BioShares Biotechnology Clinical Trials (BBC): $21.43, -$0.67, -26.2% YTD

BioShares Biotechnology Products (BBP): $34.97, -$1.02, +13.5% YTD

 

 

MARKET COMMENTARY

 

U.S. stock index futures were little changed to slightly higher, ahead of the U.S. Federal Reserve chief Janet Yellen’s congressional testimony. Markets will also watch for key economic releases, including consumer prices index, housing starts, building permits and weekly jobless claims numbers. European equities traded mixed, while Asian shares ended steadied. The dollar pulled back after eight days of back-to-back gains, while gold clawed higher. Expectations of an OPEC deal to limit production pushed oil prices up.

 

 

MARKET HIGHLIGHTS

 

AzurRx BioPharma announced that in partnership with Mayoly Spindler, a European pharmaceutical company, the initiation of a Phase II clinical trial evaluating the safety and efficacy of MS1819-SD in patients with exocrine pancreatic insufficiency (EPI) caused by chronic pancreatitis (CP). The open-label, dose escalation Phase II study is being conducted in two sites in Australia, The Royal Adelaide Hospital and Linear Clinical Research in Perth as well as two sites in New Zealand, P3 Research in Wellington and CCST in Christchurch.  The study is projected to enroll approximately 12 patients with EPI caused by chronic pancreatitis over the next several months.  Dr. Quoc Nam Ngyuen, Associate Professor in the Department of Gastroenterology at the Royal Adelaide Hospital is the principal investigator for the study.  The primary objective of this Phase II study is to investigate the safety of escalating doses of MS1819-SD in patients with chronic pancreatitis. The secondary objective is to investigate the efficacy of MS1819-SD in these patients by analysis of the coefficient of fat absorption and its change from baseline.

 

OncoSec Medical Incorporated will provide updates today on multiple key corporate initiatives at the Company’s inaugural Investor and Analyst Day in San Diego, CA, including plans for a melanoma combination directed-registration study of OncoSec’s lead product candidate, ImmunoPulse® IL-12, in combination with  anti-PD-1 therapy.  In addition, the Company will provide details on its preclinical multi-gene plasmid constructs, Tissue-based Real-time Adaptive Controlled Electroporation (TRACE™), and its Technology Access Program (TAP). OncoSec will also highlight the new Phase II melanoma data that was recently presented at the Society for Immunotherapy of Cancer Annual Meeting ("SITC 2016").

 

Immune Pharmaceuticals announced that it has secured up to $11 million in new financing from a single family office that is an existing investor. The financing includes the sale of a $1 million convertible note and up to $10 million in future equity sales at prevailing market prices in increments of at least $1 million per month.

 

ZIOPHARM Oncology announced the presentation of both clinical and nonclinical data for Ad-RTS-hIL-12 + orally-administered veledimex for recurrent brain cancer at the 21(st) Annual Scientific Meeting of the Society for Neuro-Oncology (SNO) held November 17-20, 2016 in Scottsdale, Arizona. Ad-RTS-hIL-12 + veledimex is a novel viral gene therapy candidate utilizing the proprietary RheoSwitch Therapeutic System (RTS) technology for the controlled expression of interleukin 12 (IL-12), a critical protein for stimulating a vigorous immune response against cancers.

 

Cellectar Biosciences announced that following the successful conjugation of multiple, natural product cytotoxic molecules developed by Pierre Fabre to Cellectar’s PDC delivery platform, it has initiated in vivo studies for a variety of solid tumors.

