BioShares Biotechnology Clinical Trials (BBC): $22.19, +$1.59, +20.5% YTD

BioShares Biotechnology Products (BBP): $38.40, +$1.46, +17.3% YTD

 

Key Opinion Leader Call on

Pulmonary Arterial Hypertension (PAH)

LifeSci Capital is hosting a Key Opinion Leader (KOL) Call with Dr. Ronald Oudiz (UCLA) and Dr. Jeremy Feldman (Arizona Pulmonary Specialists) to discuss the current treatment landscape for patients with PAH. The call with include a discussion of recently approved treatments for PAH, as well as therapies in clinical development.

 

Tuesday, February 28th @ 1pm Eastern Time

In the US:  888-254-3601

International:  913-905-3226

Conference ID:  3682139

Webcast:  http://public.viavid.com/player/index.php?id=123143

 

 

MARKET COMMENTARY

 

U.S. stock index futures were little changed, and world stocks hovered just off all-time highs as markets awaited a speech by U.S. President Donald Trump for signals on infrastructure spending and tax cuts. Growth, advanced goods trade, wholesale inventory, CaseShiller and consumer confidence data are scheduled to release later in the day. Oil was broadly down and prices continued to trade in a tight range with OPEC’s bullish production cuts offset by increasing crude production from the United States. Gold traded down, after falling from a 3-1/2 month high in the previous session.

 

 

MARKET HIGHLIGHTS

 

Immune Pharmaceuticals has broadened enrollment eligibility criteria based on the data reported from three patients that have completed treatment to date in its ten-patient open label study of Bertilimumab in the treatment of Bullous Pemphigoid ("BP"). In those patients, the Bullous Pemphigoid Disease Activity Index ("BPDAI") was reduced by an average of 84% and oral prednisone was tapered down to 10mg or less. No significant adverse events were reported. Based on these preliminary results, the Company has also submitted a request for orphan drug designation for Bertilimumab in BP. BP is a rare autoimmune blistering disease of the skin, which is painful and itchy, and occurs predominantly in patients more than 60 years of age.

 

Aeglea BioTherapeutics will present at the 2017 Keystone Symposia Conference on Tumor Metabolism: Mechanisms and Targets, taking place March 5 – 9 in Whistler, British Columbia.

 

Addex Therapeutics has raised gross proceeds of CHF3.0 million through a private placement in which 1.5 million treasury shares were placed at CHF2 per share. The private placement was led by Herculis Partners SA.

 

Minerva Neurosciences will present at the Cowen and Company 37th Annual Health Care Conference on March 7 at 10:40 a.m. Eastern Time.

 

After yesterday’s close, Exelixis reported 4Q16 GAAP EPS of $0.12, which compares to a loss of $0.18 for the same period a year ago. The company beat the GAAP EPS mean estimate loss of $0.01. Total revenue for 4Q16 was $77.6 million, which compares to $9.9 million for the same period a year ago. This compares to an estimate of $66.5 million. Following earnings, Stifel analyst Stephen Willey downgraded the stock to “hold” from “buy” and increased his price target to $22 from $21; Deutsche Bank analyst Andrew Peters increased his price target to $26 from $20; Leerink analyst Michael Schmidt increased his price target to $26 from $20; Piper Jaffray analyst Ed Tenthoff increased his price target to $26 from $21.

 

Medicines Co. reported 4Q16 GAAP EPS loss of $1.75, which compares to a loss of $2.99 for the same period a year ago. The company missed the GAAP EPS mean estimate loss of $1.54. Total revenue for 4Q16 was $25.2 million, which compares to $67.2 million for the same period a year ago. This compares to a mean estimate of $26.9 million.

 

Ionis reported 4Q16 GAAP EPS of $0.21, which compares to a loss of $0.59 for the same period a year ago. The company beat the GAAP EPS mean estimate loss of $0.13. Total revenue for 4Q16 was $160.3 million, which compares to $51.6 million for the same period a year ago. This compares to a mean estimate of $97.5 million.

 

Endo International reported 4Q16 non-GAAP EPS of $1.77, which compares to $1.36 for the same period a year ago. The company beat the non-GAAP EPS mean estimate of $1.63. Total revenue for 4Q16 was $1.2 billion, which compares to $1.1 billion for the same period a year ago. This compares to a mean estimate of $1.2 billion.

