BioShares Biotechnology Clinical Trials (BBC): $19.49, +$0.29, -32.9% YTD

BioShares Biotechnology Products (BBP): $33.45, +$0.39, +8.5% YTD

 

 

MARKET COMMENTARY

 

U.S. stock futures pointed to a rise at the open on Wall Street and European equities strengthened as investors shrugged off the defeat of the Italian Prime Minister’s referendum for constitutional reforms. Services and composite PMI from Markit, non-manufacturing PMI from Institute for Supply Management and employment trends data are on the economic calendar. Asian shares lost ground. The euro rebounded from 21-month lows and the dollar softened as investors took profits from its recent gains. Oil prices topped $55 for the first time in 16 months and gold erased gains.

 

 

MARKET HIGHLIGHTS

 

Zogenix announced new data demonstrating effectiveness and cardiovascular-related safety for patients treated with ZX008 (low-dose fenfluramine) as an adjunctive therapy for seizures associated with Lennox Gastaut Syndrome (LGS), and continued effectiveness and safety for the on-going open-label patients with Dravet syndrome.  The data were presented at the 70th Annual American Epilepsy Society (AES) meeting, taking place this week in Houston, Texas.

 

Onconova Therapeutics announced the presentation of data from a Phase 2 clinical trial of oral rigosertib and azacitidine in higher-risk myelodysplastic syndromes (HR-MDS) at the 58th American Society of Hematology (ASH) Annual Meeting in San Diego, California.

 

VBL Therapeutics announced that the independent Data Safety Monitoring Committee (DSMC) met to conduct its first safety review of the Phase 3 GLOBE Study investigating ofranergene obadenovec (VB-111) in recurrent glioblastoma (rGBM).  The DSMC is an independent multidisciplinary group that conducts detailed review of un-blinded study data, discusses potential safety concerns and provides recommendations regarding trial continuation. The committee reviewed the GLOBE safety data collected through a cutoff date in September 2016, and did not find any adverse events that would be cause for concern.  As a result, the DSMC recommended that the study continue as planned.

 

BrainStorm Cell Therapeutics announced today data from the Company’s Phase 2 study of NurOwn® in ALS, will be highlighted in presentations at the 27th International Symposium on ALS/MND, being held December 7-9, 2016 in Dublin, Ireland.

 

Innate Pharma announced that clinical data for lirilumab and IPH4102 were presented in two posters at the American Society of Hematology’s (ASH) 2016 Annual Meeting (December 3-6, 2016), San Diego, CA.

 

ArQule announced that preclinical data was presented on Bruton’s tyrosine kinase (BTK) inhibitor, ARQ 531, in a poster presentation by The Ohio State University at the American Society of Hematology (ASH) Annual Meeting. The presentation highlighted preclinical studies of ARQ 531 in Chronic Lymphocytic Leukemia (CLL). ARQ 531 is an investigational, orally bioavailable, potent and reversible BTK inhibitor.

 

Bellicum Pharmaceuticals announced results from the BP-004 multicenter clinical trial which includes children with Primary Immune Deficiencies (PIDs)    and hemoglobinopathies who received an add-back of BPX-501 modified T cells following a haploidentical, T cell-depleted hematopoietic stem cell transplant (haplo-HSCT). The data were presented in oral and poster sessions during the 58(th) Annual Meeting of the American Society of Hematology. Results show that all 35 children treated are alive, free from GvHD and cured of their underlying disease.

 

Global Blood Therapeutics announced the presentation of results from its ongoing Phase I/II GBT440-001 study that further support the safety and efficacy profile of GBT440 as a potentially disease-modifying therapy for sickle cell disease (SCD). These data were presented at the 58(th) ASH Annual Meeting & Exposition in San Diego. Results showing how GBT440 is metabolized in healthy subjects were also presented.

 

Cerecor announced top-line clinical results from its nicotine withdrawal Phase II clinical trial of CERC-501, an oral, potent, and selective kappa opioid receptor ("KOR") antagonist. The trial did not meet its primary objective in nicotine withdrawal. CERC-501 was well tolerated. Based on this favorable side-effect profile, and as previously planned, Cerecor intends to move forward with development of CERC-501 in its primary indication, as an adjunctive treatment of Major Depressive Disorder (MDD).

