BioShares Biotechnology Clinical Trials (BBC): $21.39, +$0.78, -26.4% YTD
BioShares Biotechnology Products (BBP): $32.15, +$0.62, +4.3% YTD
Wall Street was set to open higher as investors await a flurry of economic data including retail sales, industrial production, producer price index, business inventories and weekly jobless claims. European markets were trading modestly higher, while Asian stock markets recorded a mixed trading day. Gold slipped as the dollar edged upwards, curbing the metal’s demand ahead of next week’s U.S. Federal Reserve policy meeting. Oil prices rose after two consecutive days of losses, with gains capped by returning supplies from Nigeria and Libya.
LifeSci Advisors announced the opening of an office in Tel-Aviv, Israel. The establishment of an Israeli office, together with the recent expansion of its European team, reflects LifeSci Advisors’ increasing international focus.
Aeglea BioTherapeutics announced the dosing of the first two patients in a Phase 1 trial of AEB1102, a recombinant human enzyme designed to degrade the amino acid arginine, for the treatment of patients with Arginase I deficiency.
BioLife Solutions announced that its clinical grade CryoStor freeze media has been embedded into the manufacturing process for HepaStem, a cell-based treatment developed by Promethera Biosciences, targeting several metabolic liver disorders such as hemophilia and large clinical indications including acute or chronic liver failure (ACLF), fibrosis and nonalcoholic steatohepatitis (NASH).
Ignyta will make a presentation at the 2016 Silicon Valley Bank Healthcare Capital and Connections Summit on September 21, 2016, at 12:53 a.m. Pacific time, in Shanghai, China, and will discuss Ignyta’s Rx/Dx approach and review recent updates from ongoing clinical trials of the company’s robust pipeline of molecularly targeted therapies.
Basilea Pharmaceutica announced that Basilea Pharmaceutica International has entered into a partnering agreement with Asahi Kasei Pharma Basilea’s antifungal drug isavuconazole in Japan. Under the terms of the agreement, Asahi Kasei Pharma is granted an exclusive license to develop and commercialize isavuconazole in Japan. Basilea will receive an upfront payment of CHF 7 million and will be eligible to receive up to approximately CHF 60 million (at current exchange rate) of additional payments upon achievement of regulatory and commercial milestones. Basilea will also receive double-digit tiered royalties on product sales in Japan.
Aerie Pharmaceuticals reported the successful 90-day primary efficacy results of its 12-month Phase III “Mercury 1” clinical trial for its fixed-dose combination product candidate, Roclatan. The study achieved its primary efficacy endpoint demonstrating statistical superiority over each of its components, including Aerie product candidate Rhopressa (netarsudil ophthalmic solution) 0.02%, and market leading prostaglandin analogue (PGA) latanoprost, all of which were dosed once daily in the evening. The study evaluated patients with maximum baseline intraocular pressures (IOPs) ranging from above 20 to below 36 mmHg (millimeters of mercury). The IOP-lowering effect of Roclatan was 1 to 3 mmHg greater than monotherapy with either latanoprost or Rhopressa throughout the duration of the study. Following the news, Cantor Fitzgerald analyst Elemer Piros increased his price target to $50 from $44; Brean analyst Difei Yang increased her price target to $48 from $33; Needham analyst Serge Belanger increased his price target to $52 from $45; Canaccord analyst John Newman increased his price target to $50 from $44; RBC analyst Adnan Butt increased his price target to $55 from $51; Stifel analyst Annabel Samimy increased her price target to $50 from $40.
AcelRx Pharmaceuticals announced that in an open-label Phase III trial (SAP303), investigational product candidate ARX-04 (sufentanil sublingual tablet 30 mcg) was well tolerated in the management of moderate-to-severe acute pain in post-operative study patients, including elderly patients and those with organ impairment. Regardless of age and organ function, approximately 2 in 3 patients had no adverse events during the study (63% of all patients, 63% of those aged ≥65 years, 62% of those with hepatic impairment, 70% of those with renal impairment). The most common adverse events were nausea and headache. On a global assessment of ARX-04 as a method of pain control, 90% of healthcare professionals and 87% of patients responded “good” or “excellent.”
