BioShares Biotechnology Clinical Trials (BBC): $20.05, +$0.26, -31.0% YTD

BioShares Biotechnology Products (BBP): $30.72, +$0.35, -0.3% YTD

 

 

MARKET COMMENTARY

 

U.S. stock index futures were little changed after hawkish comments from Federal Reserve Chair Janet Yellen left the door open to a U.S. interest rate hike as early as next month. Data from the U.S.
Commerce Department is expected to show that adjusted personal consumption inched up 0.3 percent in July, driven by demand for motor vehicles. Global markets were down, while Japan’s Nikkei bucked the trend as the yen weakened. The dollar edged up and gold
was trading lower. Oil slipped as Iraq’s production rose and as Iran said it would only cooperate in talks to freeze output if fellow exporters recognized its right to fully regain market share.

 

 

MARKET HIGHLIGHTS

 

Santhera Pharmaceuticals will announce financial results for the first half-year 2016 on Tuesday, September 6th. 

 

Aeglea BioTherapeutics announced the dosing of the first patient in a Phase 1 trial of AEB1102, a recombinant human enzyme designed to degrade the amino acid arginine, for the treatment of the
hematological malignancies acute myeloid leukemia and myelodysplastic syndrome.

 

ERYTECH Pharma announced that it has reached full patient enrollment in the Phase IIb trial of eryaspase, also known as ERY-ASP or GRASPA, for the treatment of acute myeloid leukemia (AML). 
The open-label, randomized, multi-center clinical trial, which is being conducted at more than 20 sites in Europe, has completed enrollment of a total of 123 patients and is on track for reporting of primary data in the second half of 2017. Patients enrolled
in the trial are over the age of 65, newly-diagnosed with AML, and unable to receive intensive chemotherapy. The primary endpoint is overall survival (OS) at one year.

 

CTI BioPharma announced top-line results from PERSIST-2, a randomized, controlled Phase III clinical trial comparing pacritinib, an investigational oral multikinase inhibitor, with physician-specified
best available therapy (BAT), including ruxolitinib, for the treatment of patients with myelofibrosis whose platelet counts are less than 100,000 per microliter — a patient population with high-risk advanced disease. Three hundred eleven (311) patients were
enrolled in the study, which formed the basis for the safety analysis. Two hundred twenty-one (221) patients who had a chance to reach Week 24 (the primary analysis time point) at the time the clinical hold was imposed and constituted the intent-to-treat (ITT)
analysis population utilized for the evaluation of efficacy. Preliminary results demonstrated that the PERSIST-2 trial met one of the co-primary endpoints showing a statistically significant response rate in spleen volume reduction (SVR) in patients with myelofibrosis
treated with pacritinib compared to BAT, including the approved JAK2 inhibitor ruxolitinib (p<0.01). Although the PERSIST-2 trial did not meet the other co-primary endpoint of greater than 50 percent reduction in Total Symptom Score (TSS), the preliminary
analysis approached marginal significance compared to BAT (p=0.0791).

 

Tonix Pharmaceuticals Holding announced that it has received the final meeting minutes from the FDA from an End-of-Phase II/Pre-Phase III meeting. These minutes confirmed the FDA’s acceptance
of Tonix’s proposed Phase III studies and the planned NDA data package to support the registration of TNX-102 SL (cyclobenzaprine HCl sublingual tablets) for the treatment of PTSD.

 

Inovio Pharmaceuticals announced it has initiated a clinical study of its preventive Zika vaccine (GLS-5700) in 160 subjects in Puerto Rico, where the Zika virus outbreak has been declared a
public health emergency. The CDC estimates Zika will infect more than 25% of the Puerto Rican population by year end, providing the potential for this study’s placebo control design to provide exploratory signals of vaccine efficacy.

