liver (EASL).  The data demonstrated that an RNA interference (RNAi) therapeutic can achieve deep durable, and dose dependent reductions of alpha-1 antitrypsin (AAT), with the highest dose studied in healthy volunteers achieving near full suppression of the liver production of the AAT protein. In addition, at doses of 2 and 4 mg/kg in AATD patients, ARC-AAT produced similar levels of knockdown as seen in healthy volunteers. These data support the continued advancement of ARO-AAT, Arrowhead’s follow-on product candidate that utilizes the company’s next generation, proprietary subcutaneously administered delivery vehicle.

 

BioTime announced that an abstract related to the Company’s retinal tissue implant has been accepted for a paper presentation at the Annual Meeting of the Association for Research in Vision and Ophthalmology (ARVO) in Baltimore, Maryland, May 7-11.

 

Paratek Pharmaceuticals announced today that an analysis of microbiology data from its Phase 3 study of omadacycline in acute skin infections found that once-daily treatment with IV-to-oral omadacycline is effective in treating the most frequently isolated bacterial pathogens associated with skin infections, including methicillin-resistant Staphylococcus aureus (MRSA). These findings were presented for the first time at the annual meeting of the European Congress of Clinical Microbiology and Infectious Diseases (ECCMID 2017) in Vienna, Austria.

 

Paratek Pharmaceuticals announced today that two cost benefit analyses presented at the annual meeting of the European Congress of Clinical Microbiology and Infectious Diseases (ECCMID 2017) in Vienna, Austria, highlight the potential of IV-to-oral omadacycline to confer cost savings relative to the current standard of care for the treatment of patients with acute bacterial skin and skin structure infection (ABSSSI).

 

Paratek Pharmaceuticals announced that it has entered into a collaboration with Zai Lab (Shanghai) Co., Ltd., a biopharmaceutical company based in China, to support the development and commercialization of omadacycline for patients in China.  Under the agreement, Paratek has granted to Zai Lab an exclusive license to develop, manufacture and commercialize omadacycline for the greater China territory, specifically the People’s Republic of China, Hong Kong, Macau, and Taiwan markets.  The companies will establish a joint steering committee to review and oversee all development, manufacturing, and commercialization plans. Paratek will receive a $7.5 million upfront payment in connection with the signing of the agreement and is eligible for additional milestone payments related to development, regulatory, and commercial milestones.  In addition, Paratek will be eligible to receive royalty payments on sales of omadacycline in the territory.

 

Alcobra today mailed proxy materials for the Extraordinary General Meeting of Shareholders, called by Brosh Capital L.P. and certain of its affiliates ("the Brosh Group"), to describe the Board’s opposition to the Brosh Group’s calling of the meeting and to provide shareholders with the ability to vote on the Company’s proxy card to defend Alcobra against an opportunistic takeover by a new investor with no track record of managing pharmaceutical companies.  As previously announced, Alcobra believes that the Brosh Group’s proposals violate the organizational documents of the Company and applicable law, and therefore Alcobra respectfully rejected the Brosh Group’s request to convene an extraordinary general meeting of shareholders. Alcobra believes the Extraordinary Meeting of Shareholders should not be held in light of applicable law, but is nevertheless committed to defending shareholders against an opportunistic takeover should the meeting be deemed legally valid.

 

Achaogen announced multiple presentations that highlight the effectiveness of plazomicin against MDR gram-negative bacteria in multiple settings. Results were presented at the European Congress of Clinical Microbiology and Infectious Disease (ECCMID) which is being held in Vienna, Austria from April 22-25, 2017.

 

Albireo Pharma announced that the embargo on its late-breaker abstract discussing preliminary data from a Phase 2 clinical trial of its lead product candidate A4250 in children with cholestatic liver disease has been lifted and the data was presented on Saturday at The International Liver Congress™ 2017 in Amsterdam. The data demonstrated improvement in pruritus and reduction in serum bile acids (sBA) in most patients, particularly patients with progressive familial intrahepatic cholestasis (PFIC), after four weeks of treatment with A4250.

