BioShares Biotechnology Clinical Trials (BBC): $23.19, +$0.04, +26.0% YTD
BioShares Biotechnology Products (BBP): $37.75, -$0.24, +15.3% YTD
U.S. stock index futures were trading modestly higher as focus shifted towards a meeting between the U.S. and Chinese presidents scheduled later in the week. Investors will keep an eye on total vehicle sales data along with latest reading on manufacturing activity from Markit and ISM. The dollar index edged up and was holding above four-month lows hit last week, while gold prices slipped. European shares ticked higher helped by stronger oil stocks as investors turned more confident in a sector that has been a worst performer in Europe this year. Asian stocks ended higher after upbeat economic data from China and Japan. A rebound in Libyan oil production weighed on oil prices.
OncoSec Medical Incorporated presented a poster titled "Intratumoral Delivery of a P2A-linked Bicistronic IL-12 Construct Leads to High Intratumoral Expression and Systemic Anti-tumor Response" (Abstract ID # 1614) at the American Association of Cancer Research (AACR) Annual Meeting, in Washington, D.C. The poster included preclinical data demonstrating the latest developments of OncoSec’s gene delivery platform in a murine melanoma model.
DURECT Corporation will be presenting at the H.C. Wainwright NASH Investor Conference on Monday, April 3 at 4:40 p.m. Eastern Time.
VBL Therapeutics announced that in a post-hoc, hypothesis-driven analysis of data from completed Phase 2 studies, VB-201 appears to reduce certain liver enzymes. The analysis was conducted following the effect seen with VB-201 in pre-clinical models for non-alcoholic steatohepatitis (NASH) and renal fibrosis, in which VB-201 and VB-703, a next-generation Lecinoxoid drug candidate, reduced inflammation and fibrosis without affecting the lipid profile or steatosis. The data will be presented today at the H.C. Wainwright 1st Annual NASH Investor Conference, at the St. Regis Hotel in New York City.
Cellect Biotechnology has received a formal notice of Intention to Grant for a patent (Application No. 11751949.6-1466) covering a key method of treatment from the European Patent Office. The allowed claims relate to the engineering of regulatory immune cells with enhanced apoptotic activity to be used for immunomodulation in treating or preventing immune-related disorders.
Kitov Pharmaceuticals Holdings announced today that the U.S. Food and Drug Administration has granted Kitov a waiver related to the $2,038,100 New Drug Application (NDA 210045) filing fee for KIT-302. KIT-302 is Kitov’s patented combination of Amlodipine Besylate-Celecoxib tablets, intended to treat osteoarthritis pain and hypertension simultaneously. The fee waiver, which Kitov requested in accordance with sections 736(d)(1)(D) of the Federal Food, Drug and Cosmetic Act, is granted to a small business for its first human drug application submitted to the FDA for review. The Company will not be required to remit the NDA filing fee, provided that the marketing application for KIT-302 is filed prior to March 27, 2018.
Kiadis Pharma N.V. has obtained regulatory approval from the national authority in Belgium (the FAGG, the Federal Agency for Medicines and Health Products) to start its randomized, controlled, transatlantic Phase III clinical trial with ATIR101™ for acute leukemia (CR-AIR-009) in Belgium. In addition, the Company has received regulatory approval from the national authority in Germany (the PEI, the Paul-Ehrlich-Institute) to start its Phase I/II clinical trial with ATIR201™ for thalassemia (CR-BD-001).
Prothena presented clinical results from its Phase Ib multiple ascending dose study of PRX002/RG7935 in patients with Parkinson’s disease. PRX002, also known as RG7935, is an antibody under investigation as a potentially disease-modifying treatment for Parkinson’s disease and is the focus of a worldwide collaboration between Prothena and Roche. The study results were presented as part of a late-breaking oral session at the 13th International Conference on Alzheimer’s and Parkinson’s Diseases (AD/PD) in Vienna, Austria, by Joseph Jankovic, MD, Professor of Neurology, Distinguished Chair in Movement Disorders at Baylor College of Medicine, Houston, Texas. As highlighted in the November 2016 topline press release, PRX002/RG7935 was found to have an acceptable safety and tolerability profile in patients with Parkinson’s disease, meeting the primary objective of this study. Robust CNS penetration was demonstrated by a dose-dependent increase in PRX002/RG7935 levels in CSF, and a mean concentration of PRX002/RG7935 in CSF of 0.3 percent relative to serum across all dose levels. Additional results showed a rapid, dose- and time dependent mean reduction of free serum alpha-synuclein levels of up to 97 percent after a single dose, which were statistically significant (p<0.0001), and maintained following two additional monthly doses.