 

Reuters reported that Valeant Pharmaceuticals International reported a better-than-expected quarterly profit, helped by lower costs and strength in its Bausch and Lomb eyecare business, but its net loss widened and the company said it was feeling pricing pressure. Net loss attributable to Valeant widened to $515 million, or $1.47 per share in the fourth quarter ended Dec. 31, from $385 million, or $1.12 per share, a year earlier. Excluding items, it earned $1.26 per share, beating analysts’ average estimate of $1.19, according to Thomson Reuters I/B/E/S. Revenue fell 12.9 percent to $2.40 billion, but exceeded the average expectation of $2.33 billion. The company said it expected 2017 revenue of $8.90 billion-$9.10 billion. Analysts were expecting revenue of $8.96 billion.

 

INC Research Holdings reported 4Q16 non-GAAP EPS of $0.67, which compares to $0.54 for the same period a year ago. The company met the non-GAAP EPS mean estimate of $0.67. Total revenue for 4Q16 was $263.0 million, which compares to $241.4 million for the same period a year ago. This compares to a mean estimate of $269.0 million.

 

Kite Pharma announced positive data from the primary analysis of ZUMA-1 for its lead CAR-T candidate, axicabtagene ciloleucel (previously referred to as KTE-C19), in patients with chemorefractory aggressive B-cell non-Hodgkin lymphoma (NHL). The study met the primary endpoint of objective response rate (ORR), or rates of tumor response (complete response + partial response) recorded after a single infusion of axicabtagene ciloleucel, with 82 percent (p<0.0001). These results demonstrate the treatment effect of axicabtagene ciloleucel in a patient population with multiple types of aggressive NHL, including diffuse large B-cell lymphoma (DLBCL) enrolled in Cohort 1, as well as primary mediastinal B-cell lymphoma (PMBCL) and transformed follicular lymphoma (TFL) enrolled in Cohort 2.

 

aTyr Pharma announced that its product candidate Resolaris was granted Orphan Drug Designation by the FDA for the treatment of limb girdle muscular dystrophy (LGMD) patients.

 

AstraZeneca announced that the FDA has approved once-daily QTERN (10mg dapagliflozin and 5mg saxagliptin) for the treatment of type-2 diabetes. The new medicine is indicated as an adjunct to diet and exercise to improve glycemic (blood sugar level) control in adults with type-2 diabetes who have inadequate control with dapagliflozin (10mg) or who are already treated with dapagliflozin and saxagliptin.

 

Amgen announced positive results from a planned overall survival (OS) interim analysis of the Phase III head-to-head ENDEAVOR trial. The study met the key secondary endpoint of OS, demonstrating that patients with relapsed or refractory multiple myeloma treated with KYPROLIS (carfilzomib) and dexamethasone (Kd) lived 7.6 months longer than those treated with Velcade (bortezomib) and dexamethasone (Vd) (median OS 47.6 months for Kd versus 40.0 for Vd, HR = 0.79, 95 percent CI, 0.65 – 0.96). This Kd regimen administered with 56 mg/m(2) KYPROLIS twice weekly is already approved in the U.S., European Union and other countries based on the primary analysis of progression-free survival (PFS) in the ENDEAVOR study.

 

Eagle Pharmaceuticals announced that the 505(b)(2) NDA for its novel Pemetrexed Injection, 25 mg/mL has been accepted for filing by the FDA. Under the PDUFA, the FDA has set a target date of October 30, 2017 to complete its review of the NDA.

 

Eagle Pharmaceuticals announced that the USPTO has issued an 11(th) patent related to Bendeka. Patent number 9,579,384 will expire March 2033. The USPTO has allowed a total of 14 patents in the Bendeka family of patents expiring from 2026 to 2033, of which three will issue over the next few months.

 

Affinivax and Astellas Pharma announced they have entered into an exclusive worldwide license agreement to develop and commercialize a vaccine targeting Streptococcus pneumoniae (pneumococcus). The partnership will utilize Affinivax’s proprietary vaccine technology platform – Multiple Antigen Presenting System (MAPS) – to advance a novel MAPS vaccine targeted to prevent and reduce the spread of pneumococcal disease.

 

Achillion Pharmaceuticals announced that it has entered into an agreement with Imperial College London to conduct a natural history study of C3 glomerulopathy (C3G), a rare renal disorder which includes dense deposit disease (DDD) and C3 glomerulonephritis (C3GN). C3G affects an estimated 8,000 people across Europe and the United States.

 

PAREXEL announced the appointment of Simon Harford as Senior Vice President and Chief Financial Officer effective June 1, 2017. He will serve on PAREXEL’s Business Review Committee, the Company’s senior-most leadership team, and assist in developing and implementing PAREXEL’s strategic objectives.

