BioShares Biotechnology Clinical Trials (BBC): $21.52, -$0.52, +16.9% YTD

BioShares Biotechnology Products (BBP): $37.17, -$0.31, +13.5% YTD

 

 

MARKET COMMENTARY

 

U.S. stock index futures were little changed as focus shifted to the economy after pro-EU centrist Emmanuel Macron’s widely expected win in the French presidential election. European shares and the euro retreated from recent highs as the widely anticipated result of the French presidential vote spurred some profit-taking. Asian bourses mostly rose with Japanese shares hitting its highest level in 17 months. Oil prices were little changed, as the market weighed news from OPEC and other producers about prolonging output cuts against data showing the recovery in U.S. drilling. Gold prices rose on bargain-hunting.

 

 

MARKET HIGHLIGHTS

 

DURECT Corporation announced a development and commercialization agreement with Sandoz AG, a division of Novartis, to develop and market in the United States DURECT’s POSIMIR® (SABER®-Bupivacaine), an investigational locally-acting, non-opioid analgesic intended to provide up to three days of continuous pain relief after surgery.  Under the terms of the agreement, Sandoz will make an upfront payment to DURECT of $20 million, with the potential for up to an additional $43 million in development and regulatory milestones, up to an additional $230 million in sales based milestones, as well as a tiered double digit royalty on product sales in the United States. DURECT will remain responsible for the completion of the ongoing PERSIST Phase 3 clinical trial for POSIMIR as well as FDA interactions through approval.

 

TherapeuticsMD announced that it received a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) regarding the New Drug Application (NDA) for TX-004HR, the company’s investigational applicator-free estradiol vaginal softgel capsule for the treatment of moderate-to-severe vaginal pain during sexual intercourse (dyspareunia), a symptom of vulvar and vaginal atrophy (VVA) due to menopause.  In the CRL, the only approvability concern raised by the FDA was the lack of long-term endometrial safety data for TX-004HR beyond the 12-weeks studied in the pivotal phase 3 Rejoice Trial. No cases of endometrial hyperplasia were observed in the Rejoice Trial at the end of week 12 for all the doses studied and included in the NDA.  The CRL did not identify any issues related to the efficacy of TX-004HR and did not identify any approvability issues related to chemistry, manufacturing, and controls.  The Company believes that the NDA was approvable as filed and intends to meet with the FDA as soon as possible to address the concerns raised by the FDA.

 

Paratek Pharmaceuticals has completed enrollment in its pivotal Phase 3 clinical study evaluating oral-only omadacycline for the treatment of Acute Bacterial Skin and Skin Structure Infections (ABSSSI). The Company expects to report top-line data from this study in mid-July. This study is designed to assess the efficacy and safety of once-daily oral-only omadacycline compared with twice-daily oral-only linezolid in subjects with ABSSSI.

 

DelMar Pharmaceuticals announced new mechanism of action data for its anti-cancer product candidate, VAL-083 (dianhydrogalactitol), a "first-in-class" small-molecule DNA-targeting agent in temozolomide (Temodar™)-resistant GBM, at the World Federation of Neuro-Oncology Societies (WFNOS).

 

Eiger BioPharmaceuticals announced HDV/HBV co-infection prevalence data from an International Classification of Diseases (ICD) analysis presented at Digestive Disease Week (DDW) in Chicago, Illinois.  Results from two longitudinal patient databases showed a consistently growing number of newly diagnosed HDV patients in the US and a markedly higher than previously estimated incidence of HDV co-infection among chronic HBV patients in the US.

 

Lipocine announced financial results for the first quarter ended March 31, 2017.  Lipocine reported a net loss of $4.9 million, or $0.26 per diluted share, for the first quarter ended March 31, 2017, compared with a net loss of $7.0 million, or $0.38 per diluted share, in the first quarter ended March 31, 2016.  As of March 31, 2016, Lipocine had cash, cash equivalents and marketable investment securities of $26.8 million, compared with cash and cash equivalents of $26.8 million as of December 31, 2016.

 

MediWound reported financial results for the three months ended March 31, 2017.  For the first quarter of 2017, the Company’s net loss was $4.3 million, or $0.20 per share, compared with a net loss of $3.8 million, or $0.17 per share, for the first quarter of 2016.  As of March 31, 2017 the Company had cash and short-term deposits of $25.2 million, compared with cash and short-term deposits of $30.0 million as of December 31, 2016.  The Company remained on budget and utilized $4.8 million in cash to fund operating activities during the first quarter of 2017.

