BioShares Biotechnology Clinical Trials (BBC): $22.31, +$0.11, +21.2% YTD

BioShares Biotechnology Products (BBP): $36.94, -$0.25, +12.8% YTD

 

 

MARKET COMMENTARY

 

U.S. stock index futures were trading mostly higher, supported by a bounce in oil and other commodity prices. Oil prices jumped after Saudi Arabia and Russia agreed that there was a need to extend oil supply cuts until 2018, a step in-line with the OPEC-led deal to support prices for longer than originally agreed. In forex market, commodity-linked currencies such as the Canadian dollar and the Australian dollar recorded major gains. Major European markets trended lower, while Asian stocks shrugged off worries over the ‘ransomware’ cyber attack to reach a two-year high. Gold traded modestly higher.

 

 

MARKET HIGHLIGHTS

 

Minerva Neurosciences announced plans for its Phase 3 and Phase 4 clinical development of MIN-101, a drug targeting negative symptoms in schizophrenia patients.  Following a recent "end-of-Phase 2" meeting with the U.S. Food and Drug Administration (FDA), the Company`s next step is the planned initiation of a pivotal Phase 3 trial with MIN-101 in the second half of 2017.  The Phase 3 trial design will be a 12-week, double-blind, randomized, placebo-controlled, monotherapy study testing two doses of MIN-101 in patients with negative symptoms and a diagnosis of schizophrenia.  To be eligible for this study, patients will be required to have stable negative and positive symptoms over several months prior to enrollment, with a specified minimum threshold baseline score on the Positive and Negative Syndrome Scale (PANSS) negative sub-scale. After the double-blind phase, patients may enter a 36-week open label extension phase in which all patients will receive active treatment. This multi-center, international trial is expected to enroll approximately 500 patients at approximately 60 clinical sites across the U.S. and Europe.  The primary endpoint will be improvement in negative symptoms at 12 weeks as measured by the PANSS Marder negative factor score, a widely recognized instrument for quantifying severity of negative symptoms.   Secondary efficacy endpoints will include the Clinical Global Impression of Severity (CGI-S) scale and Personal and Social Performance (PSP) total score.

 

Advaxis today published online a poster previously presented at the National Cancer Research Institute (NCRI) Cancer Conference in Liverpool that showed axalimogene filolisbac achieved durable response in a patient with persistent or recurrent metastatic (squamous or non-squamous cell) carcinoma of the cervix (PRmCC).

 

Alcobra today announced that Dr. Yaron Daniely will be stepping down on May 31, 2017 as President and Chief Executive Officer to become the Chief Executive Officer of Yissum, the technology transfer company of the Hebrew University in Jerusalem, Israel. David Baker, currently Alcobra’s Chief Commercial Officer, will assume the position of interim CEO. Dr. Daniely will remain on the Board of Directors of Alcobra and will be actively involved in ensuring a seamless transition of duties and responsibilities.  The Company’s Board of Directors also announced that it has appointed Dr. Daniely as Chairman of the Board of Directors, effective June 1, 2017.

 

Eiger BioPharmaceuticals announced completion of enrollment of the Phase 2 LIBERTY study.  The LIBERTY study is designed to evaluate the effects of ubenimex added to current standard of care in patients with pulmonary arterial hypertension (PAH).  Ubenimex is a well-characterized, oral, small-molecule inhibitor of leukotriene A4 hydrolase, which blocks the production of leukotriene B4 (LTB4), an inflammatory mediator implicated in PAH disease.  A total of 61 patients were enrolled across 45 sites in the U.S. and Canada in less than one year.

 

Aurinia Pharmaceuticals released its financial results for the first quarter ended March 31, 2017.  For the first quarter ended March 31, 2017, we reported a consolidated net loss of $51.9 million or $0.92 per common share.  This compared to a consolidated net loss of $4.3 million or $0.13 per common share, which included a non-cash decrease on revaluation of derivative warrant liability of $664,000 at March 31, 2016.  Cash used in operating activities for the three months ended March 31, 2017 was $9.7 million. Cash provided by financing activities was $172.2 million comprised of net proceeds of $162.3 million from the public offering and $9.9 million from the exercise of warrants and stock options during the three month period ended March 31, 2017.

