BioShares Biotechnology Clinical Trials (BBC): $19.84, -$0.89, -31.7% YTD

BioShares Biotechnology Products (BBP): $34.03, -$0.65, +10.4% YTD

 

 

MARKET COMMENTARY

 

After Wall Street ended with big gains for November, U.S. stock index futures edged lower, ahead of a flurry of key economic data. Auto sales, Challenger layoffs, weekly jobless claims, construction spending and manufacturing PMI from Markit and Institute for Supply Management are on the economic calendar. Most European stocks dipped into negative territory, shrugging off the gains in Asian shares. The dollar backed away from a 9-1/2-month high against the yen. Oil prices extended gains, after an OPEC-led surge. Gold fell to its lowest since February, adding to losses after its biggest monthly decline in more than three years.

 

 

MARKET HIGHLIGHTS

 

Aradigm Corporation announced top-line results from its two Phase 3 clinical trials (ORBIT-3 and ORBIT-4) evaluating the safety and efficacy of Pulmaquin®, the Company’s investigational proprietary formulation of once daily ciprofloxacin for inhalation, in patients with non-cystic fibrosis bronchiectasis (“non-CF BE”) with chronic lung infections with Pseudomonas aeruginosa (P. aeruginosa).  The ORBIT-3 and ORBIT-4 pivotal trials were identical in design except for a pharmacokinetics sub-study that was conducted in one of the trials. The primary endpoint in both ORBIT-3 and ORBIT-4 was an increase in the median time to first mild, moderate or severe pulmonary exacerbation (“PE”). The key secondary efficacy endpoint in both trials was the frequency of PE’s over the 48-week double-blind treatment period.  In ORBIT-4 the median time to first mild, moderate or severe PE was 230 days in the Pulmaquin treatment group as compared to 163 days in the placebo group. This increase in the median time to first PE was statistically significant (p=0.0462) using non-stratified log-rank analysis.  In ORBIT-3 the median time to first mild, moderate or severe PE was 221 days in the Pulmaquin treatment group as compared to 136 days in the placebo group. This increase in the median time to first PE was similar to ORBIT-4 but was not statistically significant (p=0.8488) using non-stratified log-rank analysis.

 

Ignyta announced data from an ongoing Phase 1/1b study of RXDX-105 – Ignyta’s VEGFR-sparing, potent RET inhibitor – at the 2016 EORTC-NCI-AACR (ENA) Molecular Targets and Cancer Therapeutics Symposium in Munich, Germany, highlighting RXDX-105’s clinical activity in patients harboring RET molecular alterations, with five out of nine patients with RET fusion-positive cancers who were RET inhibitor-naïve achieving a RECIST response (1 complete response, 3 partial responses, and 1 unconfirmed partial response), for a preliminary objective response rate (ORR) of 56% (Abstract number 437, Poster number P116).

 

AzurRx BioPharma will be presenting at the 9th annual LD Micro Main Event on Tuesday, December 6th at 12:30PM PST.

 

Valneva SE announced that MVM Life Science Partners LLP (`MVM`), a specialist healthcare investor based in London and Boston with more than $700 million under management, has acquired a 7.5% shareholding in the Company. MVM will be entitled to an investor director representative, to be appointed to Valneva`s Supervisory Board by the next annual shareholders meeting.

 

Windtree Therapeutics announced that two abstracts have been accepted for presentation at the Hot Topics in Neonatology conference being held December 5 – 7, 2016 in Washington, DC.  For over 30 years, Hot Topics has been a premiere neonatal conference, with more than 1000 neonatologists and perinatologists from around the world attending each year.

 

Palatin Technologies announced the pricing of an underwritten public offering of 25,384,616 shares of its common stock and warrants to purchase 12,692,310 shares of its common stock for anticipated gross proceeds of $16.5 million, before deducting underwriting discounts and commissions and estimated offering expenses payable by Palatin.  The shares of common stock and warrants will be offered as Series A Units, with each Series A Unit consisting of one share of common stock and a Series J warrant to purchase 0.50 of a share of common stock, and will be priced at $0.65 per unit.

 

Aimmune Therapeutics announced the membership of its Scientific Advisory Board, comprised of internationally recognized food allergy experts.  The Scientific Advisory Board members and their academic affiliations are: Dr. Kirsten Beyer, University Hospital Charité, Berlin, Germany; Dr. A. Wesley Burks, University of North Carolina, Chapel Hill; Dr. Thomas Casale, University of South Florida, Tampa; Dr. Jonathan Hourihane, University College Cork, Ireland; and, Dr. Stacie Jones, University of Arkansas for Medical Sciences, Little Rock.

