BioShares Biotechnology Clinical Trials (BBC): $21.69, +$0.36, +17.8% YTD

BioShares Biotechnology Products (BBP): $37.93, +$0.10, +15.8% YTD

 

 

MARKET COMMENTARY

 

Wall Street was set to follow gains in European markets after upbeat manufacturing surveys lifted investor sentiment. Euro zone private sector and manufacturing growth unexpectedly accelerated to near a six-year high in February and job creation reached its fastest since August 2007, propelled by strong demand and optimism about the future. Markit’s U.S. PMI data is due later in the day. A steep decline in shares of HSBC weighed on markets, while Asian stocks recorded a mixed trading day. Gold remained under pressure as expectations of an interest rate hike by the U.S. Federal Reserve in March helped the dollar to record broad gains against major currencies. Oil prices rose as OPEC production cuts tightened supply.

 

 

MARKET HIGHLIGHTS

 

Rockwell Medical provided an update on the commercial launch progress of Calcitriol, stating that the FDA has requested that the Company resubmit the application for the new contract manufacturer as a Prior Approval Supplement (PAS) and pay a fee for the Agency review. As a result, the Company anticipates Calcitriol to become commercially available in the U.S. market in approximately 4-6 months. Calcitriol is Rockwell’s FDA approved active Vitamin D injection for the management of hypocalcemia in patients undergoing chronic renal dialysis.

 

Minerva Neurosciences will host a Research and Development Day to highlight unmet needs, including negative symptoms and cognitive impairment, and emerging treatment strategies in schizophrenia in New York City on March 2, 2017 from 8:00 am to 9:30 am Eastern Time.

 

Neovacs has signed with BioSense Global LLC a commercial license option agreement for its IFNα Kinoid vaccine to treat lupus and dermatomyositis in China, worth up to €65 million in upfront and milestone payments, not including double-digit sales royalties.

 

Cellect Biotechnology announced positive final results from its clinical trial of ApoGraft™ in healthy donors. The study’s primary objective was to validate the Company’s propriety method of stem cell selection by going through the process of production and characterization with ApoGraft™, and was conducted on samples obtained in collaboration with two leading medical centers in Israel, The Schneider Children’s Medical Center and the Rambam Medical Center.

 

VBL Therapeutics announced full results from its exploratory Phase 2 study of VB-111 (ofranergene obadenovec) in patients with advanced, differentiated thyroid cancer. The data will be presented today by Dror Harats, M.D., CEO of VBL Therapeutics, at the Federation of the Israel Societies for Experimental Biology (FISEB) conference in Eilat, Israel.  The primary endpoint of the trial, defined as 6-month progression-free-survival (PFS-6) of 25%, was met with a dose response. Forty-seven percent (47%; 8/17) of patients in the therapeutic-dose cohort reached PFS-6, versus 25% (4/12) in the sub-therapeutic cohort, both groups meeting the primary endpoint. Reduction in tumor measurement after the first dose was seen in 44% (7/16) of patients in the therapeutic-dose cohort, compared to 9% (1/11) in the sub-therapeutic-dose cohort. An overall survival benefit was seen with a tail of more than 40% at 3.7 years for the therapeutic-dose cohort (mOS 684 days). This is similar to historical data for pazopanib* (Votrient®), a tyrosine kinase inhibitor; however, most patients in the VB-111 study had tumors that previously had progressed on pazopanib or other kinase inhibitors.

 

BrainStorm Cell Therapeutics signed an agreement with CCRM, a Toronto-based leader in developing and commercializing regenerative medicine technologies, and cell and gene therapies, to support the market authorization request for NurOwn®.  At this time, CCRM is helping Brainstorm explore the opportunity to access Health Canada’s early access pathway for treatment of patients with Amyotrophic Lateral Sclerosis (ALS).  If NurOwn® qualifies for Health Canada’s "Notice of Compliance with Conditions" pathway, it could be authorized in Canada for distribution in early 2018.