 

Arena Pharmaceuticals announced the completion of its previously announced underwritten public offering of 69,000,000 shares of its common stock, including 9,000,000 shares sold pursuant to the full exercise of an option previously granted to the underwriters. All of the shares were sold at a price to the public of $1.15 per share. Including the option exercise, the aggregate gross proceeds from the offering were approximately $79.4 million, before deducting the underwriting discounts and commissions and offering expenses payable by Arena. Arena anticipates using the net proceeds from the offering for clinical and preclinical development of drug candidates, for general corporate purposes, including working capital and costs associated with manufacturing services, and for capital expenditures. Citigroup and Leerink Partners acted as joint book-running managers for the offering.

 

Cara Therapeutics announced summary results from its Phase I safety trial showing that I.V. CR845 did not significantly differ from placebo across three quantitative measures of respiratory drive in healthy individuals. Respiratory depression remains the most life-threatening side effect of traditional, centrally acting, opioid analgesics, the most commonly used drug class for current treatment of postoperative pain in the United States.

 

Sanofi announced that the FDA approved Thymoglobulin [anti-thymocyte globulin (rabbit)], for use in conjunction with concomitant immunosuppression in the prophylaxis, or prevention, of acute rejection in patients receiving a kidney transplant.

 

Akari Therapeutics announced that it will present data from an interim analysis of its ongoing Phase II trial of Coversin in paroxysmal nocturnal hemoglobinuria (PNH), as well as preclinical data for additional indications and other opportunities, at their Research and Development Day.

 

PDL BioPharma announced that on April 21, 2017, the Company entered into a settlement agreement with certain subsidiaries of Merck  to resolve the patent infringement lawsuit between the parties pending in the U.S. District Court for the District of New Jersey related to Merck’s Keytruda humanized antibody product. Under the terms of the agreement, Merck will pay the Company a one time, lump-sum payment of $19.5 million, and the Company will grant Merck a fully paid-up, royalty free, non-exclusive license to certain of the Company’s Queen et al. patent rights for use in connection with Keytruda as well as a covenant not to sue Merck for any royalties regarding Keytruda.  In addition, the parties agreed to dismiss all claims in the relevant legal proceedings.

 

Auris Medical Holding announced key results from AMPACT2 (AM-101 in the Post-Acute Treatment of Peripheral Tinnitus 2), the open-label extension study of the Phase III TACTT3 clinical trial. The study confirms the long-term safety of Keyzilen and further supports early treatment from onset of inner ear tinnitus.

 

XOMA announced that it has achieved positive Phase II proof-of-concept results for X213 in physiological hyperprolactinemia (HPRL). X213 is a monoclonal antibody that neutralizes prolactin action.

 

Inovio Pharmaceuticals announced that it has commenced a Phase II trial to evaluate the efficacy of VGX-3100 in patients with pre-cancerous lesions of the vulva, or vulvar intraepithelial neoplasia (VIN). VGX-3100 is an immunotherapy that targets human papillomavirus (HPV) 16 and 18 and is being studied for the treatment of HPV-related pre-cancerous lesions and persistent HPV infection that causes these lesions.

 

Medivir communicated an update on the status of the development of JNJ-4178, the triple combination of simeprevir, odalasvir and AL-335, following The International Liver Congress 2017 of the European Association for the Study of the Liver (EASL), which was held in Amsterdam, on 19-23 April.

 

MOLOGEN AG announced the key results of the exploratory phase II IMPULSE study. The randomized study evaluated the efficacy and safety of lefitolimod in patients with extensive-disease small-cell lung cancer (SCLC). IMPULSE shows positive results regarding overall survival (OS) in two subgroups of patients in comparison to the control group (standard therapy). Additional, potentially promising subgroups will be identified. The results of this SCLC study provide significant guidance for defining patient populations that, even beyond this study, are most likely to benefit from the immune surveillance reactivator lefitolimod, even though in this highly challenging indication the primary endpoint OS was not met in the overall study population.