 

Galmed Pharmaceuticals reported financial results for the three months ended March 31, 2017.  Net loss of $3.2 million, or $0.26 per share, for the three months ended March 31, 2017, compared to a net loss of $4.0 million, or $0.36 per share, for the three months ended March 31, 2016.  Cash and cash equivalents and marketable securities totaled $11.7 million as of March 31, 2017, compared to $15.5 million at December 31, 2016. This decrease primarily resulted from $3.9 million used in operating activities, mainly due to our ongoing clinical studies and operational activities. Galmed continues to believe that its cash balance will be sufficient to maintain its current operations through the first half of 2018, and allow the Company to complete the ARREST study as scheduled.

 

Bellerophon Therapeutics reported financial results for the first quarter ended March 31, 2017.  Net loss for the first quarter of 2017 included a change in fair value of common stock warrant liability of $14.4 million.  There was no change in fair value of common stock warrant liability for the first quarter of 2016 as the warrants were issued in November 2016.  Net loss per share was $0.60 in first quarter of 2017 compared to $0.54 net loss per share in the prior year period.  At March 31, 2017, the Company had cash and cash equivalents, restricted cash and marketable securities of $17.1 million compared to cash and cash equivalents, restricted cash and marketable securities of $20.5 million at December 31, 2016.

 

VBL Therapeutics announced financial results for the first quarter ended March 31, 2017.  The Company reported a net loss for the quarter ended March 31, 2017 of $5.0 million, or ($0.19) per share, compared to a net loss of $4.7 million, or ($0.21) per share in the quarter ended March 31, 2016.  At March 31, 2017, the Company had cash, cash equivalents and short-term bank deposits totaling $39.6 million and working capital of $37.2 million. The Company expects that its cash, cash equivalents and short-term bank deposits will enable it to fund operating expenses and capital expenditure requirements into 2019 and is expected to be sufficient to enable it to complete its ongoing Phase 3 clinical trial of VB-111 in rGBM, to support our planned potential registration trial in ovarian cancer and an exploratory clinical study of VB-111 in combination with a checkpoint inhibitor in lung cancer, as well as to support the investment in the new Modiin facility to which the Company intend to relocate in a few months.

 

BrainStorm Cell Therapeutics announced financial results for the first quarter ending March 31, 2017.  Net loss for the three months ended on March 31, 2017 was $1.8 million, or ($0.10) per share, as compared to a net loss of $1.8 million or ($0.10) per share for the three months ended March 31, 2016.  At March 31, 2017, Brainstorm had net working capital of $7.9 million including cash, cash equivalents and short-term bank deposits amounting to $8.3 million.  This compares to cash, cash equivalents, and short-term deposits of approximately $10.0 million at December 31, 2016.

 

Aradigm Corporation announced financial results for the first quarter and three months ended March 31, 2017.  Net loss for the first quarter of 2017 was $3.7 million or $0.25 per share, compared with a net loss of $8.1 million or $0.55 per share in the first quarter of 2016.  As of March 31, 2017, the Company reported cash and cash equivalents of $17.2 million.

 

Ignyta announced that the U.S. Food and Drug Administration (FDA) has granted a Breakthrough Therapy Designation (BTD) to entrectinib “for the treatment of NTRK fusion-positive, locally advanced or metastatic solid tumors in adult and pediatric patients who have either progressed following prior therapies or who have no acceptable standard therapies.” Entrectinib is the company’s investigational, orally available, CNS-active tyrosine kinase inhibitor targeting tumors that harbor NTRK1/2/3, ROS1, or ALK gene fusions.  The FDA’s Breakthrough Therapy Designation is intended to expedite development and review timelines of potential new medicines for use in the treatment of a serious or life-threatening condition when preliminary clinical evidence indicates the drug may demonstrate substantial improvement over available therapies on one or more clinically significant endpoints.

