BioShares Biotechnology Clinical Trials (BBC): $21.77, $0.00, +18.3% YTD

BioShares Biotechnology Products (BBP): $36.45, -$0.26, +11.3% YTD

 

 

MARKET COMMENTARY

 

U.S. stock index futures were little changed as investors abstained from betting on riskier assets amid rising global geopolitical tensions. Safe-haven assets benefited, with the yen recording broad gains against major currencies and gold hit its highest level since November. On the economic front, the U.S. Labor Department is expected to issue its job openings and labor turnover survey for February. Weakness in shares of banking and tech companies weighed on European bourses, while Asian markets ended on mixed. Oil eased as rising U.S. shale oil production reduced concerns over geopolitical tensions in the Middle East.

 

 

MARKET HIGHLIGHTS

 

Windtree Therapeutics announced that the AEROSURF® (lucinactant for inhalation) phase 2b independent Data Safety Monitoring Board (DSMB) has completed its second and final interim safety review and has recommended continuing the trial without modification. The final DSMB interim review was convened following achievement in mid-March 2017 of a pre-specified patient enrollment milestone.  In addition, the Company reaffirms its plan to announce top-line results from the AEROSURF phase 2b clinical trial in mid-year 2017.

 

Catalyst Biosciences has achieved a key milestone under its collaboration with ISU Abxis to advance the Factor IX program towards its first human clinical trial. Completion of the CB 2679d/ISU304 toxicology studies supported the recent Investigational New Drug (IND) approval by the Korean Ministry of Food and Drug Safety (MFDS) and triggered a milestone payment to Catalyst.

 

AzurRx BioPharma provided an update on the ongoing open-label Phase IIa trial of lead candidate MS1819 for the treatment of exocrine pancreatic insufficiency (EPI) associated with chronic pancreatitis (CP). The trial is being conducted in partnership with Mayoly Spindler, a European pharmaceutical company.  Early results indicate that MS1819 exhibits a favorable safety profile, with no moderate or severe adverse events having been reported to date.  Additionally, no patients have dropped out of the study once they have qualified to take the active drug.  Regarding efficacy, results to date indicate that MS1819 is active, as measured by coefficient of fat absorption (CFA), which is the parameter being measured for the primary efficacy endpoint.  There was a favorable dose response of greater than 20% improvement in CFA seen in all patients at either the second or third of the four escalating dosage levels of MS 1819. 

 

Ohr Pharmaceutical announced that it plans to amend the ongoing clinical trial investigating Squalamine in wet-AMD (the MAKO Study) to enable efficacy analyses by the end of calendar 2017 or early 2018.  The study remains a multi-center, randomized, double-masked, placebo controlled clinical trial.  The subjects enrolled in the study, over 200 in total, will continue to receive their assigned study treatment of monthly Lucentis® and either Squalamine or placebo drops twice daily, and undergo scheduled visits and assessments through nine months.  The primary endpoint will be an assessment of visual acuity at nine months.  Separately, Ohr announced announced that it has closed its previously announced public offering of common stock and warrants. The Company sold an aggregate 20,250,032 shares of common stock at a price to the public of $0.70 per share. Investors also received warrants to purchase up to an aggregate of 14,175,059 shares of common stock with an exercise price of $1.00. The warrants are immediately exercisable and have a term of five years. Gross proceeds to the Company from the sale of the shares were approximately $14,175,000, excluding any proceeds from the exercise of warrants. The transaction was led by existing investors and included the participation of management and the board of directors.

 

TRACON Pharmaceuticals announced that the Company’s Phase 3 TAPPAS clinical trial of TRC105 in angiosarcoma was awarded Most Innovative Clinical Trial Design at the 2017 Clinical and Research Excellence (CARE) Awards, which are presented by Informa’s Pharma Intelligence, a leading provider of drug, device, company, clinical trial, and market intelligence.

 

CTD Holdings issued an Open Letter to address false and misleading statements, recently made by another public company, regarding CTD’s hydroxypropyl beta cyclodextrin (HPBCD) product, Trappsol® Cyclo™, and its clinical use to treat Niemann-Pick Disease Type C (NPC). The false and misleading statements were made as part of another public company’s announcement of their acquisition of a hydroxypropyl beta cyclodextrin product.

 

Aurinia Pharmaceuticals announced that it will host an Investor Event during the National Kidney Foundation Spring Clinical Meeting on April 20, 2017 at 6:00pm Eastern Time in Orlando, FL.

 

Transgene announced that management will participate in the Kempen 10th Life Sciences Conference on April 19th in Amsterdam, Netherlands, and the Kepler Biotech Days on June 9th in Paris, France.  Transgene will host a “R&D Day”, on June 22, 2017 in Paris, France.

 

Tonix Pharmaceuticals announced the receipt of official minutes from its Initial Cross-Disciplinary Breakthrough Meeting held with the FDA on March 9, 2017. Upon being awarded Breakthrough Therapy designation in December 2016, Tonix was invited to meet with the FDA to evaluate the feasibility of accelerating the development and registration of TNX-102 SL for the treatment of posttraumatic stress disorder (PTSD).