 

Pfizer announced positive results of the REPROVE1 Study that showed patients diagnosed with hospital-acquired pneumonia (HAP), treated with Zavicefta, a novel combination antibiotic for the treatment of certain known or suspected Gram-negative bacterial infections, or Meropenem (meropenem for injection), a broad spectrum carbapenem antibiotic currently considered the standard of care, experienced comparable rates of clinical cure at test-of-cure 21-25 days after randomization. Clinical cure was the primary endpoint of the study and defined as a complete resolution of all signs and symptoms of infection.

 

Eli Lilly announced that following a pre-planned interim analysis for MONARCH 3, the trial met its primary endpoint of demonstrating statistically significant improvement in progression-free survival (PFS). In addition, improvement was shown in a key secondary endpoint of objective response rate (ORR). The Phase III study evaluated abemaciclib, a cyclin-dependent kinase (CDK)4 and CDK6 inhibitor, in combination with an aromatase inhibitor (letrozole or anastrozole) compared to treatment with an aromatase inhibitor alone in women with hormone-receptor-positive (HR+), human epidermal growth factor receptor 2-negative (HER2-) advanced breast cancer. Detailed efficacy and safety results will be presented at a medical meeting in the second half of the year.

 

Sage Therapeutics announced the appointment of Michael Cloonan as Chief Business Officer. Additional executive appointments include Christopher Silber, M.D., as Senior Vice President, Clinical Development, Paul Hodgkins, Ph.D., as Vice President, Health Economics and Outcomes Research and Value Demonstration and Frank Sanders as Vice President, Sales & Marketing. In addition, Amy Schacterle, Ph.D., has been promoted to Senior Vice President, Regulatory Affairs and Quality Assurance.

 

Matinas BioPharma Holdings announced that it presented positive preclinical data at the 27(th) Annual European Congress of Clinical Microbiology and Infectious Diseases, being held April 22-25, 2017 in Vienna, Austria.

 

American Regent, a member of the Daiichi Sankyo Group, announced that the first patient has been enrolled into the phase III clinical trial, HEART-FID. This double-blind, multicenter, prospective, randomized, placebo-controlled study will assess the efficacy and safety of iron therapy using intravenous (IV) ferric carboxymaltose (FCM), relative to placebo, in the treatment of patients with heart failure, iron deficiency and a reduced ejection fraction.

 

RedHill Biopharma announced enrollment of the last patient in the Phase II study with BEKINDA (RHB-102)(1) 12 mg for the treatment of diarrhea-predominant irritable bowel syndrome (IBS-D).

 

Denovo Biopharma announced that it has obtained an exclusive license to liafensine (DB104), a serotonin-norepinephrine-dopamine reuptake inhibitor (SNDRI), a late-stage CNS drug, from Albany Molecular Research.  The agreement between Denovo Biopharma and AMRI concludes AMRI’s divesture of all its legacy intellectual property assets.  Under the terms of the definitive agreement, Denovo Biopharma gains an exclusive license to all rights to develop, manufacture and commercialize liafensine globally. Liafensine represents the third late stage product in Denovo Biopharma’s pipeline.

 

Biogen will present Phase III end of study SPINRAZA (nusinersen) data from CHERISH, which demonstrated a highly statistically significant and clinically meaningful improvement in motor function in children with later-onset (most likely to develop Type 2 or Type 3) spinal muscular atrophy (SMA) compared to untreated children. The overall findings continue to support the robust efficacy and favorable safety profile of SPINRAZA across a broad range of individuals with SMA. The SPINRAZA development program represents the largest body of clinical data of its kind in SMA. SPINRAZA data will be presented at the American Academy of Neurology (AAN) annual meeting in Boston, Mass., April 22-28, 2017.

