BioShares Biotechnology Clinical Trials (BBC): $22.68, +$0.61, +23.2% YTD

BioShares Biotechnology Products (BBP): $37.70, +$0.59, +15.1% YTD

 

 

MARKET COMMENTARY

 

U.S. stock index futures were trading modestly higher as investors await further details on U.S. President Donald Trump’s tax cutting plans expected to be announced later in the day. Amgen, Raymond James and Goldcorp are some of the companies scheduled to report results after markets close. European shares pulled back slightly from 20-month highs as some disappointing corporate results weighed on the market, but Asian stocks powered ahead. The Australian and New Zealand dollars were among the biggest movers in currency markets, hitting its lowest level in two weeks after Australian inflation data pointed to an economy under strain from mediocre growth in China. Oil prices fell after data from American Petroleum Institute, issued late on Tuesday, showed a rise in U.S. crude inventories. Gold was little changed as easing political risks prompted investors to bet on riskier assets.

 

 

MARKET HIGHLIGHTS

 

TxCell SA will present in the following upcoming cell therapy conferences:  the 5th ARM Annual Cell & Gene Therapy Investor Day on April 27th in Boston, the Investment for Advanced Therapies Summit on May 3rd in London, and the International Society for Cellular Therapy May 3-6th in London.

 

Transgene reported first quarter 2017 operating revenue of €1.9 million versus €0.0 million in the same period last year.  As of March 31, 2017, Cash, cash equivalents, available-for-sale financial assets and other financial assets stood at €50.7M, compared to €56.2M as of  December 31, 2016. Cash burn was €5.5M in the first quarter of 2017, versus €8.2M for the same quarter last year.  Transgene confirms that it expects 2017 cash burn to be ~ €30M, which includes the development plan as currently programmed, as well as a $4M milestone payment to SillaJen.

 

Foamix Pharmaceuticals will report its financial results for the first quarter ended March 31, 2017 on Tuesday, May 9th after market close, and will host a corporate update conference call and webcast on Wednesday, May 10th, at 8:30am Eastern Time.

 

Onconova Therapeutics will present an overview at PIONEERS 2017 presented by Joseph Gunnar & Co on Tuesday, May 2nd.

 

iCAD, Inc. announced the Company will showcase the Xoft® Axxent® Electronic Brachytherapy (eBx®) System® at The American Society of Breast Surgeons (ASBrS) Annual Meeting in Las Vegas, Nevada from April 26-30, 2017. The Company will also host a series of presentations by leading clinicians at its booth (#101) to discuss strategies and outcomes in early-stage breast cancer patient care with intraoperative radiation therapy (IORT) using the Xoft System.

 

BioDelivery Sciences International will present at the Deutsche Bank 42nd Annual Health Care Conference on Wednesday, May 3, 2017 at 11:20 AM Eastern Time.

 

BioTime will release first quarter financial and operating results on Wednesday, May 10, 2017, after the close of the U.S. financial markets. The Company will host a conference call and webcast on Wednesday, May 10, 2017, at 4:30 p.m. Eastern Time / 1:30 p.m. Pacific Time to discuss the results and recent corporate developments.

 

Evogene will release its financial results for the first quarter of 2017 on Wednesday, May 17, 2017.

 

Kiadis Pharma announces it has submitted a MAA to the EMA for its lead product, ATIR101. The Company is seeking marketing approval in the European Union for ATIR101 as an adjunctive treatment in haematopoietic stem cell transplantation (HSCT) for malignant disease. The filing is based on Kiadis Pharma’s existing clinical data and follows positive interactions with the EMA Rapporteur and Co-Rapporteur which indicated support for the filing using the Company’s single dose Phase II trial with ATIR101 as the pivotal study. The Company submitted the application under the European Union’s centralized procedure, which permits the agency to issue a single marketing authorization that is valid across all EU countries.

 

MediciNova announced that principal investigator Dr. Benjamin Rix Brooks, Director, Carolinas HealthCare System’s Neuromuscular/ALS-MDA Center, today presented exploratory interim data from MediciNova’s ongoing clinical trial of MN-166 (ibudilast) in amyotrophic lateral sclerosis (ALS) at the American Academy of Neurology (AAN) 69th Annual Meeting in Boston.

 

Paratek Pharmaceuticals will host a conference call and live audio webcast on Thursday, May 4, 2017 at 8:30 AM ET to report first quarter 2017 financial results and provide an update on its clinical development efforts for omadacycline for skin infections, community-acquired bacterial pneumonia, and urinary tract infections.