 

Heat Biologics announced that it presented topline data from its 94-patient Phase II trial evaluating HS-410 (vesigenurtacel-L) in combination with standard of care, Bacillus Calmette-Guérin (BCG), or as a monotherapy, for the treatment of non-muscle invasive bladder cancer, at the Society of Urologic Oncology Annual Meeting in San Antonio, TX.

 

Reuters reported that most patients who previously were not helped by repeated treatments for multiple myeloma showed strong benefits from a new type of therapy in a small study, and with no worrisome side effects, drugmaker Bluebird Bio said. The Phase I study enrolled patients who had basically run out of other options for the blood cancer, after failing on average six previous rounds of treatment, including with prior stem cell transplants. Data from nine patients was evaluated for safety and efficacy and slated for presentation at a medical meeting in Munich. Among three patients given the lowest dose of the experimental treatment, one showed at least a 50 percent reduction in signs of the disease, including of a protein considered a hallmark of the cancer. All three patients receiving a medium dose of the treatment, and all three receiving a high dose showed at least a 50 percent reduction in signs of cancer. Among those six patients, two had no minimum residual disease (MRD), meaning they had no remaining detectable trace of the cancer as evaluated by the most highly sensitive available diagnostic. The treatment, called bb2121 and being developed in partnership with Celgene Corp, targets a protein called BCMA that is found on cancerous blood plasma cells. Normal plasma cells are found in the bone marrow and are an important part of the immune system.

 

Cellectar Biosciences provided a data update on the first two cohorts of the company’s Phase I clinical study of CLR 131 in patients with relapsed or refractory multiple myeloma.

 

Biostage announced that its Cellspan Esophageal Implant was granted Orphan Drug Designation by the FDA to restore the structure and function of the esophagus subsequent to esophageal damage due to cancer, injury or congenital abnormalities.

 

Amgen and Cytokinetics announced The Lancet published results from a Phase II clinical trial evaluating omecamtiv mecarbil in patients with chronic heart failure. The COSMIC-HF (Chronic Oral Study of Myosin Activation to Increase Contractility in Heart Failure) trial met its primary pharmacokinetic objective and showed statistically significant improvements in all pre-specified secondary measures of cardiac function in the treatment group receiving pharmacokinetic-based dose titration. The results were initially presented as a Late-Breaking Clinical Trial at the AHA Scientific Sessions 2015.

 

Cytokinetics announced the activation of the first clinical site and the start of GALACTIC-HF (Global Approach to Lowering Adverse Cardiac Outcomes Through Improving Contractility in Heart Failure), the Phase III cardiovascular outcomes clinical trial of omecamtiv mecarbil which is being conducted by Amgen, in collaboration with Cytokinetics. Coincident with the start of the trial, Amgen will make a $26.7 million milestone payment to Cytokinetics. In clinical studies, omecamtiv mecarbil, a novel investigational cardiac myosin activator, has been shown to enhance cardiac function by increasing cardiac contractility and is being developed for the potential treatment of patients with chronic heart failure.

 

Merck announced that the FDA has accepted for review the sBLA for KEYTRUDA (pembrolizumab), the company’s anti-PD-1 therapy, for the treatment of patients with refractory classical Hodgkin lymphoma (cHL) or for patients who have relapsed after three or more prior lines of therapy. The FDA granted Priority Review with a PDUFA, or target action, date of March 15, 2017. The sBLA will be reviewed under the FDA’s Accelerated Approval program. In April 2016, KEYTRUDA was granted Breakthrough Therapy Designation by the FDA for this indication.

 

Pfizer announced positive top-line results of a study that evaluated the use of LYRICA (pregabalin) Capsules CV and Oral Solution CV as adjunctive therapy for pediatric epilepsy patients four to 16 years of age with partial onset seizures. Results showed that adjunctive treatment with LYRICA 10 mg/kg/day resulted in a statistically significant reduction in seizure frequency versus placebo, the primary efficacy endpoint. Treatment with LYRICA 2.5 mg/kg/day resulted in a numerical reduction in seizure frequency, which was not statistically significant. LYRICA is not approved as adjunctive therapy for pediatric epilepsy patients with partial onset seizures.

 

Daiichi Sankyo Company, Limited announced that the FDA has granted Fast Track designation to its investigational HER2-targeting antibody drug conjugate DS-8201 for the treatment of HER2-positive unresectable and/or metastatic breast cancer in patients who have progressed after prior treatment with HER2-targeted therapies including ado-trastuzumab emtansine (T-DM1).

 

Cyclacel Pharmaceuticals announced the presentation of preclinical data demonstrating the therapeutic potential of the Company’s novel polo-like kinase (PLK) 1 inhibitor, CYC140, as a targeted anti-cancer agent. The data demonstrates that CYC140 is a selective PLK1 inhibitor which preferentially induces growth inhibition and cell death in malignant versus non-malignant cells. The data were presented at the 28(th) EORTC-NCI-AACR Molecular Targets and Cancer Therapeutics Symposium in Munich, Germany.

