BioShares Biotechnology Clinical Trials (BBC): $21.66, -$0.74, +17.7% YTD

BioShares Biotechnology Products (BBP): $36.45, -$0.85, +11.3% YTD

 

 

MARKET COMMENTARY

 

Global markets extended losses and U.S. stocks pointed to a lower start for Wall Street as looming uncertainty over U.S. President Donald Trump’s political future continued to weigh on markets. Safe-haven gold hovered near a two-week high, prompted by the weaker dollar. Oil prices fell on signs that the market remained well supplied with crude despite efforts by OPEC and other big exporters to curb production and support prices.

 

 

MARKET HIGHLIGHTS

 

MediWound announced that the Board of Directors has appointed Vickie R. Driver, DPM and Stephen T. Wills, CPA to its Board of Directors, effective immediately. In addition, the Board recommends that shareholders elect Nissim Mashiach and Sharon Kochan as external directors at the upcoming shareholder meeting scheduled for June 22, 2017, as the terms of the following directors will expire in June: Sarit Firon, Avri Havron, Ph.D., Marian Gorecki, Ph.D. and Meron Mann.

 

Advaxis announced that it will host its annual Investor & Analyst Day on Monday, June 12, at 1:00 p.m. in New York, NY. The event will feature presentations followed immediately by a reception.

 

Albireo Pharma announced key study design details for its planned Phase 3 program of lead product candidate, A4250, in patients with progressive familial intrahepatic cholestasis (PFIC) determined following consultations with the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA). PFIC is a rare and life-threatening genetic liver disorder for which there are currently no approved drug therapies.

 

Aurinia Pharmaceuticals announced that the first patient has been dosed in AURORA, the Company’s Phase 3 confirmatory clinical trial evaluating voclosporin for the treatment of lupus nephritis (LN), an autoimmune disease caused by lupus that involves extreme inflammation and failure of the kidneys.

 

Oramed Pharmaceuticals announced today that  Chief Executive Officer, Nadav Kidron, will present at three conferences next week in Tel Aviv, Israel:  Oppenheimer’s 17th Annual Israeli Conference, Biomed Conference for Capital Markets, MIXiii Biomed 2017.

 

Onconova Therapeutics announced details relating to a poster presentation addressing Intravenous Rigosertib in Second-line Higher Risk MDS at the upcoming American Society of Clinical Oncology Annual Meeting taking place June 2nd-6th in Chicago.

 

Arena Pharmaceuticals will host a key opinion leader (KOL) event for investors focused on the treatment of pulmonary arterial hypertension (PAH) in New York City on Thursday, May 25, from 8C9:30 a.m. EDT.

 

The 2017 ASCO Annual Meeting abstracts were published on May 17, 2017 at 5:00 PM (EDT). A link to the abstracts can be found here.

 

GlycoMimetics announced that the FDA has granted Breakthrough Therapy designation for treatment of adult relapsed/refractory acute myeloid leukemia (AML) to the company’s drug candidate GMI-1271, an E-selectin antagonist currently being evaluated in the Phase II portion of a Phase I/II clinical trial in patients with AML. The FDA had previously granted Orphan Drug designation and Fast Track Status for GMI-1271 in AML.

 

Incyte announced the publication of new data from the ongoing ECHO-202 trial, evaluating epacadostat, Incyte’s selective IDO1 enzyme inhibitor, in combination with Keytruda (pembrolizumab), Merck’s anti-PD-1 therapy. Abstracts published online by the American Society of Clinical Oncology (ASCO) in advance of its annual meeting in Chicago, Illinois, June 2-6, 2017 include ECHO-202 Phase I/II efficacy and safety data from the following cohorts: non-small cell lung cancer (NSCLC), renal cell carcinoma (RCC), bladder cancer, squamous cell carcinoma of the head and neck (SCCHN), triple-negative breast cancer (TNBC), and ovarian cancer (OVC). Pooled Phase II safety data across cohorts were also released.