 

Kamada Ltd. will release financial results for the first quarter ended March 31, 2017 prior to the open of the U.S. financial markets on Tuesday, May 16.

 

Galmed Pharmaceuticals will host a conference call and webcast on Monday, May 15th, 2017, to discuss results for the period ended March 31, 2017 and to provide an update on current developments with respect to its clinical programs for Aramchol™.

 

BioTime Co-Chief Executive Officer Michael D. West, Ph.D. will present at Terrapinn’s World Advanced Therapies & Regenerative Medicine Congress, May 17-19 at Business Design Centre in London.

 

Rexahn Pharmaceuticals will present preliminary data from the Phase IIa study of RX-3117 in advanced and metastatic bladder cancer at the American Society of Clinical Oncology (ASCO) 2017 Annual Meeting, which is being held June 2-6 in Chicago, Illinois.

 

Horizon Pharma reported 1Q17 non-GAAP EPS of $0.21, which compares to $0.25 for the same period a year ago. The company missed the non-GAAP EPS mean estimate of $0.23. Total revenue for 1Q17 was $220.9 million, which compares to $204.7 million for the same period a year ago. This compares to a mean estimate of $247.9 million. 

 

Ironwood reported 1Q17 GAAP EPS loss of $0.36, which compares to a loss of $0.09 for the same period a year ago. The company missed the GAAP EPS mean estimate loss of $0.16. Total revenue for 1Q17 was $52.2 million, which compares to $66.0 million for the same period a year ago. This compares to a mean estimate of $69.7 million. 

 

Kite Pharma reported 1Q17 GAAP EPS loss of $1.74, which compares to a loss of $0.90 for the same period a year ago. The company missed the First Call GAAP EPS mean estimate loss of $1.68. Total revenue for 1Q17 was $9.8 million, which compares to $5.1 million reported for the same period a year ago. This compares to a mean estimate of $12.3 million.

 

Reuters reported that Mallinckrodt reported earnings results for first quarter of fiscal 2017. The company announced Q1 GAAP Earnings per share of $0.28 from continuing operations; Q1 sales of $810.9 million; Q1 adjusted earnings per share of $1.68. The company had Q1 earnings per share view of $1.63 and revenue view of $780.5 million, according to Thomson Reuters I/B/E/S.

 

Axsome Therapeutics received Fast Track designation from the FDA for AXS-05 for the treatment of agitation in patients with Alzheimer’s disease (AD). There are currently no approved treatments for this condition. Axsome previously received IND clearance from the FDA to proceed with a Phase II/III trial of AXS-05 in this indication.

 

OncoMed Pharmaceuticals announced first quarter financial results.  As of March 31, 2017, cash, and short-term investments totaled $156.9 million. The company is reporting top-line results from the three arm randomized Phase II “DENALI” clinical trial of demcizumab (anti-DLL4, OMP-21M18) in combination with carboplatin and pemetrexed in front-line non-squamous non-small cell lung cancer (NSCLC).  DENALI was designed to assess the efficacy and safety of either one or two 70 day truncated courses of demcizumab plus carboplatin and pemetrexed versus carboplatin and pemetrexed plus placebo.  The primary endpoint of the study was overall response rate (ORR) and secondary endpoints were clinical benefit rate (CBR; rate of complete and partial responses and stable disease), progression-free survival (PFS), overall survival (OS) and safety.  The statistical plan for DENALI was based on the expectation of enrolling 200 patients, but enrollment was discontinued at 82 patients due to the evolving treatment landscape in NSCLC. Of these 82 patients, 25 patients received carboplatin and pemetrexed plus placebo, while 57 received carboplatin, pemetrexed plus either one or two 70 day courses of demcizumab.  Although these data were not fully mature at the time of analysis, demcizumab treatment failed to meet its efficacy endpoints when compared to placebo, with better outcomes apparent in the placebo group. Specifically, the ORR was 28% versus 52% (p=0.04) and CBR was 79% versus 92% (p=0.17) in the pooled demcizumab arms and the placebo arm, respectively. Median PFS was 5.5 months versus 8.7 months (p=0.02) and mOS was 15.5 months versus not reached (p=.06) in the pooled demcizumab arms and the placebo arm, respectively.  No statistically significant differences in efficacy were observed between patients receiving one course or two courses of demcizumab. Demcizumab was generally well tolerated in combination with chemotherapy with nausea, fatigue, constipation, anemia and hypertension being the most common toxicities. There were no cases of Grade 3 or greater heart failure or pulmonary hypertension in this study.  The overall safety profile was consistent with that observed in our other studies, and no new safety signals were identified. OncoMed is discontinuing the dosing of all patients on the demcizumab trials, including the demcizumab plus pembrolizumab Phase Ib study, and will conduct a complete program review in the near term with its partner Celgene.

