BioShares Biotechnology Clinical Trials (BBC): $20.81, +$0.31, -28.4% YTD

BioShares Biotechnology Products (BBP): $31.80, +$0.85, +3.2% YTD

 

MARKET COMMENTARY

U.S. stock index futures dipped despite comments from dovish Federal Reserve Governor Lael Brainard, which cautioned against removing support for the U.S. economy too quickly. The Treasury department is expected to release the Federal budget for August. European markets held steady ahead of a speech from European Central Bank Chief Mario Draghi in Italy. Asian stocks closed mostly lower despite relatively upbeat Chinese industrial output data. The yen’s safe-haven appeal held it steady and the dollar edged up. Oil plunged after the International Energy Agency said the crude market will be oversupplied at least through the first six months of 2017. Gold edged up.

 

MARKET HIGHLIGHTS

DelMar Pharmaceuticals announced that the Company and its collaborators from the University of Texas MD Anderson Cancer Center presented new data in a research poster entitled “Activity of dianhydrogalactitol (VAL-083) in ovarian tumor models, sensitive or resistant to cisplatin” at the 11th Biennial Ovarian Cancer Research Symposium. The data support a distinct mechanism of action for VAL-083 versus platinum-based chemotherapy currently used in the treatment of ovarian cancer.

 

Cellect Biotechnology announced that the Company has received the Israeli Ministry of Health’s approval to begin a Phase I/II clinical trial in leukemia patients. The trial, which Cellect expects will be initiated in a few weeks, is planned to be conducted in the bone marrow transplantation (“BMT”) unit of Rambam Hospital, and led by Clinical Assistant Professor, Zila Zuckerman, the Director of the unit.  This trial is the first of its kind in leukemia patients in need of BMT, and will evaluate Cellect’s cell selection technology, “Powered by Cellect,” with the aim of assessing the technology’s safety and efficacy.

 

Can-Fite BioPharma announced that it has received a Notice of Allowance from the European Patent Office indicating the patent titled, “Pharmaceutical Composition Comprising A3 Adenosine Receptor Agonist (IB-MECA/CF-101) for Treatment of Psoriasis” will be granted. This key patent addresses Can-Fite’s lead drug candidate Piclidenoson (CF101), an Adenosine Receptor Agonist (A3AR), in the treatment of psoriasis.

 

Santhera Pharmaceuticals announced that additional data from the pivotal phase III trial (DELOS) demonstrating efficacy of Raxone (idebenone) on inspiratory function in patients with DMD were published online in the medical journal Pediatric Pulmonology (http://dx.doi.org/10.1002/ppul.23547).

 

Basilea Pharmaceutica announced that Basilea Pharmaceutica International has entered into a supply, distribution and license agreement with Grupo Biotoscana S.L., for Basilea’s antifungal CRESEMBA (isavuconazole) and antibiotic Zevtera (ceftobiprole) in 19 countries in Latin America including Brazil, Mexico, Argentina and Colombia. Under the terms of the agreement, GBT holds an exclusive license to commercialize isavuconazole and ceftobiprole in key Latin American countries. In total, Basilea will receive upfront payments of CHF 11 million for both drugs and will be eligible to receive payments upon achievement of regulatory and commercial milestones. GBT will be responsible for marketing authorization applications, market access, commercialization and distribution of isavuconazole and ceftobiprole in the countries covered by the agreement.

 

Sanofi announced that adults with Type II diabetes treated with Toujeo (insulin glargine 300 Units/mL) experienced a consistently lower rate of confirmed or severe hypoglycemia both at night and at any time of the day compared with those treated with Lantus (insulin glargine 100 Units/mL), at all levels of HbA(1c) (average blood glucose over the previous three months) achieved at month 6. The results of this new patient-level meta-analysis from the EDITION 1, 2 and 3 Phase III clinical trials in patients with Type II diabetes were presented at the 52(nd) Annual Meeting of the European Association for the Study of Diabetes (EASD) in Munich, Germany.

 

Sanofi announced that iGlarLixi, the investigational, titratable fixed-ratio combination of insulin glargine 100 Units/mL and GLP-1 receptor agonist lixisenatide, offers greater control of mealtime blood sugar (post-prandial glucose, PPG) in adults living with Type II diabetes compared to insulin glargine 100 Units/mL alone. A new post-hoc analysis of data from the LixiLan-L pivotal Phase III clinical trial found more patients who received the fixed-ratio combination reached their daily PPG target than those who received only insulin glargine 100 Units/mL. The new analysis was presented at the European Association for the Study of Diabetes (EASD) 52nd Annual Meeting in Munich, Germany. iGlarLixi is currently under review in the United States and Europe.

 

Novo Nordisk presented data showing the odds of reaching fasting plasma glucose (FPG) targets without hypoglycaemia and weight gain were significantly greater for Xultophy (IDegLira) compared to up-titration with insulin glargine U100 in adults with Type II diabetes uncontrolled on insulin glargine U100 (20-50 units). Xultophy is the first once-daily combination of a long-acting insulin (insulin degludec) and a glucagon-like peptide-1 (GLP-1) receptor agonist (liraglutide) in Europe. Results were presented at the 52nd Annual Meeting of the European Association for the Study of Diabetes (EASD) 2016.