A new post-hoc analysis from the pivotal DECIDE study shows that a significantly greater number of people treated with ZINBRYTA (daclizumab) achieved no evidence of disease activity (NEDA) compared to those taking AVONEX (interferon beta-1a) intramuscular injection. The findings continue to support the positive impact of ZINBRYTA on NEDA status. Additional new interim data from the long-term extension study, EXTEND, further affirm ZINBRYTA’s efficacy on clinically meaningful measures of multiple sclerosis (MS) disease activity and provide additional information supporting ZINBRYTA’s safety profile. These results were presented by Biogen and AbbVie at the 32nd Congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) in London.
BioMarin Pharmaceutical announced that the EMA validated the MAA for Brineura (cerliponase alfa) to treat children with CLN2 disease, a form of Batten disease. Validation of the MAA confirms that the submission is accepted and starts the formal review process by the EMA’s CHMP.
Merck in partnership with Pfizer announced that a Phase III study (VERTIS SITA2) of ertugliflozin, an investigational oral SGLT2 inhibitor for the treatment of patients with Type II diabetes, met its primary endpoint. Both 5 mg and 15 mg daily doses of ertugliflozin showed significantly greater reductions in A1C* of 0.69 percent and 0.76 percent, respectively, compared with placebo (p<0.001, for both comparisons), when added to patients on a background of sitagliptin (100 mg/day) and stable metformin (≥1500 mg/day). These study results were presented for the first time during an oral session today at the 52nd Annual Meeting of the European Association for the Study of Diabetes (EASD) in Munich, Germany. Merck and Pfizer plan to submit New Drug Applications to the FDA for ertugliflozin and two fixed-dose combinations (ertugliflozin plus JANUVIA (sitagliptin) and ertugliflozin plus metformin) by the end of 2016, with additional regulatory submissions outside of the U.S. to follow in 2017.
ArQule announced the publication of a paper detailing the preclinical profile of ARQ 087, an orally available fibroblast growth factor receptor (FGFR) inhibitor. The findings, published on-line by PLOS ONE, demonstrate that ARQ 087 has anti-proliferative activity in cell lines driven by FGFR dysregulation, including amplifications, fusions, and mutations.
Cara Therapeutics announced the initiation of patient enrollment in a Phase IIb trial of an oral tablet formulation of its peripherally selective kappa opioid agonist, CR845, for the treatment of pain associated with osteoarthritis (OA). The Company expects to report top-line data from this trial during the first half of 2017.
Akcea Therapeutics, a wholly-owned subsidiary of Ionis Pharmaceuticals, focused on developing and commercializing drugs to treat patients with serious cardiometabolic diseases caused by lipid disorders, announced that the company will present new data on the clinical characteristics, impact on quality of life and psychosocial consequences of living with familial chylomicronemia syndrome (FCS). The findings will be presented in two poster presentations at the American Society for Preventative Cardiology (ASPC) 2016 Congress being held September 16-18 in Boca Raton, Florida. These new data reflect findings from the largest study in patients with FCS conducted to date and provide new insights on the disease burden from the perspective of patients and caregivers.
Celsion announced that the independent Data Safety Monitoring Board (DSMB) has completed its safety review of data from the first three patient cohorts in the ongoing Phase Ib OVATION Study. Based on the DSMB’s recommendation, the study will continue as planned and the Company will proceed with dosing in its fourth and final patient cohort at an escalated dose. The OVATION Study is a dose-escalating clinical trial combining GEN-1, the Company’s DNA-based immunotherapy, with the standard of care for the treatment of newly-diagnosed patients with advanced ovarian cancer who will undergo neoadjuvant chemotherapy followed by interval debulking surgery.
CTD Holdings announced that on September 12, 2016 the UK Medicines and Healthcare Products Agency (MHRA) approved its Clinical Trial Application (CTA) for its orphan drug product, Trappsol Cyclo, in the treatment of Niemann-Pick Type C (NPC) disease. CTD will launch a Phase I/II study to investigate safety, tolerability, pharmacokinetic parameters and pharmacological effects of the intravenous administration of Trappsol Cyclo, CTD’s proprietary formulation of hydroxypropyl beta cyclodextrin. Trappsol Cyclo has Orphan Drug Designation in both the EU and the US.
