BioShares Biotechnology Clinical Trials (BBC): $21.33, -$0.11, +15.9% YTD

BioShares Biotechnology Products (BBP): $37.83, -$0.29, +15.5% YTD

 

 

MARKET COMMENTARY

 

Tracking weakness in global stocks, U.S. stock index futures indicated a lower start as investors looked for clarity on President Donald Trump’s policies on tax and trade. Leading index is on the economic calendar. The greenback gained against a basket of currencies. Oil was down as crude supplies grew, while gold edged up.

 

 

MARKET HIGHLIGHTS

 

Takeda Pharmaceutical and TiGenix NV announced new data from the Phase III ADMIRE-CD clinical trial, which indicated that investigational compound Cx601, a suspension of allogeneic adipose-derived stem cells (eASC), maintained long-term remission of treatment refractory complex perianal fistulas in patients with Crohn’s disease over 52 weeks. Results were presented at the 12th Congress of the European Crohn’s and Colitis Organization (ECCO). The ADMIRE-CD trial is a randomized, double-blind, controlled, Phase III trial, designed to investigate the efficacy and safety of the investigational compound Cx601 for the treatment of complex perianal fistulas in patients with Crohn’s disease. Patients were randomized to a single administration of Cx601 cells or placebo (control), both added to standard of care. A significantly greater proportion of patients in the Cx601 group versus in the control group achieved clinical and radiological combined remission, and clinical remission at week 52 in the modified intention-to-treat population. Of those mITT patients who had shown combined remission at week 24, a greater number in the Cx601 group versus the control group reported no relapse at week 52. The rates and types of treatment related adverse events (non-serious and serious) and discontinuations due to adverse events were indicated to be similar in both groups.

 

Aimmune Therapeutics announced an AR101 Phase 3 program update, based on recent feedback from the U.S. Food and Drug Administration (FDA). AR101 is Aimmune’s investigational biologic oral immunotherapy for desensitization of patients with peanut allergy.  The primary efficacy analysis in the ongoing Phase 3 PALISADE trial will now be conducted on ages 4-17 years, which aligns with the Breakthrough Therapy Designation population, based on the company’s discussions with the FDA. The company expects to conduct separate analyses for older patients in PALISADE. Additionally, based on FDA feedback, the company anticipates that the safety database for a potential Biologics License Application (BLA) will include data from at least 600 patients treated with AR101.

 

Aeglea BioTherapeutics announced the appointment of Suzanne L. Bruhn, Ph.D. to its Board of Directors. Dr. Bruhn previously served as chief executive officer, president and director at Promedior, Inc., a clinical-stage biotechnology company, from 2012 to 2015.

 

Immune Pharmaceuticals will announce on Monday, February 20th, 2017 at the Acute Leukemia XVI symposium in Munich, Germany that it will be launching REMAIN™, an international Overall Survival clinical study with the combination of Ceplene®, the Company’s flagship product for Acute Myeloid Leukemia (AML) remission maintenance and relapse prevention, and low dose Proleukin®. Patient recruitment is expected to start in later 2017.

 

ArQule and Daiichi Sankyo announced that the METIV-HCC Phase III study of tivantinib in hepatocellular carcinoma (HCC) did not meet its primary endpoint of improving overall survival. METIV-HCC is a biomarker-selected, double-blind, placebo-controlled, randomized phase III study evaluating tivantinib (2:1) versus best supportive care in patients with MET-overexpressing, inoperable HCC intolerant to or previously-treated with systemic therapy. A total of 340 patients with MET-overexpressing HCC analyzed by a validated immunohistochemical assay were randomized in the intent-to-treat population for efficacy analysis. The primary endpoint of the study is overall survival. Secondary endpoints include progression-free survival and safety. Full results from the trial will be presented at an upcoming scientific forum.

 

AstraZeneca announced positive results from its Phase III OlympiAD trial comparing LYNPARZA (olaparib) tablets (300mg twice daily) to physician’s choice of a standard of care chemotherapy in the treatment of patients with HER2-negative metastatic breast cancer harboring germline BRCA1 or BRCA2 mutations. Patients treated with LYNPARZA showed a statistically-significant and clinically-meaningful improvement in progression-free survival (PFS) compared with those who received chemotherapy (capecitabine, vinorelbine or eribulin).