 

Biogen announced new real-world data that show treatment with its leading multiple sclerosis (MS) therapies, TECFIDERA (dimethyl fumarate) and TYSABRI (natalizumab), early in the course of the disease may improve outcomes for people living with relapsing MS. These data were presented at the 69th annual meeting of the American Academy of Neurology (AAN) in Boston.

 

aTyr Pharma announced promising clinical results from its Phase Ib/II 003 trial assessing the safety and potential activity of Resolaris in patients with early onset facioscapulohumeral muscular dystrophy (FSHD).

 

Mustang Bio, a Fortress Biotech company, announced that Manuel Litchman, M.D., has been appointed President and CEO. Dr. Litchman has also joined Mustang’s Board of Directors. Michael S. Weiss, who oversaw Mustang’s corporate operations on an interim basis, will continue to serve as Chairman of the Board of Directors.

 

CytoSorbents announced that it was awarded a two-year $999,996 Small Business Innovation Research (SBIR) Phase II contract to continue development of CytoSorb for fungal mycotoxin blood purification. This follows the successful completion of its previously announced $150,000 Phase I SBIR contract for mycotoxin removal.  This contract is funded through the Chemical and Biological Defense (CBD) SBIR Program which represents a component of the Joint Chemical and Biological Defense Science & Technology Program.  This SBIR project is being managed by the Joint Program Executive Office for Chemical and Biological Defense (JPEO-CBD), the U.S. Department of Defense focal point for research, development, acquisition, fielding, & life-cycle support of Chemical, Biological, Radiological, Nuclear (CBRN) equipment and medical countermeasures.

 

Acasti Pharma announced the granting of additional patents by the Taiwanese and Australian patent offices, further expanding the intellectual property position of CaPre. The granted patents are valid until 2030 and relate to concentrated therapeutic krill oil-based phospholipid omega-3 compositions covering methods for treating or preventing disorders associated with cardiovascular diseases. These patents add to Acasti’s growing portfolio of issued patents in the United States, China, Mexico, Saudi Arabia, Panama, and South Africa. Patent applications with similar claim sets are being pursued in many other jurisdictions worldwide.

 

Alder BioPharmaceuticals presented additional data for eptinezumab (formerly ALD403), which is in Phase III clinical development for the prevention of migraine, at the 69(th) Annual American Academy of Neurology (AAN) meeting in Boston.

 

Oryzon Genomics announced that the Board of Directors of the Alzheimer’s Drug Discovery Foundation (ADDF) has approved a grant of $300,000 to Oryzon Genomics S.A. in support of the project "Clinical development of a companion marker for treatment with the dual LSD1/MAO-B inhibitor ORY-2001."

 

Theravance Biopharma announced that new preliminary data from the ongoing Telavancin Observational Use Registry (TOUR) study are the focus of three poster presentations at the 27th European Congress of Clinical Microbiology and Infectious Diseases (ECCMID). TOUR, which has enrolled its target of 1,000 patients, is designed to report how VIBATIV (telavancin) is being used by healthcare practitioners to treat patients in real-world clinical settings. The presented findings, which focus on data from registry patients with diagnoses of complicated skin & skin structure infections (cSSSIs), bone and joint infections, or lower respiratory tract infections (LRTIs), report positive clinical responses for VIBATIV treatment ranging from 58.3% to 75.3% in these infection types. Positive clinical response was defined as cure or improvement leading to step-down oral therapy. The Company plans to present additional collections of data from the ongoing TOUR study at appropriate upcoming scientific conferences. The 27(th) ECCMID is being held in Vienna, Austria, April 22-25, 2017.

 

Adamas Pharmaceuticals announced the presentation of two separate analyses of pooled data from the placebo-controlled Phase III clinical trials of ADS-5102 (amantadine) extended-release capsules in oral platform and poster presentation sessions at the 69(th) American Academy of Neurology (AAN) Annual Meeting in Boston. The pooled data results, which are consistent with the original findings from EASE LID and EASE LID 3, demonstrated that people with Parkinson’s disease treated with ADS-5102 had a significant reduction in levodopa-induced dyskinesia (LID), as measured by the Unified Dyskinesia Ratings Scale (UDysRS), and showed statistically significant reduction in OFF time, as reported by Parkinson’s disease home diary data.