Novocure announced final results from its Phase III pivotal EF-14 trial adding Optune to standard temozolomide chemotherapy for the treatment of newly diagnosed glioblastoma (GBM). Landmark analyses show a consistent and maintained improvement in overall survival at two, three, four and five years. The final results include data from all 695 patients included in the EF-14 trial with a median follow-up of 40 months. The two-year survival rate increased from 30 percent to 43 percent for patients treated with Optune together with temozolomide versus patients treated with temozolomide alone. The five-year survival rate increased from five percent to 13 percent for patients treated with Optune together with temozolomide versus patients treated with temozolomide alone. These are the best results reported for newly diagnosed GBM patients in a Phase III trial to date and represent clinically meaningful increases in landmark survival rates (hazard ratio, 0.63; p <0. 00006).
AVEO Oncology announced receipt of a $500,000 milestone payment from CANbridge Life Sciences related to a technology transfer milestone for AV-203, AVEO’s clinical-stage ErbB3 (HER3) inhibitory antibody candidate. AV-203 has demonstrated preclinical activity in a number of different tumor models including breast, head and neck, lung, ovarian and pancreatic cancers. CANbridge is planning clinical development of AV-203 in squamous cell esophageal cancer as its initial indication.
Dynavax Technologies announced today that the FDA has informed the company that the Vaccines and Related Biological Products Advisory Committee (VRBPAC) will review HEPLISAV-B [Hepatitis B Vaccine, Recombinant (Adjuvanted)] at its meeting scheduled for July 28, 2017. The scheduled VRBPAC meeting is close to the HEPLISAV-B Prescription Drug User Fee Act (PDUFA) date of August 10, 2017 solely as a function of meeting logistics. The PDUFA date remains unchanged. The FDA will communicate specific questions for the VRBPAC to address closer to the meeting date, and will post a draft agenda and draft questions on its website 48 hours prior to the meeting. HEPLISAV-B is the company’s vaccine candidate for immunization against hepatitis B infection in adults ages 18 years of age and older.
BioCryst Pharmaceuticals announced that Mundipharma has obtained regulatory approval of Mundesine (Forodesine hydrochloride) for the treatment of relapsed/refractory PTCL (Peripheral T-Cell Lymphoma) by the Ministry of Health, Labor and Welfare in Japan. Mundesine is a Purine-nucleoside phosphorylase (PNP) inhibitor developed by BioCryst, under an exclusive license with Albert Einstein College of Medicine and Victoria Link Limited. The Ministry’s decision follows successful clinical trials and makes Japan the first country in the world to make Mundesine available for treatment of PTCL.
Kite Pharma announced two plenary presentations of positive data from the primary analysis of ZUMA-1 for its lead CAR-T candidate, axicabtagene ciloleucel, in patients with refractory aggressive B-cell non-Hodgkin lymphoma (NHL) at the 2017 American Association of Cancer Research Annual Meeting in Washington, D.C. Both presentations were given by Frederick L. Locke, M.D., the ZUMA-1 Co-Lead Investigator, and Director of Research for the Immune Cell Therapy Program at Moffitt Cancer Center in Tampa, Florida. The study met the primary endpoint of objective response rate (ORR) recorded after a single infusion of axicabtagene ciloleucel, with 82 percent (p <0.0001). These results demonstrate the treatment effect of axicabtagene ciloleucel in diffuse large B-cell lymphoma (DLBCL), primary mediastinal B-cell lymphoma (PMBCL) and transformed follicular lymphoma (TFL), which are types of aggressive NHL.