 

Innate Pharma SA announced its revenues and cash position for the first three months of 2017.  Revenues for the first three months of 2017 amounted to €7.3 million (€5.7 million for the same period in 2016).  Cash, cash equivalents and financial assets of the Company amounted to €223.8 million as of March 31, 2017.

 

Onconova Therapeutics reported financial results for the first quarter ended March 31, 2017.  The first quarter net loss was $8.3 million, compared to a net loss of $7.2 million in the first quarter of 2016.  Cash and cash equivalents as of March 31, 2017, totaled $15.4 million, compared to $21.4 million as of December 31, 2016.  This excludes the proceeds from the financing completed in April 2017, in which the Company raised approximately $5.2 million before underwriting discounts and commissions and estimated offering costs in a public offering of  common stock through Laidlaw & Company (UK) Ltd. This also excludes the proceeds from the exercise of the underwriter’s over-allotment option which is expected to raise an additional $0.8 million before deducting underwriting discounts and commissions and estimated offering costs. Onconova believes that its current cash and cash equivalents will be sufficient to fund its ongoing trials and operations to the end of 2017.

 

Lysogene announced that Karen Aiach, CEO and Founder of Lysogene, won the Women’s Entrepreneurship’s Innovation & New Technologies Trophy – co-organized by the Women’s Entrepreneurship Association and the CPME – rewarding her “spectacular breakthrough in the exclusive arena of rare diseases.”

 

ADMA Biologics announced its financial results for the quarter ended March 31, 2017.  The consolidated net loss for the first quarter ended March 31, 2017 was approximately $6.5 million, or ($0.51) per share, as compared to a consolidated net loss of approximately $4.6 million, or ($0.43) per share for the first quarter ended March 31, 2016.  At March 31, 2017, ADMA had cash, cash equivalents and short-term investments of approximately $8.8 million, as compared to approximately $15.3 million at December 31, 2016.

 

Ionis Pharmaceuticals announced that the Phase III NEURO-TTR study of inotersen (IONIS-TTR(Rx)) in patients with familial amyloid polyneuropathy (FAP) met both primary endpoints. Over the 15-month period of the study, inotersen-treated patients achieved statistically significant benefit compared to placebo in the modified Neuropathy Impairment Score +7 (mNIS+7) and the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN) (p <0.0001 and p=0.0006, respectively). Statistically significant differences were also observed for both endpoints at eight months.

 

Alexion Pharmaceuticals announced that researchers presented data showing that the rapid benefits of Strensiq (asfotase alfa) achieved in adolescents and adults (ages 13-66 years at study entry) with hypophosphatasia (HPP) within the first 6 months were sustained through 5 years of treatment. These are the final data from the extension phase of a randomized, open-label, dose-ranging Phase II trial of Strensiq and they confirm previously presented interim results.

 

Biogen announced that it has completed an asset purchase of Remedy Pharmaceuticals’ Phase III candidate, CIRARA (intravenous glyburide). The target indication for CIRARA is large hemispheric infarction (LHI), a severe form of ischemic stroke where brain swelling (cerebral edema) often leads to a disproportionately large share of stroke-related morbidity and mortality. The FDA recently granted CIRARA Orphan Drug Designation for severe cerebral edema in patients with acute ischemic stroke. The FDA has also granted CIRARA Fast Track designation.

 

Cytokinetics announced that the Office of Orphan Products Development of the FDA has granted orphan drug designation to CK-2127107 for the potential treatment of spinal muscular atrophy (SMA). In collaboration with Astellas Pharma, Cytokinetics is developing CK-2127107, a next-generation fast skeletal muscle troponin activator (FSTA), as a potential treatment for people living with SMA, chronic obstructive pulmonary disease (COPD) and certain other debilitating diseases and conditions associated with skeletal muscle weakness and/or fatigue.