Abeona Therapeutics announced that preclinical data supporting clinical trials for ABO-102 (AAV-SGSH), the AAV-based single intravenous gene therapy program for MPS IIIA, (Sanfilippo Type A), were published in the June issue of Molecular Therapy Methods & Clinical Development.
Voyager Therapeutics announced a co-exclusive worldwide license agreement with the California Institute of Technology (Caltech) related to novel adeno-associated virus (AAV) capsids. The license agreement covers all fields of use and includes novel AAV capsids that have demonstrated enhanced blood-brain barrier penetration for the potential treatment of CNS diseases following systemic administration of an AAV gene therapy vector.
Rigel Pharmaceuticals announced plans to build a commercial organization to support the potential launch of fostamatinib, its oral SYK inhibitor, for the treatment of chronic immune thrombocytopenia (ITP). The company recently reported that fostamatinib met the primary endpoint in the first of two identical Phase III studies in chronic ITP. Results of the second Phase III clinical study are expected in late October or early November. Rigel has reduced its workforce by 38%, resulting in the elimination of 46 positions, mostly in the research area. A smaller research department will continue Rigel’s mission to identify and develop novel small molecule therapeutics and will maintain active programs in immunology and oncology. This reduction and refocus is expected to provide approximately $17-20 million in savings annually going forward. Rigel is still assessing the full charges associated with this measure including approximately $5.7 million in cash-related restructuring expenses, which are expected to be recorded predominantly in the third quarter of 2016.
Caladrius Biosciences through its subsidiary PCT, a Caladrius Company, announced that on September 14, 2016, it entered into Securities Purchase Agreements with several accredited investors for the sale of 4,449,153 shares of its common stock in private placements of $21 million. Caladrius has a pre-existing relationship with each of these investors, including its now largest institutional shareholder and its strategic collaborator, Sanford Health. The private placements were priced at $4.72 per share of common stock, which represented the closing price of the Company’s common stock on September 13, 2016. These private placements do not include any stock purchase warrants or any future price adjustments. Sanford, which includes Sanford Research, a non-profit research organization focused on finding a cure for Type I diabetes, agreed to invest $5 million in this transaction.
Otonomy announced the successful completion of an open-label Phase IIIb clinical trial that evaluated OTIPRIO (ciprofloxacin otic suspension) in 501 pediatric patients with a history of otitis media requiring tympanostomy tube placement (TTP) surgery. The Phase IIIb trial was designed primarily to evaluate OTIPRIO in a broader pediatric patient population than the previous Phase III trials by including patients undergoing common concurrent surgeries and patients without bilateral effusion on the day of surgery. The study also collected patient insurance information to evaluate differences in outcomes for patients with Medicaid versus commercial insurance, and assessed caregiver burden via a questionnaire.
Calithera Biosciences announced that the first patient has been dosed in a Phase I clinical trial assessing the safety and efficacy of CB-1158, a first-in-class arginase inhibitor for the treatment of advanced solid tumors. Arginase is an enzyme in myeloid-derived suppressor cells (MDSCs), which prevents T-cell and natural killer (NK) cell activation in tumors.
Sanofi announced the key results of a new pharmacokinetic / pharmacodynamic (PK/PD) study in people with Type I diabetes comparing Toujeo (insulin glargine 300 Units/mL) to insulin degludec U100. The study demonstrated a more stable PK/PD profile over the dosing interval of 24 hours at steady state, in favor of Toujeo. When dosed at 0.4 Units/kg/day, patients showed a more even distribution of insulin exposure and activity with Toujeo compared to insulin degludec. With Toujeo, 67% of the study participants also achieved lower within-day variability of metabolic activity than with insulin degludec, measured by changes in the glucose infusion rate. Both insulins were measurable until the end of the observation period, at 30 hours.
Teva Pharmaceutical Industries announces a collaboration with Intel to develop a unique wearable device and machine learning platform for use in Huntington disease (HD). This platform will continuously monitor and analyze key symptoms that impact daily living, in an effort to better understand disease progression and improve treatment evaluation.