 

Mylan announced that its U.S. subsidiary will launch the first generic to EpiPen Auto-Injector (epinephrine injection, USP) at a list price of $300 per generic EpiPen two-pack carton, which represents
a discount of more than 50% to the Mylan list price, or wholesale acquisition cost (“WAC”), of the branded medicine. The authorized generic will be identical to the branded product, including device functionality and drug formulation. Mylan expects to launch
the product in several weeks, pending completion of labeling revisions. Upon launch, the product will be available as a two-pack carton in both 0.15 mg and 0.30 mg strengths. Mylan also intends to continue to market and distribute branded EpiPen.

 

Leap Therapeutics and
Macrocure announced the signing of a definitive merger agreement. Under the terms of the agreement, Macrocure will become a wholly owned subsidiary of Leap, and Leap will become a public company. In connection with the transaction, Leap will apply to
have the shares of the combined entity listed for trading on NASDAQ upon completion of the merger. Under the terms of the agreement, Macrocure shareholders will exchange their Macrocure shares for newly issued shares of Leap common stock. In addition, existing
Leap investors, including entities affiliated with HealthCare Ventures, have committed to invest an additional $10 million at the closing of the transaction. On a pro forma basis, after giving effect to the merger and the investment, Macrocure equity holders
are expected collectively to own approximately 31.8%, and Leap equity holders are expected collectively to own approximately 68.2%, of the combined company, subject to certain possible adjustments based on Macrocure’s net cash level at closing. Existing Leap
shareholders will receive the right to a royalty, under certain circumstances, based on future net sales. The combined company is expected to have a minimum of $30 million of cash at closing to finance future operations.

 

Abeona Therapeutics announced that enrollment has been completed for the low-dose cohort (n=3) in its ongoing Phase I/II trial for ABO-102 (AAV-SGSH). The first-in-man clinical trial utilizes
a single injection of AAV gene therapy for patients with MPS IIIA (Sanfilippo syndrome type A), a rare autosomal recessive disease that causes neurocognitive decline, speech loss, loss of mobility, and premature death.

 

Cynapsus Therapeutics announced that the FDA has granted Fast Track Designation for APL-130277, a product candidate for the treatment of OFF episodes in patients with Parkinson’s disease (PD).

 

Regeneron Pharmaceuticals and
Sanofi announced detailed positive results from ODYSSEY ESCAPE, a Phase III trial which evaluated Praluent (alirocumab) Injection in patients with an inherited form of high cholesterol known as heterozygous familial hypercholesterolemia (HeFH) who require
regular weekly or bi-weekly apheresis treatment. The trial demonstrated that adding Praluent to existing therapy reduced LDL cholesterol by approximately 50 percent from baseline (compared to 2 percent increase for placebo). Praluent significantly reduced
the need for apheresis treatment by 75 percent compared to placebo (p less than 0.0001), the primary endpoint of the study. Results will be presented today at a Hot Line session at the ESC Congress 2016 in Rome, Italy.

 

Amgen announced data presented at the European Society of Cardiology (ESC) Congress 2016 showing Repatha (evolocumab) consistently reduced low-density lipoprotein cholesterol (LDL-C) in patients
across cardiovascular (CV) risk subgroups or with familial hypercholesterolemia (FH). In other company news, Amgen announced positive top-line results from the primary analysis conducted in a Phase III randomized, double-blind, double-dummy, active-controlled
study evaluating the safety and efficacy of Prolia (denosumab) compared with risedronate in patients receiving glucocorticoid treatment. The study met all primary and secondary endpoints at 12 months. The data showed that treatment with Prolia for 12 months,
compared to risedronate, led to significantly greater gains in bone mineral density (BMD) at the lumbar spine and total hip, both in patients receiving continuing glucocorticoid therapy and in patients newly initiating glucocorticoid therapy.