 

Oncodesign announced the effective transfer to Oncodesign of François Hyafil Research Centre (FHRC) from GSK and the appointment of two new activity directors. In order to accelerate its in-house programs of therapeutic molecules and expand its service offering, Oncodesign signed in September 2016 an agreement for the acquisition of François Hyafil Research Centre from GSK in Les Ulis.

 

TNK Therapeutics, a subsidiary of Sorrento Therapeutics, provided an update on its lead chimeric antigen receptor (CAR) expressing T cells (CAR-T) programs for the treatment of hematological malignancies. Adoptive immunotherapy utilizing CAR-T cells represents a promising new paradigm in the treatment of cancer. Sorrento intends to present these findings at upcoming clinical conferences and in scientific publications.

 

Kura Oncology presented preclinical data highlighting the identification and characterization of KO-947, its development candidate targeting ERK1/2 kinases. The company has also presented preclinical data relating to the identification and optimization of potent and selective inhibitors of the menin-MLL interaction. Both presentations took place at the EORTC-NCI-AACR Symposium on Molecular Targets and Cancer Therapeutics (EORTC) in Munich, Germany.

 

DiaMedica announced the sale and transfer of its DM-71 product and related intellectual property. The sale of this non-core asset provides DiaMedica with a total of $300,000 in upfront payments, of which $50,000 had been previously received, and a royalty stream linked to future sales.

 

Aequus Pharmaceuticals announced that it has successfully obtained provincial formulary coverage from Alberta Health and Manitoba Health for Vistitan (bimatoprost 0.03% w/v, ophthalmic solution). Alberta Health have additionally listed Vistitan as interchangeable to Lumigan RC for the purposes of its formulary coverage, giving Aequus rapid access to an existing market in Alberta.

 

Agile Therapeutics announced that it has received net proceeds of approximately $3 million in non-dilutive financing through the State of New Jersey’s Technology Business Tax Certificate Transfer Program. The Program enables biotechnology companies to raise funds to finance their growth and operations and is administered by the New Jersey Economic Development Authority (NJEDA) and the New Jersey Department of the Treasury’s Division of Taxation.

 

Roche announced an expansion of their distribution alliance with the life science business of Merck KGaA to now include the qPCR and endpoint PCR portfolios from Kapa Biosystems. Merck KGaA, Darmstadt, Germany operates as MilliporeSigma in the US and Canada. Kapa Biosystems was previously acquired by Roche in December of 2015.

 

Bicycle Therapeutics announced that it has entered into a collaboration with AstraZeneca for the identification and development of bicyclic peptides (Bicycles) for the treatment of respiratory, cardiovascular and metabolic diseases. Under the terms of the agreement, Bicycle is responsible for identifying Bicycles for an undisclosed number of targets specified by AstraZeneca while AstraZeneca is responsible for further development and product commercialization. If all planned programs reach the market, Bicycle will be eligible for over $1 billion in payments, including an upfront payment, future R&D funding, development, regulatory and commercialization milestone payments. Bicycle would also be entitled to receive royalties on sales of products resulting from the collaboration.

 

IBM Watson Health and Pfizer announced a collaboration that will utilize IBM Watson for Drug Discovery to help accelerate Pfizer’s research in immuno-oncology, an approach to cancer treatment that uses the body’s immune system to help fight cancer.

 

ACADIA Pharmaceuticals announced the initiation of CLARITY, a Phase II study to evaluate pimavanserin for adjunctive treatment in patients with major depressive disorder (MDD) who have an inadequate response to first-line therapies for clinical depression. Pimavanserin is a selective serotonin inverse agonist (SSIA) preferentially targeting 5-HT(2A) receptors that may play a role in depression.

 

Inovio Pharmaceuticals announced that it has been awarded a $6.1 million sub-grant through The Wistar Institute to develop a DNA-based monoclonal antibody designed to provide a fast-acting treatment against Zika infection and its debilitating effects. The goal of this program, which is funded by a grant to The Wistar Institute from the Bill & Melinda Gates Foundation, is for the researchers to develop a Zika dMAb therapy ready for human clinical trials in less than two years.

 

PharmaMar announced that its antibody-drug conjugate (ADC) has demonstrated a remarkable in vitro antitumoral activity in a CD13, fibrosarcoma positive animal model. CD13 is an aminoectopeptidase, involved in the modulation of several vasoactive peptides and is known to influence major biological events, such as cell proliferation, invasion and angiogenesis. MI130110 is formed by a compound of marine origin (PM050489) that has been conjugated to a monoclonal anti-CD13 antibody through a non-hydrolysable linker.