 

Omeros announced that its small-molecule inhibitors against GPR174, an orphan G protein-coupled receptor (GPCR) narrowly expressed in immune cells and linked to cancers, substantially and statistically significantly boost levels of cytokines and reduce the population of a subset of T cells known as regulatory T cells (Tregs), both activities known to be important in cancer immunotherapy. Based on these findings, the compounds being advanced by Omeros hold potential advantages over both chimeric antigen receptor (CAR) T-cell therapy and checkpoint inhibitors, each highly touted immunotherapies in development for the treatment of multiple cancers. Part of its orphan GPCR program, Omeros believes that the company alone has identified compounds that inhibit GPR174, and Omeros is establishing a broad patent position directed to any GPR174 inhibitor for the treatment of cancer.

 

Dynavax Technologies announced clinical data from an ongoing Phase I/II, two-part clinical trial evaluating intratumoral administration of SD-101 in the treatment of low-grade lymphoma. The combination of intratumoral SD-101 and low-dose irradiation resulted in tumor regression in untreated tumor sites as well as in the treated tumors. Treatment was well-tolerated and changes in T cell populations consistent with stimulation of anti-tumor immunity were observed in the treated lesions. These data were presented in a poster session on Sunday at the 58(th) American Society of Hematology (ASH) Annual Meeting in San Diego, California.

 

Sunesis Pharmaceuticals announced the presentation of results from the Company’s Phase IA study in healthy volunteers evaluating oral non-covalent reversible BTK inhibitor SNS-062. The results were presented in a poster session titled "CLL: Therapy, excluding Transplantation: Poster I" at the 58th American Society of Hematology Annual Meeting in San Diego, California.

 

Akari Therapeutics announced several corporate updates that were discussed during an Analyst & Investor Symposium held during the 58(th) American Society of Hematology meeting.

 

GeneNews Limited announced that it has engaged All Group Financial Services to complete a brokered private placement of convertible unsecured debentures for gross proceeds of up to $3 million on a "best efforts" basis. The Debentures will have a term of three years and bear interest at a rate of 8% per annum, payable semi-annually in arrears, in cash. Payment of principal is payable in cash or common shares of the Company at the discretion of the Company, subject to the approval of the Toronto Stock Exchange. If the Company elects to pay the principal in Common Shares, the number of Common Shares issued will be determined based on a 10% discount to the 5 day volume weighted average trading price ending on the trading day immediately preceding the date that the principal amount is due.

 

Juno Therapeutics announced in a presentation at the 58(th) ASH Annual Meeting encouraging early data for JCAR014 in patients with chronic lymphocytic leukemia (CLL) who failed treatment with ibrutinib. Insights from studies of the translational product, JCAR014, are being applied to the development of    JCAR017 for the treatment of B-cell malignancies. Both JCAR014 and JCAR017 use a 4-1BB co-stimulatory domain and defined 1:1 cell ratio of CD4:CD8 T cells.

 

bluebird bio announced the presentation of new data from the ongoing HGB-205 clinical study evaluating its LentiGlobin product candidate in patients with transfusion-dependent ?-thalassemia (TDT) and severe sickle cell disease (SCD) at the 58th American Society of Hematology Annual Meeting.

 

Alnylam Pharmaceuticals announced positive initial results from Cohorts 1 and 2 of Part C of its Phase I study with givosiran (gi-VOH-si-ran), the    International Nonproprietary Name for ALN-AS1, an investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) for the treatment of acute hepatic porphyrias. These results were presented in a poster at the 58(th) Annual Meeting of the American Society of Hematology (ASH), held December 3 – 6, 2016 in San Diego, California.

 

Trillium Therapeutics presented data from an ongoing study with its lead drug candidate, TTI-621, in patients with relapsed or refractory hematologic malignancies, at the ASH 58(th) Annual Meeting in San Diego.

