BioShares Biotechnology Clinical Trials (BBC): $19.12, -$0.47, -34.2% YTD

BioShares Biotechnology Products (BBP): $33.50, -$0.43, +8.7% YTD

 

 

MARKET COMMENTARY

 

U.S. stock index futures rose, the dollar held steady, while gold slipped ahead of a key two-day U.S Federal Reserve meeting, which is almost certain to conclude with the second rise in U.S. interest rates since the global financial crisis. Investors are expected to examine the Fed’s statement and economic forecasts for any signs of how the central bank thinks Trump’s election has affected the outlook for growth and inflation. Import and export data for November are on the economic calendar. European shares rose, helped by gains in banks after UniCredit unveiled a 13 billion euro share issue. Asian markets closed higher. Oil prices were stable, supported by strong demand in Asia and a supply cut by Abu Dhabi as part of production curbs organized by OPEC and other exporters.

 

 

MARKET HIGHLIGHTS

 

ExpreS2ion Biotechnologies ApS and ABIVAX S.A. have entered into a service agreement and a term sheet for a commercial license agreement under which ExpreS2ion and ABIVAX collaborate in the development of their proprietary prophylactic and treatment for Ebola virus infection, ABX544. The financial terms of the agreement were not disclosed.

 

Aurinia Pharmaceuticals announced that it has received the final End of Phase II (EOP2) meeting minutes from the U.S. Food & Drug Administration Division of Pulmonary, Allergy and Rheumatology Products. The minutes are consistent with the preliminary responses that were issued to the Company prior to the meeting which took place on October 25, 2016.

 

Aurinia Pharmaceuticals announced the appointment of Lorin Jeffry “Jeff” Randall to its board of directors and Chairman of the Audit Committee. Mr. Randall currently serves on the boards of directors of Athersys, Inc. where he serves as Chairman of the Audit and Compensation Committees, and Acorda Therapeutics, Inc. where he serves on the Audit, Compliance and Nominations and Governance Committees.

 

Paratek Pharmaceuticals projects its ongoing pivotal Phase 3 clinical study evaluating omadacycline for the treatment of community acquired bacterial pneumonia (CABP) will report top-line data during the second quarter of 2017.  The company had previously estimated that this trial would report top-line data as early as the third quarter of 2017.  The revised estimate reflects faster-than-expected patient enrollment in the study. This study, which is designed to assess the efficacy and safety of IV to once-daily oral omadacycline compared with moxifloxacin in subjects with CABP, enrolled its first patient in November 2015.

 

BioDelivery Sciences International announced that its Phase 2b clinical study assessing the efficacy and safety of Clonidine Topical Gel for the management of painful diabetic neuropathy failed to show a statistically significant difference in pain relief between Clonidine Topical Gel and placebo.  As a result, BDSI is discontinuing further development of the product at this time.

 

Zealand Pharma announced that it has dosed the first patients in its Phase IIa clinical trial with dasiglucagon in a dual-hormone artificial (or bionic) pancreas system from Beta Bionics. Dasiglucagon is a Zealand-invented glucagon analogue with a unique stability profile in liquid formulation. The multiple-dose version of dasiglucagon is intended for use in a dual-hormone artificial pancreas system to better control hypoglycaemia and, potentially, hereby provide insulin treated diabetes patients with options for easier and more effective management of their disease.

 

Addex Therapeutics announced today that the pharmacological profile of ADX71441, gamma-aminobutyric acid subtype B (GABAB) receptor positive allosteric modulator (PAM), was published in Neuropharmacology, a leading peer reviewed journal.

 

Achaogen announced that it has commenced an underwritten public offering of up to 5,750,000 shares of its common stock. All of the shares to be sold in the offering will be offered by Achaogen. In addition, Achaogen expects to grant the underwriters of the offering a 30-day option to purchase up to an additional 862,500 shares of common stock at the public offering price, less underwriting discounts and commissions. The offering is subject to market and other conditions, and there can be no assurance as to whether or when the offering may be completed, or as to the actual size or terms of the offering.

 

Proteon Therapeutics announced that its first Phase III clinical trial with investigational vonapanitase, PATENCY-1, did not meet its primary endpoint of improved primary unassisted patency compared to placebo (p=0.254). However, the top-line results for the trial’s secondary endpoint suggested that vonapanitase may improve secondary patency compared to placebo (p=0.048). Data from one of the trial’s three tertiary endpoints also suggested vonapanitase may improve unassisted fistula use for hemodialysis (p=0.035) and any use of the fistula (unassisted or assisted) for hemodialysis (p=0.006). Adverse events reported for vonapanitase were similar to placebo.