 

Abeona Therapeutics announced updated data from the ongoing gene therapy clinical trial for Sanfilippo syndrome Type A (MPS IIIA), at the 13th Annual WORLDSymposium 2017 lysosomal storage disorders conference in San Diego, CA. The ongoing Phase I/II trial for ABO-102 (AAV-SGSH) is a first-in-man clinical trial utilizing a single intravenous injection of AAV gene therapy for subjects with MPS IIIA.

 

Adamas Pharmaceuticals will present results from its Phase II proof-of-concept study of ADS-5102 (amantadine hydrochloride) extended-release capsules for the treatment of walking impairment in patients with multiple sclerosis (MS) at the Americas Committee for Treatment and Research in Multiple Sclerosis (ACTRIMS) Forum 2017. In the study, patients on ADS-5102 showed an approximate 17% placebo-adjusted improvement in walking speed in the Timed 25 Foot Walk (T25FW) test and an approximate three second placebo-adjusted improvement in the Timed Up and Go (TUG) test. In addition, ADS-5102 was generally well tolerated.

 

Theravance Biopharma announced the presentation of positive clinical data for TD-1473, a novel, potent, and orally administered pan-Janus kinase (JAK) inhibitor designed to be intestinally restricted, at the 12th Congress of the European Crohn’s and Colitis Organization (ECCO). In a poster presentation, the Company reported further data from its completed Phase I study of single-ascending and multiple-ascending doses of TD-1473. Results of the study were first announced in June 2016. Theravance Biopharma is developing TD-1473 as an investigational compound with the potential to treat a range of inflammatory intestinal diseases and is currently conducting a Phase 1b study of the compound in patients with moderate to severe ulcerative colitis. The 12th Congress of ECCO is being held in Barcelona, Spain, February 15-18, 2017.

 

After yesterday’s close, Ultragenyx Pharmaceuticals reported 4Q16 GAAP EPS loss of $1.75 which compares to a loss of $1.42 for the same period a year ago. The company missed the GAAP EPS mean estimate loss of $1.73. Total revenue for 4Q16 was $0.5 million, which compares to no revenue reported for the same period a year ago. This compares to a mean estimate of $0.1 million.

 

After yesterday’s close, Cytokinetics reported total revenues for the fourth quarter of 2016 were $33.1 million, compared to $9.8 million, during the same period in 2015. Net income for the fourth quarter was $7.2 million, or $0.18 and $0.16 per basic and diluted share, respectively. This is compared to a net loss for the same period in 2015 of $9.2 million, or $0.24 per basic and diluted share. As of December 31, 2016, cash, cash equivalents and investments totaled $163.9 million.  The 2016 year-end cash and cash equivalents does not include $100 million received from a royalty monetization deal with Royalty Pharma that closed in February 2017. Cytokinetics also announced financial guidance for 2017. The company anticipates cash revenue will be in the range of $21 to $23 million, cash R&D expenses will be in the range of $108 to $112 million, and cash G&A expenses will be in the range of $30 to $32 million. This guidance excludes approximately $7.0 million in unearned revenue from the 2014 amendment of our collaboration with Astellas, which will be recognized in 2017 under generally accepted accounting principles, as well as any potential future milestones that may be achieved in accordance with our collaboration agreements with our partner Astellas.  This guidance also excludes an estimated $8.9 million in non-cash related operating expenses primarily related to stock compensation expense.

 

Global Blood Therapeutics announced initiation of the Phase I Basecamp (GBT440-011) clinical trial, which is evaluating the physiologic effects of GBT440 in fit healthy volunteers under hypoxic conditions and exercise conditions that maximally stress the function of the heart and lungs.

 

Ascendis Pharma announced the publication of its previously presented pediatric Phase II trial of TransCon Growth Hormone in the Journal of Clinical Endocrinology and Metabolism. The authors concluded that once-weekly, sustained release TransCon Growth Hormone was comparable to daily growth hormone in terms of efficacy, safety and tolerability in children with growth hormone deficiency (GHD).

 

Perrigo announced it has received final approval from the FDA for hydrocodone bitartrate and homatropine methylbromide oral solution (syrup), 5 mg/1.5 mg per 5 mL.