Infinity Pharmaceuticals reported updated Phase I clinical data for IPI-549, an orally administered immuno-oncology development candidate that selectively inhibits phosphoinositide-3-kinase gamma (PI3K-gamma). These Phase I clinical dose-escalation data demonstrated that IPI-549 was well tolerated both as a monotherapy and in combination with Opdivo (nivolumab), a PD-1 immune checkpoint inhibitor. Additionally, data from the more advanced monotherapy module of the study showed IPI-549 has a favorable pharmacokinetic (PK) and pharmacodynamic (PD) profile that supports once daily (QD) dosing. The data included 15 evaluable patients who received IPI-549 as a monotherapy and six evaluable patients who received IPI-549 in combination with Opdivo. The data were presented at the American Association for Cancer Research (AACR) Annual Meeting 2017 taking place in Washington, D.C., April 1 – 5. IPI-549 is believed to be the only selective PI3K-gamma inhibitor in clinical development.
Antares Pharma announced that data from the 52-week pharmacokinetics and safety Phase III study of subcutaneous testosterone enanthate delivered through the QuickShot auto injector was selected for a moderated poster presentation at the Endocrine Society Annual Meeting (ENDO 2017). The poster will be presented today, April 3, 2017.
Cyclacel Pharmaceuticals announced the presentation by independent investigators of preclinical data demonstrating therapeutic potential of CYC065, the Company’s second-generation, cyclin-dependent kinase (CDK) 2/9 inhibitor, as a targeted anti-cancer agent. The data show that CYC065 substantially inhibited growth, triggered apoptosis, and induced anaphase catastrophe in murine and human lung cancer cells with known high metastatic potential. This was in marked contrast to effects in immortalized pulmonary epithelial murine and human cells. CYC065 markedly inhibited migration and invasion of lung cancer cells and affected distinctive pathways involved in DNA damage response, apoptosis, cell cycle regulation and cell migration. The data were presented at the American Association for Cancer Research (AACR) Annual Meeting 2017, April 1 – 5, 2017, in Washington, D.C.
Seattle Genetics highlighted multiple data presentations that support the company’s advancing antibody-drug conjugate (ADC) and immuno-oncology programs at the upcoming 108(th) Annual Meeting of the American Association for Cancer Research (AACR) being held April 1-5, 2017, in Washington, D.C. The presentations describe the ability of ADCETRIS (brentuximab vedotin) to activate antitumor immune responses, supporting continued clinical evaluation in combination with checkpoint inhibitors. Additionally, preclinical data feature two immuno-oncology agents, SEA-CD40 and SGN-2FF, both of which are in Phase I trials. Seattle Genetics and Unum Therapeutics are presenting preclinical data evaluating combination treatment with Antibody-Coupled T cell Receptor (ACTR) engineered autologous T cells and an antibody targeting B-cell maturation antigen (BCMA), SEA-BCMA, for multiple myeloma. Further data highlight clinical biomarker analyses for vadastuximab talirine (SGN-CD33A; 33A), an ADC under evaluation in the global Phase III CASCADE trial for acute myeloid leukemia (AML).
Sunovion Pharmaceuticals announced that Utibron Neohaler (indacaterol/glycopyrrolate) inhalation powder is now available at pharmacies in the United States for the long-term maintenance treatment of airflow obstruction in people with chronic obstructive pulmonary disease (COPD), including chronic bronchitis and/or emphysema. UTIBRON NEOHALER is not indicated to treat asthma or for the relief of sudden symptoms of COPD.
Five Prime Therapeutics announced that a poster featuring preclinical data related to Five Prime’s CSF-1R antibody, cabiralizumab (FPA008), was presented at the 2017 American Association for Cancer Research (AACR) Annual Meeting in Washington, D.C.