 

Gilead Sciences announced top-line results from two Phase III clinical trials (SIMPLIFY 1 and 2) evaluating momelotinib, an investigational inhibitor of Janus kinase (JAK) compared to ruxolitinib or best alternative therapy (BAT) in patients with myelofibrosis. The SIMPLIFY-1 study achieved its pre-specified primary endpoint of non-inferiority to ruxolitinib for splenic response rate at Week 24 (SRR24), defined as the percentage of patients experiencing a ≥  35 percent reduction in spleen volume (momelotinib: 26.5%; ruxolitinib: 29.0%; 95 percent CI: -11.2% to +5.6%; p=0.011). Non-inferiority was not achieved for the key secondary endpoint of response rate in total symptom score (TSS). Greater improvements in all three pre-specified anemia-related secondary endpoints (proportion of patients who are transfusion independent, or transfusion dependent and transfusion rate) were observed in patients receiving momelotinib compared to ruxolitinib. However, because the TSS response rate did not meet the non-inferiority test, formal sequential statistical testing was not undertaken for these three additional anemia secondary endpoints.

 

Ra Pharmaceuticals announced that the European Commission has designated RA101495 as an orphan medicinal product for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). RA101495, Ra Pharma’s lead clinical candidate, is a synthetic macrocyclic peptide inhibitor of complement component C5. The molecule is currently in development as a self-administered subcutaneous injection for the treatment of PNH, a serious hematologic disease characterized by pathological red blood cell lysis due to unregulated activity of the complement system.

 

Asterias Biotherapeutics announced the appointment of Ryan Chavez to the position of Chief Financial Officer. In his expanded role, Mr. Chavez, who will also retain the title of General Counsel, will oversee all financial aspects of the company.

 

Eisai announced that it has received FDA Fast Track designation for the development of E2609, a Beta-secretase Cleaving Enzyme (BACE) inhibitor currently being evaluated in Phase III clinical trials for early Alzheimer’s disease. E2609 was discovered by Eisai and is being jointly developed by Eisai and Biogen as a potential Alzheimer’s disease (AD) modifying treatment.

 

Moleculin Biotech announced it has received verbal positive guidance from the FDA regarding its planned IND submission indicating that the Company may incorporate by reference the IND established by a prior developer.

 

Merck announced the launch of the two new innovative fertility technologies Gidget and Geri+ to extend its innovative portfolio to support all steps performed by fertility laboratories during assisted reproductive treatment (ART), where technologies play a vital role for treatment success. Gidget is designed as an easy-to-use witnessing and tracking system to reduce the potential for error and improve lab workflows, and Geri+ is the basis to combine the Geri embryo incubator and the innovative Eeva algorithm. Both new products underline Merck’s healthcare strategy to provide innovation to patients / customers through best-in-class products.

 

Sanofi Genzyme announced that the first patient in the UK has been enrolled and received an infusion in a pivotal Phase III clinical trial named COMET for the investigational therapy neoGAA. NeoGAA is a second-generation enzyme replacement therapy being studied for the treatment of Pompe disease. The first patient was dosed at the University of Newcastle upon Tyne.

 

Egalet announced the issuance of a United States patent covering OXAYDO (oxycodone HCI, USP) tablets for oral use only–CII.  Egalet licensed OXAYDO from Acura Pharmaceuticals and launched the product in the United States in September 2015.

 

ProMetic Life Sciences announced positive interim results from its Open Label Phase II clinical trial in patients suffering from idiopathic pulmonary fibrosis ("IPF"). In addition to demonstrating that PBI-4050 is safe and well tolerated in patients suffering from IPF, the objective of this study was to provide early evidence of clinical benefits of PBI-4050 treatment whether used alone or in addition to either nintedanib or pirfenidone. Forty patients are enrolled in the study in 6 sites across Canada. At this time, the Corporation is reporting on the first 30 patients that have completed their 12 weeks of treatment.

 

Arog Pharmaceuticals announced that the FDA has granted Fast Track designation for crenolanib for the treatment of patients with unresectable or metastatic gastrointestinal stromal tumors (GIST) harboring a platelet-derived growth factor receptor alpha (PDGFR?) D842V mutation.

 

Amgen announced positive top-line results for erenumab from a global Phase III, randomized, double-blind, placebo-controlled STudy to evaluate the efficacy and safety of erenumab in migRaIne preVEntion (STRIVE). These data showed the STRIVE study met the primary endpoint, demonstrating statistically significant reductions from baseline in monthly migraine days in patients with episodic migraine treated with either 70 mg or 140 mg erenumab compared with placebo. Erenumab is specifically designed to prevent migraine by blocking the Calcitonin Gene-Related Peptide (CGRP) receptor, which is believed to have a critical role in mediating the incapacitating pain of migraine.