 

G1 Therapeutics announced a clinical trial collaboration with Genentech, a member of the Roche Group. A Phase II clinical trial is expected to begin in the first half of 2017 and will evaluate the combination of Genentech’s immune checkpoint, anti-PD-L1 antibody Tecentriq (atezolizumab) with G1’s CDK4/6 inhibitor trilaciclib (G1T28) as a first-line treatment for patients with small-cell lung cancer (SCLC) receiving chemotherapy.

 

Mission Therapeutics announced that Dr Colin Goddard has been appointed as non-executive Chairman, effective January 1st 2017. Dr Colin Goddard joined the board of Mission Therapeutics as a non-executive Director in July 2015. Michael Moore, Mission’s Founder Chairman, will continue on the board and transition to the role of Deputy Chairman.

 

Boehringer Ingelheim announced results from a new analysis of the GLORIA-AF Registry Program, which showed that newly diagnosed non-valvular atrial fibrillation (NVAF) patients treated with Pradaxa® (dabigatran etexilate) had a 76.6 percent probability of remaining on treatment at one year and a 69.2 percent probability at two years. The results were presented at the American Society of Hematology (ASH) Annual Meeting in San Diego, CA.

 

Aduro Biotech announced that an anti-CD27 antibody developed by Aduro Biotech Europe and derived from its proprietary B-select technology has been selected to be advanced into clinical development by Merck, through a subsidiary.  CD27 has been recognized as having a critical role in activating a productive anti-cancer immune response and has demonstrated the potential to be combined with checkpoint inhibitors in pre-clinical studies.

 

Bristol-Myers Squibb announced updated findings from the Phase Ib trial, CheckMate -012, in chemotherapy-naïve advanced non-small cell lung cancer patients evaluating Opdivo monotherapy, or in combination with Yervoy, at different doses and schedules. Data from this trial have been previously reported. These updated results, with a median follow-up of 16 months, include pooled efficacy findings for the Opdivo and Yervoy combination cohorts (Opdivo 3 mg/kg every two weeks plus Yervoy 1 mg/kg every six [Q6W] or 12 weeks [Q12W]). In the pooled combination cohorts, the median progression-free survival in patients with PD-L1 expression ≥1% (n=46) was 12.7 months (95% CI: 7.8, 23.0) and was not reached in patients with PD-L1 expression ≥50% (n=13; 95% CI: 7.8, NR). For patients with ≥50% PD-L1 expression (n=13), the one-year overall survival rate was 100% in the pooled combination cohorts. In addition, the confirmed objective response rates in all treated patients (n=77) was 43%, nearly double the response rate reported with Opdivo monotherapy (23%; n=52), with six patients (8%) achieving a complete response, three of which were in patients with PD-L1 expression <1%. The Grade 3/4 treatment-related adverse events were 42% and 31% for the Q12W and Q6W combination cohorts, respectively.

 

Oculus Innovative Sciences announced that on December 6, 2016, it will officially change the name of the company to Sonoma Pharmaceuticals. In conjunction with the name change, the company’s ticker symbol listed on the NASDAQ will change from OCLS to SNOA (warrants SNOAW), effective December 6, 2016.

 

Cesca Therapeutics announced that on November 21, 2016 the Company has submitted an IDE Supplement to the FDA for its previously approved pivotal trial. The study is designed to demonstrate the safety and effectiveness of the Company’s point-of-care, SurgWerks system, in the treatment of late stage, “no-option” and “poor-option”, CLI patients. The Company expects the FDA to submit its response to the IDE supplement within 30 days from submission.

 

NeuroDerm announced that following its End-of-Phase II meeting with the FDA in late October, the Company intends to pursue a comparative bioavailability regulatory path for the Company’s lead product candidate ND0612 based on comparative pharmacokinetic (PK) data in place of data from Phase III clinical efficacy trials. The FDA also reaffirmed that long-term safety data from the Company’s ongoing BeyoND trial (trial 012) should be part of the eventual New Drug Application (NDA) submission.  ND0612 is the Company’s continuous, subcutaneously delivered levodopa/carbidopa (LD/CD) proprietary liquid formulation for the treatment of Parkinson’s disease.