 

BioTime announced the deconsolidation of OncoCyte’s financial statements from BioTime’s consolidated financial statements effective February 17, 2017. As a result of the deconsolidation, BioTime will report a pro forma, non-cash gain of approximately $56 million. The actual amount of the gain will be determined and recorded during the quarter ending March 31, 2017. The deconsolidation occurred due to the exercise of 625,000 warrants by certain OncoCyte warrant holders on February 17, 2017. As a result of the warrant exercises, OncoCyte’s shares outstanding increased, so that BioTime no longer owns a majority of OncoCyte’s shares. BioTime owns 14,674,244 OncoCyte common shares, which currently represent approximately 49.9% of OncoCyte’s common stock outstanding. The OncoCyte shares that BioTime holds had a market value of approximately $71 million at the close of trading on February 17, 2017.

 

 

Kamada announced a collaboration with Massachusetts General Hospital (MGH) to conduct a proof of concept study evaluating the potential benefit of the Company’s Liquid Alpha-1 Antitrypsin (AAT) on liver preservation.  The study will be led by James F. Markmann M.D., Ph.D., Chief, Division of Transplant Surgery, MGH, who is the Claude E. Welch Professor of Surgery at Harvard Medical School.

 

SciClone Pharmaceuticals announced that it plans to report financial results for the fourth quarter and full year 2016 on Monday, March 6, 2017. SciClone will host a conference call and webcast to provide a business update at 8:30 am ET (5:30 am PT) that day. The call will be hosted by Friedhelm Blobel, Ph.D., President and CEO, and Wilson W. Cheung, Senior Vice President and CFO.

 

Delcath Systems announced that a retrospective, multicenter study demonstrated that 45.7 percent of patients with ocular melanoma that metastasized to the liver who underwent percutaneous hepatic perfusion (PHP) using investigational Melphalan/HDS experienced a complete or partial response. The study further showed that among those who responded to treatment, overall survival was projected to be more than three years.

 

Cidara Therapeutics reported that the randomized, controlled Phase II RADIANT clinical trial in acute vulvovaginal candidiasis (VVC) did not show sufficient efficacy to justify further development of the tested topical formulations. RADIANT was designed to evaluate gel and ointment topical formulations of the novel echinocandin antifungal CD101 in women with moderate-to-severe acute VVC.

 

Trevena announced positive top-line results from its Phase III APOLLO-1 and APOLLO-2 pivotal efficacy studies of oliceridine in moderate-to-severe acute pain following bunionectomy and abdominoplasty, respectively. In both studies, all dose regimens achieved their primary endpoint of statistically greater analgesic efficacy than placebo, as measured by responder rate. In addition, oliceridine showed dose-related trends of improvements vs. morphine on numerous measures of respiratory safety and gastrointestinal tolerability – both key unmet needs in acute pain management.

 

Valeant Pharmaceuticals International and EyeGate Pharmaceuticals announced that they have entered into an exclusive, worldwide licensing agreement through which EyeGate has granted a subsidiary of Valeant exclusive, worldwide commercial and manufacturing rights to the EyeGate II Delivery System and EGP-437 combination product candidate for the treatment of post-operative pain and inflammation in ocular surgery patients.

 

Momenta Pharmaceuticals announced that Sandoz’s contracted fill/finish manufacturing partner, Pfizer, has received an FDA warning letter. The Pfizer facility is a key part of the supply chain for the Company’s Glatopa products. Pfizer has indicated that the warning letter does not restrict the production or shipment of the Glatopa 20 mg (glatiramer acetate injection) product that is currently marketed by Sandoz in the United States.

 

Vericel announced that the FDA has designated the investigation of ixmyelocel‑T for reduction in the risk of death and cardiovascular hospitalization in patients with chronic advanced heart failure due to ischemic dilated cardiomyopathy as a Fast Track Development Program.

 

Sarepta Therapeutics announced it has entered into an agreement to sell its Rare Pediatric Disease Priority Review Voucher (PRV). Sarepta received the PRV when EXONDYS 51 was approved by the FDA for the treatment of patients with Duchenne muscular dystrophy amenable to exon 51 skipping.

 

RedHill Biopharma announced that the last patient enrolled in the Phase III study with BEKINDA 24 mg for the treatment of acute gastroenteritis and gastritis (the GUARD study) has completed the treatment course and observation period for the primary endpoint evaluation.

 

Veracyte announced that ten Blues plans have recently issued new positive coverage policies for the Afirma Gene Expression Classifier (GEC), deeming it a medically necessary benefit for their nearly 24 million collective members. The genomic test, which is used in thyroid cancer diagnosis, is now covered for nearly 225 million Americans, including more than 70 million Blues plan members.

