BioShares Biotechnology Clinical Trials (BBC): $22.70, +$0.10, +23.3% YTD

BioShares Biotechnology Products (BBP): $38.62, +$0.43, +17.9% YTD

 

 

MARKET COMMENTARY

 

U.S. stock index futures fell, as geopolitical tensions in Asia and U.S. President Donald Trump’s accusation that former president Barack Obama wiretapped him, weighed on market sentiment. Durable goods and factory orders data are on the economic calendar. European shares tanked following a slump in Deutsche Bank shares, which overshadowed a flurry of M&A activity, while Asian stocks ended higher. Gold edged higher on its safe haven appeal, while the dollar was little changed. Oil prices fell on concern over Russia’s compliance with a global deal to cut oil output and China’s lower growth target.

 

 

MARKET HIGHLIGHTS

 

Advaxis announced that the FDA has indicated the IND application for ADXS-NEO, a personalized neoantigen-targeted approach to cancer immunotherapy that is being developed in collaboration with Amgen, can proceed. This ground-breaking IND paves the way for bringing a new precision immunotherapy for the treatment of cancers into the clinic this year. ADXS-NEO employs Advaxis’ proprietary Listeria monocytogenes (Lm)-based antigen delivery technology, its Lm Technology™, to target multiple patient-specific neoantigens in each individual patient’s tumor that are not present in normal cells. ADXS-NEO is designed to stimulate both the innate and adaptive arms of the immune system.

 

BrainStorm Cell Therapeutics announced the appointment of Ralph Z. Kern, MD, MHSc to the positions of Chief Operating Officer and Chief Medical Officer, effective March 6, 2017. He joins BrainStorm Cell Therapeutics from Biogen, where he is currently Senior Vice President and Head of Worldwide Medical. His previous industry appointments include Head of Neuroscience Medical Unit at Novartis and Global Medical Director of Personalized Genetic Health at Genzyme Corporation.

 

TiGenix NV announced that it has received the Day 180 List of Outstanding Issues (LoOI) from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) and expects to receive a Marketing Authorization decision for Cx601 in 2017.

 

Rockwell Medical announced that poster and oral presentations for Triferic have been selected by the Annual Dialysis Conference for presentation at their Annual Meeting, March 11-14, 2017 in Long Beach, CA.  Triferic is the Company’s late-stage investigational iron-replacement drug for the treatment of iron deficiency in chronic kidney disease patients receiving hemodialysis. Triferic is the only FDA approved therapy indicated to replace iron and maintain hemoglobin in hemodialysis patients.

 

SciClone Pharmaceuticals reported financial results for the fourth quarter and year ended December 31, 2016.  In the fourth quarter of 2016, SciClone reported revenues of $44.1 million, compared to $42.9 million for the same period in 2015. Revenues for the full year 2016 were $160.1 million, compared to $157.3 million for the full year of 2015. In the fourth quarter of 2016, SciClone reported GAAP diluted net income per share of $0.12, compared to GAAP diluted net income per share of $0.24 for the same period in 2015. GAAP diluted net income per share for the full year 2016 was $0.58, compared to $0.56 for the full year of 2015.  As of December 31, 2016, cash and cash equivalents totaled $134.4 million, compared to $101.4 million as of December 31, 2015, excluding the $12.8 million of restricted cash held in escrow as of December 31, 2015 for the SEC settlement which was released and paid in February 2016.

 

Attune Pharmaceuticals announced the first preclinical data results for ATN-249, a novel orally administered plasma kallikrein inhibitor for the treatment of Hereditary Angioedema (HAE). The data was presented in a late-breaking poster presentation at the 2017 American Academy of Allergy, Asthma & Immunology Annual Meeting (AAAAI 2017) and highlighted a profile which suggests high potency with a wide therapeutic window and the potential for once daily dosing of ATN-249.

 

Minerva Neurosciences will release financial results and business updates for the fourth quarter and fiscal year ended December 31, 2016 on Monday, March 13, 2017. 

 

Eiger BioPharmaceuticals will present a corporate overview and business update at two investor conferences in March 2017:  the 29th Annual ROTH Conference: March 13, @ 9am PT; and the 27th Annual Oppenheimer Healthcare Conference: March 21 @ 4:30pmET.

 

Achaogen will report its fourth quarter 2016 financial results on Tuesday, March 14, 2017 after the close of the U.S. financial markets.

