BioShares Biotechnology Clinical Trials (BBC): $22.83, +$0.85, +24.0% YTD

BioShares Biotechnology Products (BBP): $38.02, +$0.08, +16.1% YTD

 

 

MARKET COMMENTARY

 

U.S. stock index futures were little changed, after the Dow went past the 21,000 mark in the previous session, lifted by the U.S. President Donald Trump’s measured tone in his first speech to Congress. Growing expectations of an interest rate hike later this month by the Federal Reserve pushed the dollar higher against a basket of major currencies, while gold prices fell. European equities edged down despite strong quarterly results from top companies, while Asian stocks closed the trading day on a mixed note. Oil fell on record U.S. crude inventories.

 

 

MARKET HIGHLIGHTS

 

BIOPHYTIS announced the complete results of the SARA-PK study, in particular the favorable pharmacokinetics and pharmacodynamics of Sarconeos.  The analyses confirm the good pharmacokinetic profile in healthy elderly volunteers, the therapeutic window of Sarconeos, and confirms the dosages that will be tested in the Phase 2b trial SARA-INT. The study’s complete results will be presented at the International ICFSR on April 28th in Barcelona.

 

VBL Therapeutics will host a conference call and live audio webcast on Monday, March 27, 2017 at 8:30am Eastern Time to report fourth quarter and full-year ended December 31, 2016 financial results.

 

Zogenix will report its financial results for the fourth quarter and full-year ended December 31, 2016, after the market close, and will host a corporate update conference call and webcast on Thursday, March 9th, at 4:30 PM Eastern Time.

 

Advaxis will host a Research Reception during the Society of Gynecologic Oncology (SGO) Annual Meeting on Women’s Cancer. The reception will be held from 5:30-7:00 p.m. ET on Monday, March 13, in National Harbor Room 15 at the Gaylord Convention Center in National Harbor, MD.

 

Rockwell Medical announced that David Domzalski, has been nominated to join the Company’s Board of Directors. Mr. Domzalski is nominated to replace Mr. Ken Holt, whose term is expiring. Mr. Holt has served the Rockwell Board for several years and will remain engaged through the end of his term.

 

Cellect Biotechnology announced the appointment of Dr. Michael Berelowitz to the Company’s Board of Directors. Cellect’s Board now includes seven members, five of whom are independent.  Dr. Berelowitz is a former Head of Clinical Development and Medical Affairs at Pfizer and brings over 40 years of clinical, development and academic research experience. Prior to Pfizer, he spent a number of years in academia and has held appointments at the University of Chicago, University of Cincinnati College of Medicine, SUNY at Stony Brook and most recently, Mount Sinai School of Medicine.

 

OncoSec Medical will host its fiscal second quarter 2017 financial results conference call on Thursday, March 16 at 1:15 PM PST/4:15 PM EST.

Innate Pharma SA announced that data on monalizumab will be presented at the American Association for Cancer Research (AACR) Annual Meeting, April 1 – 5, 2017, in Washington, D.C. Abstracts are available on the AACR website. Monalizumab is Innate Pharma`s investigational first-in-class anti-NKG2A antibody partnered with AstraZeneca.

 

Aurinia Pharmaceuticals announced top-line results from its Phase IIb AURA-LV (AURA) study in lupus nephritis (LN). At 48 weeks, the trial met the complete and partial remission (“CR”/ “PR”) endpoints, demonstrating statistically significantly greater CR and PR in patients in both low dose (23.7mg of voclosporin twice daily (p<.001)) and high dose (39.5mg twice daily (p=.026)) cohorts versus the control group.  Each arm of the study included the current standard of care of mycophenolate mofetil (MMF) as background therapy and a forced steroid taper to 5mg/day by week 8 and 2.5mg by week 16. No unexpected safety signals were observed and there were no additional deaths in the voclosporin treated patients; however, there were three deaths and one malignancy reported in the control arm after completion of the study treatment period. Additional data analyses for the AURA study at 48 weeks will be released at future corporate, medical and scientific meetings.

 

ASPiRA LABs, a Vermillion company, announced a major contracted agreement with Blue Cross Blue Shield of Michigan for ASPiRA’s U.S. FDA cleared, American College of Obstetricians and Gynecologists (ACOG) recommended ovarian cancer risk assessment test, OVA1 (MIA). Blue Cross Blue Shield of Michigan serves over six million beneficiaries throughout Michigan and surrounding states.

 

Onconova Therapeutics will present an overview at the Maxim Inaugural Healthcare Investor and Partnering Conference in China on Tuesday, March 7th, and the 29th Annual ROTH Conference on March 15th.