 

Akebia Therapeutics and Otsuka Pharmaceutical announced that they have expanded their collaboration for vadadustat by entering into a collaboration and license agreement for Europe, China and other territories. Vadadustat is an oral hypoxia-inducible factor (HIF) stabilizer currently in Phase III development for the treatment of anemia associated with chronic kidney disease (CKD). Anemia related to CKD arises from the kidney’s failure to produce adequate amounts of erythropoietin, a key hormone stimulating the production of red blood cells. Left untreated, anemia significantly accelerates patients’ overall deterioration of health with increased morbidity and mortality. This agreement follows a previously announced collaboration between the companies in which they equally share the costs of developing and commercializing vadadustat in the U.S, as well as the profits from potential future sales of vadadustat in the $3.5 billion renal anemia market. The total committed development funding from all vadadustat collaborations, combined with Akebia’s cash, is expected to exceed $600 million. Under the terms of this collaboration agreement, Akebia will receive $208 million or more in committed capital from Otsuka, including $73 million upon signing and $135 million or more of development funding. In addition, Akebia is eligible to receive up to $657 million in milestone payments, representing a total transaction value of approximately $865 million. Otsuka will also make tiered, double-digit royalty payments of up to 30% on net sales of vadadustat in Otsuka’s territory, which includes Europe, Russia, China, Canada, Australia and the Middle East, but excludes Latin America and other previously licensed countries.

 

Medicines Co. reported 1Q17 GAAP EPS loss of $1.44, which compares to a loss of $1.34 for the same period a year ago. The company missed the GAAP EPS mean estimate loss of $1.23. Total revenue for 1Q17 was $24.2 million, which compares to $50.3 million for the same period a year ago. This compares to a mean estimate of $35.1 million.

 

United Therapeutics reported 1Q17 GAAP EPS of $3.89, which compares to $4.84 for the same period a year ago. The company beat the GAAP EPS mean estimate of $3.23. Total revenue for 1Q17 was $370.5 million, which compares to $369.0 million for the same period a year ago. This compares to a mean estimate of $399.6 million.

 

After yesterday’s close, PRA Health Sciences reported 1Q17 non-GAAP EPS of $0.62, which compares to $0.55 for the same period a year ago. The company beat the non-GAAP EPS mean estimate of $0.60. Total revenue for 1Q17 was $487.8 million, which compares to $430.2 million for the same period a year ago. This compares to a mean estimate of $419.5 million. 

 

After yesterday’s close, Illumina reported 1Q17 non-GAAP EPS of $0.64, which compares to $0.71 for the same period a year ago. The company matched the non-GAAP EPS mean estimate of $0.64. Total revenue for 1Q17 was $598.0 million, which compares to $571.8 million for the same period a year ago. This compares to a mean estimate of $590.7 million. Following earnings, Cantor analyst Bryan Brokmeier increased his price target to $170 from $155; Piper Jaffray analyst William Quirk increased his price target to $200 from $187; Baird analyst Catherine Ramsey increased her price target to $171 from $159; Leerink analyst Puneet Souda increased his price target to $201 from $195; Canaccord Genuity analyst Mark Massaro increased his price target to $170 from $150; Deutsche Bank analyst Daniel Leonard increased his price target to $175 from $165; JP Morgan analyst Tycho Peterson increased his price target to $200 from $175; Evercore analyst Ross Muken increased his price target to $204 from $197.

 

Reuters reported that GlaxoSmithKline reported slightly better than expected first-quarter results, keeping the company on track to hit financial targets in 2017 as it braces for generic competition to blockbuster lung drug Advair. Sales and adjusted earnings per share (EPS) rose 19 and 31 percent respectively to 7.38 billion pounds ($9.46 billion) and 25.0 pence. Analysts, on average, had forecast sales of 7.26 billion pounds and EPS of 24.5p, according to Thomson Reuters data.

 

Xenon Pharmaceuticals reported the expansion of its ion channel, neurology-focused product pipeline with XEN1101, an innovative potassium channel modulator for the potential treatment of epilepsy. Xenon acquired worldwide development and commercialization rights to XEN1101 from 1st Order Pharmaceuticals. XEN1101 is a next-generation Kv7 potassium channel opener that preclinically demonstrated improved pharmacokinetics, selectivity, and pharmacology from a new chemical platform over first-generation potassium channel modulators, such as ezogabine. Xenon anticipates filing an IND, or IND equivalent, to initiate a Phase I first-in-man clinical trial in the fourth quarter of 2017.