 

Novartis announced positive topline results from the global Phase III STRIVE study, evaluating the efficacy and safety of the fully human monoclonal antibody AMG 334 (erenumab) in episodic migraine prevention. Once-monthly subcutaneous AMG 334 was evaluated at 70mg and 140mg doses, with both doses meeting the study’s primary endpoint, demonstrating a statistically significant reduction from baseline in mean monthly migraine days at six months versus placebo.[1] AMG 334 is specifically designed to target and block the Calcitonin Gene-Related Peptide (CGRP) receptor that is believed to have a critical role in mediating the incapacitating pain of migraine.

 

Pfizer announced that detailed results from the Phase III PALOMA-2 trial were published in The New England Journal of Medicine. These data, presented at the 52(nd) ASCO Annual Meeting in June, further demonstrate the clinical benefit of IBRANCE (palbociclib) as initial therapy for postmenopausal women with estrogen receptor-positive, human epidermal growth factor receptor 2-negative (ER+, HER2-) metastatic breast cancer. The PALOMA-2 study showed the combination of IBRANCE and letrozole extended progression-free survival (PFS), or the amount of time before tumor growth, by more than 10 months    compared with letrozole plus placebo. Further, the study demonstrated that the median PFS of the IBRANCE and letrozole combination exceeded two years – making it the first and only treatment for this population of women to do so in a randomized Phase 3 study.

 

BeiGene announced a public offering of $175,000,000 of its ADSs, each representing 13 of its ordinary shares, par value $0.0001 per share. BeiGene is proposing to sell $160,000,000 of its ADSs, and certain selling stockholders are proposing to sell $15,000,000 of BeiGene ADSs. In addition, the underwriters have been granted a 30-day option to purchase up to an additional $26,250,000 of ADSs from BeiGene. Morgan Stanley, Goldman Sachs and Cowen are acting as joint book-running managers.

 

Regeneron Pharmaceuticals and Sanofi announced that the ongoing Praluent (alirocumab) ODYSSEY OUTCOMES trial will continue as planned, based on the recommendation of an independent Data Monitoring Committee (DMC) after it completed a second pre-specified interim analysis. The DMC will continue to monitor the ongoing safety and efficacy of Praluent as planned.

 

Pharnext SA announced that the independent Data Safety Monitoring Board (DSMB) has completed its first pre-specified safety evaluation of PXT3003 in the ongoing PLEO-CMT Phase III clinical trial. Based on a review of safety data from 100 patients who completed at least three months of study treatment, the DSMB recommended continuing the PLEO-CMT study as planned.

 

Novocure announced that data will be presented from STELLAR, its ongoing Phase II pilot clinical trial in mesothelioma, at the International Association for the Study of Lung Cancer (IASLC) 17th World Conference on Lung Cancer on Wednesday, Dec. 7, 2016, in Vienna, Austria. The data show that treatment with Tumor Treating Fields (TTFields) plus standard chemotherapy, pemetrexed and cisplatin or carboplatin, was tolerable and safe in patients with unresectable, previously untreated mesothelioma. The interim data also suggest improvements in the 12-month survival rate and progression free survival among patients who received TTFields with standard chemotherapy compared to historical control data of patients who received standard chemotherapy alone.

 

XBiotech announced commencement of a collaboration with a research team at Case Western Reserve University (CWRU) School of Medicine headed by leading gastroenterologist, Fabio Cominelli, M.D., Ph.D. Dr. Cominelli and his research team will conduct pre-clinical studies to help develop new treatments for Inflammatory Bowel Disease (IBD) using the company’s pioneering approach to using natural human antibody therapy to neutralize harmful inflammation. Dr. Cominelli is a world-leading expert in inflammatory bowel disease. His group was the first to report that specific blockade of interleukin-1 (IL-1) was effective in reducing disease severity in colitis and that deregulated inflammation is a cause of auto-inflammatory diseases, including IBD.