 

Array BioPharma announced that it has entered into a clinical trial collaboration agreement with Merck to investigate the safety and efficacy of Array’s MEK inhibitor, binimetinib, with Merck’s anti-PD-1 therapy, KEYTRUDA (pembrolizumab), in metastatic colorectal cancer patients with microsatellite stable tumors (MSS CRC). Under the terms of the agreement, Array and Merck will collaborate on a clinical trial to investigate the safety and efficacy of the combination of binimetinib with KEYTRUDA, in MSS CRC patients. The trial is expected to establish a recommended dose regimen of binimetinib and KEYTRUDA, as well as explore the preliminary anti-tumor activity of several novel regimens. The study is expected to begin in the second half of 2017. Results from this first study will be used to determine optimal approaches to further clinical development of these combinations. The collaboration agreement is between Array BioPharma and Merck, through a subsidiary. Under the agreement, the trial will be sponsored by Merck. Additional details of the collaboration were not disclosed.

 

Aptose Biosciences announced the presentation of preclinical data demonstrating that CG’806, a highly potent pan-FLT3/BTK inhibitor, exerts compelling activity against acute myeloid leukemia (AML) cells harboring mutant forms of FLT3 and eradicates AML tumors in a murine xenograft model. The data were presented in a poster on Sunday, May 7, 2017 at the 2017 American Association for Cancer Research (AACR) Conference Hematologic Malignancies: Translating Discoveries to Novel Therapies, held May 6-9 in Boston, MA.

 

Synergy Pharmaceuticals announced new insights that highlighted the frustrations many patients with chronic idiopathic constipation (CIC) feel with managing their condition. Detailed results from the BURDEN-CIC Study (Better Understanding and Recognition of the Disconnects, Experiences and Needs of Patients with Chronic Idiopathic Constipation) were presented today at Digestive Disease Week (DDW), in Chicago. Findings are based on results from an online survey of more than 1,200 patients and 325 healthcare providers (HCPs).

 

Novocure announced results from health-related quality of life analyses from its Phase III, pivotal EF-14 trial adding Optune to standard temozolomide chemotherapy for the treatment of newly diagnosed glioblastoma. The data, presented at this year’s World Federation of Neuro-oncology Societies 5(th) Quadrennial Meeting in Zurich, showed that Optune with temozolomide did not negatively impact health-related quality of life, except for itchy skin, which was most likely due to skin irritation beneath Optune’s transducer arrays. The combination treatment of Optune with temozolomide improved deterioration-free survival of several predefined health-related quality of life scales, compared to temozolomide alone, likely related to improved progression free and overall survival.

 

Intra-Cellular Therapies announced it had two presentations on its novel investigational agent lumateperone at the 19th Annual Conference of the International Society for Bipolar Disorders (ISBD) held in Washington, DC, May 4-7, 2017.

 

Ipsen announced that it has completed its previously announced acquisition of a portfolio of five consumer healthcare products from Sanofi

 

Oncolytics Biotech announced that the FDA has granted Fast Track designation for REOLYSIN, the Company’s proprietary immuno-oncology viral agent, for the treatment of metastatic breast cancer.

 

Inovio Pharmaceuticals and Regeneron Pharmaceuticals announced a clinical study agreement for a Phase Ib/IIa immuno-oncology trial. The study will be conducted by Inovio in patients with newly diagnosed glioblastoma multiforme (GBM) and will evaluate Regeneron’s PD-1 inhibitor, REGN2810, in combination with Inovio’s INO-5401 T cell activating immunotherapy encoding multiple antigens and INO-9012, an immune activator encoding IL-12.

 

Ocular Therapeutix presented new data from its most recent Phase III study evaluating the safety and efficacy of DEXTENZA (dexamethasone insert) 0.4 mg for the treatment of ocular pain and inflammation following cataract surgery. The data were released at the American Society of Cataract and Refractive Surgery Annual Symposium (ASCRS) in Los Angeles, CA.

 

CRISPR Therapeutics announced they have signed an exclusive license with the Massachusetts Institute of Technology (MIT) for a family of Lipid Nanoparticle (LNP) technologies developed in the lab of Dr. Daniel G. Anderson, a scientific founder and advisory board member of CRISPR Therapeutics. Under the terms of the license, CRISPR Therapeutics obtains the exclusive rights to use the LNP technologies in their therapeutic development programs focused on in vivo gene editing applications. MIT receives an upfront technology access fee, milestones, and royalties on licensed products that reach the market.