 

Actinium Pharmaceuticals announced that the Company received a notice of allowance from the USPTO for a patent claiming the methods for generating a radioimmunoconjugate comprised of actinium-225, an alpha emitting radioisotope, conjugated to monoclonal antibodies. Actinium-225 is the radioisotope used in Actinium’s Actimab-A, a drug candidate in a Phase II clinical trial for patients newly diagnosed with acute myeloid leukemia (AML) who are over the age of 60; Actimab-M, a drug candidate in a Phase 1 trial for patients with relapsed or refractory multiple myeloma (MM); and the Company’s alpha particle immunotherapy technology platform. Actinium currently has 61 issued and pending patents that are owned or licensed relating to isotope production methods, drug preparation methods and the Company’s platform technology. This patent is expected to expire in July of 2030.

 

Opiant Pharmaceuticals announced that the Company has received supportive feedback from the FDA on a proposed development plan for OPNT002, an intranasally-dosed opioid antagonist, for the treatment of Alcohol Use Disorder (AUD). The feedback was received pursuant to a recent Type B meeting with the FDA.

 

EMD Serono, the biopharmaceutical business of Merck KGaA, and Pfizer announced that the FDA has accepted for Priority Review EMD Serono’s BLA for avelumab as a treatment for patients with locally advanced or metastatic urothelial carcinoma (mUC) with disease progression on or after platinum-based therapy. The FDA has set a PDUFA target action date of August 27, 2017, for avelumab in this indication.

 

Neuralstem announced the recent publication of preclinical data on NSI-189 in Journal of Cellular Physiology. The study demonstrated that NSI-189 improved behavioral function recovery and enhanced hippocampal synaptic plasticity in stroke-induced animals during the 12-week treatment period, which was maintained up to 24 weeks post-stroke.

 

Novo Nordisk announced the submission of a NDA to the Japanese Ministry of Health, Labour and Welfare for semaglutide, a new glucagon-like peptide-1 (GLP-1) analogue administrated once-weekly, for the treatment of adults with Type II diabetes. The Japanese filing follows the recent once-weekly semaglutide regulatory submissions to the FDA, the EMA, Health Canada and SwissMedic.

 

AC Immune SA announced that its partner Genentech, member of Roche group, has decided to start a second Phase III clinical trial of the Alzheimer’s therapy crenezumab, an anti-Abeta antibody. This new trial CREAD2 will recruit 750 patients with prodromal or mild Alzheimer’s disease. This new trial complements the current Phase III CREAD1 trial of 750 participants with prodromal or mild Alzheimer’s disease, expected to read out in 2020. Trial design details of CREAD2 are not yet available but will be posted on ClinicalTrials.Gov in due course. Crenezumab was discovered by AC Immune using its SupraAntigen technology platform and out-licensed to Genentech in 2006 as a potential therapy for Alzheimer’s disease. AC Immune will not receive any milestone payments for the start of this second Phase III trial since the company already received a milestone payment when the CREAD1 trial started.

 

Titan Pharmaceuticals announced that the FDA has completed its initial review of the ropinirole implant IND and has requested that Titan hold the initiation of the clinical study pending submission of the requested information and the agency’s 30-day review.

 

Sangamo Therapeutics announced the appointment of Kathy Yi as senior vice president and chief financial officer, to succeed current executive vice president and chief financial officer H. Ward Wolff, who will be retiring from the company in early March.

 

Perrigo announced it has signed a definitive agreement to divest its rights to the royalty stream from the global net sales of the multiple sclerosis drug Tysabri (natalizumab) to RPI Finance Trust, an affiliate of Royalty Pharma. This transaction is valued at a total consideration of up to $2.85 billion, composed of $2.2 billion in cash at closing and up to $650 million in potential milestone payments based upon future global net sales of Tysabri in 2018 and 2020. This transaction is expected to close within 30 business days, subject to certain specified closing deliverables.

 

Global Blood Therapeutics announced the closing of its previously announced underwritten public offering of 5,867,347 shares of its common stock at a price to the public of $24.50 per share, which includes 765,306 additional shares of common stock issued upon the exercise in full of the underwriter’s option to purchase additional shares. The aggregate gross proceeds to GBT from this offering are $143.8 million, before deducting underwriting discounts and commissions and other estimated offering expenses payable by GBT. Cantor Fitzgerald & Co. was the sole book-running manager for the offering.

 

Perrigo announced that Ron Winowiecki, Senior Vice President, Business Finance, has been appointed acting Chief Financial Officer, effective immediately, following the resignation of Judy L. Brown, Executive Vice President, Business Operations and Chief Financial Officer, who is leaving Perrigo to take a position with another company in the pharmaceutical industry, beginning on April 1, 2017. Perrigo is conducting a thorough search process to identify a permanent Chief Financial Officer, which includes Mr. Winowiecki as a key candidate.