 

Perrigo Company announced that the company’s Board of Directors, after consultation with management and discussion with Ernst & Young, the company’s independent accounting firm, and following the recommendation of the company’s Audit Committee, concluded that the company’s previously issued financial statements for the following periods should no longer be relied upon: the fiscal years ended June 28, 2014 and June 27, 2015; the transition period from June 28, 2015 to December 31, 2015; and the quarterly periods ended December 28, 2013, March 29, 2014, September 27, 2014, December 27, 2014, March 28, 2015, September 26, 2015, April 2, 2016, July 2, 2016 and October 1, 2016. The company determined that it is necessary to restate certain previously issued financial statements to address these issues. However, the company does not expect these changes to have a material impact on net cash flows and does not expect an impact on the previously closed sale of Tysabri. Perrigo announced that preliminary unaudited first quarter 2017 consolidated net sales were approximately $1.2 billion comprised of Consumer Healthcare Americas (CHCA) net sales of $0.58 billion, Consumer Healthcare International (CHCI) net sales of $0.37 billion and Rx net sales of $0.22 billion. These results do not include any contributions from Tysabri. The company is issuing select guidance metrics to exclude Tysabri and expects 2017 net sales to be in the range of $4.6 billion to $4.8 billion and cash flow from operations of greater than $575 million. The company also announced that effective April 19, 2017, the company’s Board of Directors appointed Ronald L. Winowiecki as the company’s principal accounting officer.

 

Valeant Pharmaceuticals International’s wholly owned subsidiary, Bausch + Lomb, announced it has received 510(k) clearance from the FDA for Vitesse, the first and only hypersonic  device for vitreous removal,  which uses a novel, single-needle design and a continuously open-port system. This new technology represents the first major vitreous removal innovation in 40 years, and one that will be available exclusively on the company’s new Stellaris Elite Vision Enhancement System.

 

Shire announced that the EC has granted Conditional Marketing Authorization for Natpar (rhPTH 1-84), the first recombinant human protein with the full length 84-amino acid sequence of endogenous parathyroid hormone (PTH). Natpar will be the first and only approved hormone therapy indicated as adjunctive treatment for adult patients with chronic hypoparathyroidism who cannot be adequately controlled with standard therapy alone. Hypoparathyroidism is a rare endocrine disease resulting from an inappropriately low circulating PTH concentration.

 

Galapagos announced a new Phase II Proof-of-Concept study investigating filgotinib and another investigational agent in cutaneous lupus erythematosus (CLE). This study is being led by filgotinib collaboration partner Gilead Sciences. Galapagos and Gilead entered into a global collaboration for the development and commercialization of filgotinib in inflammatory indications. This study in CLE is in addition to the ongoing Phase II studies in Sjögren’s syndrome, ankylosing spondylitis (TORTUGA), and psoriatic arthritis (EQUATOR), as well as the ongoing FINCH Phase III program in rheumatoid arthritis, the DIVERSITY Phase III study in Crohn’s disease (also Phase II in small bowel and fistulizing Crohn’s disease), and the SELECTION Phase IIb/III study in ulcerative colitis.

 

Neurocrine Biosciences announced its intention to offer, subject to market and other conditions, $450.0 million aggregate principal amount of convertible senior notes due 2024 in a private offering to qualified institutional buyers pursuant to Rule 144A under the Securities Act of 1933, as amended. Neurocrine also expects to grant the initial purchasers of the notes a 30-day option to purchase up to an additional $67.5 million aggregate principal amount of notes. The notes will be senior unsecured obligations of Neurocrine and will accrue interest payable semiannually in arrears. The notes will be convertible in certain circumstances into cash, shares of Neurocrine’s common stock, or a combination of cash and shares of Neurocrine’s common stock, at Neurocrine’s election. The interest rate, initial conversion rate and other terms of the notes will be determined at the time of the pricing of the offering. Neurocrine intends to use the net proceeds from the offering for general corporate purposes, which may include commercialization expenses, clinical trial and other research and development expenses, capital expenditures, working capital and general and administrative expenses.

 

Flexion Therapeutics announced its intention to offer, subject to market and other conditions, $125 million principal amount of convertible senior notes due 2024 in a private offering to qualified institutional buyers pursuant to Rule 144A of the Securities Act of 1933, as amended. Flexion expects to grant the initial purchasers of the Notes a 30-day option to purchase up to an additional $18.75 million aggregate principal amount of Notes, solely to cover over-allotments. 

 

Antibe Therapeutics announced that is has formally commenced IND enabling pre-clinical studies for its second pipeline drug, ATB-352, a non-addictive, potent analgesic for the treatment of severe pain.