 

Fibrocell Science announced that the Data Safety Monitoring Board (DSMB) has recommended continuation of the Phase I/II clinical trial of FCX-007 for the treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB), following a review of safety data from the first patient treated.  No product-related adverse events were reported.

 

InVivo Therapeutics announced that a new patient has been enrolled into The INSPIRE Study (InVivo Study of Probable Benefit of the Neuro-Spinal Scaffold for Safety and Neurologic Recovery in Subjects with Complete Thoracic AIS A Spinal Cord Injury) at Oregon Health & Science University (OHSU) in Portland, Oregon. Jason J. Chang, M.D., Assistant Professor of Neurological Surgery and co-study investigator, performed the surgery and implantation approximately 77 hours after the injury occurred.

 

Takeda Pharmaceutical that data from the pivotal Phase II ALTA (ALK in Lung Cancer Trial of AP26113) clinical trial evaluating ALUNBRIG (brigatinib) in patients with crizotinib-refractory anaplastic lymphoma kinase-positive (ALK+) locally advanced or metastatic non-small cell lung cancer (NSCLC) were published in the Journal of Clinical Oncology. The study found that, for the patients who received brigatinib at 180 mg once daily following a seven-day lead-in at 90 mg once daily, the Independent Review Committee (IRC) assessed confirmed objective response rate (ORR) was 53 percent. Additionally, 67 percent of patients with measurable brain metastases who received this dosing regimen achieved a confirmed intracranial objective response. Takeda recently received Accelerated Approval from the FDA for ALUNBRIG for the treatment of ALK+ metastatic NSCLC patients who have progressed on or are intolerant to crizotinib. This indication is approved under Accelerated Approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

 

Audentes Therapeutics announced the appointment of John T. Gray, Ph.D. to Senior Vice President and Chief Scientific Officer.  Dr. Gray joined Audentes in 2014 as Vice President, Research and Development, and has successfully built and led teams across the Company’s molecular biology, process development, and nonclinical research and development functions.

 

Capricor Therapeutics announced that it has signed definitive agreements for the sale of approximately $3.7 million of the Company’s common stock with certain accredited investors. The private placement is being led by Cedars-Sinai Medical Center and includes select members of the Company’s Board of Directors. The investors have agreed to purchase 1,196,291 shares of common stock at a price of $3.10 per share. Gross proceeds from the private placement are expected to be approximately $3.7 million. The closing of the offering is expected to take place on or about May 10, 2017.

 

Syros Pharmaceuticals announced new preclinical data that further elucidates the mechanism of action of SY-1425, its oral first-in-class selective retinoic acid receptor alpha (RAR?) agonist currently in a Phase II clinical trial in genomically defined subsets of patients with acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS). These data demonstrate that SY-1425 represses genes known to be associated with the proliferation of AML cells, while activating genes critical for driving normal cell differentiation. These data were highlighted in an oral presentation during a plenary session at the American Association for Cancer Research (AACR) Hematologic Malignancies: Translating Discoveries to Novel Therapies conference.

 

Rasna Therapeutics provided an update from further studies towards the development of formulated RASP-101, a novel modality for treatment of NPM1-mutated acute myeloid leukemia (AML).

 

AVEO Oncology announced that it received a Notice of Allowance from the USPTO for U.S. patent application number 14/653,684, entitled "notch binding agents and antagonists and methods of use thereof." Allowed under the application are composition of matter and method of use claims related to the Company’s humanized anti-Notch 3 antibodies, including AV-353. The U.S. patent scheduled to issue from this application will expire December 19, 2032, with the potential for extension to December 19, 2037.

 

BriaCell Therapeutics announced dosing of the first patient in BriaCell’s Phase I/IIa clinical trial evaluating the Company’s lead product candidate, BriaVax, a genetically engineered whole-cell vaccine derived from a human breast tumor cell line, in patients with advanced breast cancer.

 

Therapix Biosciences announced that it entered into a trial agreement with the Hannover Medical School to conduct a proof-of-concept Phase II clinical study with its lead compound, THX-TS01, for patients suffering from Tourette Syndrome.