 

Pfizer announced that a BLA for inotuzumab ozogamicin has been accepted for filing and granted Priority Review by the FDA. Inotuzumab ozogamicin is being evaluated for the treatment of adult patients with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL).

 

XBiotech announced that an Independent Data Monitoring Committee (IDMC) has performed a prospectively planned, unblinded analysis of the Phase III XCITE study for the Company’s novel candidate antibody therapy for colorectal cancer. The IDMC reported that the FDA Fast-Tracked study had no safety concerns and that indications of efficacy were sufficient to recommend proceeding with the study without modification.

 

Exelixis announced results from a Phase I trial of cabozantinib in combination with either nivolumab or nivolumab plus ipilimumab in patients with refractory genitourinary tumors. The primary endpoint of the trial is to determine the dose limiting toxicity (DLT) and recommended Phase II doses of the doublet and triplet combinations. The findings were presented during a poster session at the 2017 Genitourinary Cancers Symposium, which was being held in Orlando, Florida.

 

Genentech, a member of the Roche Group, announced encouraging results from the Phase II IMmotion150 study that compared TECENTRIQ (atezolizumab) plus Avastin (bevacizumab) and TECENTRIQ monotherapy to sunitinib alone in people with previously untreated, locally advanced or metastatic renal cell carcinoma (mRCC). These results were presented at the 2017 Genitourinary Cancers Symposium in Orlando, Fla. IMmotion150 is the first randomized clinical trial to evaluate the combination of TECENTRIQ and Avastin in mRCC. The study was designed to inform further clinical development of this combination, and these study results reinforce the potential of this combination in this setting.

 

AstraZeneca announced that it has entered into an agreement with TerSera Therapeutics for the commercial rights to Zoladex (goserelin acetate implant) in the U.S and Canada. Zoladex is an injectable luteinising hormone-releasing hormone agonist, used to treat prostate cancer, breast cancer and certain benign gynaecological disorders. It was first approved in the U.S and Canada in 1989. TerSera will pay AstraZeneca $250 million upon completion. AstraZeneca will also receive sales-related income through milestones totalling up to $70 million, as well as recurring quarterly sales-based payments at mid-teen percent of Product Sales. AstraZeneca will also manufacture and supply Zoladex to TerSera, providing a further source of ongoing income from Zoladex in the US and Canada.

 

Reuters reported that BrainStorm Cell Therapeutics is seeking early approval in Canada for its adult stem cell treatment for patients with amyotrophic lateral sclerosis (ALS), a neuro-degenerative disease, even before it completes late-stage clinical trials. BrainStorm said that it had signed an agreement with CCRM, a Canadian not-for-profit organization that supports development of regenerative medicine, to support a market authorization request for its ALS treatment, called NurOwn.

 

GenSight Biologics announced that enrollment in REVERSE, a Phase III clinical trial of GS010 in the treatment of Leber’s Hereditary Optic Neuropathy (LHON), has been successfully completed. REVERSE is the first of two parallel randomized, double-masked, sham-controlled, multi-center pivotal Phase III trials designed to evaluate the efficacy of a single intravitreal injection of GS010 (rAAV2/2-ND4) in subjects affected by LHON due to the G11778A mutation in the mitochondrial ND4 gene. REVERSE enrolled 36 patients with an onset of vision loss of 7-12 months, while RESCUE, the second trial, is currently enrolling 36 patients with an onset of vision loss of less than 6 months. Both studies are being conducted in 7 centers in Europe and in the U.S.

 

Protalex provided an update on its U.S. Phase I/II and European Phase Ib studies of PRTX-100 in adults with persistent, chronic Immune Thrombocytopenia (ITP). PRTX-100, Protalex’s lead drug candidate, is a highly-purified form of Staphylococcal protein A (SpA) and is the subject of ongoing clinical development in ITP and rheumatoid arthritis (RA). PRTX-100 has been granted Orphan Drug Designation in the U.S. and in Europe for the treatment of ITP.