 

Novo Nordisk announced that NovoSeven (rFVIIa), a portable room temperature stable recombinant activated factor VIIa, resolved 96.5% of bleeds when initiated within one hour after onset of bleeding, demonstrating efficacy of early treatment in people with haemophilia A or B with inhibitors. Efficacy also remained high for bleeds treated after 4 hours. A subanalysis of the SMART-7 study, evaluating the efficacy of NovoSeven in a real-world setting, was presented today at the 58(th) ASH annual meeting.

 

Seattle Genetics presented data from a Phase II clinical trial evaluating ADCETRIS (brentuximab vedotin) combination therapy in frontline diffuse large B-cell lymphoma (DLBCL) at the 58(th) ASH Annual Meeting and Exposition taking place in San Diego, California, December 3-6, 2016. ADCETRIS is an antibody-drug conjugate (ADC) directed to CD30, expressed on several types of non-Hodgkin lymphoma. ADCETRIS is currently not approved for the    treatment of DLBCL.

 

Pfizer announced new data from a randomized Phase II study of glasdegib (PF-04449913), an oral, smoothened (SMO) inhibitor, showing the addition of glasdegib to low-dose cytarabine (LDAC) significantly increased overall survival (OS) when compared to LDAC alone in patients with acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS) who were ineligible for intensive chemotherapy (HR: 0.501, 80% CI: 0.384, 0.654, one-sided log rank    p-value 0.0003). Glasdegib is the first SMO inhibitor to show clinical benefit in this patient population. These data were presented today at the 58th American Society of Hematology (ASH) Annual Meeting and Exposition in San Diego, CA.

 

uniQure N.V. announced new and updated results from its ongoing, dose-ranging Phase I/II trial of AMT-060, its proprietary, investigational gene therapy in patients with severe hemophilia B. The data includes up to 52 weeks of follow-up from the low-dose cohort and up to 31 weeks of follow-up from the second dose cohort. New data presented from the second-dose cohort show a dose response with substantial improvement in disease state in all five patients, including the discontinuation of precautionary Factor IX (FIX) infusions in all four patients that previously required chronic replacement therapy.  To date, only one spontaneous bleed was reported after discontinuation of prophylactic FIX replacement therapy.

 

CSL Behring announced new results from its Phase III clinical development program evaluating IDELVION [Coagulation Factor IX (Recombinant), Albumin Fusion Protein], the company’s novel, long-acting recombinant albumin fusion protein for the treatment of hemophilia B. The results, from a pooled analysis of clinical studies from the global PROLONG-9FP program, assessed the relationship between estimated factor IX activity levels and clinical bleeding risk in adult hemophilia B patients treated with IDELVION using prophylaxis or on-demand (episodic) treatment. Analysis of factor IX activity versus efficacy showed that adult patients achieving sustained factor IX activity levels above 5 or 10 percent, have approximately 80 percent lower risk of bleeding events over one year compared with patients having factor IX activity levels below these thresholds. The findings were presented at the 58(th) annual meeting of the American Society of Hematology (ASH) in San Diego, December 3-6.

 

Alexion Pharmaceuticals announced that new data from an ongoing Phase I/II dose-escalation study of ALXN1210 in patients with paroxysmal nocturnal hemoglobinuria (PNH) showed rapid and sustained reductions in lactate dehydrogenase (LDH), a direct marker of hemolysis (the destruction of red blood cells), in patients treated with once-monthly dosing. In the interim analysis of 13 patients, reductions in LDH were observed at the first evaluable time point (week 1) and were sustained over the study analysis period of up to 24 weeks. Patients also had improvements in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue score from baseline, with patients in the higher-dose cohort achieving a two-fold greater improvement compared with the lower-dose cohort. These findings were presented in a poster session at the 58(th) American Society of Hematology (ASH) annual meeting in San Diego.