 

DelMar Pharmaceuticals will participate in a Key Opinion Leader forum highlighting the Company’s novel treatment for patients suffering from glioblastoma multiforme ("GBM"). The luncheon forum takes place on Thursday, March 9th at 12:00pm EST at the Lotte New York Palace Hotel in New York City.

 

After yesterday’s close, Juno Therapeutics reported 4Q16 GAAP EPS loss of $0.51, which compares to a $0.89 loss for the same period a year ago. The company beat the GAAP EPS mean estimate loss of $0.62. Total revenue for 4Q16 was $21.2 million, which compares to $4.2 million for the same period a year ago. This compares to a mean estimate of $14.6 million. Following earnings, Morgan Stanley analyst Matthew Harrison increased his price target to $27 from $25; JP Morgan analyst Cory Kasimov decreased his price target to $31 from $34.

 

After yesterday’s close, Nektar Therapeutics reported 4Q16 GAAP EPS loss of $0.28, which compares to a loss of $0.40 for the same period a year ago. The company missed the GAAP EPS mean estimate loss of $0.26. Total revenue for 4Q16 was $37.5 million, which compares to $39.4 million for the same period a year ago. This compares to a mean estimate of $40.7 million.

 

After yesterday’s close Intrexon reported 4Q16 GAAP EPS loss of $0.37, which compares to a loss of $0.28 for the same period a year ago. The company missed the GAAP EPS mean estimate loss of $0.22. Total revenue for 4Q16 was $46.0 million, which compares to $41.5 million for the same period a year ago. This compares to a mean estimate of $52.5 million. Following earnings, JP Morgan analyst Tycho Peterson decreased his price target to $25 from $34.

 

After yesterday’s close Opko Health reported 4Q16 non-GAAP EPS loss of $0.04, which compares to no EPS for the same period a year ago. The company met the non-GAAP EPS mean estimate loss of $0.04. Total revenue for 4Q16 was $275.5 million, which compares to $276.2 million for the same period a year ago. This compares to a mean estimate of $310.8 million. Following earnings, Jefferies analyst Brandon Couillard decreased his price target to $8 from $10. 

 

Tonix Pharmaceuticals, working with researchers from the University of Alberta, announced the successful synthesis of a potential smallpox-preventing vaccine. This vaccine candidate, TNX-801, is a live form of horsepox virus (HPXV) that has been demonstrated to have protective vaccine activity in mice.

 

Amgen announced the New England Journal of Medicine published results from the Phase III TOWER study evaluating the efficacy of BLINCYTO (blinatumomab) versus standard of care (SOC) chemotherapy in high-risk adult patients with Philadelphia chromosome-negative (Ph-) relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL), one of the most aggressive B-cell malignancies. Results from the analysis showed that median overall survival (OS) was 7.7 months (95 percent CI: 5.6, 9.6) for BLINCYTO versus four months (95 percent CI: 2.9, 5.3) for SOC (hazard ratio


for death=0.71; p=0.012). The TOWER study is the confirmatory study for the Phase II trial that supported the FDA’s accelerated approval designation for BLINCYTO in 2014.

 

Genentech, a member of the Roche Group, the Breast International Group (BIG), Breast European Adjuvant Study Team (BrEAST) and Frontier Science Foundation (FS) announced positive results from the Phase III APHINITY study. The study met its primary endpoint and showed that adjuvant (after surgery) treatment with the combination of Perjeta (pertuzumab), Herceptin (trastuzumab) and chemotherapy (the Perjeta-based regimen) achieved a statistically significant reduction in the risk of recurrence of invasive disease or death (invasive disease-free survival; iDFS) in people with HER2-positive early breast cancer (EBC) compared to Herceptin and chemotherapy alone. The safety profile of the Perjeta-based regimen was consistent with that seen in previous studies, and no new safety signals were identified. Full results from the APHINITY trial will be presented at an upcoming medical meeting in 2017.

 

Spark Therapeutics and Pfizer announced that SPK-9001, the lead investigational candidate in the companies’ SPK-FIX program has been granted support through the EMA PRIority MEdicines (PRIME) program. According to the EMA, PRIME is intended to enhance support for new treatments in development “that may offer a major therapeutic advantage over existing treatments, or benefit patients without treatment options.”

 

Myovant Sciences announced it has initiated a Phase III clinical trial, HERO, to evaluate the safety and efficacy of relugolix in treating men with advanced prostate cancer. Relugolix is an oral, once-daily, small molecule, gonadotropin-releasing hormone (GnRH) receptor antagonist that lowers testosterone by inhibiting pituitary release of luteinizing hormone and follicle-stimulating hormone.