 

Actinium Pharmaceuticals announced that the company has submitted an application with the EMA seeking Orphan Designation for Actimab-A for patients newly diagnosed with acute myeloid leukemia (AML) age 60 and above who are ineligible for currently used induction therapies. Actimab-A is currently in a 53-patient, multicenter open label Phase II trial where it is being studied as a monotherapy in these patients who have low peripheral blast (PB) burden. In a previously completed Phase I trial, Actimab-A showed a 50% composite response rate at the dose level of 2.0 μCi/kg/fraction, which is the dose level being studied in the current Phase II trial, in patients with low PB burden.

 

aTyr Pharma announced clinical results from exploratory trials assessing the safety and potential activity of Resolaris, including: Top-line results from a completed Phase Ib/II trial for adult patients with limb-girdle muscular dystrophy 2B (LGMD2B/dysferlinopathy) or facioscapulohumeral muscular dystrophy (FSHD) (the "004 Trial"); Interim data from an ongoing Phase Ib/II trial with early onset FSHD (the "003 Trial"); and Interim data from an ongoing long-term safety extension study (the "005 Trial") for patients from aTyr’s adult FSHD trial completed earlier this year (the "002 Trial"). The results announced today highlight the potential of Resolaris, an immuno-modulator of activated T cells, as a single treatment for multiple rare myopathies with an immune component (RMIC).

 

Arbutus Biopharma reported new results from the ongoing Phase II study of ARB-1467 in chronically infected HBV patients on stable nucleos(t)ide therapy, and plans for a new cohort to be added to the Phase II study. Arbutus reported multiple dose results from Cohort 2, which showed a significant reduction in HBsAg. In both Cohort 1 and Cohort 2, an additive, step-wise reduction in HBsAg was observed with each dose. The HBsAg reduction achieved after three monthly doses of 0.4mg/kg in Cohort 2 was greater than that seen at 0.2 mg/kg in Cohort 1, demonstrating a dose-response seen with repeat dosing. In Cohort 2, three of the five patients who received three doses of ARB-1467 experienced a greater than 1 log(10) IU/mL reduction in serum HBsAg. The Company plans to add another cohort to this study to explore bi-weekly administration of the 0.4 mg/kg dose.

 

Synthetic Biologics announced that the United States Patent Office has granted U.S. Patent No. 9,512,204 which provides composition of matter coverage for SYN-005, the Company’s preclinical drug candidate designed to treat or prevent the development of Pertussis (whooping cough), especially in infants. This patent, which is co-owned with Synthetic Biologics’ Pertussis academic collaborator, The University of Texas at Austin (UT Austin), is also exclusively licensed to Synthetic Biologics. The patent covers, among others, the Company’s monoclonal antibody, hu1B7, a component of SYN-005 which is the subject of an ongoing study funded by a grant from the Bill & Melinda Gates Foundation to UT Austin. The exclusivity provided by this new patent has an expiration date of 2035, which does not include a potential patent term extension.

 

Sage Therapeutics announced the initiation of Phase II clinical development of SAGE-217, a novel, internally-developed, next generation oral GABA(A) receptor modulator that Sage is developing in both mood and movement disorders. Dosing has now been initiated in the first of two movement disorder indications. Patients are receiving SAGE-217 in a Phase II proof-of-concept trial in Parkinson’s disease. In addition, trial sites are open and screening patients for a Phase IIa trial in essential tremor. Patients in this trial are expected to be dosed imminently. Top-line results from the Part A open-label study in Parkinson’s disease are expected in the first half of 2017 and the essential tremor study is anticipated to report results in the second half of 2017. The Company also plans to initiate Phase II clinical trials of SAGE-217 in two mood disorders — major depressive disorder (MDD) and postpartum depression (PPD).

 

AcelRx Pharmaceuticals announced that it has submitted a NDA under section 505(b)(2) with the FDA for ARX-04 (sufentanil sublingual tablet, 30 mcg) for the treatment of patients experiencing moderate-to-severe acute pain in a medically supervised setting.

 

Moleculin Biotech announced its scientific presentation at the 28(th) EORTC-NCI-AACR Symposium on Molecular Targets and Cancer Therapeutics. Waldemar Priebe, PhD, Moleculin’s Founder, Founding Scientist, and Chair of the Scientific Advisory Board presented the Abstract on November 30(th) at the Symposium in Munich, Germany.

 

SCYNEXIS announced two new development milestones for SCY-078, a novel antifungal agent. The SCY-078 Phase I intravenous (IV) Program consists of single-ascending and multiple-ascending dose studies to test different IV formulations and doses in healthy volunteers followed by oral regimens. Based on the available safety and pharmacokinetics (PK) data from this program, we have identified an IV formulation and dose regimen that, pending discussions with the FDA, we plan to test in the upcoming clinical trials of SCY-078. Additionally, the SCY-078 toxicology program was expanded to include three-month oral dose studies in two species (rats and dogs). Consistent with findings from prior non-clinical toxicology studies of shorter durations, these longer-term toxicity studies confirmed the favorable safety profile of oral SCY-078. These results would allow flexible treatment regimens of SCY-078 for up to three months in our next stages of clinical development, which is particularly relevant for patients with invasive aspergillosis or refractory fungal infections.