 

Alnylam Pharmaceuticals announced new positive results from its ongoing Phase II open-label extension (OLE) study with fitusiran, an investigational RNAi therapeutic, in patients with hemophilia A or B without inhibitors. These results were presented today in a poster at the 58(th) Annual Meeting of the American Society of Hematology (ASH) in San Diego, California.

 

Acceleron Pharma and Celgene announced preliminary Phase II results from the ongoing three-month base and long-term extension studies with investigational drug luspatercept in patients with lower risk myelodysplastic syndromes (MDS) at the 58(th) Annual Meeting of the American Society of Hematology (ASH) in San Diego, California. Luspatercept is being developed as part of the global collaboration between Acceleron and Celgene.

 

Karyopharm Therapeutics announced updated results from the Phase II SAIL study, in which deep responses to selinexor (KPT-330) allowed patients with heavily pretreated acute myeloid leukemia (AML) to proceed onto stem cell transplantation or donor lymphocyte transfusion, at the ASH 2016 annual meeting held December 3-6, 2016 in San Diego. Selinexor is the Company’s lead, novel, oral Selective Inhibitor of Nuclear Export (SINE™) compound, in development for the treatment of AML and a variety of additional malignancies.

 

Incyte Corporation announced an exploratory pooled analysis of data from the five-year follow-up of the Phase III COMFORT-I and COMFORT-II trials which further supports previously published overall survival findings and suggests that earlier treatment with Jakafi (ruxolitinib) may result in an improved survival advantage for patients with intermediate-2 or high-risk myelofibrosis (MF) than best available therapy (BAT) or placebo. These data also reinforce previous long-term results observed with ruxolitinib compared with controls (BAT or placebo).

 

AbbVie announced encouraging efficacy and safety findings from two separate studies evaluating ibrutinib (IMBRUVICA) as a combination therapy in two of the most common types of non-Hodgkin’s lymphoma: diffuse large B-cell lymphoma (DLBCL) and follicular lymphoma (FL). IMBRUVICA, a first-in-class Bruton’s tyrosine kinase (BTK) inhibitor, is jointly developed and commercialized by Pharmacyclics, an AbbVie company, and Janssen Biotech.

 

Blueprint Medicines announced data from its ongoing Phase I trial evaluating BLU-285, an investigational medicine for the treatment of patients with advanced systemic mastocytosis (SM).

 

Kite Pharma announced that 82 percent of patients (9 out of 11) achieved complete remission or complete remission with incomplete or partial hematological recovery in a preliminary analysis of the Phase I ZUMA-3 and ZUMA-4 trials of KTE-C19 in adult and pediatric relapsed/refractory acute lymphoblastic leukemia (r/r ALL). In these patients, 100 percent of responders tested negative for minimal residual disease (MRD), which has been shown to correlate with risk of disease relapse in ALL. The data were presented at the ASH Annual Meeting in San Diego, CA.

 

Novelion Therapeutics announced that its subsidiary, Aegerion Pharmaceuticals, has entered into a licensing agreement with Amryt Pharma for the exclusive rights to LOJUXTA (lomitapide) hard capsules in certain European and Middle Eastern territories. LOJUXTA, marketed as JUXTAPID in the U.S, is currently approved by the EC as an adjunct to a low-fat diet and other lipid-lowering medicinal products with or without low density lipoprotein (LDL) apheresis in adult patients with homozygous familial hypercholesterolemia (HoFH).

 

SCILEX Pharmaceuticals, a subsidiary of Sorrento Therapeutics, announced that its pivotal study for lead investigational product, ZTlido (lidocaine patch 1.8%), has met a primary endpoint establishing comparative pharmacokinetics and a secondary endpoint of bioequivalence for ZTlido as compared to the reference product, Lidoderm (lidocaine patch 5.0%). The full data will be resubmitted to the FDA as part of the 505(b)(2) NDA in mid-2017.

 

CASI Pharmaceuticals announced that China’s Food and Drug Administration has accepted for review the Company’s import drug registration application for EVOMELA (melphalan) for Injection.