 

TG Therapeutics announced positive topline results from its Phase III GENUINE clinical trial of TG-1101 (ublituximab) plus ibrutinib in patients with previously treated high risk Chronic Lymphocytic Leukemia (CLL).  For the study, high risk was defined as having any one or more of the following: 17p deletion, 11q deletion or p53 mutation. The multicenter, randomized trial (NCT02301156), which assessed the efficacy and safety of TG-1101 plus ibrutinib, met its primary endpoint, demonstrating a statistically significant improvement in Overall Response Rate (ORR) compared to ibrutinib alone in both the Intent to Treat (ITT) population (p=0.001) and Treated population (p <0.001).  The ITT population includes all 126 randomized patients (64 in the TG-1101 + ibrutinib arm and 62 in the ibrutinib alone arm) while the Treated population includes all ITT patients that received at least one dose of either study drug (59 in the TG-1101 + ibrutinib arm and 58 in the ibrutinib alone arm).

 

Concert Pharmaceuticals announced that it has signed a definitive asset purchase agreement with Vertex Pharmaceuticals under which Vertex will acquire CTP-656. CTP-656 is an investigational cystic fibrosis transmembrane conductance regulator (CFTR) potentiator that has the potential to be used as part of future once-daily combination regimens of CFTR modulators that treat the underlying cause of CF. As part of the agreement, Vertex will pay Concert $160 million in cash for all worldwide development and commercialization rights to CTP-656. If CTP-656 is approved as part of a combination regimen to treat CF, Concert could receive up to an additional $90 million in milestones based on regulatory approval in the U.S. and reimbursement in the UK, Germany or France. The agreement is subject to approval by Concert’s shareholders and the expiration of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act. Concert’s Board of Directors unanimously support the transaction and recommend that Concert’s shareholders vote in favor of it.

 

PTC Therapeutics announced that the FDA has acknowledged the filing over protest of PTC’s NDA for Translarna (ataluren), an oral, first-in-class, protein restoration therapy for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD). The Company is seeking approval to market the drug for the treatment of nmDMD patients in the United States. Translarna received marketing authorization for patients with nmDMD in the European Union in August 2014 and is now available in over 25 countries. The FDA has granted standard review and assigned a PDUFA date of October 24, 2017. The PDUFA date is the target date for the FDA to complete its review of the NDA.

 

Galectin Therapeutics announced results from an exploratory, Phase IIa clinical trial with GR-MD-02 in patients with moderate-to-severe plaque psoriasis. Data to be presented at Maui Derm for Dermatologists on March 20 to March 23, 2017 in Maui, Hawaii, showed no serious adverse events and achieved an average PASI (Psoriasis Area and Severity Index) reduction of over 50% in all patients that participated in the 24-week trial, further demonstrating the safety and efficacy of this novel investigational drug in this patient population.

 

Amgen announced positive results from a planned overall survival (OS) interim analysis of the Phase III head-to-head ENDEAVOR trial. The study met the key secondary endpoint of OS, demonstrating that patients with relapsed or refractory multiple myeloma treated with KYPROLIS (carfilzomib) and dexamethasone (Kd) lived 7.6 months longer than those treated with Velcade (bortezomib) and dexamethasone (Vd) (median OS 47.6 months for Kd versus 40.0 for Vd, HR=0.79; 95 percent CI: 0.65 – 0.96; p=0.01). The OS benefit was consistent regardless of prior Velcade therapy (HR 0.75 for no prior Velcade; HR 0.84 for prior Velcade). In other company news, Amgen announced positive data from the Phase III ‘482 study, the largest international multiple myeloma trial ever conducted. In this study, XGEVA (denosumab) met the primary endpoint, demonstrating non-inferiority to zoledronic acid in delaying the time to first on-study skeletal-related event (SRE) in patients with multiple myeloma (HR=0.98, 95 percent CI: 0.85, 1.14; p=0.01). The median time to first on-study SRE was similar between XGEVA (22.83 months) and zoledronic acid (23.98 months).

 

UCB and Dermira announced 16-week, investigational results from the CIMPASI-1 and CIMPASI-2 Phase III trials at the 75(th) Annual Meeting of the American Academy of Dermatology (AAD) in Orlando, Florida. Results from the trials showed that CIMZIA (certolizumab pegol) demonstrated significant improvements in patients with moderate-to-severe chronic plaque psoriasis versus placebo. In addition to previously reported co-primary endpoints, new data presented in an oral presentation at AAD showed the percentage of patients who achieved 90% or greater disease improvement from baseline, as measured by the Psoriasis Area and Severity Index (PASI 90). Additionally, data analyses from the RAPID-PsA Phase III study were presented evaluating the long-term effect of CIMZIA in adult patients with active psoriatic arthritis (PsA).