 

InVivo Therapeutics Holdings announced that a new patient has been enrolled into The INSPIRE Study (InVivo Study of Probable Benefit of the Neuro-Spinal Scaffold for Safety and Neurologic Recovery in Subjects with Complete Thoracic AIS A Spinal Cord Injury) at Oregon Health & Science University (OHSU) in Portland, Oregon. Ahmed M. Raslan, M.D. and Jason J Chang, M.D., Assistant Professors of Neurological Surgery and study investigators, performed surgery and the implantation on the T9 neurologically complete patient approximately 36 hours after the injury occurred.

 

Tetraphase Pharmaceuticals announced the publication of results from IGNITE1, the company’s Phase III clinical trial which evaluated eravacycline compared to ertapenem for the treatment of complicated intra-abdominal infection (cIAI), in the Journal of the American Medical Association (JAMA) Surgery. The article, titled "IGNITE1: A Phase III, Randomized Multicenter Prospective Study to Assess the Efficacy and Safety of Eravacycline vs. Ertapenem in Complicated Intra-Abdominal Infections," is available online and will appear in a forthcoming print issue of the journal. In the trial, eravacycline was well tolerated and demonstrated statistical non-inferiority to ertapenem using the primary endpoint of clinical response at the test-of-cure (TOC) visit, under the guidance set by the FDA and the EMA.

 

Cellvation, a majority-owned subsidiary of Fortress Biotech, announced data demonstrating a patient’s own stem cells may be safe and effective in diminishing neuroinflammatory response and preserving brain tissue in adults following severe TBI. Data from a Phase I study of Cellvation’s CEVA101, an autologous bone marrow-derived mononuclear cell therapy, were published online this month in the journal STEM CELLS.

 

Chimerix presented data from a preclinical study of intravenous (IV) brincidofovir to determine tissue distribution after single IV or oral administration of the drug. These data were presented at the American Association of Pharmaceutical Scientists’ Annual Meeting in Denver, Colorado. Data from this study showed that oral administration of brincidofovir provided consistent drug exposure to organs that are often the target of viral infection, such as the liver and kidney, but delivered much higher drug exposures in the small intestine than in other anatomical sites. In contrast, the new intravenous formulation of brincidofovir demonstrated much lower, yet constant exposure levels to the small intestines. The IV formulation delivered more uniform drug exposure levels to key organs including the small intestine, liver and kidney. Importantly, IV administration of brincidofovir resulted in higher central nervous system concentrations, which may support testing of this formulation in viral infections in the brain.

 

Momenta Pharmaceuticals announced that data from preclinical studies on its M230 program were published in Science Translational Medicine. The article describes fundamental insights into the mechanism of Fc? receptor (Fc?R) modulation that facilitated the design of multivalent Fc therapeutics, including M230, a selective immunomodulator of Fc receptors (SIFs).

 

Neos Therapeutics announced that it has submitted a NDA to the FDA for its Amphetamine XR oral suspension drug candidate, NT-0201, an ADHD medication based on its proprietary modified–release drug delivery technology platforms.

 

Resverlogix announced the collection of successful data from the New Zealand based Phase I PK study with lead drug candidate apabetalone in patients with severe renal impairment.

 

Janssen Biotech announced the submission of a BLA to the FDA seeking approval of guselkumab for the treatment of adults living with moderate to severe plaque psoriasis. Guselkumab is a human monoclonal antibody that targets interleukin (IL)-23, a protein which has been shown to play a key role in the development of immune-mediated inflammatory diseases. Psoriasis is a chronic, autoimmune inflammatory disorder that results in the overproduction of skin cells, characterized by raised, inflamed, scaly, red lesions, or plaques, which can cause itching, discomfort and pain. It is estimated that 7.5 million Americans have psoriasis, which can range from mild to severe and disabling.

 

Aytu BioScience announced that clinical data demonstrating the reproducibility and reliability of its novel infertility diagnosis platform MiOXSYS has been published in the peer-reviewed, scientific journal Reproductive BioMedicine Online. The data establish a clear protocol for reproducible sampling and a clinical cut-off value for distinguishing infertile men from healthy ones.