 

RedHill Biopharma reported that, following a pre-planned review of safety data from its ongoing Phase III study with RHB-104 for Crohn’s disease (the MAP US study) by an independent Data and Safety Monitoring Board (DSMB), it has received a unanimous recommendation to continue the study as planned, without any modifications.

 

CytomX Therapeutics announced that Bristol-Myers Squibb has selected a clinical candidate for its CTLA-4 Probody program under the strategic oncology collaboration established in May 2014. Achieving this milestone results in a $2 million payment to CytomX.

 

Dimension Therapeutics announced that the FDA accepted Dimension’s IND application for DTX301 for the treatment of Ornithine Transcarbamylase (OTC) Deficiency. Dimension anticipates commencing a global, multi-center Phase I/II clinical trial to evaluate DTX301 in patients with OTC Deficiency by the end of 2016.

 

Spotlight Innovation announced that the Company has obtained from the Florida State University Research Foundation exclusive, worldwide rights to develop and commercialize certain compounds for the treatment of viral infections, including Zika virus infection. Included among the licensed compounds are those identified in a study co-authored by Florida State University Professor Hengli Tang that was published in Nature Medicine in August of this year. Prof. Tang and his research team collaborate with Spotlight Innovation as part of an existing sponsored research agreement (SRA), and he is a member of the Company’s Scientific Advisory Board.

 

Kite Pharma and Vitruvian Networks announced a strategic partnership to create a software solution to support commercial availability of T-cell therapies. Together, the parties will design and develop a platform for patients, physicians and treatment centers that enables commercial-scale ordering, logistics, monitoring and delivery of autologous cell therapies if they are FDA-approved, including axicabtagene ciloleucel (formerly known as KTE-C19), Kite’s lead investigational engineered T-cell therapy for aggressive non-Hodgkin lymphoma.

 

Anthera Pharmaceuticals announced that the Data Monitoring Committee (DMC) completed its pre-planned safety review of the SIMPLICITY clinical study of Sollpura supplied as a powder for oral solution in an initial cohort of cystic fibrosis patients seven years of age and older with exocrine pancreatic insufficiency. The DMC was in unanimous agreement "that there are no safety concerns and that the study can move forward to enrolling Part B," which will enroll patients from 28 days to 6 years of age.

 

Novocure presented data from its Phase II pilot INNOVATE clinical trial at its research and development day suggesting that Tumor Treating Fields (TTFields) in combination with weekly paclitaxel is tolerable and safe in patients with recurrent ovarian cancer. The data also suggested more than doubling of the progression free survival and an improvement in overall survival among patients who received TTFields therapy with paclitaxel compared to a recent Phase III trial of patients who received paclitaxel alone.  Novocure will submit the data for presentation at an upcoming medical conference and will begin planning a Phase III pivotal trial in recurrent ovarian cancer.

 

Repros Therapeutics announced it has requested a meeting to discuss Phase III requirements for the development of Proellex (telapristone acetate) for the treatment of symptomatic uterine fibroids. The Company anticipates a meeting will be scheduled during the first half of 2017.

 

Revance Therapeutics announced positive interim results from its U.S. Phase II open-label, dose-escalating clinical study of DaxibotulinumtoxinA Injectable (RT002) to treat moderate-to-severe isolated cervical dystonia in adults, a movement disorder of the neck.

 

MorphoSys AG announced that the first patient was dosed in a Phase II combination trial of MOR208 with idelalisib (Zydelig). The trial, which has been named COSMOS (CLL patients assessed for ORR & Safety in MOR208 Study), is designed to evaluate the safety and efficacy of MOR208 in combination with the PI3K delta inhibitor idelalisib in adult patients with relapsed or refractory chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL). Patient enrolled must have been refractory or shown relapse or intolerance to a prior, most recent, therapy with a Bruton’s Tyrosine Kinase (BTK) inhibitor (e.g. ibrutinib). MOR208 is an investigational Fc-engineered monoclonal antibody targeting CD19 that is being developed for the treatment of patients with B cell malignancies. CLL is the most common type of leukemia in Western populations.

 

Pharnext SA presented positive exploratory Phase II data for PXT864, a PLEODRUG? being developed for the treatment of Alzheimer’s disease (AD), at the 9th Clinical Trials on Alzheimer’s Disease (CTAD) conference, on December 8-10, 2016 in San Diego (United States).