 

Eli Lilly announced that patients with moderate-to-severe plaque psoriasis treated with Taltz (ixekizumab) demonstrated superior efficacy at 24 weeks compared to patients treated with Stelara (ustekinumab). Detailed results from the IXORA-S study were presented during the American Academy of Dermatology (AAD) Annual Meeting.

 

Regeneron Pharmaceuticals and Sanofi presented detailed results from the one-year Phase III CHRONOS study, which showed that patients receiving the investigational drug DUPIXENT with topical corticosteroids (TCS) achieved significantly improved measures of overall disease severity compared to TCS alone in adults with uncontrolled moderate-to-severe atopic dermatitis (AD).

 

Celgene announced that results from its Phase IV UNVEIL trial evaluating OTEZLA (apremilast), the Company’s oral, selective inhibitor of phosphodiesterase 4 (PDE4), in patients with moderate plaque psoriasis with a body surface area (BSA) of 5-10 percent, were presented at the American Academy of Dermatology’s Annual Meeting in Orlando, Florida.

 

Ablynx announced that its partner, Merck KGaA, has presented new data from a Phase Ib study demonstrating strong efficacy with the bi-specific anti-IL-17A/F Nanobody (M1095; ALX-0761) in patients with moderate-to-severe chronic plaque psoriasis. The results were presented at the 75(th) Annual Meeting of the American Academy of Dermatology Conference.

 

Sandoz, a Novartis division, presented data for its proposed biosimilar adalimumab (GP2017). The Phase III confirmatory efficacy, safety and immunogenicity study met its primary endpoint demonstrating GP2017 has equivalent efficacy to the reference medicine, Humira. Results were presented at the American Academy of Dermatology (AAD) in Orlando, Florida.

 

CEL-SCI announced that it has received the official minutes from its February 8, 2017 meeting with the FDA in regards to the partial clinical hold placed on the Phase III head and neck cancer study with CEL-SCI’s investigational drug Multikine (Leukocyte Interleukin, Injection) on September 26, 2016. Pursuant to this partial clinical hold, patients currently receiving study treatments can continue to receive treatment at the discretion of their physicians and with their consent, and patients already enrolled in the study will continue to be followed. 928 patients are enrolled in this study.

 

Exelixis announced that the FDA has granted orphan drug designation to cabozantinib for the treatment of hepatocellular carcinoma (HCC). This information was posted to FDA’s website on March 4, 2017. A pivotal Phase III trial (CELESTIAL) of cabozantinib is ongoing in patients with advanced HCC, and Exelixis has guided that data from the trial are expected in 2017.

 

Merck and Pfizer announced that the FDA has accepted for review three NDAs for medicines containing ertugliflozin, an investigational SGLT2 inhibitor in development to help improve glycemic control in adults with Type II diabetes: one for monotherapy, one for the fixed-dose combination of ertugliflozin and JANUVIA (sitagliptin), and one for the fixed-dose combination of ertugliflozin and metformin. The PDUFA action date from the FDA is in December 2017 for the three NDAs. Additionally, the EMA has validated for review three MAAs for ertugliflozin monotherapy and the two fixed-dose combination products.

 

Daiichi Sankyo announced that the first patient has been enrolled into the ENTRUST-AF PCI study. The multinational, randomized Phase IIIb study will evaluate a treatment regimen based on the company’s oral, once-daily direct factor Xa-inhibitor edoxaban (known by the brand name LIXIANA outside the US and SAVAYSA in the US) against a vitamin K antagonist based regimen in patients with atrial fibrillation following successful percutaneous coronary intervention (PCI) with stent placement to investigate the incidence of major or clinically relevant non-major ISTH-defined bleeding (MCRB). 1,500 patients will be enrolled in ENTRUST-AF PCI from 200 clinical sites across Europe, Korea, Taiwan and the Ukraine.

 

Teligent announced it has received approval of the Company’s ANDA from the FDA of Triamcinolone Acetonide Ointment USP, 0.5%.  This is Teligent’s first approval for 2017, and its twelfth approval from its internally-developed pipeline of topical generic pharmaceutical medicines.