 

Northwest Biotherapeutics joined the University Medical Center (UMC) of the Johannes Gutenberg University Mainz in Germany in announcing a Phase II clinical trial program combining DCVax(R)-L and Pembrolizumab (Keytruda) for colorectal cancer with liver metastases.

 

Endo International announced positive results from its Phase IIb study of collagenase clostridium histolyticum (or "CCH") for the treatment of edematous fibrosclerotic panniculopathy ("EFP"), commonly known as cellulite. CCH is known in its currently approved indications in the U.S. as XIAFLEX for adult Dupuytren’s contracture and Peyronie’s disease. Trial subjects receiving CCH showed statistically significant levels of improvement in the appearance of cellulite with treatment, as measured by the trial’s primary endpoint, compared to those subjects receiving placebo. CCH was well-tolerated in the actively treated subjects with most adverse events (AEs) being mild to moderate in severity, and primarily limited to the local injection area.

 

OPKO Health announced that data on RAYALDEE (calcifediol) extended-release capsules as a treatment for secondary hyperparathyroidism (SHPT) in stage 3 and 4 chronic kidney disease patients with vitamin D insufficiency will be presented in a poster presentation at the American Society of Nephrology Kidney Week Meeting, underway in Chicago, IL. The data showed that RAYALDEE has similar effectiveness and safety in controlling SHPT in both African-American (AA) and non-African-American (nAA) patients with stage 3 or 4 chronic kidney disease (CKD) and vitamin D insufficiency (defined as serum total 25-hydroxyvitamin D levels less than 30 ng/mL). Serum total 25-hyrdroxyvitamin D levels showed a similar increase in both patient populations beyond the target of 30 ng/mL, a level considered sufficient for CKD patients in published clinical practice guidelines, despite the well established tendency for lower levels in AA patients. Plasma intact parathyroid hormone (iPTH) levels were effectively suppressed by RAYALDEE in AA patients and nAA patients versus placebo treatment.

 

AmpliPhi Biosciences announced the pricing of an underwritten public offering of 5,335,000 shares of its common stock and warrants to purchase up to an aggregate of 5,335,000 shares of common stock. Each share of common stock is being sold together with a warrant to purchase one share of common stock at a combined price to the public of $0.75 per share and accompanying warrant. The warrants will be immediately exercisable at a price of $0.75 per share of common stock and will expire five years from the date of issuance. The shares of common stock and warrants will be issued separately and will be immediately separable upon issuance. The offering is expected to close on or about November 22, 2016, subject to customary closing conditions. The net proceeds to AmpliPhi from this offering are expected to be approximately $3.8 million, after deducting underwriting discounts and commissions and before deducting other offering expenses payable by AmpliPhi, and excluding any proceeds that may be received upon exercise of the warrants. AmpliPhi anticipates using the net proceeds from the offering for general corporate purposes, including manufacturing expenses, clinical trial expenses, research and development expenses and general and administrative expenses. Roth Capital Partners is acting as the sole book-running manager for the offering and Griffin Securities is acting as the co-manager.

 

MorphoSys announced that its licensee Janssen Research & Development, through one of its affiliaties, has submitted a BLA, to the FDA for guselkumab, an investigational fully human monoclonal antibody against IL-23 identified from MorphoSys’s HuCAL antibody library. MorphoSys will receive a milestone payment from Janssen in connection with the BLA filing which seeks approval for the treatment of patients with moderate to severe plaque psoriasis. Financial details were not disclosed.

 

ContraFect announced that it has been granted a $2.1 million Technology/Therapeutic Development Award through the US Department of Defense Peer Reviewed Medical Research Program (PRMRP).

 

Aequus Pharmaceuticals announced that is has entered into a service agreement with Corium International for the manufacturing of clinical trial materials for Aequus’ transdermal doxylamine/pyridoxine long-acting patch, AQS1303. This product is being developed for the treatment of nausea and vomiting of pregnancy (NVP), and is expected to enter into a Proof of Concept clinical trial in the first half of 2017.