 

Spark Therapeutics and Selecta Biosciences announced a license agreement that provides Spark Therapeutics with exclusive worldwide rights to Selecta’s proprietary Synthetic Vaccine Particles (SVP) platform technology for co-administration with gene therapy targets, including FVIII for hemophilia A, as well as exclusive options for up to four additional undisclosed genetic targets.

 

GW Pharmaceuticals announced additional positive Epidiolex (cannabidiol or CBD) Phase III data in poster presentations at the 70(th) Annual Meeting of the American Epilepsy Society. These data are from the positive pivotal Phase III study of Epidiolex in Dravet syndrome and the first pivotal Phase III study of Epidiolex in Lennox-Gastaut syndrome (LGS), both reported earlier this year.

 

Acceleron Pharma and Celgene announced preliminary results from an ongoing investigator initiated Phase II study with investigational drug sotatercept in patients with myelofibrosis at the 58th Annual Meeting of the American Society of Hematology (ASH) in San Diego, California. Sotatercept is being developed as part of the global collaboration between Acceleron and Celgene.

 

Evogen and UCB announced a collaboration for further development of EvoScore START, Evogen’s new proteomics-based blood test designed to accurately distinguish epileptic seizures from other events.

 

BioLineRx presented positive Phase IIa correlative data, as well as detailed mechanism-of-action data, for BL-8040, the Company’s leading oncology platform at the ongoing 58th American Society of Hematology (ASH) Annual Meeting and Exhibition in San Diego, California.

 

Spark Therapeutics announced the appointment of John Furey to the newly created position of chief operating officer. Mr. Furey will be responsible for global commercial operations, medical affairs, technology development and technical operations.

 

Xenetic Biosciences announced it has appointed Jeffrey F. Eisenberg as Chief Operating Officer.

 

Matinas BioPharma Holdings announced that it has commenced dosing in the Phase I clinical study of its lead antibiotic product candidate MAT2501, under development for the treatment of nontuberculous mycobacterium infections (NTM) as an initial indication. Data from this study is expected to be announced in the first half of 2017.

 

Adaptimmune Therapeutics announced that it has initiated a study of its NY–ESO SPEAR T–cells in myxoid/round cell liposarcoma (MRCLS). Patient screening is underway, and the results from this study in up to 15 patients will inform a potential future registration trial. The initiation of screening in this study meets a milestone set forth in the Company’s strategic collaboration agreement with GlaxoSmithKline.

 

KaloBios Pharmaceuticals announced that Cameron Durrant, MD, chairman and CEO, has issued a Letter to Stakeholders highlighting the company’s priorities and milestones.

 

TG Therapeutics announced the presentation of two preclinical data sets, one oral presentation and one poster presentation, for TGR-1202, the Company’s once-daily PI3K delta inhibitor, at the 58(th) American Society of Hematology (ASH) annual meeting in San Diego, California.

 

ARIAD Pharmaceuticals announced clinical data on brigatinib, its investigational anaplastic lymphoma kinase (ALK) inhibitor, from the ongoing Phase I/II and pivotal ALTA trials in patients with ALK-positive (ALK+) non-small cell lung cancer (NSCLC) and intracranial central nervous system (CNS) metastases. These data, being presented at the International Association for the Study of Lung Cancer (IASLC) 17(th) World Conference on Lung Cancer (WCLC) being held in Vienna, showed that in patients with measurable brain metastases, the confirmed intracranial objective response rate (ORR) was 53 percent in the Phase I/II trial, and the confirmed intracranial ORR was 67 percent in Arm B (brigatinib 180 mg with seven-day lead-in at 90 mg once daily) in the ALTA trial. Median intracranial progression-free survival (PFS) in ALTA Arm B was 18.4 months.

 

Cempra announced that Toyama Chemical a subsidiary of FUJIFILM Holdings, has begun Phase III clinical trials with solithromycin in Japan, the world’s second largest antibiotic market, for patients with community-acquired bacterial pneumonia (CABP) and other respiratory infections.

 

Helsinn and MEI Pharma announced final results from a Phase II clinical study of the investigational drug candidate Pracinostat and azacitidine in elderly patients with acute myeloid leukemia (AML) who were not eligible for induction chemotherapy, including evidence of prolongation of survival in the overall population and across a number of patient subgroups.