 

Syros Pharmaceuticals announced that its collaborator, Andrew Lowy, M.D., Professor of Surgery and Chief of the Division of Surgical Oncology at the University of California San Diego Moores Cancer Center, presented Syros’ drug discovery research in immuno-oncology at the Moores Cancer Center Industry/Academia Translational Oncology Symposium. As part of a research collaboration in pancreatic cancer with the Lowy laboratory at the Moores Cancer Center, Syros scientists discovered alterations in regulatory regions of the genome in tumor-associated macrophages in a subset of patient tissues. Because these alterations are unique to the immunosuppressive state, they could point to genes critical for driving immunosuppression in the tumor microenvironment, as well as potential drug targets to reactivate the immune system to fight cancer.

 

Dow Jones reported that Bristol-Myers Squibb, under pressure from an activist investor, said it would name new members to its board and increase its share buybacks. Activist firm Jana Partners has been building a stake in Bristol in recent months and said last week in a filing that late last year it owned 3.9 million shares in the company. Bristol said the board appointments reflect discussions with the hedge fund. Bristol recruited former senior executives from Bausch & Lomb and Vertex Pharmaceuticals Robert Bertolini and Matthew Emmens will join the board effective immediately, along with Theodore Samuels, who currently sits on the board of Perrigo and Stamps.com. The board will be temporarily expanded to 14 seats, but only 11 directors will stand for election at the company’s annual meeting in May. Current Chairman Lamberto Andreotti will retire as previously announced. The company also said it would implement an accelerated share repurchase program.

 

CEL-SCI announced that Dr. Daniel Zimmerman, its Senior V.P of Research, Cellular Immunology, presented data with a LEAPS Rheumatoid Arthritis therapeutic (treatment) vaccine at Madridge’s International Conference on Vaccines held in Baltimore, MD on February 20-22, 2017. At the conference Dr. Zimmerman presented a potentially novel approach to fighting the inflammation in the joints caused by Rheumatoid Arthritis. He introduced animal data that demonstrates that using CEL-SCI’s L.E.A.P.S CEL-4000 Rheumatoid Arthritis treatment vaccine downregulates pro-inflammatory T-cell responses and upregulates anti-inflammatory T-cell responses. This finding was confirmed by an examination of cytokines in the serum of the animals.

 

Swedish Orphan Biovitrum announced that the company has entered into a 3-year agreement with Valeant Pharmaceuticals for the distribution of Ammonul (sodium phenyl acetate and sodium benzoate) injection in Europe, the Middle East and North Africa. The new agreement replaces the current distribution agreement with Valeant Pharmaceuticals North America LLC for the same territory. Under the new agreement, Sobi will have exclusive rights and license for sales and distribution of Ammonul in Europe, the Middle East and North Africa until 31 December 2019 for named patient use (NPU) programs.

 

Applied DNA Sciences announced a significant expansion of its U.S. and Global patent portfolio in 2016, demonstrating its ongoing commitment to innovation and leadership in the marketplace.

 

Amphastar Pharmaceuticals announced that the FDA issued a CRL for its NDA for Naloxone Hydrochloride 2mg/0.5mL Nasal Spray, indicated for the emergency treatment of known or suspected opioid overdose, as manifested by respiratory and/or central nervous system depression.

 

OncoCyte announced the receipt of $2,031,250 through the early exercise of 625,000 common stock purchase warrants.  Each warrant was exercised to purchase one share of common stock for $3.25 per share. The warrants were issued during August 2016 through a private placement of 3,246,153 units, with each unit consisting of one share of common stock and one warrant to purchase one share of common stock.

 

Loxo Oncology announced that the company has completed clinical trial enrollment for the larotrectinib NDA primary efficacy analysis. This determination is based on written feedback from the FDA, which affirmed the target enrollment goal for the primary efficacy analysis data set to support an NDA filing.

 

Bellicum Pharmaceuticals announced it has dosed the first patient with BPX-601, the first CAR T-cell product candidate to enter clinical studies that is designed to enable control over the expansion and stimulation of the cells. BPX-601 targets solid tumors that express PSCA (prostate stem cell antigen), with an initial indication in non-resectable pancreatic cancer.

 

Assembly Biosciences announced it has received early termination of the waiting period required by the Hart-Scott-Rodino Antitrust Improvements Act of 1976, closed the transaction and received the upfront payment from Allergan plc for the research, development, collaboration and license agreement between the two companies announced on January 9, 2017 (see 8-K filing dated January 10, 2017).  

 

The Medicines Company announced that the FDA has accepted for priority review the Company’s NDA filing for Carbavance for the treatment of complicated urinary tract infections. The FDA does not currently plan to hold an advisory committee meeting to discuss the application.