 

Tocagen announced its randomized, controlled Phase II/III clinical trial, called Toca 5, is now enrolling patients in Israel. Enrollment in the trial is ongoing in the United States and Canada. The study compares a cancer-selective virus, Toca 511 (vocimagene amiretrorepvec), in combination with Toca FC (extended-release 5-fluorocytosine) to standard of care in patients with first or second recurrence of glioblastoma or anaplastic astrocytoma who are undergoing resection.

 

GlaxoSmithKline announced the submission of a regulatory application to the Japanese Ministry of Health, Labor and Welfare (MHLW) for belimumab, in adult patients with active, autoantibody-positive systemic lupus erythematosus (SLE) who have an inadequate response to standard therapy.

 

Eli Lilly announced that people who use Lilly insulin will be able to access discounted prices for their purchases starting January 1, 2017 via mobile and web platforms hosted by Blink Health. The discounts, provided by Lilly through a partnership with Express Scripts, may reduce costs for people who pay full retail prices at the pharmacy, such as those who have no insurance or are in the deductible phase of their high-deductible insurance plans. 

 

Eagle Pharmaceuticals announced positive results from its pivotal animal study conducted under the Animal Rule1, for Ryanodex for the treatment of Exertional Heat Stroke (“EHS”), an investigational new indication for the product. Due to the rare and unpredictable nature of EHS, the animal work is an essential part of the hybrid development program in support of the efficacy of Ryanodex for the treatment of EHS. The hybrid program is comprised of safety and efficacy data from a human study in EHS patients, and efficacy data from an established animal model. Both components are necessary to fulfill the NDA submission requirements. Eagle previously filed human safety and efficacy clinical data as part of the rolling NDA submission.

 

Soligenix announced today the pricing of an underwritten public offering of 1,670,000 shares of its common stock and warrants to purchase up to an aggregate of 2,087,500 shares of its common stock at a combined offering price of $3.16. The warrants will have an initial per share exercise price of $3.95, subject to customary adjustment in connection with stock splits, stock dividends, reclassifications, combinations and other similar events, are exercisable immediately and will expire five years from the date of issuance. The gross proceeds to Soligenix, Inc. from this offering are expected to be approximately $5,277,200, before deducting underwriting discounts and commissions and other estimated offering expenses. Soligenix, Inc. has granted the underwriters a 45-day option to purchase up to an additional 250,500 shares of common stock and/or 313,125 additional warrants to cover over-allotments, if any. The offering is expected to close on December 16, 2016, subject to customary closing conditions. The Company’s common stock and warrants are expected to begin trading on The Nasdaq Capital Market under the symbols "SNGX" and "SNGXW," respectively, on December 13, 2016. Aegis Capital Corp. is acting as the sole book-running manager for the offering. Maxim Group LLC is acting as co-manager for the offering.

 

CytRx announced that it has entered into securities purchase agreements with existing institutional investors to purchase shares of our common stock at a purchase price of $0.42 per share in a registered direct offering with gross proceeds of $8.1 million. To the extent that the purchase of shares of common stock would result in beneficial ownership above 4.99%, an investor may elect to purchase shares of Series B convertible preferred stock in lieu of common stock. Each share of preferred stock has a conversion price of $0.42 and no dividend rights. The offering is expected to close on or about December 16, 2016, subject to the satisfaction of customary closing conditions. Rodman & Renshaw, a unit of H.C. Wainwright acted as exclusive placement agent for the offering.

 

 

ANALYST RECOMMENDATIONS

 

Following Achaogen’s announcement of better than expected results from its EPIC and CARE trials, Aegis analyst Difei Yang increased her price target to $20 from $10; William Blair analyst Katherine Xu increased her price target to $25 from $11; Needham analyst Alan Carr upgraded the stock to “buy” from “hold.”

 

Morgan Stanley analyst Matthew Harrison downgraded Alexion to “equal-weight” from “overweight” and removed his price target, citing lack of clarity despite solid fundamentals based on the next-gen Soliris strategy and potential pipeline drivers.

 

Chardan analyst Madhu Kumar downgraded Arbutus to “neutral” from “buy” and decreased his price target to $3 from $4, citing Liver safety risk overwhelms promising efficacy.

 

Oppenheimer analyst Leah Rush Cann initiated coverage of the following companies: Amgen with an “outperform” rating and $166 price target; Acceleron with an “outperform” rating and $44 price target.

 

Following Opthotech’s announcement that Phase III studies of Fovista treatment in combination with Lucentis for wet Age-related Macular Degeneration patients failed to achieve their primary efficacy endpoints, Morgan Stanley analyst Matthew Harrison downgraded the stock to “equal-weight” from “overweight” and decreased his price target to $7 from $99; Goldman analyst Jami Rubin upgraded the stock to “neutral” from “sell” and decreased her price target to $5 from $22.