 

MabVax Therapeutics Holdings announced the expansion of the Company’s MVT-5873 Phase I clinical trial to include the HonorHealth Research Institute located in Scottsdale, Arizona.

 

CytomX Therapeutics announced the selection of the fourth target by Bristol-Myers Squibb under the companies’ current strategic oncology collaboration established in 2014. As a result, Bristol-Myers Squibb will pay CytomX $15 million. This constitutes the final target selection under this agreement.

 

Lipocine announced it plans to initiate a dosing validation study after receiving feedback from the FDA of its protocol for LPCN 1021. LPCN 1021 is an oral testosterone product candidate for testosterone replacement therapy ("TRT") in adult males for conditions associated with a deficiency of endogenous testosterone, also known as hypogonadism.

 

Dimension Therapeutics announced an oral poster presentation that highlights findings of in vivo preclinical studies demonstrating that the selection of capsid, a key component of AAV vectors, can enhance product efficiency and potentially reduce host immune responses to therapy. The presentation took place during the 58th American Society of Hematology (ASH) Annual Meeting, which is being held in San Diego, California, December 3-6, 2016.

 

Minerva Neurosciences announced data presentations at the 55(th) Annual Meeting of the American College of Neuropsychopharmacology (ACNP), December 4-8, 2016. Conclusions from these data analyses are summarized below.

 

Pfizer and its partner Avillion announced results from the Phase III BFORE (Bosutinib trial in First line chrOnic myelogenous leukemia tREatment) trial demonstrating superiority of BOSULIF (bosutinib) over imatinib as a first-line treatment for patients with chronic phase Philadelphia chromosome positive (Ph+) chronic myeloid leukemia (CML). The study met its primary endpoint of major molecular response (MMR) at 12 months. No new or unexpected safety issues were identified. BOSULIF is currently indicated in the U.S. and EU for the treatment of adult patients with Ph+ CML with resistance or intolerance to prior therapy.

 

The FDA approved a new indication for Jardiance (empagliflozin) tablets to reduce the risk of cardiovascular death in adults with Type II diabetes and established cardiovascular disease. JARDIANCE is the first Type II diabetes treatment approved with this additional indication and the only oral Type II diabetes medicine shown in a clinical trial to provide a life-saving cardiovascular benefit. JARDIANCE is marketed by Boehringer Ingelheim and Eli Lilly.

 

Merck announced that it will provide its Provantage End-to-End services to Acticor Biotech SAS for accelerated development and manufacturing of Acticor’s antibody fragment used for the primary treatment of ischemic stroke.

 

Celldex Therapeutics presented data on new product candidate CDX-1140, a fully human antibody targeted to CD40 that has demonstrated potent agonist and anti-lymphoma activity. Found on antigen presenting cells, such as dendritic cells, macrophages and B cells, CD40 is a key activator of the immune response and is expressed on many cancer cells, in particular B cell lymphomas. The data were presented at the American Society of Hematology (ASH) Annual Meeting on Saturday, December 3 in a poster titled "CDX-1140, A Novel Agonist CD40 Antibody with Potent Anti-lymphoma Activity" (Abstract #1848). CDX-1140 is expected to be ready to enter clinical studies in patients with advanced cancers, including lymphoma, in 2017.

 

Syros Pharmaceuticals announced that new preclinical data on SY-1425, its first-in-class selective retinoic acid receptor alpha agonist currently in a Phase II clinical trial in genomically defined subsets of patients with acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS), were presented at the 58(th) American Society of Hematology (ASH) Annual Meeting and Exhibition in San Diego.

 

Cancer Genetics announced that it is on Merck‘s list of national reference laboratories offering the PD-L1 22C3 pharmDx immunohistochemistry (IHC) assay, the first FDA-approved companion diagnostic test for pembrolizumab, KEYTRUDA(R) , for PD-L1 testing in non-small cell lung cancer (NSCLC). The company first started offering the DAKO PD-L1 IHC 22C3 pharmDx test in early 2016, and was recently listed as a national reference laboratory.