Janssen Pharmaceuticals announced it has collaborated with Premier on the first and largest study of its kind to address an unmet medical need for hospitalized patients with atrial fibrillation (AF) who are at risk for ischemic stroke. Named QUANTUM AF (Quantify Use of ANTicoagUlation to improve Management of AF), the study will evaluate the effect of a structured hospital quality improvement (QI) program on oral anticoagulant (OAC) use in these patients.
Sunesis Pharmaceuticals announced results from an Ohio State University-sponsored preclinical study evaluating the efficacy of non-covalent BTK inhibitor SNS-062 in chronic lymphocytic leukemia (CLL) proprietary cell lines and patient samples. The study demonstrated that, unlike ibrutinib, SNS-062 inhibition of BTK signaling is unaffected by the presence of the C481S mutation and may address acquired resistance to covalent BTK inhibitors. The results are being presented in a poster session titled “Reversal of Drug Resistance” on Monday, April 3, 2017 from 8:00 AM to 12:00 PM ET at the American Association for Cancer Research Annual Meeting in Washington, D.C.
Tetraphase Pharmaceuticals announced completion of enrollment in IGNITE4, its ongoing Phase III clinical trial evaluating the efficacy and safety of intravenous (IV) eravacycline compared to meropenem in complicated intra-abdominal infections (cIAI). The Company expects to report top-line data from this trial in the third quarter of 2017.
XBiotech announced top-line results today from its double-blind, placebo-controlled, Phase I-II study evaluating the safety and efficacy of its FDA Fast Tracked true human antibody (514G3) for the treatment of Staphylococcus aureus bloodstream infections.
MEI Pharma announced the appointment of Brian Drazba as Chief Financial Officer.
Halozyme Therapeutics demonstrated in preclinical models that its investigational drug PEGPH20 increases the number of cancer-fighting white blood cells accumulating in the tumor and the effectiveness of immunotherapies. The research was presented at the 108(th) annual meeting of the American Association of Cancer Research (AACR) and builds upon prior preclinical findings.
Audentes Therapeutics announced that the FDA has cleared the IND application for AT132, the Company’s gene therapy product candidate to treat X-Linked Myotubular Myopathy (XLMTM). The IND is now active and Audentes plans to initiate ASPIRO, the multicenter, multinational, open-label, ascending dose Phase I/II clinical study of AT132. Preliminary data from ASPIRO is expected to be available in the fourth quarter of 2017.
Syros Pharmaceuticals announced that SY-1365, its first-in-class selective cyclin-dependent kinase 7 (CDK7) inhibitor, shows significant anti-proliferative activity in multiple in vitro and in vivo models of difficult-to-treat solid tumors, including triple negative breast, small cell lung and ovarian cancers. Leveraging its expertise in transcriptional biology and chemistry, Syros also showcased its work to further elucidate the biology of cyclin-dependent kinase 12 (CDK12) and cyclin-dependent kinase 13 (CDK13), advancing its aim of designing the first highly selective CDK12 and CDK13 inhibitors suitable for clinical development. These data were presented at the American Association of Cancer Research (AACR) Annual Meeting in Washington, D.C.
Peregrine Pharmaceuticals announced the presentation of positive new data from the company’s ongoing collaboration with researchers from Memorial Sloan Kettering Cancer Center (MSK). Presented preclinical study results highlighted the potential of the company’s phosphatidylserine (PS)-targeting antibodies to enhance the anti-tumor activity of adoptive T cell transfer therapy without triggering any off-target toxicities. Data were presented this morning by MSK researchers at the 2017 Annual Meeting of the American Association for Cancer Research (AACR), which is being held April 1-5, 2017 in Washington, D.C.
Aytu BioScience announced the divestiture of Primsol, an oral antibiotic solution for urinary tract infections, to Allegis Holdings. The sale price of $1.75 million was paid in cash upon closing on March 31, 2017. Primsol will remain on the market during the transition. Aytu and Allegis will work collectively over the coming weeks to effectively transition Primsol to Allegis.