 

PDL BioPharma announced that it has agreed to sell $150.0 million aggregate principal amount of its 2.75% Convertible Senior Notes due 2021 (the Notes) in an underwritten public offering. The conversion rate of the Notes will initially be 262.2951 shares of common stock per $1,000 principal amount of the Notes, equivalent to an initial conversion price of approximately $3.81 per share of common stock, subject to adjustment. The net proceeds from the offering, after deducting the underwriters discount and other estimated offering expenses are expected to be approximately $145.8 million. PDL granted the underwriters an option to purchase up to an additional $22.5 million aggregate principal amount of the Notes to cover overallotments. PDL expects that the offering will be completed, subject to customary closing conditions, on November 22, 2016. RBC Capital Markets is acting as sole book-running manager and sole structuring advisor for the Notes offering. Piper Jaffray is acting as lead manager and Roth Capital Partners is acting as co-manager.

 

Aclaris Therapeutics announced that it intends to offer and sell, subject to market conditions, up to $65,000,000 of shares of its common stock in an underwritten public offering. Aclaris also intends to grant the underwriters a 30-day option to purchase up to an additional $9,750,000 of shares of its common stock sold in the public offering on the same terms and conditions. Aclaris intends to use the net proceeds from the offering to fund the submissions for regulatory approval of its drug candidate A-101 Topical Solution (A-101) for the treatment of seborrheic keratosis (SK), to develop a commercial sales force to market A-101 for the treatment of SK, if approved, to fund the continued research and development of A-101 for the treatment of common warts and its JAK inhibitor drug candidates ATI-50001 and ATI-50002 for the treatment of alopecia areata, to fund preclinical development and formulation of a JAK inhibitor drug candidate for the treatment of androgenetic alopecia and vitiligo, and for working capital and general corporate purposes. Jefferies, Leerink Partners and Guggenheim Securities are acting as joint book-running managers for the offering. William Blair and JMP Securities are acting as co-managers for the offering.

 

Invitae announced the pricing of an underwritten public offering of 7,333,333 shares of its common stock at a price to the public of $6.00 per share. All of the shares are being sold by Invitae. The gross proceeds to Invitae from the offering, before deducting the underwriting discounts and commissions and other offering expenses, are expected to be approximately $44 million. The offering is expected to close on or about November 22, 2016, subject to customary closing conditions. In addition, Invitae has granted the underwriters a 30-day option to purchase up to an additional 1,099,999 shares of its common stock at the public offering price, less the underwriting discounts and commissions. J.P. Morgan Securities is acting as the sole book-running manager for the offering. Cowen and Company is acting as a co-manager for the offering.

 

 

ANALYST RECOMMENDATIONS

 

LifeSci Capital analyst Jerry Isaacson initiated coverage of Albireo.

 

Mizuho analyst Irina Koffler decreased her price target of Allergan to $273 from $293, citing expectations are already low and the business is less levered than its peers, positioning AGN towards a faster recovery in a challenging space.

 

Wells Fargo analyst John Baumgartner downgraded Enzymotec to “market perform” from “outperform” and decreased his valuation range to $5-$6 from $11-$12, citing lack of a catalyst.

 

HC Wainwright analyst Shaunak Deepak initiated coverage of Mirati Therapeutics with a “buy” rating and $12 price target, citing Mirati could be a high-reward story with the otherwise high-risk offset by a relatively strong balance sheet.

 

BTIG analyst Dane Leone increased his price target of Corvus Pharmaceuticals to $26 from $23, citing increased market penetration expectations for CPI- 444.

 

Following Gilead’s mixed Phase III trials of momelotinib, the following analysts increased their price target of Incyte: Leerink analyst Michael Schmidt increased his price target to $115 from $101; BMO analyst Ian Somaiya increased his price target to $126 from $121.

 

Following Endologix’s news of Nellix being delayed, Stephens analyst Chris Cooley decreased his price target to $10 from $17.