 

Novelion Therapeutics announced that on December 3, 2016, Aegerion Pharmaceuticals, an indirect, wholly-owned subsidiary of Novelion, entered into an agreement in principle to settle all claims in the class action shareholder lawsuit pending in the United States District Court for the District of Massachusetts (the "Class Action Litigation"). The agreement provides for an aggregate settlement payment by or on behalf of Aegerion of $22.25 million. The Company expects $22 million of the settlement will be funded by Aegerion’s insurance proceeds and $250,000 will be funded by Aegerion. The settlement would include the dismissal of all claims against Aegerion and the named individuals in the Class Action Litigation without any liability or wrongdoing attributed to them. The settlement remains subject to further documentation, court approval, and other customary conditions, including Aegerion’s right to terminate the settlement in the event an agreed upon percentage of class members do not participate.

 

OncoMed Pharmaceuticals announced that Jakob Dupont, M.D., has resigned as the company’s Senior Vice President and Chief Medical Officer, due to personal and family-related issues. Dr. Dupont expects to return to Genentech/Roche in a position of significant responsibility in oncology clinical development. Dr. Dupont’s resignation will be effective on or about January 1, 2017 in order to facilitate a smooth transition.

 

EyeGate Pharmaceuticals announced promising data from the third stage of its Phase Ib/IIa trial assessing lead product candidate, iontophoretic EGP-437, for the treatment of ocular inflammation and pain in post-surgical cataract patients.

 

Theravance Biopharma announced that GlaxoSmithKline and Innoviva, have filed a MAA in the European Union for the Closed Triple (the combination of fluticasone furoate, umeclidinium, and vilanterol in a single ELLIPTA inhaler) for patients with chronic obstructive pulmonary disease (COPD). The Closed Triple is one of the drug development programs for which Theravance Biopharma has an economic interest in future payments that may be made by GSK or one of its affiliates pursuant to its agreements with Innoviva (formerly Theravance, Inc.). Should the Closed Triple be approved and commercialized, Theravance Biopharma is entitled to receive an 85% economic interest in the royalties paid by GSK on worldwide net sales. Those royalties are upward-tiering from 6.5% to 10%. Additionally, Theravance Biopharma is not responsible for any costs related to the Closed Triple.

 

GTx announced a reverse stock split of its shares of common stock at a ratio of one-for-ten. The reverse stock split will be effective at 5:00 p.m. Eastern Time on December 5, 2016. At the opening of trading on December 6, 2016, GTx’s common stock will begin trading on a split-adjusted basis and the number of shares of GTx’s common stock outstanding will decrease from approximately 159.2 million pre-split shares to approximately 15.9 million post-split shares.

 

AmpliPhi Biosciences reported final results from its Phase I trial to evaluate the safety and tolerability of AB-SA01, its proprietary investigational phage cocktail targeting Staphylococcus aureus (S. aureus) infections. Overall, treatment with AB-SA01 was well tolerated when administered topically to the intact skin of healthy adults. The trial was conducted under a Collaborative Research and Development Agreement with the U.S. Army at the Walter Reed Army Institute of Research Clinical Trials Center in Silver Spring, Maryland.

 

AbbVie and Northwestern University announced they signed a five-year collaboration agreement with the goal of advancing research and discovery in oncology. Together, AbbVie and the Robert H. Lurie Comprehensive Cancer Center of Northwestern University will work in several areas of oncology research, which in addition to others could include, lung, colorectal, breast, prostate and hematological cancer.

 

AbbVie and the Johns Hopkins University School of Medicine announced that they signed a five-year collaboration agreement with the goal of advancing medical oncology research and discovery at both organizations.

 

ANALYST RECOMMENDATIONS

 

Leerink analyst Jason Gerberry decreased his price target of Allergan to $259 from $266, citing generic entry of Minastrin and Namenda XR in 2017.

 

H.C. Wainwright analyst Ed Arce initiated coverage of The Medicines Company with a “buy” rating and $57 price target, citing the robust interim results of the Phase II ORION-1 study presented at AHA recently.