Lion Biotechnologies announced that it has entered into an exclusive license agreement with PolyBioCept AB, a corporation organized under the laws of Sweden, and a related clinical trials agreement to sponsor two clinical studies in glioblastoma and pancreatic cancer at the Karolinska University Hospital in Sweden. Under the terms of the license agreement, Lion gained exclusive worldwide rights to two international patent applications related to a specific combination of cytokines for use in the expansion, selection and enrichment of TIL products for the treatment of multiple cancer indications. Lion also has co-exclusive worldwide rights (with PolyBioCept) to make genetically engineered TIL using the cytokine cocktail for use in multiple cancer indications. Under the terms of the clinical trials agreement, Lion will fund two clinical studies in glioblastoma and pancreatic cancer to be conducted at the Karolinska University Hospital in which TIL is manufactured using the licensed combination of cytokines.
Alder BioPharmaceuticals announced the presentation of data from Phase IIb and Phase I clinical trials of ALD403 for the prevention of migraine as well as preclinical data from a case study of ALD403 and other CGRP-antibodies at the 5th European Headache and Migraine Trust International Congress (EHMTIC 2016) in Glasgow, Scotland.
Xencor announced that the first patient has been dosed in a Phase Ib multi-dose trial of subcutaneously administered XmAb 7195, a potential treatment for allergic disease.
Eisai, the U.S. pharmaceutical subsidiary of Eisai Co., Ltd., announced the launch of the Eisai Andover innovative Medicines (AiM) Institute, a discovery innovation unit within the greater Boston biopharma hub. With 90 integrated scientists at a state-of-the-art research facility in Andover, Massachusetts, the Institute is focused on executing novel therapeutic targets validated by human genetics and related data to discover and develop precision medicines for some of the hardest-to-treat medical conditions including dementia, cancer and auto-immune diseases.
TapImmune announced that its Board of Directors has approved a 1-for-12 reverse stock split of the Company’s authorized, issued, and outstanding common stock in preparation for its proposed listing of its common stock on the NASDAQ Capital Market. The Company believes that it will meet all of the listing requirements for listing the company’s common stock on the NASDAQ Capital Market. The reverse stock split will become effective with the Financial Industry Regulatory Authority (FINRA) and in the marketplace on September 16, 2016, whereupon the shares of common stock will begin trading on a split-adjusted basis.
Teva Pharmaceutical Industries announced the launch of an authorized generic of Cubicin 1 (daptomycin for injection) 500 mg per vial in the United States. Daptomycin for injection is an antibacterial drug indicated in adults for the treatment of complicated skin and skin structure infections (cSSSI) caused by susceptible isolates of the following Gram-positive bacteria: Staphylococcus aureus (including methicillin-resistant isolates), Streptococcus pyogenes, Streptococcus agalactiae, Streptococcus dysgalactiae subspecies equisimilis, and Enterococcus faecalis (vancomycin-susceptible isolates only).
OncoImmune announced that it has entered into an exclusive option and license agreement with Pfizer for ONC-392, a novel, differentiated preclinical anti-CTLA4 monoclonal antibody in a deal worth up to $250 million in upfront and potential milestone payments. Under the terms of the agreement, Pfizer plans to evaluate ONC-392 up until a certain agreed-upon time to determine whether it will exercise its option to exclusively license ONC-392 as well as any other OncoImmune anti-CTLA4 antibodies. If Pfizer exercises its option under the agreement, Pfizer would be responsible for all development and potential commercialization of the program, and OncoImmune would be eligible to receive potential developmental and commercial milestone payments as well as royalties, tiered from mid-single up to low-double digits, on sales of any potential resulting products.
Proteostasis Therapeutics announced the closing of its follow-on public offering of 5,000,000 shares of its common stock at a public offering price of $13.00 per share, before the underwriting discount. All of the shares sold were offered by Proteostasis Therapeutics, Inc. The gross proceeds from the offering are $65 million, before deducting the underwriting discount and expenses associated with the offering payable by the company. In addition, the company has granted the underwriters a 30-day option to purchase up to an additional 750,000 shares of its common stock at the public offering price, less the underwriting discount.
Global Blood Therapeutics announced publication of preclinical data supporting the development of GBT440 for the treatment of hypoxemic pulmonary disorders, including idiopathic pulmonary fibrosis (IPF), in Physiological Reports. To date, GBT has established preclinical proof of concept for GBT1118, a hemoglobin modifier and analog of GBT440, in three different animal models of hypoxia. Data from these models support the potential beneficial effects of hemoglobin modification as a promising therapeutic strategy to treat hypoxemia (abnormally low levels of oxygen in the blood) associated with chronic fibrotic lung disorders, such as IPF.