 

Novo Nordisk announced that semaglutide, an investigational glucagon-like peptide-1 (GLP-1) analogue administered once-weekly, significantly improved glycaemic control compared to placebo, as add-on to basal insulin alone or in combination with metformin, in adults with a mean Type II diabetes duration of 13 years. Results from SUSTAIN 5 were presented at the 52nd Annual Meeting of the European Association for the Study of Diabetes (EASD) 2016. The 30-week trial showed that, from a mean baseline HbA1c of 8.4%, adults treated with 0.5 mg and 1.0 mg semaglutide achieved statistically significant and superior HbA1c reductions of 1.4% and 1.8%, respectively, vs 0.1% reduction with placebo. In addition, more adults treated with 0.5 mg and 1.0 mg semaglutide achieved HbA1c targets compared with placebo: HbA1c <7% (61% and 79% vs 11%) and ≤6.5% (41% and 61% vs 5%).

 

REGENXBIO provided an update on the recent publication of data from ongoing preclinical studies of NAV Gene Therapy for the treatment of Mucopolysaccharidosis Type I (MPS I) and Mucopolysaccharidosis Type II (MPS II).

 

Flex Pharma announced that it has initiated a Phase II efficacy study in amyotrophic lateral sclerosis (ALS) patients in Australia with FLX-787, its chemically synthesized, single molecule, transient receptor potential (TRP) ion channel activator, formulated as an oral tablet. The randomized, controlled, blinded, cross-over study is designed to evaluate the safety and efficacy of FLX-787 in patients who suffer from cramps and/or spasticity as a consequence of ALS.

 

Exemplar Genetics, a wholly owned subsidiary of Intrexon, announced two publications in the current issue of the Journal of Clinical Investigation (JCI) demonstrating the potential of ExeGen CFTR miniswine research models to help define and develop effective gene therapies for cystic fibrosis.

 

Kezar Life Sciences announced the initiation of its Phase I randomized, double-blind clinical program for KZR-616, a selective inhibitor of the immunoproteasome. Since enrollment of the first cohort on August 9, Kezar has completed dosing of 24 subjects across three cohorts in the single ascending dose (SAD) portion of the Company’s Phase Ia study, which includes both SAD and multiple ascending dose (MAD) portions. The study is expected to enroll a total of 64-80 subjects, with key endpoints including pharmacokinetics and biomarkers of target engagement, as well as safety and tolerability. In the Phase I program, KZR-616 is administered once-weekly as a subcutaneous injection. Pending progress of the study, the Company anticipates that it will report topline pharmacokinetics, safety, and target engagement data by the end of 2016. Additionally, in the first half of 2017 the Company anticipates commencing a Phase 1b study of KZR-616, which is expected to enroll up to 40 patients with autoimmune disorders and include a cohort with a placebo control.

 

EyeGate Pharmaceuticals announced that Jade Therapeutics, a wholly owned subsidiary of EyeGate, has received the second year of funding of $448,185 from the U.S. Army Medical Research and Materiel Command (USAMRMC) to continue the development of its proprietary thiolated hyaluronic acid (CMHA-S) for use as an ocular bandage film entitled “Novel Hyaluronic Acid Delivery Polymer for Repair of Ocular Injuries”. The second year of funding brings the total awarded to Jade under the U.S. Army’s Small Business Innovation Research (SBIR) program to $1.25 million. This work is supported by the U.S. Army Medical Research and Materiel Command under Contract No. W81XWH-14-C-0025.

 

Ligand Pharmaceuticals announced the initiation of a Phase II clinical trial with the company’s glucagon receptor antagonist LGD-6972 for the treatment of Type II diabetes mellitus (T2DM). This randomized, double-blind, placebo-controlled study will evaluate the safety and efficacy of LGD-6972, as an adjunct to diet and exercise, in subjects with T2DM whose blood glucose levels are inadequately controlled with metformin.

 

RedHill Biopharma announced that a Phase Ib/II clinical study evaluating YELIVA (ABC294640) in patients with refractory or relapsed multiple myeloma has been initiated. The open-label, dose escalation Phase Ib/II study is being conducted at Duke University Medical Center and will enroll up to 77 patients with refractory or relapsed multiple myeloma who have previously been treated with proteasome inhibitors and immunomodulatory drugs.

 

LEUKOCARE AG and PaxVax announced that both partners have entered into a licensing agreement granting PaxVax access to LEUKOCARE’s proprietary SPS formulation technology platform for the clinical development and subsequent commercialization of one of PaxVax’s live viral vaccine products.

 

Ablative Solutions announced that the FDA has allowed the Company’s IND application for the Peregrine System Kit for catheter-based renal denervation, with targeted delivery of alcohol, for the treatment of uncontrolled hypertension to proceed. Under the IND, Ablative Solutions plans to conduct a multi-center, double-blind, randomized, sham-controlled trial to evaluate its drug-device combination therapy for the treatment of persistent hypertension despite optimal medical treatment, in conjunction with antihypertensive medical therapy.