 

Otsuka Pharmaceutical and Teva Pharmaceutical Industries announce an agreement covering Japan for Otsuka to develop and commercialize Teva’s investigational drug candidate fremanezumab (TEV-48125), an anti-calcitonin gene-related peptide (CGRP) monoclonal antibody for the prevention of migraine. Fremanezumab is administered monthly as a subcutaneous injection. Through the agreement, Otsuka secures exclusive rights in Japan to fremanezumab, which Teva is globally developing in other countries.

 

Biohaven Pharmaceutical Holding announced that the FDA has granted the company Fast Track Designation for Biohaven’s product candidate trigriluzole (BHV-4157) for the potential treatment of Spinocerebellar Ataxia (SCA). Trigriluzole previously received Orphan Drug Designation from the FDA for the treatment of SCA in 2016. Biohaven is currently conducting a Phase II/III clinical trial in patients with SCA, with topline results expected in 2018.

 

Cellular Biomedicine Group announced the addition of a new independent Phase I clinical trial of the Company’s ongoing CARD-1 study in patients with chemorefractory and aggressive DLBCL. The Company and Shanghai Tongji Hospital (Tongji) are conducting a single arm, non-randomized study to evaluate the safety and efficacy of C-CAR011 (Anti-CD19 single-chain variable fragment (scFv) (41BB-CD3ζ)) therapy in relapsed or refractory B cell Non-Hodgkin Lymphoma (NHL). The trial will enroll 15 patients comprised of DLBCL, Primary Mediastinal Large B-Cell Lymphoma (PMBCL) and Follicular Lymphoma (FL).

 

Aerie Pharmaceuticals reported that it has received notification from the FDA that the FDA has completed its initial 60–day review of the Rhopressa NDA and determined that the application is sufficiently complete to permit a substantive review. The PDUFA goal date for the completion of the FDA’s review of the Rhopressa NDA is set for February 28, 2018. This date reflects a standard 12-month review period and is consistent with management’s expectations. The notification also indicated that the FDA has not identified any potential review issues, and that the FDA is currently planning to hold an advisory committee.

 

Selecta Biosciences announced new preclinical data regarding non-immunogenic gene therapies that were presented at the American Society of Gene & Cell Therapy (ASGCT) 2017 Annual Meeting, which took place last week in Washington, D.C.

 

Spring Bank Pharmaceuticals announced that the Data Safety Monitoring Board (DSMB), an independent group of medical experts closely monitoring Spring Bank’s ACHIEVE global Phase IIa clinical trial evaluating SB 9200 in treatment-naïve chronic Hepatitis B virus (HBV) patients without cirrhosis, has reviewed the initial safety and tolerability data from the 25mg monotherapy dose cohort (n=20) in the Phase IIa clinical trial of SB 9200. Based on their assessment of the safety data, the DSMB approved proceeding with the enrollment for the second cohort of the Phase IIa segment of the ACHIEVE trial with a dose escalation to 50mg once a day. Spring Bank also announced that study investigators have already begun screening patients for the second cohort of the ACHIEVE trial, which will also enroll 20 patients.

 

Syros Pharmaceuticals announced that the first patient has been dosed in the Phase I clinical trial of SY-1365, its first-in-class selective cyclin-dependent kinase 7 (CDK7) inhibitor, in patients with advanced solid tumors, including transcriptionally dependent cancers such as triple negative breast, small cell lung and ovarian cancers.

 

Aytu BioScience announced that it presented two posters demonstrating new clinical findings that further validate and expand the potential utility of its MiOXSYS System as an advanced tool for assessing oxidative stress in human semen, which is broadly implicated as a major cause of male infertility.

 

Eagle Pharmaceuticals announced that Pete A. Meyers has joined the Company as Chief Financial Officer, effective today. Mr. Meyers will also serve as the Company’s principal financial officer and principal accounting officer, effective today.

 

Summit Therapeutics announced that it has completed enrolment in PhaseOut DMD, a Phase II proof of concept clinical trial of the utrophin modulator, ezutromid, in patients with DMD. PhaseOut DMD aims to provide proof of concept for ezutromid through measures of a number of endpoints related to muscle structure, health and function. The Company believes the trial could provide valuable insight into utrophin modulation as a potential disease-modifying treatment for all patients with DMD, regardless of the underlying dystrophin mutation.