 

Roche announced that the FDA has issued an Emergency Use Authorization (EUA) for the LightMix Zika rRT-PCR Test. The product is for use in patients meeting CDC Zika virus clinical criteria and/or
CDC Zika virus epidemiological criteria. The test is used for the detection of Zika virus in EDTA plasma or serum samples using Roche’s LightCycler 480 Instrument II or cobas z 480 Analyzer.

 

Kadmon Holdings announced that the first patient has been dosed in a Phase II clinical trial of tesevatinib, the Company’s oral tyrosine kinase inhibitor, for the treatment of recurrent glioblastoma.
The open-label, multicenter study examines tesevatinib monotherapy administered at 300 mg once daily in up to 40 patients in the United States.

 

Exalenz Bioscience announced a collaboration with
Conatus Pharmaceuticals to use the BreathID Methacetin Breath Test (MBT) to monitor patients in a planned Phase IIb clinical trial evaluating emricasan. Emricasan is an investigational treatment for patients with chronic liver disease, being developed
by Conatus.

 

Elite Pharmaceuticals announced it has entered into a Development and License Agreement with
SunGen Pharma to collaborate to develop and commercialize four generic pharmaceutical products. Under the terms of the agreement, Elite and SunGen will share in the responsibilities and costs in the development of the products. Upon approval, the products
will be owned jointly by Elite and SunGen. SunGen shall have the exclusive right to market and sell two of the products using SunGen’s label and Elite shall have the exclusive right to market and sell two of the products using Elite’s label. Elite will manufacture
and package all four products on a cost plus basis.

 

RedHill Biopharma announced that it has received from the European Patent Office a Notice of Intention to Grant for a new patent covering the use of RHB-104 in the treatment of multiple sclerosis
(MS). Upon grant by the European Patent Office, the patent can be officially validated in up to 38 European countries.

 

Intersect ENT announced that CareFirst BlueCross BlueShield, the regional blues provider for Maryland and the National Capital area, has issued a positive coverage decision for PROPEL and PROPEL
mini steroid releasing sinus implants for use in patients following endoscopic sinus surgery.

 

AcelRx Pharmaceuticals announced that the company and its investigators will be presenting results from the Phase III SAP302 study of ARX-04 (sufentanil sublingual tablet, 30 mcg) in 76 patients
who were treated for moderate-to-severe acute pain in the emergency department. An oral presentation reviewing these results, including the types of injuries that were sustained by patients in the study, will be made at the International Society for Burn Injuries
(ISBI), which is taking place August 29 — September 1 in Miami, Florida.

 

RegeneRx Biopharmaceuticals announced that it has received notice of Intent to Grant a patent from the European Patent Office for the treatment of patients with MS. The patent covers use of the
Company’s proprietary molecule Thymosin beta 4 in a composition for treating or reducing deterioration of, injury or damage to tissue due to MS. The patent expiry is January 13, 2026.

 

M Pharmaceutical announced findings  from the second of three proprietary pilot studies, previously conducted, as it relates to the recently acquired Chelatexx technology, referred to as the
Company’s C-103 project.  In this study, which was conducted in the USA, C-103 was over 98% effective in eliminating the GI adverse events associated with orlistat. This finding is consistent with the first of the three pilot study findings announced on August
11th, 2016.

 

Ferring Pharmaceuticals announced that it has signed an agreement with
Seikagaku granting Ferring the exclusive worldwide rights (excluding Japan) to SI-6603 (condoliase), a chemonucleolytic product in Phase III development for the treatment of radicular leg pain (e.g. sciatica) due to lumbar disc herniation.

 

Linkage Biosciences announced that it has been awarded a four-year contract by NHS Blood and Transplant (NHSBT) in the UK for implementation of the LinkS?q Real-Time PCR HLA typing product. 
The new tissue typing platform, including Real-Time PCR instrumentation, will be installed and adopted by various laboratories across the country.