Biocept announced that it has entered into a Preferred Provider Collaboration and Services Agreement with Oregon Health & Sciences University on behalf of the OHSU Knight Cancer Institute (collectively "OHSU"). The multiphase agreement grants OHSU the rights to commercially offer Biocept’s Target Selector liquid biopsy testing services exclusively throughout the state of Oregon. Additionally, Biocept and OHSU plan to engage in technology transfer, whereby OHSU will have the ability to use Target Selector assays in-house, and act as a secondary laboratory for Biocept’s research and testing activities. Biocept and OHSU also plan to co-develop additional liquid biopsy assay technologies and platform capabilities including highly sensitive, multiplexed assay panels for molecular biomarker detection and assessment.
Bristol-Myers Squibb announced the first report of five-year overall survival (OS) data from the Phase I dose-ranging study CA209-003 evaluating Opdivo in patients with previously treated advanced non-small cell lung cancer (NSCLC; n=129). Overall survival was an exploratory endpoint in this study. The estimated OS rate at five years was 16% in heavily pre-treated NSCLC patients; survival was observed across PD-L1 expression levels and tumor histologies. The safety profile of Opdivo from this study was previously reported; no new safety signals were identified in this analysis. These data were featured today during the official press program at the American Association for Cancer Research (AACR) Annual Meeting 2017 in Washington, D.C.
Bristol-Myers Squibb announced the first overall survival (OS) data from the Phase III CheckMate -067 clinical trial. With a minimum follow-up of 28 months, the median OS had not yet been reached in either of the two Opdivo treatment groups and was 20 months for the Yervoy monotherapy group (95% CI: 17.1-24.6). Opdivo in combination with Yervoy and as a monotherapy reduced the risk of death 45% [hazard ratio (HR) 0.55; 95% CI: 0.42-0.72; P <0.0001] and 37% (HR 0.63; 95% CI: 0.48-0.81; P <0.0001), respectively, compared with Yervoy alone. The two-year OS rates were 64% for the Opdivo plus Yervoy combination, 59% for Opdivo alone and 45% for Yervoy alone. Results will be presented in the press program and an oral presentation during the Update, Novel Indication, and New Immuno-oncology Clinical Trials session from 3:35 to 3:50 p.m. ET (Late-Breaking Abstract CT075) at the American Association for Cancer Research Meeting 2017 in Washington, D.C.
AVEO Pharmaceuticals announced the closing of its previously announced underwritten public offering of 34,500,000 shares of common stock, including the exercise in full by the underwriter of its option to purchase 4,500,000 shares at the public offering price of $0.50 per share. The exercise of the option increased the amount of net proceeds raised in the offering, after underwriting discounts and estimated offering expenses, to approximately $15.5 million. The proceeds of the offering are expected to be used for working capital and general corporate purposes, including development and pre-commercial expenses incurred in connection with the TIVO-3 trial, AVEO’s ongoing Phase III clinical trial of tivozanib in the third-line treatment of patients with refractory renal cell carcinoma (RCC), for which top line data is anticipated in the first quarter of 2018, and the TiNivo trial, AVEO’s ongoing Phase I/II clinical trial of tivozanib in combination with Opdivo (nivolumab). Piper Jaffray & Co.acted as sole manager for the offering. Joseph Gunnar & Co. acted as financial advisor to AVEO in connection with the offering.
Bayer announced positive data on its investigational compound copanlisib, an intravenous pan-Class I phosphatidylinositol-3-kinase (PI3K) inhibitor with predominant inhibitory activity against PI3K-α and PI3K-δ isoforms. The Phase II CHRONOS-1 trial, an open-label, single-arm study evaluating patients with relapsed or refractory indolent non-Hodgkin’s lymphoma (iNHL), met its primary endpoint of a pre-specified objective response rate (ORR). The results across all patient groups show an ORR of 59.2%, with a 12% complete response (CR) rate and a median
Mylan announced that Meridian Medical Technologies, a Pfizer company and Mylan’smanufacturing partner for EpiPen Auto-Injector, has expanded a voluntary recall of select lots of EpiPen (epinephrine injection, USP) and EpiPen Jr (epinephrine injection, USP) Auto-Injectors to now include additional lots distributed in the U.S. and other markets in consultation with the FDA.