Immune Design announced that it plans to offer and sell shares of its common stock in an underwritten public offering. All of the shares in the proposed offering are to be sold by Immune Design. The offering is subject to market conditions, and there can be no assurance as to whether or when the offering may be completed, or the actual size or terms of the offering.
Cumberland Pharmaceuticals announced the initiation of a new Phase II clinical program of Portaban for patients with portal hypertension associated with liver disease. The FDA has cleared Cumberland’s IND for a multicenter, randomized, double-blind, placebo-controlled Phase II clinical study.
Thermo Fisher Scientific announced that it has priced an offering of $1.2 billion aggregate principal amount of its 2.950% senior notes due 2026 at the issue price of 98.787% of the principal amount. The issuance of the notes is expected to close on or about September 19, 2016, subject to customary closing conditions. The notes will pay interest on a semi-annual basis.
Virtus Pharmaceuticals is voluntarily recalling seven batches of Hyoscyamine sulfate (0.125mg) listed in the table below, to the consumer level which include the tablet, sublingual, and orally disintegrating tablet form. This recall is being initiated due to both superpotent and subpotent test results. All of these batches were manufactured by Pharmatech for distribution by Virtus throughout the United States and Puerto Rico.
GlaxoSmithKline announced the publication of detailed results from a randomized phase III study (ZOE-70) of its investigational shingles vaccine, Shingrix, showing 90% efficacy in adults aged 70 years and older that is maintained for at least four years. The results were published in the New England Journal of Medicine (NEJM). The study, from which headline results were reported in October 2015, showed that the two-dose candidate shingles vaccine had 90% efficacy (95% confidence interval: 84-94%) compared to placebo in people over 70 years old. Vaccine efficacy was maintained across the various age groups included in the study, ranging between 90% in people aged 70-79 years (95% confidence interval: 83-94%) and 89% in those aged 80 years and above (95% confidence interval: 74-96%).The high efficacy is in line with the results of the ZOE-50 trial, a study in people over 50 years old which was presented and published last year showing a 97% efficacy (95% confidence interval: 93-99%)2. A pooled analysis of data from both trials showed the vaccine demonstrated 91% efficacy against shingles (95% confidence interval: 86-95%) in adults aged 70 years and older compared to placebo. This efficacy was maintained with an 88% reduction in the risk of shingles (95% confidence interval: 73-95%) in the fourth year after vaccination.
Lexicon Pharmaceuticals announced that the FDA will require additional time to complete its review of the NDA for telotristat etiprate, an oral drug for the treatment of carcinoid syndrome. In a notice received from the FDA, the PDUFA date for its Priority Review of telotristat etiprate has been extended by three months, from November 30, 2016 to February 28, 2017. In response to an information request from the FDA, additional clinical data analyses have been submitted and the extension will provide the FDA time for a full review of the submission.
A joint meeting of the FDA Psychopharmacologic Drugs Advisory Committee and Drug Safety Risk Management Advisory Committee reviewed data from EAGLES (Evaluating Adverse Events in a Global Smoking Cessation Study) evaluating the neuropsychiatric safety of CHANTIX (varenicline). The Committees recommended by a majority vote to remove the boxed warning regarding serious neuropsychiatric adverse events from the CHANTIX labeling. The role of the Advisory Committees is to provide recommendations to the FDA; however, the FDA makes the final labeling decisions. Earlier this year, Pfizer submitted to the FDA a sNDA requesting updates to the CHANTIX labeling based on the safety and efficacy outcomes of EAGLES. In addition to requesting removal of the boxed warning, Pfizer proposed retaining the Warnings and Precautions section in the labeling regarding serious neuropsychiatric events occurring in patients attempting to quit smoking and updating it with EAGLES data. Pfizer believes that such a warning would sufficiently inform prescribers of the possibility that these types of events may occur.
Eisai announced that The European Commission (EC) has issued a marketing authorisation for Kisplyx (lenvatinib) in combination with everolimus for the treatment of adult patients with advanced renal cell carcinoma (RCC) following one prior vascular endothelial growth factor (VEGF)-targeted therapy. Lenvatinib was granted an accelerated assessment by the European Medicines Agency in October 2015.