 

Caprion Biosciences announced that the company has acquired the assets of the immune monitoring laboratory of ImmuneHealth. Located in Gosselies, Belgium, the ImmuneHealth laboratory will provide a fully operational immune monitoring laboratory infrastructure with well-established expertise, quality processes and industry services track record, which Caprion will leverage to expand its immune monitoring offering and strengthen its business partnerships and research collaborations throughout Europe. The ImmuneHealth lab also possesses complementary expertise and assays for cellular and humoral immunity that will help support Caprion’s efforts to further diversify its services offering in the areas of infectious diseases and vaccine development.

 

InMed Pharmaceuticals announced that Andrew Hull has been appointed to the Board of Directors, effective September 12, 2016.

 

Oxford Gene Technology has launched two new CE-IVD labelled fluorescence in situ hybridisation (FISH) probes, further expanding its extensive lung cancer portfolio. The Cytocell Aquarius ROS1 Plus Breakapart and RET Breakapart probes specifically and accurately detect rearrangements in the genome associated with the most common form of lung cancer – non-small cell lung cancer (NSCLC). The new additions complement OGT’s existing NSCLC probe range (including ALK Breakapart, EGFR Amplification and EML Breakapart), providing a comprehensive solution for diagnosis and patient stratification.

 

Innovus Pharma’s RecalMax was tested in a four-month human clinical trial on 72 patients with low memory and learning. After taking one capsule twice a day for 16 weeks, patients reported a 115% increase in the recall of words and names, a 49.5% increase in maintaining thoughts when distracted, and a 35% increase in processing speed. The supplement was also well-tolerated with no serious adverse events reported.

 

Invitae announced that it entered into a provider agreement effective November 1, 2016 for laboratory services with the California affiliate of a major U.S. payer. The Company expects to enter into agreements with other regional affiliates of this payer as well.

 

AmpliPhi Biosciences announced topline data from a Phase I trial to evaluate the safety and tolerability of AB-SA01, its proprietary investigational phage cocktail targeting Staphylococcus aureus (S. aureus) infections. A safety committee reviewed the trial data and concluded AB-SA01 was well-tolerated by subjects in the trial and there were no drug-related serious adverse events.

 

Matinas BioPharma Holdings announced the final closing of an $8.0 million private placement equity financing, which represents the full amount of the offering. The Company sold to accredited investors an aggregate of 1,600,000 Series A Preferred Shares at a purchase price of $5.00 per share. The net proceeds to the Company from the offering were approximately $6.9 million. The net proceeds will be used for research and development activities, a new manufacturing facility and for working capital and general corporate purposes. SternAegis Ventures, through Aegis Capital, acted as the exclusive Placement Agent for the offering. Participants in the offering included members of the Board of Directors and management of the Company and affiliates of the Placement Agent.

 

OPKO Health announced the appointment of Jane Pine Wood, Esq. as Chief Legal and Compliance Officer of its wholly owned subsidiary, BioReference Laboratories, effective October 1, 2016.

 

 

ANALYST RECOMMENDATIONS

 

LifeSci Capital analyst Jerry Isaacson, Ph.D. initiated coverage of Kamada, citing its lead product Glassia is approved in a large and growing AAT deficiency market.

 

Raymond James analyst Christopher Raymond initiated coverage of the following companies: Vertex Pharmaceuticals with a “market perform” rating; Nivalis Therapeutics with an “outperform” rating and $20 price target; Cempra with an “outperform” rating and $32 price target; Sage Therapeutics with an “outperform” rating and $75 price target.

 

Jefferies analyst Jeffrey Holford revised his price target of the following companies: Abbott decreased to $48 from $52; Bayer AG increased to €104 from €102; GlaxoSmithKline decreased to 1,900p from 2,000p; Johnson & Johnson decreased to $115 from $119; Novo Nordisk decreased to DKK315 from DKK380; Pfizer decreased to $39 from $40; Sanofi decreased to €76 from €80.

 

Following Horizon Pharma’s announcement of the acquisition of Raptor Pharmaceuticals, Mizuho analyst Irina Koffler increased her price target of Horizon Pharma to $33 from $30; Piper Jaffrayanalyst David Amsellem increased his price target to $35 from $33.

 

JP Morgan analyst Jessica Fye initiated coverage of Alder Biopharmaceuticals with an “overweight” rating and $40 price target, citing ALD403’s clear efficacy thus far will enable it to capture share and generate north of $1bn in peak sales.

 

Gabelli & Co. analyst Kevin Kedra initiated coverage of the following companies: Collegium Pharmaceuticals with a “buy” rating and $23 price target; Egalet Corporation with a “buy” rating and $9.50 price target.

 

Gabelli & Co. analyst Kevin Kedra downgraded Pain Therapeutics to “hold” from “buy,” citing while the FDA’s recent favorable actions on extended-release abuse deterrent opioids are a positive, these are offset by some red flags related to abuse liability studies and the product’s history.