 

Dr. Reddy’s Laboratories announced that it has launched Nitroglycerin sublingual tablets USP, 0.3 mg, 0.4 mg and 0.6 mg, a therapeutic equivalent generic version of Nitrostat (Nitroglycerin)
sublingual tablets in the US market on August 26, 2016, having been approved by the FDA.

 

OvaScience announced that it has finalized its commercial agreement with the
IVF JAPAN GROUP and that the AUGMENT treatment is now available to women through the clinic. The AUGMENT treatment is designed to improve the health of a woman’s existing eggs and enhance in-vitro fertilization (IVF) procedures. The commercial agreement
follows a non-commercial preceptorship training program at IVF JAPAN GROUP, which was approved by the Japan Society of Obstetrics and Gynecology (JSOG).

 

IMS Health Holdings and
Quintiles Transnational Holdings announced key senior management roles in the future Quintiles IMS corporate leadership team, subject to the completion of the merger. Senior functional leaders reporting to Chairman and Chief Executive Officer Ari Bousbib
will include Michael McDonnell, EVP and Chief Financial Officer; James Erlinger, EVP and General Counsel; and Trudy Stein, EVP and Chief Human Resources Officer.

 

PDL BioPharma announced that PDL has received approximately $57.4 million in connection with the termination of PDL’s credit agreement with
Paradigm Spine, which included a repayment of the full principal amount outstanding of $54.7 million as well as accrued interest and a prepayment fee. In February 2014, PDL entered into a credit agreement with Paradigm Spine in which it made available
up to $75.0 million of debt financing with a five-year term, and initially provided $50.0 million, net of fees. PDL subsequently provided an additional $4.0 million in October 2015 as part of an amendment to the credit agreement.

 

Nichi-Iko Pharmaceutical Co announced the results of its tender offer to purchase all of the outstanding shares of common stock of
Sagent Pharmaceuticals at $21.75 per share, net to the holder in cash, without interest, less any applicable withholding taxes. The tender offer was effected by Nichi-Iko’s wholly-owned subsidiary,
Shepard Vision. The depositary for the tender offer has advised Nichi-Iko and Sagent that, as of the expiration of the tender offer at one minute following 11:59 p.m. (12:00 a.m.), New York City time, on August 26, 2016, a total of 28,229,440 shares
of Sagent common stock had been validly tendered and not withdrawn, representing approximately 85.6 percent of Sagent’s outstanding shares of common stock. In addition, the depositary advised that Notices of Guaranteed Delivery have been delivered with respect
to 589,169 additional shares of common stock, representing approximately 1.8 percent of Sagent’s outstanding shares of common stock. All shares of common stock that were validly tendered and not validly withdrawn during the offer period have been accepted
for payment.

 

CEL-SCI announced that it has closed its previously announced registered direct offering with institutional investors. The Company has received gross proceeds of $5 million.
Rodman & Renshaw, a unit of H.C. Wainwright, acted as the exclusive placement agent in connection with the offering.

 

 

ANALYST RECOMMENDATIONS

 

Rodman & Renshaw
analyst Raghuram Selvaraju assumed coverage of Can-Fite BioPharma with a “buy” rating and $6 price target, citing Can-Fite constitutes an underrated opportunity given its late-stage clinical development status with CF101 for treatment of psoriasis and
rheumatoid arthritis.

 

SunTrust analyst Sandy Draper decreased his price target of
Premier to $38 from $40, citing lower margins.

 

Janney analyst Ken Trbovich initiated coverage of
Recro Pharma with a “buy” rating and $21 price target, citing REPH is on track to gain FDA approval in 1H18 and launch in 2H18.

 

Evercore analyst Umer Raffat initiated coverage of
Theravance Biopharma with a “buy” rating and $41, citing JAK inhibitor can offer a proof of concept super early and market potential well understood.

 

Roth Capital
analyst Joseph Pantginis initiated coverage of Neuralstem with a “buy” rating and $1.20 price target, citing new management has “righted the ship” as it is now focused on small molecule approaches to neurological disorders.