 

Cytokinetics announced that it is joining the global initiative with the European Organization for Rare Diseases (EURORDIS) and the National Organization for Rare Disorders (NORD) to raise awareness of Rare Disease Day, an international campaign dedicated to elevating the public understanding of rare diseases. This year’s theme, Research, calls attention to the importance of scientific research to better understand rare diseases and to develop innovative treatments to better patients’ lives.

 

Alliqua BioMedical announced that investors have agreed to purchase unregistered shares of the Company’s common stock in a private placement. Under the terms of the private placement, Alliqua agreed to sell an aggregate of 5,540,000 shares of its common stock at the price of $0.50 per share.

 

Teva Pharmaceutical Industries announced the FDA has accepted the NDA and granted Priority Review for SD-809 (deutetrabenazine) for the treatment of tardive dyskinesia (TD). The FDA has assigned a PDUFA goal date of August 30, 2017.

 

Baxter International and ScinoPharm Taiwan announced a strategic partnership to develop, manufacture and commercialize five injectable drugs used in a range of cancer treatments, including lung cancer, multiple myeloma and breast cancer, as well as medication to treat nausea and vomiting, common side effects of chemotherapy. The arrangement also provides Baxter the option to partner with ScinoPharm—one of the world’s leading active pharmaceutical ingredient (API) manufacturers—on as many as 15 additional injectable molecules.

 

Checkpoint Therapeutics, a Fortress Biotech company, announced that the USPTO has issued a composition of matter patent for CK-101 (also known as RX518), Checkpoint’s oral, third-generation epidermal growth factor receptor (EGFR) inhibitor product candidate under development for the treatment of patients with EGFR mutation-positive non-small cell lung cancer (NSCLC).

 

International Stem Cell Corporation announced the third patient in the clinical trial for Parkinson’s Disease was successfully transplanted with ISC-hpNSC cells and is already discharged from the Royal Melbourne Hospital (RMH). The operation was successfully performed without complications by the team of the RMH neurosurgeons.

 

Adamas Pharmaceuticals announced the presentation of additional data from EASE LID 2, its Phase III open-label, long-term safety and efficacy study of ADS-5102 (amantadine hydrochloride) extended-release capsules for the treatment of levodopa-induced dyskinesia (LID) in patients with Parkinson’s disease, at the first Pan American Parkinson’s Disease and Movement Disorders Congress. The updated analysis demonstrates the safety and tolerability of ADS-5102 and a reduction in LID and OFF time symptoms for up to 64 weeks. Symptoms of LID are characterized by involuntary movements that are non-rhythmic, purposeless, and unpredictable. Symptoms of OFF time are characterized by slowness of movement, rigidity, impaired walking, tremor and postural instability.

 

Neurotrope announced the conclusion of dosing and patient monitoring in its Phase II double blind, placebo controlled clinical trial of bryostatin-1 in the treatment of moderate to severe Alzheimer’s dementia.  Patients underwent a 12 week treatment with bryostatin-1, followed by a 30-day post-treatment evaluation. The study is designed to assess the therapeutic efficacy of bryostatin-1, a PKC epsilon activator.  Prior animal studies have demonstrated bryostatin’s efficacy for restorative synaptogenesis, prevention of neuronal death, and anti-amyloid, anti-tau metabolism via the activation of PKC epsilon.

 

 

ANALYST RECOMMENDATIONS

 

HC Wainwright analyst Ed Arce assumed coverage of DURECT with a “buy” rating and $3 price target, citing DURECT’s epigenetic regulator program, now preparing to enter Phase II clinical trials, has demonstrated broad applicability and potential utility in several chronic metabolic diseases including primary biliary cholangitis (PBC), primary sclerosing cholangitis (PSC) and non-alcoholic steatohepatitis (NASH); as well as acute organ injuries such as acute kidney injury (AKI).

 

Following BioCryst’s earnings, JP Morgan analyst Jessica Fye increased her price target to $6 from $5; Jefferies analyst Brian Abrahams increased his price target to $6 from $3.50.

 

Following Horizon’s earnings, UBS analyst Marc Goodman decreased his price target to $22 from $23; Mizuho analyst Irina Koffler decreased her price target to $22 from $25.

 

The following analysts revised their price targets and ratings of Esperion Therapeutics: Citi analyst Joel Beatty upgraded the stock to “buy” from “neutral” and increased his price target to $39 from $14; JP Morgan analyst Jessica Fye increased her price target to $22 from $15.