 

TG Therapeutics announced the publication of clinical data from a Phase I/II trial of TG-1101 (ublituximab), the Company’s novel glycoengineered anti-CD20 monoclonal antibody, in patients with B-cell non-Hodgkin Lymphoma (NHL) or Chronic Lymphocytic Leukemia (CLL) previously exposed to rituximab. The data demonstrates single agent TG-1101 to be well tolerated with the most common adverse event observed being grade 1/2 infusion related reactions (IRR), with no grade 3/4 IRRs.  TG-1101 monotherapy was active, with a 45% overall response rate (ORR) observed among heavily pretreated patients with NHL and CLL, including those who were refractory to prior anti-CD20 based therapy.  These data are described further in the manuscript titled, “A phase 1/2 trial of ublituximab, a novel, glycoengineered anti-CD20 monoclonal antibody, in patients with B-cell non-Hodgkin lymphoma or chronic lymphocytic leukaemia previously exposed to rituximab,” which was published online in the British Journal of Haematology.

 

OncoGenex Pharmaceuticals announced that apatorsen results from two randomized Phase II clinical trials were presented at the American Society of Clinical Oncology (ASCO) 2017 Genitourinary Cancers Symposium, held February 16(th) – 18(th) in Orlando. Clinical data from trials in bladder and prostate cancers demonstrated apatorsen was well-tolerated and improved patient outcomes when administered in combination with standard-of-care treatments.

 

MannKind confirmed that it received $16.7 million on February 17, 2017, upon closing the sale of real estate owned by the Company in Valencia, California, net of closing costs.

 

Aldeyra Therapeutics announced the closing of its previously announced underwritten public offering. Aldeyra sold 2,555,555 shares of its common stock, including 333,333 shares sold in connection with the exercise in full by the underwriters of their option to purchase additional shares. The net proceeds of the offering, including the full exercise of the option, were approximately $10.5 million, after deducting the underwriting discounts and commissions and the other estimated offering expenses payable by Aldeyra. Stifel and Canaccord Genuity acted as joint book-running managers and Laidlaw & Company acted as the lead manager for the offering.

 

Endocyte announced that Michael T. Andriole has been appointed as CFO.

 

 

ANALYST RECOMMENDATIONS

 

Rodman & Renshaw analyst Joseph Pantginis assumed coverage of the following companies: Inspyr Therapeutics with a “buy” rating and $2 price target; STRATA Skin Sciences with a “buy” rating and $1.50 price target.

 

The following analysts initiated coverage of Jounce Therapeutics: Baird analyst Michael Ulz with an “outperform” rating and $30 price target; Cowen analyst Boris Peaker with an “outperform” rating; Wells Fargo analyst Jim Birchenough with an “outperform” rating and valuation range of $30-$33; JP Morgan analyst Cory Kasimov with an “outperform” rating and $28 price target.

 

The following analysts initiated coverage of AnaptysBio: Wedbush analyst David Nierengarten with an “outperform” rating and $28 price target; Credit Suisse analyst Kennen MacKay with an “outperform” rating and $34 price target; JMP analyst Michael King with an “outperform” rating and $45 price target; Stifel analyst Thomas Shrader with a “buy” rating and $30 price target.

 

The following analysts initiated coverage of Observa SA: Leerink analyst Jason Gerberry with an “outperform” rating and $21 price target; Credit Suisse analyst Kennen MacKay with an “outperform” rating and $27 price target; Jefferies analyst Biren Amin with a “buy” rating and $21 price target.

 

Wedbush analyst Liana Moussatos initiated coverage of miRagen Therapeutics with an “outperform” rating and $19 price target, citing miRagen represents an attractive investment opportunity in a microRNA pipeline demonstrating early clinical efficacy with near-term catalysts and potential for multi-billion dollars in product sales.

 

Citi analyst Yigal Nochomovitz decreased his price target of Ophthotech to $5 from $7, citing model updates.

 

Following Ardelyx’s earnings, Leerink analyst Jason Gerberry increased his price target to $25 from $24.

 

Credit Suisse analyst Vamil Divan increased his price target of Teva to $42 from $41, citing news that Pfizer has received a warning letter at a plant that is involved with the production of Sandoz and Momenta’s Glatopa (generic Copaxone) products is a surprising positive for TEVA.