Amgen and UCB announced results from the fourth year of a Phase II study showing the efficacy and safety of a second course of treatment with EVENITY (romosozumab), an investigational agent for postmenopausal women with osteoporosis. The results were presented in an oral session (OR08-1) at ENDO 2017, the Endocrine Society’s Annual Meeting in Orlando, Fla.
Bristol-Myers Squibb announced the companies have agreed to advance their clinical development program evaluating the combination of epacadostat, Incyte’s investigational oral selective IDO1 enzyme inhibitor, with Opdivo, Bristol-Myers Squibb’s PD-1 immune checkpoint inhibitor, into Phase III registrational studies in first-line non-small cell lung cancer across the spectrum of PD-L1 expression and first-line head and neck cancer. Additionally, the companies are expanding the ECHO-204 Phase I/II study, established under a collaboration between the companies in 2014, to include anti-PD-1/PD-L1 relapsed/refractory melanoma cohorts. The expanded clinical development program, including the Phase III registrational studies, will be co-funded by the two companies.
Puma Biotechnology announced that interim results from the Phase Ib/II FB-10 clinical trial of Puma’s investigational drug PB272 (neratinib) given in combination with the antibody drug conjugate T-DM1 (Kadcyla, ado-trastuzumab emtansine) were presented at the 2017 American Association for Cancer Research Annual Meeting (AACR) that is currently taking place in Washington, D.C. The presentation entitled, “NSABP FB-10: Phase Ib dose-escalation study evaluating trastuzumab emtansine (T-DM1) with neratinib in women with metastatic HER2-positive breast cancer” was selected for an oral presentation.
United Therapeutics announced that regulatory delays will result in postponement of the planned U.S. launch of the RemoSynch Implantable System for Remodulin until 2018.
Piramal Imaging and AC Immune presented the pre-clinical profile and first clinical data of the investigational next generation tau PET-imaging tracer PI-2620 at the International conference on Alzheimer’s and Parkinson’s Diseases and Related Neurological Disorders (AD/PD) , in Vienna, Austria. These results are an important step in Piramal Imaging’s mission to develop molecular imaging innovations for an earlier and more accurate diagnosis of Alzheimer’s disease and other neurodegenerative disorders. PI-2620 is the result of a research collaboration between the two companies.
Sucampo Pharmaceuticals and Vtesse announced that Sucampo has acquired Vtesse for upfront consideration of $200 million. Sucampo funded the acquisition through the issuance of 2,782,678 shares of Sucampo Class A common stock and $170 million of cash on hand; no external financing was utilized.
Ascendis Pharma announced data supporting its product pipeline presented at ENDO 2017, the annual meeting of the Endocrine Society in Orlando, Florida.
Radius Health presented results from new analyses of the Phase III ACTIVE clinical trial of abaloparatide-SC, one during a late-breaker oral session at ENDO 2017, in Orlando, Fla., and the other in a late-breaker poster presentation.
Intrexon, ZIOPHARM Oncology, and Merck KGaA, announced an update on the development of next-generation chimeric antigen receptor T cell (CAR-T) therapy for cancer as part of their strategic collaboration and license agreement.
Tonix Pharmaceuticals announced that it has regained compliance with the minimum bid price requirement for continued listing on the NASDAQ Global Market. On March 17, 2017, Tonix effected a 1-for-10 reverse stock split of its outstanding common stock intended to increase the per share trading price of Tonix’s common stock to satisfy the $1.00 minimum bid price requirement of $1.00 per share for continued listing on the NASDAQ Global Market, as set forth in NASDAQ Listing Rule 5450(a)(1) (the “Bid Price Rule”).
Anavex Life Sciences announced the presentation of new mechanism of action data related to ANAVEX compounds targeting the sigma-1 receptor at the AD/PD(TM) 2017 Meeting.
ProQR Therapeutics announced the grant of two key patents protecting QR-010 in the US and EU. These patents provide the Company exclusive rights for QR-010 for the treatment of cystic fibrosis (CF) until at least July 2033.