 

Syndax Pharmaceuticals announced results from the melanoma cohort of the ongoing Phase II ENCORE 601 trial of entinostat in combination with KEYTRUDA (pembrolizumab), Merck‘s anti-PD-1 (programmed death receptor-1) therapy, which will be presented at the American Society of Clinical Oncology (ASCO) Annual Meeting being held June 2-6, 2017 in Chicago, Illinois. The Company recently reported that the first cohort of 13 melanoma patients who had progressed on or after prior immune checkpoint inhibitor therapy in ENCORE 601 met the pre-specified objective response criteria, with a minimum of 2 patients demonstrating a confirmed or unconfirmed objective response, to advance into the second stage of the trial. Data from the first cohort of patients indicate that 4 patients achieved an objective response (ORR) by irRECIST criteria (3 patients had a confirmed response; 1 patient had an unconfirmed response; 31% ORR, 95% CI: 9 C 61%). Of the 4 responders, 2 patients had stable disease (SD) and 2 patients had progressive disease (PD) as best response to their prior anti-PD-1 therapy prior to progressing, with a median duration on prior anti-PD-1 therapy of 4.9 months (range 2.7-12.5). Three patients remain on treatment, without progression, as of the data cutoff, 1 with a partial response (PR), and 2 with SD. ENCORE 601 (NCT02437136) is a Phase Ib/II trial evaluating the efficacy and safety of entinostat in combination with KEYTRUDA in melanoma patients whose disease had progressed despite prior treatment with anti-PD-1/PD-L1 therapies. Responses were seen both in patients who had, as well as those who had not, received prior treatment with YERVOY (ipilimumab) in combination with an anti-PD-1 therapy, either (OPDIVO (nivolumab) or KEYTRUDA). Of note, 1 patient with a confirmed PR converted from a PD-L1 negative, non-inflamed gene signature in a pre-treatment tumor biopsy to PD-L1 positive, inflamed gene signature post-treatment with the entinostat-KEYTRUDA combination. Correlative analyses of peripheral blood and tumor tissue biomarkers across the entire patient cohort are ongoing. The combination of entinostat and KEYTRUDA also appears to have a manageable toxicity profile, with 8 patients having a treatment emergent adverse event with severity of Grade ≥ 3, and with 1 patient discontinuing treatment due to an adverse event (transaminitis that was deemed to be likely related to KEYTRUDA).  Baseline demographic data in the first cohort of 13 melanoma patients included: Median age of 62; ECOG status of 0 (62% of patients) or 1 (38% of patients), PD-L1 expression negative (31%) or positive (46%) with 23% not evaluable; visceral metastases (46%); all patients had disease progression on or after prior treatment with a PD-1 antagonist, either KEYTRUDA therapy (54%) or OPDIVO therapy (46%); prior YERVOY therapy (62%); and prior B-Raf inhibitor therapy (15%).

 

Shire announced positive topline Phase III results for the HELP study, a global, multi-center, randomized, double-blind placebo-controlled parallel group trial that evaluated the efficacy and safety of subcutaneously administered lanadelumab versus placebo over 26 weeks in patients 12 years of age or older with Hereditary Angioedema (HAE). Lanadelumab is an investigational treatment being evaluated for the prevention of angioedema attacks in patients with HAE, a rare genetic disease characterized by recurrent swelling of extremities, gastrointestinal tract, and upper airways. This study met its primary endpoint and all secondary endpoints with highly statistically significant and clinically meaningful results for all three lanadelumab treatment arms compared to placebo. The 300 mg dose administered once every two weeks resulted in a statistically significant reduction in mean HAE attack frequency of 87% compared to placebo (p <0.001). Results were consistent regardless of baseline attack rate. Notably for each of the three lanadelumab regimens studied, whether administered biweekly or monthly, a significantly higher proportion of patients-compared to placebo-were attack free throughout the entire 26 week study period.

 

Spark Therapeutics announced the completion of the rolling submission of a BLA with the FDA for voretigene neparvovec, an investigational, one-time gene therapy for the treatment of patients with vision loss due to confirmed biallelic RPE65 mutation-associated retinal disease. The BLA submission includes data from three clinical trials that enrolled 41 participants with RPE65-mediated IRD, including the first randomized, controlled Phase III trial for a gene therapy for a genetic disease. FDA will have 60 days to review the submission to determine if it is complete. If deemed complete, the application will be considered filed and the review period will begin.

 

Salix Pharmaceuticals announced the peer-reviewed publication of a study in the Journal of Crohn’s & Colitis evaluating the safety and efficacy of budesonide multimatrix (MMX), which was examined for induction of remission in patients with mild to moderate ulcerative colitis (UC) refractory to baseline mesalamine therapy. Budesonide MMX or UCERIS tablets, is a prescription corticosteroid medicine used to help get mild to moderate UC under control.

 

NANOBIOTIX announced its first set of clinical data from its immuno-oncology (IO) program, showing the potential ability of NBTXR3 to transform “cold” tumors into “hot” tumors.