 

Janssen-Cilag International announced new two-year data from the ongoing IM-UNITI long-term extension (LTE) study evaluating the efficacy and safety of subcutaneous (SC) STELARA (ustekinumab) in patients with moderate to severe Crohn’s disease. The data presented at the 12th Congress of the European Crohn’s and Colitis Organization (ECCO) showed that treatment with ustekinumab maintained clinical response and remission for up to two years with no new safety signals observed. Of the 1,281 patients enrolled in the maintenance study, 397 patients who achieved a response to ustekinumab at week 8 following an induction phase were randomised to receive SC ustekinumab 90mg every 8 weeks (Q8W) or every 12 weeks (Q12W), or placebo during a maintenance (0–44 week) period, before entering the LTE (44–252 week) period. A one-time dose adjustment to ustekinumab 90mg Q8W was permitted in patients in the randomised group who met loss of response criteria between weeks 8–32. Clinical efficacy data were collected every 12 weeks and safety data were collected every 4 weeks from the end of the maintenance trial (week 44) until the maintenance study was unblinded and then at Q8W or Q12W dosing visits during the LTE period; data at week 92 are reported here.

 

Janssen-Cilag International announced the publication of data revealing radiographic progression-free survival (rPFS) of 16.5 months (95% CI, 13.5–20.0) and treatment duration of 11.6 months (95% CI, 10.2–12.8) in men treated with ZYTIGA (abiraterone acetate) plus prednisone (AAP), in the real-world, outside the clinical trial setting. The study assessed men being treated for asymptomatic and mildly symptomatic metastatic castration-resistant prostate cancer (mCRPC), following androgen deprivation therapy (ADT). These valuable insights were shown despite the real-world study population including those who had a poor prognosis or were difficult-to-treat patients, usually excluded from clinical trials. These data are part of a comprehensive real-world evidence (RWE) portfolio being presented by Janssen at this year’s American Society of Clinical Oncology Genitourinary Cancers Symposium (ASCO GU) in Orlando, Florida.

 

Heat Biologics announced that it presented a poster of its immunological data from its 94-patient Phase II trial evaluating vesigenurtacel-L (HS-410) either alone or in combination with BCG in patients with non-muscle invasive bladder cancer (NMIBC) at the 2017 Genitourinary (GU) Cancers Symposium.  Researchers reported that HS-410, in combination with BCG, continues to be generally well-tolerated, that HS-410 activates CD8+ T cells and that these immune responders appear to have a lower recurrence rate than non-immune responders. Taken together, these data strengthen support for the vaccine mechanism of action and clinical proof-of-concept of immune activation.

 

Myriad Genetics announced new data demonstrating the utility of the Prolaris test to more accurately classify mortality risk and guide the management of newly diagnosed men with prostate cancer. The data are being presented at the 2017 Genitourinary Cancers Symposium (ASCO GU) meeting in Orlando, Fla.

 

Celgene announced that its phase III SUNBEAM trial, evaluating the efficacy and safety of ozanimod, an investigational oral, selective S1P 1 and 5 receptor modulator, in patients with relapsing multiple sclerosis (RMS), met the primary endpoint in reducing annualized relapse rate (ARR), compared to weekly interferon (IFN) β-1a (Avonex). SUNBEAM evaluated two orally administered treatment doses (0.5 mg and 1 mg) of ozanimod, with patients treated for at least one year. The randomized phase III trial enrolled 1,346 RMS patients in 20 countries. Top-line data show that both the ozanimod 1 mg and 0.5 mg treatment arms demonstrated statistically significant and clinically meaningful improvements compared to Avonex for the primary endpoint of ARR and the measured secondary endpoints of the number of gadolinium-enhancing MRI lesions and the number of new or enlarging T2 MRI lesions at month 12. As agreed to in the Special Protocol Assessment (SPA) with the U.S. Food and Drug Administration, a pre-specified analysis on the time to onset of disability progression will be conducted using pooled results from both the SUNBEAM and RADIANCE phase III trials. The overall safety and tolerability profile was consistent with results from previously reported phase II RMS (RADIANCE) and phase II ulcerative colitis (TOUCHSTONE) trials.

 

Momenta Pharmaceuticals announced the termination of the applicable waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976 in connection with the collaboration and license agreement with CSL to develop Fc multimer programs, including M230, executed by the Company on January 5, 2017.

 

Capricor Therapeutics announced that it has elected to terminate its license agreement with the Mayo Clinic relating to natriuretic peptide receptor agonists, including Cenderitide.