Agios Pharmaceuticals announced that it has priced an underwritten public offering of 3,370,786 shares of common stock at a price to the public of $44.50 per share, before underwriting discounts, which would result in aggregate gross proceeds of approximately $150 million. All of the shares in the offering are to be sold by Agios. Agios has also granted the underwriters a 30-day option to purchase up to an additional 505,617 shares of common stock on the same terms and conditions. Closing of the offering is expected to occur on or about September 20, 2016, subject to customary closing conditions.
M Pharmaceutical announced findings from the third of three proprietary pilot studies previously conducted. The three studies relate directly to the recently acquired Chelatexx technology, referred to as the Company’s C-103 project. The third study, conducted in India, found that C-103 was over 97% effective in eliminating the GI adverse events associated with orlistat.
BONE THERAPEUTICS announced that it has received a positive ruling regarding the application of the Belgian Patent Income Deduction (PID). Under this favourable regime, 80% of all revenues from patents relating to its products PREOB and ALLOB plus two other earlier-stage products, MXBTM and JTATM, will be exempt from Belgian income tax. PREOB and ALLOB are currently in Phase II and Phase III clinical trials for a variety of indications including osteoporosis, delayed-union, spinal fusion and osteonecrosis. The 80% exemption results in a net effective tax rate of 6.8% before incorporation of losses carried forward and tax credits.
POXEL SA announced the presentation of novel data on its lead drug candidate, Imeglimin, and its direct adenosine monophosphate-activated protein kinase (AMPK) activator, PXL770, at the European Association for the Study of Diabetes (EASD) Annual Meeting in Munich, Germany. The data discussed in an oral presentation and during two poster sessions highlight important new insights on the potential of each compound for the treatment of Type II diabetes as well as related metabolic diseases.
Epizyme announced it has earned a $6 million milestone payment from GlaxoSmithKline. The milestone payment follows GSK’s initiation of patient dosing in a Phase I clinical trial of GSK3326595 (formerly EPZ015938), a first-in-class protein arginine methyltransferase-5 (PRMT5) inhibitor discovered by Epizyme and licensed to GSK. PRMT5 is a protein methyltransferase that is associated with a number of human cancers.
KemPharm announced several key corporate and clinical updates as a follow up to the Company’s second quarter 2016 business and financial update.
pSivida announced that its Board of Directors has appointed Nancy Lurker as its President and CEO and a member of the Board of Directors.
Stifel analyst Stephen Willey increased his price target of Exelixis to $15 from $12, citing continued momentum in EXEL shares ahead of next month’s CABOSUN data.
FBR analyst Christopher James initiated the following companies: Achillion Pharmaceuticals with an “outperform” rating and a $16 price target; Arena Pharmaceuticals with an “outperform” rating and $6 price target; Alexion Pharmaceuticals with an “underperform” and $82 price target.
Roth analyst Joseph Pantginis initiated Sophiris Bio with a “buy” rating and $8 price target, citing the company’s lead asset, PRX302 has the potential to address the large markets of benign prostate hyperplasia (BPH) with a positive Phase III in hand and localized prostate cancer.
Goldman analyst Salveen Richter added bluebird bio to the Conviction Buy List, citing BLUE is a leading gene/cell therapy company focused on rare disease and oncology with 3 integrated platforms: gene therapy, immunotherapy and gene editing.
Piper Jaffray analyst David Amsellem upgraded Akorn to “overweight” from “neutral” and increased his price target to $34 from $33, citing overall volume trends across the company’s commercial portfolio and a deep pipeline.
Following the announcement Vitae Pharmaceuticals was being acquired by Allergan; BMO analyst Matthew Luchini downgraded Vitae to “market perform” from “outperform.”
Goldman Sachs analyst Salveen Richter upgraded Seattle Genetics to “neutral” from “sell” and increased her price target to $47 from $30, citing revised Adcetris U.S. sales estimates in 1L HL to reflect greater market penetration per physician diligence.
Goldman Sachs analyst Salveen Richter downgraded Atara Biotherapeutics to “sell” from “neutral” and decreased her price target to $16 from $23, citing a lack of catalysts until late 2017 or after.