 

Galapagos announced the closing of its underwritten public offering of 4,312,500 ADSs, at a price of $90.00 per ADS, before underwriting discounts, for gross proceeds of €363.9 million. This includes the full exercise of the underwriters’ option to purchase additional ADSs. All of the ADSs were offered by Galapagos.

 

Bristol-Myers Squibb announced data from a Phase II study of BMS-986036, an investigational pegylated analogue of human fibroblast growth factor 21 (FGF21), a key regulator of metabolism, in patients with biopsy-confirmed nonalcoholic steatohepatitis (NASH) (F1-F3). The study achieved its primary endpoint of significant reduction in liver fat versus placebo. Statistically significant improvements were also seen in prespecified exploratory endpoints including biomarkers of fibrosis, metabolic parameters and markers of liver injury. These data were presented at a late-breaking oral presentation at EASL: The International Liver Congress in Amsterdam.

 

Merck announced the first sustained virologic response(1) (SVR) results 12 weeks after completion of therapy (SVR12, considered virologic cure) from C-SURGE, an ongoing, open label Phase II clinical trial evaluating MK-3682B [uprifosbuvir (MK-3682)(2)/grazoprevir(3)/rusazvir(4)], the company’s investigational triple-combination therapy in treatment-experienced patients with hepatitis C virus (HCV) genotype (GT) 1 infection for whom treatment with approved direct-acting antiviral regimens had failed. The study showed that 100 percent (43/43) of patients who completed 16 weeks of treatment plus ribavirin (RBV) achieved SVR12 and 100 percent (49/49) of patients who completed 24 weeks of treatment achieved SVR12 (abstract PS-159). These results were presented at The International Liver Congress 2017.

 

ContraVir Pharmaceuticals presented data demonstrating the synergistic antiviral activity from the combination of its two investigational drugs for the treatment of hepatitis B viral (HBV) infection, tenofovir exalidex (TXL, formerly CMX157) a nucleotide reverse transcriptase inhibitor and CRV431, a cyclophilin inhibitor. In addition, the mode of action (MOA) of CRV431 was further defined.

 

Achillion Pharmaceuticals announced the presentation of updated results from the ongoing Phase II ‘604 Study’ being conducted by Alios BioPharma, part of the Janssen Pharmaceutical Companies. These results were presented as an oral presentation during the European Association for the Study of the Liver (EASL) 2017 International Liver Congress in Amsterdam. These results demonstrate that the triple combination of simeprevir, odalasvir and AL-335 has the ability to shorten treatment duration, offer high efficacy and be generally well tolerated in those whose disease is caused by hepatitis C virus (HCV) genotype 1 (GT1), one of the most prevalent causes of hepatitis C globally.

 

Arbutus Biopharma presented results of the first three cohorts of a Phase II study of its RNAi agent, ARB-1467, at the European Association for the Study of the Liver (EASL) in Amsterdam, The Netherlands.

 

 

ANALYST RECOMMENDATIONS

 

Gabelli analyst Jing He downgraded Bioverativ to “hold” from “buy,” citing valuation.

 

Rodman & Renshaw analyst Raghuram Selvaraju increased his price target of Motif Bio to $25 from $16, citing Motif Bio Plc reported positive top-line data from the REVIVE-1 pivotal Phase III trial of its lead drug candidate, the next-generation dihydrofolate reductase inhibitor iclaprim.

 

CL King analyst David Westenberg increased his price target of Illumina to $220 from $175, citing ILMN has five to ten years of unobstructed growth in production-scale sequencing, and that investors underappreciate both Illumina’s ability to scale the SBS chemistry and Grail, Firefly and Helix.