 

Neuralstem announced it has completed dosing of the last subject in its multicenter Phase II clinical trial evaluating the efficacy of NSI-189 for the treatment of major depressive disorder (MDD).  NSI-189 is an oral antidepressant with a novel mechanism of action and is the lead compound in Neuralstem’s neurogenic small molecule program.

 

Vertex Pharmaceuticals announced that the FDA has approved KALYDECO (ivacaftor) for use in people with cystic fibrosis (CF) ages 2 and older who have one of 23 residual function mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. This precision medicine decision is based on analyses of in vitro data and is supported by more than five years of real-world clinical data that demonstrate KALYDECO’s strong safety and efficacy profile. More than 900 people ages 2 and older in the U.S. have one of these mutations. Based on this approval, Vertex today increased its guidance for 2017 product revenues of KALYDECO to a range of $740 million to $770 million. In addition to the mutations added to the label, Vertex is continuing discussions with the FDA concerning the approval for additional people who have mutations responsive to KALYDECO, including one of five “splice” mutations. These five mutations were evaluated as part of the previously disclosed Phase III EXPAND study in which the KALYDECO monotherapy arm met its primary efficacy endpoint while being generally well tolerated. More than 600 people ages 2 and older in the U.S. have one of these mutations.

 

ImmunoGen announced promising safety and efficacy data from monotherapy and combination studies with mirvetuximab soravtansine in patients with folate receptor alpha (FRα)-positive epithelial ovarian cancer (EOC). These data include results from pooled analyses of three Phase I expansion cohorts and from a Phase Ib/II study, FORWARD II, evaluating mirvetuximab soravtansine in combination with Avastin (bevacizumab), carboplatin, Doxil (pegylated liposomal doxorubicin), or Keytruda (pembrolizumab).

 

Heptares Therapeutics, a wholly owned subsidiary of Sosei Group, has been notified by its partner Teva that a preclinical candidate calcitonin gene-related peptide (CGRP) antagonist has been nominated for advancement into further preclinical studies as an investigational treatment for migraine.

 

Swedish Orphan Biovitrum announced that the Saudi Food & Drug Authority (SFDA) in the Kingdom of Saudi Arabia has approved Elocta (efmoroctocog alfa), a recombinant human factor VIII Fc-fusion protein with an extended half-life, for the treatment of haemophilia A.  Elocta is the first extended half-life and recombinant factor VIII Fc fusion protein therapy approved for the treatment of haemophilia A in Saudi Arabia.

 

PharmaMar announced an agreement with Singapore-based Specialised Therapeuticsto market the marine-based anti-tumour compound of the Company, lurbinectedin (PM1183) for the treatment of platinum-resistant ovarian cancer, small-cell lung cancer, BRCA 1/2 -associated metastatic breast cancer and other future oncology indications in Australia, New Zealand and in 12 Asian countries (Brunei, Cambodia, Indonesia, Laos, Malaysia, Myanmar, Papua New Guinea, Philippines, Singapore, Timor-Leste, Thailand and Vietnam).

 

Innovus Pharmaceuticals announced that the Company filed its Product License Application with Health Canada as a Natural Health Product (“NPN”) to market its current best-selling product Vesele with the indication to “Help Improve Symptoms of Sexual Dysfunction.” If accepted by Health Canada, Vesele will be available over-the-counter (“OTC”) in 120 capsule bottles and will not require a prescription. The product if approved will not be reimbursed by the Canadian government, therefore, the Company will be able to set the retail price as it does in the United States. If approved, the Company expects to directly launch the product itself through its Beyond Human sales and marketing platform currently being set up in Canada and through other sales channels.

 

Epizyme announced positive interim data on its first-in-class EZH2 inhibitor, tazemetostat, from the epithelioid sarcoma cohort of its ongoing Phase II study in adult patients with molecularly defined solid tumors. In addition, the Company announced that it recently conducted a positive meeting with the FDA to discuss the registration strategy for tazemetostat for the treatment of epithelioid sarcoma. Based on discussions with the FDA, the Company has identified a path to submission for accelerated approval of tazemetostat based on the 60-patient cohort from its Phase II study, and will target a NDA submission in 2018.

 

Soligenix announced that long-term follow-up data from its recent positive Phase II clinical trial, in which SGX942 (dusquetide) demonstrated a significant reduction in the median duration of severe oral mucositis in patients with head and neck cancer (HNC), have been published in the peer-reviewed journal Biotechnology Reports. These 12-month data further support the safety and tolerability of SGX942, with the 1.5 mg/kg treatment group demonstrating accelerated tumor resolution and a decreased mortality rate relative to the placebo group.