 

Mast Therapeutics reported that its wholly-owned subsidiary, Aires Pharmaceuticals, has entered into an agreement with the University of Pittsburgh related to a Phase I/II open-label safety and proof of concept clinical trial of the Company’s lead product candidate, AIR001, for the treatment of Pseudomonas aeruginosa (P. aeruginosa) infection in cystic fibrosis (CF) patients. The study is being conducted by the University of Pittsburgh and the University of Pittsburgh Medical Center. Mast’s subsidiary will provide study drug and nebulizers for the study, but no direct financial support.

 

XW Laboratories announced an exclusive licensing agreement with the University of Pittsburgh to further commercialize a class of novel compounds based on pioneering mitochondria targeted bis-nitroxide technology developed in the laboratory of Professor Peter Wipf.

 

NuvOx Pharma announced that the first patients have been dosed in a Phase Ib/II clinical trial for NVX-208, an oxygen therapeutic being developed for acute ischemic stroke. The trial is being performed at the University of Arkansas for Medical Sciences in Little Rock, Arkansas, under the direction of Dr. William Culp, the Jonathan Fitch Distinguished Chair in Stroke. Culp said, “We have studied NVX-208 in a number of pre-clinical stroke models and found that administration of NVX-208 decreases the brain damage from stroke by over 80%. The only approved drug to treat stroke is the thrombolytic drug t-PA, which has a risk of bleeding and is approved for use only up to 3 hours following stroke. Administration of NVX-208 prior to t-PA extended the time window of efficacy to at least nine hours.”

 

ImmunoCellular Therapeutics provided an update on the Company’s ICT-107 phase III registration trial in newly diagnosed glioblastoma, and announced advances in its Stem-to-T-cell Research program. As previously disclosed, ImmunoCellular submitted an amendment of the ICT-107 phase III protocol to the FDA. The key change in this amendment will enable patients to be randomized 30 days after commencing screening procedures, accelerating the time to randomization by approximately 2 months.

 

Provectus Biopharmaceuticals announced that it has extended the expiration date of its rights offering until Friday, March 17, 2017 at 5:00 p.m. Eastern Time. All other terms and conditions of the Rights Offering remain unchanged.

 

Seattle Genetics announced that the FDA has lifted the clinical hold announced on December 27, 2016 on phase I trials of vadastuximab talirine (SGN-CD33A; 33A) in acute myeloid leukemia (AML).

 

Valeant Pharmaceuticals International announced that following the successful closure of the skincare products asset sale on March 3, 2017, Valeant has used the net proceeds of the sale to pay down approximately $1.1 billion of its senior secured term loans.  This debt repayment further enhances the company’s confidence in meeting its goals, and it is taking this opportunity to refinance and amend additional portions of its outstanding debt to further create operating flexibility.

 

Coherus BioSciences announced that Amgen has filed a trade secret action in California state court on March 3, 2017 alleging trade secret misappropriation and other claims against Coherus and other parties.

 

OncoCyte reported the successful completion of a critical step in the development of its lung cancer diagnostic test. While the key performance metrics of its diagnostic cannot be revealed until they are presented at the American Thoracic Society Meeting in May, the company has locked its prediction algorithm and intends to move to the Clinical Validation Phase of development—the last phase before commercial launch. The data from the study exceed levels OncoCyte believes necessary for a commercially successful test and the Company is moving forward with plans to launch the lung cancer diagnostic test during the second half of 2017.

 

Akcea Therapeutics, a wholly owned subsidiary of Ionis Pharmaceuticals, announced that the pivotal Phase III APPROACH study of volanesorsen met its primary endpoint of reducing triglyceride levels in patients with familial chylomicronemia syndrome (FCS). APPROACH is a randomized, double-blind, placebo-controlled, 52-week Phase 3 study in 66 patients with FCS, a rare disease affecting approximately 3,000 to 5,000 patients worldwide. The average incoming triglyceride level of patients in the study was 2,209 mg/dL.

 

Kura Oncology announced updated results from the ongoing, Phase II trial of tipifarnib, a selective inhibitor of farnesyl transferase, and additional preclinical data in HRAS mutant squamous cell carcinomas of the head and neck (SCCHN). The data were presented at the 15(th) International Congress on Targeted Anticancer Therapies (TAT 2017) in Paris, France.