 

Puma Biotechnology announced that based on its recent meeting with the Rapporteur, Co-Rapporteur and review team members, as well as the EMA, the Company plans to modify the summary of product characteristics (SmPC), sometimes referred to as the European product label, in its MAA to restrict the intended population to patients initiating neratinib treatment within one year after completion of adjuvant trastuzumab therapy. The proposed SmPC will continue to include both hormone receptor positive and hormone receptor negative patients.

 

bluebird bio announced the publication in the New England Journal of Medicine of a case study on Patient 1204, the first patient with severe sickle cell disease (SCD) to be treated with gene therapy. This patient, who was 13 years old at the time of treatment, was treated with LentiGlobin drug product in the HGB-205 clinical study conducted in Necker Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France. The data in the publication reflect 15 months of follow-up, and a brief summary of this patient’s outcomes with 21 months of follow-up was presented at the 58th American Society of Hematology Annual Meeting in December 2016.

 

Actelion announced that Japan’s Ministry of Health, Labor and Welfare granted a label extension for dosage and administration for "Epoprostenol ACT" (0.5 mg and 1.5 mg) for the treatment of pediatric patients with pulmonary arterial hypertension (PAH).

 

Swedish Orphan Biovitrum has received approval from the EMA for the grouped type II 15 K Variation for Elocta (efmoroctocog alfa). The variation involves several changes, including the approval of Elocta drug substance manufacturing in 15 000 litre scale bioreactors. Elocta is a recombinant extended half-life factor VIII Fc fusion protein product for the treatment of haemophilia A. 

 

Boehringer Ingelheim announced a new collaboration with Vanderbilt University, Nashville, Tennessee. The multi-year program complements an already existing collaboration by focusing on the research and development of small molecule compounds targeting the protein SOS (Son Of Sevenless). This molecule activates KRAS, a molecular switch that plays a central role in the onset of some of the deadliest cancers.

 

Horizon Pharma announced support of a program developed by the National Organization for Rare Disorders (NORD) to help people with Urea Cycle Disorders (UCDs) seeking assistance with high out-of-pocket costs associated with the purchase of medical foods and supplements for their low-protein dietary needs.

 

PTC Therapeutics announced that the Ataluren Confirmatory Trial (ACT CF) in nonsense mutation cystic fibrosis (nmCF) did not achieve its primary or secondary endpoints. Ataluren was generally well tolerated and ACT CF confirmed a favorable safety profile for ataluren, which has now been used by more than 1,000 patients across multiple indications. PTC plans to discontinue current clinical development of ataluren in cystic fibrosis, close ongoing extension studies and withdraw its application for marketing authorization in cystic fibrosis in Europe.

 

Regen BioPharma has granted CheckPoint Immunology an exclusive worldwide license to develop and commercialize Regen’s NR2F6 technology for human therapeutic use. The objective of the license grant is the separation of Regen’s small molecule technology from its other intellectual property in order to facilitate any future transactions involving small molecule therapies focused on the NR2F6 checkpoint.

 

Akorn announced that it has received approval from the FDA for its NDA for Ephedrine Sulfate Injection, USP 50 mg/mL in 1 mL single dose ampule.  Ephedrine sulfate injection is indicated for the treatment of clinically important hypotension occurring in the setting of anesthesia.

 

Coherus BioSciences announced that CHS-1420, its proposed biosimilar of adalimumab (Humira), met the primary endpoint in a clinical pharmacokinetic (PK) bioequivalence study that compared CHS-1420 to Humira in healthy subjects.  The study met the criteria for clinical PK similarity on all prospectively defined PK endpoints: maximum serum concentration (Cmax), area under the time-concentration curve from first to last time point measured (AUC-0-last), and area under the time-concentration curve from first time point extrapolated to infinity (AUC0-inf).  The 90% confidence intervals of the geometric mean ratios for all PK endpoints fell well within the bioequivalence boundaries of 80% to 125%.  Both agents were well tolerated and there were no differential safety findings observed between the two agents in this study.

 

BeiGene announced the dosing of the first patient in a pivotal clinical trial of BGB-3111, an investigational Bruton’s Tyrosine Kinase (BTK) inhibitor, in Chinese patients with relapsed or refractory mantle cell lymphoma (MCL). BGB-3111 is also currently being evaluated in a global Phase III study in comparison with ibrutinib for the treatment of patients with Waldenström’s Macroglobulinemia.

 

Intercept Pharmaceuticals announced that the National Institute for Health and Care Excellence (NICE) has approved Ocaliva (obeticholic acid) for routine use by the National Health Service (NHS) in England, Wales and Northern Ireland. Ocaliva has been conditionally approved in the European Union for the treatment of primary biliary cholangitis (PBC) in combination with ursodeoxycholic acid (UDCA) in adults with an inadequate response to UDCA, or as monotherapy in adults unable to tolerate UDCA. The NHS is expected to make Ocaliva available to patients with PBC within 90 days of NICE’s final appraisal publication and Intercept will work with local reimbursement authorities to help ensure eligible patients obtain access.