 

Regeneron Pharmaceuticals and SillaJen announced a new clinical and supply agreement for a Phase Ib dose-escalation study in renal cell carcinoma (RCC), or kidney cancer. The study will evaluate Regeneron’s PD-1 inhibitor, REGN2810, in combination with SillaJen’s oncolytic vaccinia virus, Pexa-Vec, in patients with previously treated metastatic or unresectable renal cell carcinoma.

 

Janssen Pharmaceuticals announced real-world evidence showing people with Type II diabetes initiated on oral INVOKANA (canagliflozin) 300 mg (INVOKANA) were as likely as matched patients initiated on injectable GLP-1 receptor agonists (GLP-1 RAs) to achieve the recognized HEDIS standard of blood glucose control of A1C less than 8.0 percent. Patients on INVOKANA were also less likely to discontinue their medication or be prescribed a new antihyperglycemic agent (AHA). These findings with INVOKANA, a sodium glucose co-transporter 2 (SGLT2) inhibitor, were presented at the American Association of Clinical Endocrinologists’ 26th Annual Scientific & Clinical Congress in Austin, Texas.

 

MT Pharma America announced the FDA has granted approval of RADICAVA (edaravone), as an intravenous infusion treatment for amyotrophic lateral sclerosis (ALS), a rapidly progressive neurodegenerative disease in which the majority of patients die within two to five years of diagnosis. People given RADICAVA showed significantly less decline in physical function compared to placebo as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R), a validated rating instrument for monitoring the progression of disability in patients with ALS.

 

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 14(th) International Symposium on MDS in Valencia, Spain. Luspatercept is being developed as part of the global collaboration between Acceleron and Celgene.

 

Agile Therapeutics announced the presentation of additional results of its Phase III SECURE trial of its investigational low-dose combination hormone contraceptive patch, Twirla (AG200-15). Anita Nelson, MD, Professor and Chair of Obstetrics and Gynecology at the College of Osteopathic Medicine of the Pacific, presented a summary of SECURE clinical trial results, which included new data on the bleeding profile of clinical trial subjects during a poster presentation at the 2017 Annual Clinical and Scientific Meeting of the American Congress of Obstetricians and Gynecologists (ACOG) in San Diego, CA.

 

Myovant Sciences announced the presentation of data from a placebo-controlled Phase II dose-finding study conducted by Takeda Pharmaceutical in Japan evaluating the ability of relugolix to decrease heavy menstrual bleeding in women with uterine fibroids. The study met its primary endpoint with relugolix demonstrating a marked decrease in menstrual blood loss from Week 6 to Week 12 as assessed by a patient-reported outcome (p < 0.0003 at relugolix 40-mg dose vs. placebo). The findings were presented during a poster session (Abstract #17H) at the 2017 Annual Clinical and Scientific Meeting of the American Congress of Obstetricians and Gynecologists, which was held in San Diego, California.

 

PharmaCyte Biotech released an educational, interview-style Q&A article with Sarah DeMare, the Product Development Champion with Facet Life Sciences and U.S. Agent for PharmaCyte, who discusses the IND submission process for PharmaCyte’s upcoming clinical trial in locally advanced pancreatic cancer (LAPC).

 

G1 Therapeutics announced the commencement of the initial public offering of shares of its common stock pursuant to a registration statement on Form S-1 filed with the SEC. The offering consists of 6,250,000 shares of common stock being offered by the Company. In addition, the Company is expected to grant the underwriters a 30-day option to purchase up to an additional 937,500 shares of common stock at the public offering price, less the underwriting discount. The current expected initial public offering price is between $15.00 and $17.00 per share. G1 plans to list its common stock on the NASDAQ Global Market under the ticker symbol “GTHX”. The Company intends to use the net proceeds from the offering to fund the development of its two clinical-stage product candidates and a preclinical-stage product candidate, for drug manufacturing expenses, and for working capital and other general corporate purposes. J.P. Morgan Securities LLC and Cowen and Company, LLC will serve as joint book-running managers for the offering. Needham & Company, LLC and Wedbush Securities Inc. will serve as co-managers.

 

 

ANALYST RECOMMENDATIONS

 

The following analysts initiated coverage of Tocagen: Leerink analyst Michael Schmidt with a “market perform” rating and $19 price target; Stifel analyst Thomas Shrader with a “buy” rating and $24 price target.  

 

Following UltraGenyx’s earnings, Jefferies analyst Maury Raycroft increased his price target to $68 from $66; Canaccord analyst Arlinda Lee decreased her price target to $98 from $100.

 

Leerink analyst Geoffrey Porges decreased his price target of Fibrogen to $53 from $55, citing the recent capital raise and expectations of the forthcoming Q1 result.