Sierra Oncology reported that preclinical results for its Chk1 inhibitor, SRA737, were presented on April 2 in a poster at the American Association of Cancer Research (AACR) Annual Meeting being held in Washington D.C. This research was conducted in the laboratory of Professor Paul Workman, Chief Executive and President of The Institute of Cancer Research (ICR), London, UK, and funded by Wellcome.
Zymeworks announced that it has filed a registration statement on Form F-1 (the “Registration Statement”) with the U.S. Securities and Exchange Commission (the “SEC”) and a preliminary prospectus with the securities regulatory authorities in each of the provinces and territories of Canada for a proposed initial public offering of its common shares. The number of common shares to be sold and the price range for the proposed offering have not yet been determined. Zymeworks has applied to list its common shares on the New York Stock Exchange and intends to apply to list its common shares on the Toronto Stock Exchange, under the ticker symbol “ZYME” for both exchanges.
Amicus Therapeutics has completed enrollment in the ongoing Phase III clinical study (ESSENCE) of the novel topical medicine SD-101 for patients with all 3 major types of epidermolysis bullosa (EB) (Simplex, Recessive Dystrophic, and Junctional non-Herlitz EB). With the achievement of full enrollment, top-line data from this study are expected in the third quarter of 2017.
Invitae announced a program in partnership with Alnylam Pharmaceuticals to provide genetic testing for hereditary ATTR (hATTR) amyloidosis, a rare, progressive and life-threatening disease caused by a mutation in the TTR gene which causes accumulation of misfolded proteins in nerves and cells of other organs.
Versartis announced that new data on somavaratan in pediatric and adult GHD were presented during the Endocrine Society’s 99th Annual Meeting & Expo (ENDO 2017), being held April 1-4, 2017 in Orlando, Florida. Somavaratan was featured in four posters and an oral session over the weekend and three-year safety and efficacy results from the ongoing VISTA pediatric long-term safety study are scheduled to be presented during an oral session today from 11:15-12:45 ET.
Novartis announced the EC has approved Tafinlar (dabrafenib) in combination with Mekinist (trametinib) for the treatment of patients with BRAF V600-positive advanced or metastatic non-small cell lung cancer (NSCLC). The approval marks the first targeted treatment approved for the patient population, who previously had few treatment options, in all 28 member states of the EU, plus Iceland and Norway.
Diffusion Pharmaceuticals announced that it entered into subscription agreements for the sale of an aggregate of 4,558,030 shares of its Series A convertible preferred stock in a private offering for gross proceeds of approximately $9.2 million, prior to deducting placement agent fees and estimated expenses payable by Diffusion. The closing is the second and final closing of Diffusion’s previously announced private placement. The private placement was oversubscribed as the original raise targeted $15.0 million. Together with the shares issued at and gross proceeds from the initial closing, Diffusion issued an aggregate of 12,395,053 shares of Series A convertible preferred stock for aggregate gross proceeds of approximately $25.0 million in the private placement. The Series A convertible preferred stock is initially convertible into one share of the Diffusion’s common stock at a conversion price equal to the purchase price of $2.02 per share. In addition, each investor received a 5-year warrant to purchase one share of common stock for each share of Series A preferred stock purchased by such investor at an exercise price equal to $2.22, subject to adjustment thereunder. Maxim Merchant Capital acted as the Company’s placement agent in the Private Placement.
Sarepta Therapeutics announced the appointment of Catherine Stehman-Breen, M.D., M.S., as chief medical officer.
ESSA Pharma provided an update on the status of its Phase I clinical study of EPI-506 for patients with metastatic castration-resistant prostate cancer ("mCRPC"). EPI-506 targets the N-terminal domain ("NTD") of the androgen receptor (AR) – a novel approach to AR inhibition. Research into NTD AR biology is also being highlighted in three posters at the American Association for Cancer Research Annual Meeting ("AACR") in Washington, DC, April 1-5, 2017.
Kadmon Holdings announced that the Company, together with its lending syndicate, led by Perceptive Credit Opportunities Fund, entered into an amendment to its 2015 Credit Agreement and related warrants.