 

Lion Biotechnologies announced that it has entered into a new three-year Manufacturing Services Agreement and related statements of work with PharmaCell B.V., a contract manufacturing services company based in the Netherlands, to manufacture the Company’s autologous cell therapy products. PharmaCell will manufacture TIL products for Lion in its clinical and commercial facility in Geleen, the Netherlands.

 

Flex Pharma announced that the Company’s IND application for FLX-787 for patients with ALS is effective which allows the Company to commence its U.S. Phase II clinical trial of FLX-787 in ALS patients who suffer from cramps as a consequence of the disease. The Company expects to begin enrolling US patients this summer in this randomized, controlled, double-blinded, parallel design study, referred to as the COMMEND trial. In addition, the Company has exploratory Phase II studies currently ongoing in patients with MS and ALS in Australia. FLX-787 is a small molecule co-activator of the TRPA1 and TRPV1 ion channels, and has been shown to prevent and reduce the frequency and intensity of muscle cramps in a human model of electrically induced cramps.

 

Veracyte announced that new data demonstrating the strong performance of the Afirma Gene Expression Classifier (GEC) in thyroid cancer diagnosis and its long-term impact on patient care will be presented at the AACE 26(th) Annual Scientific and Clinical Congress, the annual meeting of the American Association of Clinical Endocrinologists. In addition, researchers will share data validating the company’s next-generation Afirma test, the Afirma Genomic Sequencing Classifier (GSC), at the meeting being held May 3-7 in Austin, Texas.

 

ARCA biopharma announced that the 200(th) patient has been randomized into the GENETIC-AF clinical trial evaluating Gencaro (bucindolol hydrochloride) as potentially the first genetically-targeted treatment for atrial fibrillation (AF).

 

TESARO announced that the European Commission (EC) has approved VARUBY (oral rolapitant tablets) for the prevention of delayed nausea and vomiting associated with highly and moderately emetogenic cancer chemotherapy in adults. Chemotherapy-induced nausea and vomiting (CINV) is a frequent and debilitating, yet often preventable, side effect of chemotherapy.

 

Turnstone Biologics announced that treatment has been initiated on the first patient in the company’s Phase I/II clinical trial combining its novel oncolytic viral immunotherapy agent, MG1-MAGEA3, with pembrolizumab, an approved anti-PD-1 antibody. Named the Sandpiper Trial, this study represents the second clinical trial for Turnstone’s lead product.

 

3D Systems and United Therapeutics announced plans to develop solid-organ scaffolds for human transplants.  The multi-year collaboration and development agreement combines the 3D printing and precision healthcare expertise of 3D Systems with the regenerative medicine and organ manufacturing capabilities of United Therapeutics.

 

Becton, Dickinson and Company announced that it has non-exclusively licensed its patented stochastic labelling technology to Roche for multiple commercial applications. BD’s patented stochastic labelling technology incorporating molecular indexes (known also as UMIs or molecular barcodes), enables the precise counting of individual biological molecules, including DNA or RNA, in complex and often volume-limited samples. BD is developing a suite of products utilizing this technology for accurate quantitative measurements of mRNA targets from single cells, enabling researchers to gain greater insights into the biology of complex and heterogeneous samples.

 

ArQule announced that the USPTO has issued U.S. Patent Number 9,630,968. The patent claims composition of matter of ARQ 531. ArQule will be entitled to patent protection through December 2035 in the U.S. for the allowed claims. ARQ 531 is an investigational, orally bioavailable, potent and reversible inhibitor of both wild type and C481S-mutant Bruton’s tyrosine kinase (BTK). The company plans to begin a phase I clinical trial by the third quarter of 2017 with ARQ 531 in patients with B-cell malignancies who are refractory to other therapeutic options.

 

The Medicines Company and Alnylam Pharmaceuticals announced that The Medicines Company has agreed with the FDA on plans for the Phase III clinical program for inclisiran, which is designed to support the submission of a NDA. The Company has received final, End-of-Phase II meeting minutes from the FDA.

 

Aclaris Therapeutics announced that it has received a Notice of Allowance from the USPTO for a patent application covering the formulation and methods of use of A-101 40% topical solution (A-101 40%), an investigational drug, being developed by Aclaris for the treatment of seborrheic keratosis (SK) and A-101 45% topical solution (A-101 45%), an investigational drug, being developed by Aclaris for the treatment of common warts (verruca vulgaris). Aclaris submitted a NDA to the FDA for A-101 40% as a treatment for SK in February 2017.  In August 2016, Aclaris reported positive data from a Phase II trial, WART-201, in which patients treated with A-101 45% achieved statistically significant and clinically meaningful improvement on all primary and secondary endpoints. This newly allowed patent application contains 70 allowed claims and expires in 2035.