 

XTL Biopharmaceuticals announced that it has entered into definitive agreements with institutional investors to receive gross proceeds of $2.5 million. Rodman & Renshaw, a unit of H.C. Wainwright & Co., is acting as the exclusive placement agent in connection with this offering.

 

Biostage announced the closing on February 15, 2017 of its previously announced public offering of 20,000,000 shares of common stock and warrants to purchase 20,000,000 shares of common stock, for total gross proceeds of $8.0 million. The offering was priced at $0.40 per share of common stock, with each share of common stock sold with one five-year warrant to purchase one share of common stock, at an exercise price of $0.40 per share. Rodman & Renshaw, a unit of H.C. Wainwright & Co., acted as exclusive placement agent for the offering.

 

CEL-SCI announced it has entered into a definitive agreement with institutional investors for an offering of shares of common stock with gross proceeds of approximately $1.0 million in a registered direct offering. The closing of the offering is expected to take place on or about February 23, 2017. In connection with the offering, the CEL-SCI will issue approximately 10 million registered shares of common stock at a purchase price of $0.10 per share. Concurrently in a private placement, the Company will issue warrants to purchase up to shares of its common stock. For each share of common stock purchased by an investor, such investor in the private placement will receive from the CEL-SCI an unregistered warrant to purchase one share of common stock. The warrants have an exercise price of $0.12 per share, will be exercisable upon the 6 month anniversary of the issue date, and will expire five and a half years from the issue date. Rodman & Renshaw, a unit of H.C. Wainwright & Co., LLC, is acting as the exclusive placement agent in connection with the offering.

 

The board of directors of AbbVie has authorized a $5 billion increase to AbbVie’s existing stock repurchase program.

 

Catabasis Pharmaceuticals announced the promotion of Ted Hibben to Chief Business Officer.

 

AcelRx Pharmaceuticals announced that Vincent J. Angotti has been appointed CEO and a member of the company’s board of directors, effective Monday, March 6, 2017.

 

 

ANALYST RECOMMENDATIONS

 

Natixis analyst Jean-Jacques Le Fur upgraded DBV Technologies to “buy” from “neutral” and increased his price target to €92 from €65 after switching coverage to a new analyst and given that the results of the Phase III study (PEPITES) on the Viaskin Peanut patch are due out this year and are expected to be positive.

 

CL King analyst David Westenberg initiated coverage of Quidel with a “neutral” rating and $22 fair value estimate, citing a strong player in flu, with a good management team and competitive new products.

 

Needham analyst Alan Carr downgraded Genocea Biosciences to “hold” from “buy,” citing uncertainty for GEN-003 development timelines as well as the stock.

 

Citi analyst Yigal Nochomovitz increased his price target of Portola Pharmaceuticals to $39 from $32, citing two analyses of FDA precedent support higher betrixaban approval probability given news of no panel.

 

Berenberg analyst Alistair Campbell increased his price target of Pfizer to $39 from $38, citing the acquisition of Medivation, which strengthens the oncology portfolio, and the exclusion of Hospira’s infusion business following its sale to ICU Medical.

 

Roth analyst Sa’ar Yaniv initiated coverage of Aerie Pharmaceuticals with a “buy” rating and $69 price target, citing expectations of Rhopressa and Roclatan approval in 2018 and 2019.

 

Cantor Fitzgerald analyst Chiara Russo initiated coverage of Nabriva Therapeutics with an “overweight” rating and $14 price target, citing NBRV could bring to market a new class of systemic antibiotics, transitioning from a development-stage to a fully-integrated pharmaceutical company over the next 24 months.

 

Jefferies analyst Jeffery Holford revised his price target of the following major pharma companies, limited visibility on US pricing/ politics as well as an opaque outlook for the key immuno-oncology players: Abbot increased to $51 from $48; AstraZeneca increased to 5,250p from 4,800p; Bayer AG increased to €130 from €125; Bristol-Myers Squibb increased to $64 from $60; Eli Lilly increased to $95 from $93; GlaxoSmithKline increased to 1,800p from 1,780p; Johnson and Johnson increased to $125 from $120; Novartis increased to CHF93 from CHF90; Pfizer increased to $34 from $30; Roche increased to CHF275 from CHF270; Sanofi increased to €85 from €80; Zoetis increased to $65 from $64.

 

RBC analyst Adnan Butt increased his price target of Tesaro to $202 from $142, citing optionality and M&A based multiples could approach similar valuation levels.