Morphotek, a subsidiary of Eisai, announced the launch of its Antibody Drug Conjugate (ADC) Services business based on its proprietary REsidue-SPEcific Conjugation Technology (RESPECT) and eribulin-linker toxin platforms. RESPECT is a site-specific conjugation technology that targets select amino acid residues as a way of producing investigational homogeneous ADCs with defined drug-to-antibody ratios. The platform allows for site-specific conjugation of a single cytotoxic payload or two payloads with different mechanisms of action. The platform employs eribulin as one of the cytotoxic payloads along with a proprietary high-throughput screening system that can evaluate multiple ADC products simultaneously for client-desired biophysical properties and target-specific binding. As part of our services, the eribulin-linker payload is offered as an option to develop investigational ADCs using traditional bioconjugation for companies interested in developing next-generation formats of their own antibodies.
Aptevo Therapeutics announced the presentation of new preclinical data on the Company’s next generation bispecific antibody candidates, APVO436 and APVO437, at the American Association for Cancer Research 2017 annual meeting.
Jazz Pharmaceuticals announced the completion on March 31, 2017 of a rolling submission of a NDA to the FDA for the approval of Vyxeos (cytarabine and daunorubicin) liposome for injection, an investigational treatment for acute myeloid leukemia (AML), a rapidly progressing and life-threatening blood cancer. The company has requested a priority review for the Vyxeos NDA, which, if granted, would accelerate the expected timing of the FDA’s review.
Morgan Stanley analyst Matthew Harrison initiated coverage of Loxo Oncology with an “overweight” rating and $59 price target, citing Loxo is developing larotrectinib (LOXO-101), a cancer therapy that is designed to specifically target ultra rare tropomyosin-related kinase (TRK) fusions.
Canaccord analyst Arlinda Lee increased her price target of Blueprint Medicines to $42 from $40, citing at AACR, Blueprint highlighted key features of BLU-285, and importantly, showed steadystate plasma concentration of BLU-285 at the selected expansion cohort dose of 400mg QD is in therapeutic range to inhibit both ATP binding site and activation loop KIT mutations.
Raymond James analyst Chris Raymond increased his price target of Blueprint Medicines to $50 from $41, citing ~$450M on the balance sheet after last week’s follow-on, and a recent publication suggesting that PDGFRa D842V mutations may be more prevalent in adjuvant GIST.
Janney analyst Ken Trbovich downgraded Acorda Therapeutics to “neutral” from “buy” and decreased his price target to $19 from $38, citing firm sees a generic Ampyra as likely in Q3/18.
Piper Jaffray analyst Edward Tenthoff decreased his price target of AVEO Pharmaceuticals to $1.60 from $1.75 based on dilution.
Citi analyst Robyn Karnauskas decreased her price target of Juno Therapeutics to $30 from $34, citing while JCAR015 has been discontinued for strategic reasons, the company expects to initiate a trial in adult ALL with a defined cell product next year.
Following Forward Pharma’s announcement the court decided that all FWP’s ‘187 patent claims are invalid, making Biogen the winner of the interference, JMP analyst Jason Butler downgraded Forward Pharma to “market perform” to “market outperform;” Jefferies analyst Biren Amin downgraded the stock to “hold” from “buy” and decreased his price target to $24 from $41; Leerink analyst Jason Gerberry decreased his price target to $28 from $37.
Leerink analyst Jonathan Chang resumed coverage of Calithera with an “outperform” rating and $16 price target, citing CALA is developing oncology drugs in the promising tumor and immune cell metabolism space.
Citi analyst Yigal Nochomovitz increased his price target of Portola Pharmaceuticals to $51 from $39, citing recent Ph3 successes are poised to expand the indication set for approved novel oral anticoagulants (NOAC) and should drive broader need for Portola’s bleeding reversal agent AndexXa.
Leerink analyst Michael Schmidt increased his price target of Incyte to $141 from $130, citing epacadostat is better positioned in the emerging IO combination landscape.