 

Innovus Pharmaceuticals announced that it received the CPNP notification number required to commercialize Zestra in all 28 member countries of the European Union.

 

Kite Pharma announced the commencement of an underwritten public offering of 4,750,000 shares of its common stock. In addition, Kite has granted the underwriters of the offering a 30-day option to purchase up to an additional 712,500 shares of its common stock. All shares of common stock to be sold in the offering will be sold by Kite.

 

Bioverativ and Swedish Orphan Biovitrum AB announced that interim results from the B-YOND extension trial, which studies ALPROLIX [Coagulation Factor IX (Recombinant), Fc Fusion Protein] in previously-treated subjects with severe hemophilia B, were published in the March 2017 issue of Thrombosis and Haemostasis. The study results reinforce the long-term safety and efficacy of prophylactic treatment with ALPROLIX over a median duration of more than three years in adults/adolescents and more than a year and a half in children under 12 years of age. The primary outcome measure was development of inhibitors (neutralizing antibodies that can interfere with activity of the therapy); no patients treated with ALPROLIX in the study developed inhibitors.

 

 

ANALYST RECOMMENDATIONS

 

Nomura analyst Christopher Marai initiated coverage of the following companies: Biogen with a “buy” rating and $345 price target; Alexion Pharmaceuticals with a “buy” rating and $148 price target; Incyte with a “buy” rating and $148 price target; BioMarin Pharmaceutical with a “neutral” rating and $93 price target; Galapagos with a “buy” rating and $87 price target; Prothena with a “buy” rating and $87 price target; Sarepta Therapeutics with a “buy” rating and $84 price target; Five Prime Therapeutics with a “buy” rating and $94 price target; Xencor with a “neutral” rating and $21 price target; Acceleron Pharma with a “buy” rating and $58 price target; Global Blood Therapeutics with a “buy” rating and $50 price target; Cellectis with a “buy” rating and $49 price target; MacroGenics with a “buy” rating and $41 price target; CytomX Therapeutics with a “buy” rating and $21 price target; Voyager Therapeutics with a “buy” rating and $31 price target; Syndax Pharmaceuticals with a “buy” rating and $27 price target.

 

Following Puma Biotechnology’s earnings, RBC analyst Simos Simeonidis decreased his price target to $48 from $57; Credit Suisse analyst Kennen MacKay decreased his price target of Puma Biotechnology to $58 from $107, citing the removal of de novo adjuvant therapy from the model as well as the neoadjuvant setting from the model.

 

Credit Suisse analyst Alethia Young increased her price target of Tesaro to $198 from $151, citing oncology assets are scarce and many global players are on the hunt which could increase bidding pressure for Tesaro’s PARP and they will be acquired in a competitive process that leads to a price above current trading levels.

 

Morgan Stanley analyst Matthew Harrison increased his price target of Portola to $31 from $29, citing management commentary related to the refiling of Andexanet as well as the potential for approval of betrixaban.

 

Citi analyst Robyn Karnauskas downgraded Kite Pharma to “neutral” from “buy” and increased her price target to $87 from $62, citing positive 6 month data from KITE’s pivotal trial yesterday increased confidence in approval for R/R DLBCL and expect a 2018 launch.

 

Citi analyst Robyn Karnauskas upgraded Acceleron Pharma to “buy” from “neutral” and decreased her price target to $36 from $40, citing several catalysts over next 2-3 years, robust partnership with CELG for a >$2B opportunity, near-term data that could increase conviction, and limited downside from pipeline.

 

Leerink analyst Geoffrey Porges increased his price target of Fibrogen to $55 from $52, citing the company’s progress has been impressive in the back end of 2016 and early part of 2017 and at this stage the long anticipated launch of their first branded medicine may be coming close to reality.

 

Following Pacira’s Pharmaceuticals’ earnings, Jefferies analyst David Steinberg increased his price target to $60 from $53; HC Wainwright analyst Corey Davis increased his price target to $59 from $48; Janney analyst Ken Trbovich downgraded the stock to “sell” from “neutral” and increased his price target to $38 from $34.

 

Leerink analyst Joseph Schwartz upgraded Sarepta to “outperform” from “market perform” and increased his price target to $47 from $27, citing the stock appears to have fully consolidated and reflects reasonable expectations for the Exondys launch, following management’s new revenue guidance ($13-15M in 1Q and over $80M in FY17) which looks highly conservative to us.