 

Mymetics announced that its subsidiary Mymetics B.V. has agreed on a research project with Sanofi Pasteur. The project will investigate the immunogenicity of influenza vaccines based on Mymetics’ proprietary virosome technology platform in pre-clinical settings. If this project is successful it could result in a further and more extensive collaboration between the two companies.

 

Innovus Pharmaceuticals announced the launch of UriVarx in the U.S. UriVarx is clinically proven to reduce urinary urgency, accidents and both day and night frequency in Overactive Bladder (“OAB”) and Urinary Incontinence (“UI”) patients.

 

Xencor announced the pricing of an underwritten public offering of 4,585,000 shares of its common stock, offered at a price to the public of $24.00 per share. The gross proceeds from this offering to Xencor are expected to be approximately $110 million, before deducting underwriting discounts and commissions and offering expenses payable by Xencor. The offering is expected to close on or about December 6, 2016, subject to customary closing conditions. In addition, Xencor has granted the underwriters a 30-day option to purchase up to an aggregate of 687,750 additional shares of common stock. All of the shares are being offered by Xencor. Xencor expects to use net proceeds of this offering for working capital and general corporate purposes, including additional investments in advancing its lead compound XmAb5871 through later stages of clinical development and additional investment in its immune-oncology pipeline and technology. Leerink Partners is acting as sole book-running manager in the offering.  Canaccord Genuity and Wedbush PacGrow are acting as co-managers in the offering.

 

MyoKardia announced that Marc Semigran, M.D., has joined the Company as chief medical officer.

 

Pieris Pharmaceuticals announced that it has appointed Claude Knopf as Senior Vice President and Chief Business Officer.

 

Sangamo BioSciences announced key leadership appointments to strengthen the company’s clinical and commercial capabilities. The company has appointed Edward R. Conner, M.D., as senior vice president and chief medical officer, effective immediately. Curt Herberts has been promoted to the newly created role of senior vice president and chief business officer.

 

GenVec announced that the Company’s previously declared one-for-ten reverse stock split of its outstanding shares of common stock became effective today prior to the opening of trading on the NASDAQ Capital Market. The Reverse Stock Split was previously authorized at the Company’s 2016 Annual Meeting of Shareholders and the Company’s Board of Directors approved a ratio within the approved range on November 11, 2016. The Reverse Stock Split is intended to enable GenVec to regain compliance with the $1.00 minimum bid price required for continued listing on NASDAQ Capital Market.

 

Apricus Biosciences announced that on November 30, 2016, the Company received notice from the Nasdaq Listing Qualifications Staff that due to the Company’s non-compliance with the $35 million market value of listed securities ("MVLS") requirement, the Company was subject to delisting unless it timely requests a hearing before the Nasdaq Hearings Panel. The Company intends to timely request a hearing before the Panel, which request will stay any delisting action by the Staff, pending the Panel’s decision. At the hearing, the Company will present its plan to regain compliance with the MVLS requirement within the 180-day discretionary period available to the Panel, ending May 29, 2017. In accordance with the Nasdaq Listing Rules, the Company was previously granted a 180-calendar day period within which to regain compliance with the MVLS requirement, through November 29, 2016. In order to demonstrate compliance with the MVLS requirement, the Company’s MVLS must close at $35 million or more for a minimum of 10 consecutive business days.

 

 

ANALYST RECOMMENDATIONS

 

Raymond James analyst Reni Benjamin initiated coverage of Bellicum with an “outperform” rating and $29 price target, citing the company’s most advanced product candidate, BPX-501, appears to have improved clinical outcomes for patients undergoing conventional haploidentical hematopoietic stem cell transplantation, including better rates of engraftment, lower rates of transplantation-related mortality and relapses, and controllable graft versus host disease.

 

Goldman analyst Stephen Stewart initiated coverage of the following companies: Catalent with a “neutral” rating and $25 price target; Pacira Pharmaceuticals with a “neutral” rating and $36 price target; Akorn with a “sell” rating and $20 price target.

 

Roth analyst Sa’ar Yaniv initiated coverage of Inotek with a “buy” rating and $10 price target, citing trabodenoson has the potential to become the go-to adjunctive therapy for glaucoma patients who are uncontrolled with or cannot tolerate prostaglandins, potentially capturing a significant portion of second-line prescriptions.

 

Morgan Stanley analyst David Risinger decreased his price target of Mallinckrodt to $65 from $82, citing generics competition has intensified and will pressure margins in 2017.

 

Deutsche Bank analyst Andrew Peters increased his price target of Exelixis to $20 from $17, citing opportunities beyond RCC are under-appreciated, and expect clarity on the direction forward for Cabo over the next several months as likely.

 

JMP analyst Liisa Bayko increased her price target of Ultragenyx to $90 from $84, citing reduction in major clinical events shown in 78-week data completes the data package to support a Phase III trial in 2017.