 

Insmed announced the appointment of Paolo Tombesi as Chief Financial Officer, effective June 1, 2017, and Paul Streck, M.D., as Chief Medical Officer, effective June 5, 2017. Additionally, Eugene Sullivan, M.D., has been appointed to the newly created role of Chief Product Strategy Officer.

 

Amicus Therapeutics announced positive functional data from initial patients in a global Phase I/II study to investigate ATB200/AT2221 in patients with Pompe disease. Patients who completed six months of treatment with ATB200/AT2221 showed improvements in the six-minute walk test (6MWT) distance and other measures of motor function, in addition to stability or improvements in forced vital capacity (FVC). Consistent with previous results presented at the 2017 WORLDSymposium, patients treated with ATB200/AT2221 continue to show improvements in biomarkers of muscle damage and disease substrate.

 

Novocure announced today that FDA has designated it’s Tumor Treating Fields (TTFields) delivery system as a Humanitarian Use Device (HUD) for the treatment of pleural mesothelioma.

 

Nuvo Pharmaceuticals announced that the United States District Court for the District of New Jersey has upheld the validity of Horizon Pharma plc’s U.S. patent covering Pennsaid(diclofenac sodium topical solution) 2% w/w (Pennsaid 2%), which Actavis Laboratories UT, Inc. (Actavis) has admitted that its proposed generic version of Pennsaid 2% would infringe.  Nuvo earns revenue by selling commercial bottles and physician samples of Pennsaid 2% to Horizon under an exclusive manufacturing and supply agreement that extends to 2029.

 

Radius Health announced that Jose (Pepe) Carmona has been appointed Chief Financial Officer of the Company.

 

Cardiome Pharma announced that it has amended its term loan agreement with CRG-managed funds that was originally entered into on June 13, 2016. The Amended Agreement provides Cardiome with up to $50 million of available borrowing capacity.

 

 

ANALYST RECOMMENDATIONS

 

Cantor analyst Mara Goldstein initiated coverage of Karyopharm Therapeutics with an “overweight” rating and $18 price target, citing the clinical program is large, which provides for multiple opportunities and the company has cash runway into 2019.

 

Wells Fargo analyst Jim Birchenough decreased his price target of Avexis to $89 from $102, citing new AVXS101 gene therapy manufacturing capacity estimates, following recent discussions with contract manufacturing organization (CMO) representatives.

 

BTIG analyst upgraded Foudation Medicine to “buy” from “neutral,” citing Expiration of Roche standstill provisions, strength in test volumes and acceleration in clinical sales growth among the rationale.

 

RBC analyst Randall Stanicky initiated coverage of Jazz Pharmaceuticals with an “outperform” rating and $210 price target, citing a de-risked story that is poised to accelerate growth on long-duration, highly visible cash flows.

 

Leerink analyst Puneet Souda increased his price target of Exact Sciences to $41 from $38, citing the higher penetration and subsequent estimates.

 

Following Ardelyx’s announcement of top-line results of the T3MPO-1 trial in patients with constipation associated with IBS-C, Ladenburg Thalmann analyst Matthew Kaplan decreased his price target to $18.50 from $21; BTIG analyst Timothy Chiang decreased his price target to $12 from $18; Citi analyst Yigal Nochomovitz decreased his price target to $12 from $17.

 

Following Teva’s earnings, Goldman analyst Jami Rubin decreased her price target to $34 from $35. 

 

HC Wainwright analyst Shaunak Deepak decreased his price target of OncoMed to $6 from $9, citing OncoMed announced that it was discontinuing dosing of all demcizumab trials, following the disappointing top-line results from the Phase 2 DENALI trial.

 

Evercore ISI analyst Ross Muken upgraded PAREXEL to “outperform” from “in line” and increased his price target to $85 from $73, citing recent news suggesting the company is contemplating strategic actions.