 

ObsEva announced the completion of a Phase I study which investigated the potential drug-drug interactions of OBE022 when given with magnesium sulfate (MgSO4), betamethasone, atosiban, or nifedipine, medications typically used in women with preterm labor.

 

Sage Therapeutics announced that the FDA has granted Fast Track Designation to SAGE-217 for development as a potential treatment for major depressive disorder (MDD). Fast Track is a process designed to facilitate the development and review of new treatments for serious conditions with unmet medical need such as MDD.

 

Remedy Pharmaceuticals announced that Biogen completed an asset purchase of its Phase III candidate, CIRARA. Biogen made an upfront payment of $120 million to Remedy and may also pay additional milestone payments and royalties. The target indication for CIRARA is large hemispheric infarction, a severe form of ischemic stroke where brain swelling often leads to disproportionately large share of stroke-related morbidity and mortality. The FDA recently granted CIRARA Orphan drug designation for severe cerebral edema in patients with acute ischemic stroke. The FDA has also granted CIRARA Fast Track designation.

 

Amgen announced the submission of a BLA to the FDA for erenumab to prevent migraine. Erenumab is specifically designed to prevent migraine by blocking the Calcitonin Gene-Related Peptide (CGRP) receptor. This BLA includes data from pivotal studies in patients with episodic and chronic migraine.

 

Shire announced publication in the May 17, 2017 issue of The Lancet the results from a Phase II study (NCT01620255) investigating PF-00547659 (now called SHP647), an anti-mucosal addressin cell adhesion molecule 1 (MAdCAM-1) antibody, investigated in the treatment of moderate-to-severe ulcerative colitis in adults.

 

Recro Pharma announced the presentation of clinical data from its Phase III study evaluating intravenous (IV) meloxicam 30mg for the treatment of acute postoperative pain in patients following bunionectomy surgery, at the American Pain Society 36th Annual Scientific Meeting, taking place May 17-20, 2017, in Pittsburgh, PA. The poster presentation highlights the clinical performance of IV meloxicam 30mg, including achievement of the study’s primary endpoint, a statistically significant difference in Summed Pain Intensity Difference (SPID) over the first 48 hours (SPID48) compared to placebo, along with detailed secondary endpoints and safety results.

 

Thermo Fisher Scientific announced that it has entered into an agreement with Agios Pharmaceuticals to develop and commercialize a next-generation sequencing (NGS) oncology companion diagnostic (CDx) for ivosidenib (AG-120) to identify isocitrate dehydrogenase 1 (IDH1) mutations in cholangiocarcinoma patients. Ivosidenib is an IDH1 inhibitor currently in a Phase III trial (ClarIDHy, NCT02989857) for the treatment of patients with advanced IDH1m positive cholangiocarcinoma.

 

Relmada Therapeutics and Laidlaw & Company (UK) Ltd. announced final disposition of all claims and counterclaims in all legal proceedings related to Relmada Therapeutics, Inc. v. Laidlaw & Company (UK) Ltd., et al., Case No.1:16-CV-07767, pending in the United States District Court for the Southern District of New York.

 

XOMA announced it has earned a $10 million milestone payment, reflecting the clinical advancement of an asset the Company licensed to one of its pharmaceutical partners.

 

 

ANALYST RECOMMENDATIONS

 

HC Wainwright analyst Ed Arce decreased his price target of Aurinia Pharmaceuticals to $10 from $12, citing dilution. 

 

Citi analyst Joel Beatty increased his price target to Syndax to $24 from $20, citing the ORR reported yesterday afternoon for Entinostat + Keytruda in melanoma, especially in light of the supportive update the day before for this same combo in lung cancer.

 

Leerink analyst Joseph Schwartz upgraded Achillion to “outperform” from “market perform” and increased his price target to $6 from $4, citing at the current stock price, there is limited downside for investors even if complement fails, assuming the HCV partnership remains intact.

 

Aegis analyst Difei Yang initiated coverage of Fennec Pharmaceuticals with a “buy” rating and a $6.80 price target, citing there is a potential new therapy for preventing cisplatin associated ototoxicity.

 

Ladenburg analyst Jeffrey Cohen upgraded Tenax Therapeutics to “buy” from “neutral” and established a $2.50 price target, citing the potential risk/reward of a filing for CABG alone potentially offer a very strong risk reward profile from the current valuation.

 

Cantor analyst Mara Goldstein decreased her price target of Ardelyx to $12 from $19, citing the read-out from T3MPO-1 (tenapanor in IBS-C), although statistically significant, represents a more modest effect than was anticipated.