 

uniQure presented new preclinical data on AMT-130, a gene therapy candidate for the treatment of Huntington’s disease at the 12th Annual CHDI HD Therapeutics Conference in Malta. Data from the study demonstrate widespread and effective AAV5 vector distribution and extensive silencing of the human mutant huntingtin gene (HTT) in minipigs, among the largest HD animal models available for testing. AMT-130 consists of an AAV5 vector carrying a DNA cassette encoding artificial micro-RNA (miHTT) that silences the huntingtin gene. The proof-of-concept study was performed by uniQure in collaboration with Prof. Jan Motlik, Director of the Institute of Animal Physiology and Genetics in the Czech Republic and Ralf Reilmann, Founding Director of the George Huntington Institute in Germany.

 

Asterias Biotherapeutics announced that the results from its completed Phase II clinical trial of AST-VAC1 are now available online in Cancer, a leading peer-reviewed journal of the American Cancer Society.

 

Agios Pharmaceuticals announced that the underwriters of its previously announced public offering of common stock have fully exercised their option to purchase an additional 757,575 shares at the public offering price of $49.50 per share, resulting in additional gross proceeds of approximately $37 million. After giving effect to the full exercise of the over-allotment option, the total number of shares sold by Agios in the public offering increased to 5,808,080 shares and gross proceeds increased to approximately $287 million. The exercise of the over-allotment option is expected to close on April 28, 2017, subject to customary closing conditions.

 

Teva Pharmaceutical Industries confirmed that Eyal Desheh, Group Executive Vice President, Chief Financial Officer, will depart from Teva during the coming months. The Company will immediately commence a search to identify a successor to serve as Chief Financial Officer. Mr. Desheh will take part in the company’s upcoming Q1 earnings call on May 11(th).

 

Ra Pharmaceuticals announced that it has initiated dosing in the Company’s global Phase II clinical program evaluating RA101495 for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). Ra Pharma is a clinical stage biopharmaceutical company focusing on the development of next-generation therapeutics for the treatment of complement-mediated diseases and is developing RA101495 as a novel and potent subcutaneously-administered (SC) inhibitor of complement component 5 (C5). Inhibition of C5 is a clinically validated approach for the control of complement-induced hemolysis, the process responsible for the rupture and destruction of red blood cells (RBCs) in patients with PNH.

 

Intellia Therapeutics and CRISPR Therapeutics announced that the USPTO is expected to issue a CRISPR/Cas9 genome editing patent to Vilnius University (“Vilnius”). Intellia and CRISPR are nonexclusive sublicensees for a defined field of human therapeutic, prophylactic, and palliative uses (including companion diagnostics), excluding anti-fungal and anti-microbial applications.

 

BioPharmX announced that it is has entered into securities purchase agreements with certain existing institutional investors for the purchase and sale of 6,410,258 shares of common stock at a price of $0.78 per share with 50% warrant coverage (representing warrants to purchase up to 3,205,129 shares of common stock) in a registered direct offering.  The warrants will be exercisable at an exercise price of $0.90 per share beginning six months following the closing date and will expire five years from the date they become exercisable.  The gross proceeds of the offering are expected to be approximately $5.0 million. Net proceeds of the offering will be used to advance the BioPharmX dermatology-focused drug delivery and clinical programs, including advancing the company’s lead product BPX-01, and general corporate purposes. The offering is expected to close on April 28, 2017, subject to customary closing conditions.Roth Capital Partners acted as sole placement agent.

 

 

ANALYST RECOMMENDATIONS

 

Rodman & Renshaw analyst Raghuram Selvaraju initiated coverage of Apricus Biosciences with a “buy” rating and $4 price target, citing Apricus is focused on the near-term submission and potential commercialization of Vitaros®, a next-generation erectile dysfunction (ED) topical cream based on the naturally-occurring prostaglandin alprostadil. 

 

Aegis analyst Difei Yang increased her price target of Akebia Therapeutics to $25 from $21, citing the expanded deal with Otsuka lifts a financing overhang.

 

Piper Jaffray analyst David Amsellem made price target changes to the following Specialty Pharmaceuticals companies ahead of 1Q17 earnings: Allergan to $212 from $205; Collegium to $16 from $22; Shire to $173 from $176; Teva to $29 from $31.