 

Infinity Pharmaceuticals announced that Phase I clinical data for IPI-549, an orally administered immuno-oncology development candidate that selectively inhibits phosphoinositide-3-kinase gamma (PI3K-gamma), were presented during a plenary session at the 15(th) International Congress on Targeted Anticancer Therapies (TAT 2017) taking place in Paris, France, March 6 – 8. A Phase I clinical study is ongoing to explore the safety and activity of IPI-549 both as a monotherapy and in combination with Opdivo (nivolumab), a PD-1 immune checkpoint inhibitor, in patients with advanced solid tumors. IPI-549 is believed to be the only selective PI3K-gamma inhibitor in clinical development.

 

Matinas BioPharma Holdings announced the Institutional Review Board of the National Institute of Allergy and Infectious Diseases (NIAID), part of the NIH has granted approval for a 6-month open-label safety extension of the Phase IIa study of Matinas’ lead anti-infective product candidate, MAT2203 being conducted at NIH.

 

ARCA biopharma announced that the 175(th) patient has been randomized into GENETIC-AF, a seamless design Phase IIB/III clinical trial evaluating Gencaro (bucindolol hydrochloride) as a potential treatment for atrial fibrillation (AF).

 

AMAG Pharmaceuticals announced changes to the company’s executive and commercial leadership teams, including that Frank Thomas, president and chief operating officer, intends to transition out of the organization on April 30, 2017. Following this transition, Ted Myles, chief financial officer, and Joseph Vittiglio, general counsel, along with AMAG’s business development function, will report directly to William Heiden, chief executive officer of AMAG. Mr. Vittiglio joined AMAG as chief counsel in August 2015, and Mr. Myles joined the company as chief financial officer in April 2016.

 

BONE THERAPEUTICS announces the appointment of Miguel Forte, MD, PhD as Chief Medical Officer (CMO).

 

Evoke Pharma announced that the underwriter of its previously announced public offering of approximately 2.4 million shares of its common stock has exercised in full its option to purchase an additional 362,068 shares of common stock, at a price to the public of $2.90 per share. The issuance of the additional shares closed on Friday, March 3, 2017, and all of the shares were sold by Evoke. The exercise brings the total gross proceeds from the offering to approximately $8.0 million, before underwriting discounts and commissions and estimated offering costs. Evoke intends to use the net proceeds from the offering to fund clinical development, pre-approval and pre-commercialization activities for Gimoti, including the planned comparative exposure trial and planned NDA submission, and for working capital and general corporate purposes. Laidlaw & Company (UK) acted as sole book-running manager for the offering.

 

 

ANALYST RECOMMENDATIONS

 

Following Achaogen’s R&D Day, Leerink analyst Paul Matteis raised his price target to $31; Needham Analyst Alan Carr raised his price target to $29; William Blair analyst Katherine Xu raised her price target to $41; and SunTrust Robinson Humphrey analyst Edward Nash raised his price target to $31.

 

Jefferies analyst Biren Amin assumed coverage of Minerva Neurosciences with a “buy” rating and $17 price target, citing NERV’s two candidates MIN-101 and -202 offer upside to shares.

 

Roth analyst Chris Lewis initiated coverage of AxoGen with a “buy” rating and $14.50 price target, citing market awareness, surgeon education, clinical evidence, sales execution, and new products / market expansion.

 

HC Wainwright analyst Swayampakula Ramakanth increased his price target of CytoSorbents to $13 from $11.50, citing higher-than-expected product sales, offset by increased R&D and SG&A increases.

 

Jefferies analyst Eun Yang downgraded United Therapeutics to “underperform” from “hold,” citing physician poll indicates continuing competitor Uptravi impact on UTHR’s treprostinil franchise & meaningful headwinds from generic Remodulin – potentially ~35% of current sales at risk.

 

SunTrust analyst Peter Lawson increased his price target of Kite to $90 from $80, citing Kite is well positioned to receive a positive opinion in the event of an ODAC panel — based upon the robust and durable response rate, efficacy at least 4x better than standard of care, and manageable toxicity.

 

Deutsche Bank analyst Gregg Gilbert downgraded Akorn to “hold” from “buy” and decreased his price target to $24 from $28, citing valuation.

 

Following FibroGen’s earnings, Goldman Sachs analyst Terence Flynn decreased his price target to $30 from $31. For your reference, I have included a full summary of the note below.