 

Idera Pharmaceuticals a clinical-stage biopharmaceutical company developing toll-like receptor and RNA therapeutics for patients with cancer and rare diseases, today announces successful completion of the Phase I portion of the ongoing Phase I/II clinical trial of intratumoral IMO-2125. Intratumoral IMO-2125 is an agonist of TLR9, in combination with ipilimumab for the treatment of anti-PD-1 refractory metastatic melanoma. Enrollment has begun for the Phase II portion of the trial with the 8mg dose of intratumoral IMO-2125.  The Phase I dose escalation of IMO-2125 in combination with pembrolizumab is ongoing.

 

Intellia Therapeutics announced that the European Patent Office (EPO) has decided to grant a patent broadly covering the CRISPR/Cas9 genome editing technology. The patent includes claims covering compositions of the widely adopted CRISPR single guide RNA technology for use in any non-cellular and cellular setting, including eukaryotic cells such as human or mammalian cells, as well as for use in human therapeutics.

 

Inotek Pharmaceuticals announced the completion of the active recruitment phase of the Phase II dose-ranging trial of a fixed-dose combination (FDC) of trabodenoson and latanoprost for the treatment of glaucoma. Trabodenoson is a highly selective adenosine mimetic that lowers intraocular pressure (IOP) by augmenting the natural function of the trabecular meshwork, the primary outflow pathway in the eye. Latanoprost, a prostaglandin analog, targets the secondary uveoscleral pathway and is the most commonly used drug for lowering IOP. Top-line data from the FDC study are expected in July.

 

Inovio Pharmaceuticals announced preliminary results from the expanded stage of its Phase I study, EBOV-001. The expanded study examined different regimens of its Ebola DNA vaccine INO-4201 using intradermal (skin) administration. The results across both stages of the trial, including both intramuscular and intradermal delivery, demonstrated that 95% (170/179) of evaluable subjects generated an Ebola-specific antibody immune response, with the mean antibody titer comparable or superior to those reported from viral vector-based Ebola vaccines.

 

Bristol-Myers Squibb and Apexigen announced a clinical trial collaboration to evaluate Bristol-Myers Squibb’s Opdivo  (nivolumab) in combination with Apexigen’s APX005M in patients with advanced solid tumors. APX005M is an investigational compound that is designed to activate CD40, a key immune co-stimulatory receptor essential to regulating the activation of both innate and adaptive immune responses against cancer.

 

ArQule announced that it has received clearance from the FDA for the IND application to conduct a phase I clinical trial with ARQ 531 in patients with B-cell malignancies who are refractory to other therapeutic options. ARQ 531 is an investigational, orally bioavailable, potent and reversible inhibitor of both wild type and C481S-mutant Bruton’s tyrosine kinase (BTK).

 

Agile Therapeutics announced it has received the final meeting minutes from its recent NDA pre-submission meeting with the FDA for its lead product candidate, Twirla, an investigational low-dose combined hormonal contraceptive patch. Based on the feedback from the FDA, Agile believes it has the necessary information to complete the resubmission of its NDA, which is expected to be submitted by the end of the second quarter of 2017.

 

Nabriva Therapeutics announced that it has completed enrollment in its first lefamulin evaluation against pneumonia (LEAP 1) Phase III clinical trial. LEAP 1, which is designed to assess the efficacy and safety of lefamulin (IV/oral) compared to moxifloxacin (IV/oral) in patients with moderate to severe community-acquired bacterial pneumonia (CABP), met its enrollment target of 550 patients. The company anticipates availability of top-line clinical data from LEAP 1 in the third quarter of 2017.

 

Five Prime Therapeutics announced that it has completed enrollment in the initially planned 30-patient cohort of the Phase II part of the ongoing clinical trial evaluating cabiralizumab in patients with tenosynovial giant cell tumor, diffuse type, also known as pigmented villonodular synovitis (PVNS), an aggressive tumor confined to the synovium.

 

PTC Therapeutics announced that Shane Kovacs, Chief Financial Officer, will be leaving the company in mid-May 2017 to return to the finance sector, joining RBC Capital Markets as Managing Director where he will help lead the firm’s biotech investment banking efforts.

 

Axovant Sciences announced the pricing of its underwritten public offering of 6,742,179 of its common shares at a price to the public of $18.54 per share. Gross proceeds to Axovant from the offering are expected to be approximately $125.0 million, before deducting underwriting discounts and commissions and estimated offering expenses.  All of the common shares are being offered by Axovant. In connection with this offering, Axovant has granted the underwriters a 30-day option to purchase up to an additional 1,011,326 of its common shares on the same terms and conditions.  The offering is expected to close on April 17, 2017, subject to customary closing conditions.

 

Cytori Therapeutics announced that it has priced an underwritten public offering of 8,600,000 shares of its common stock at a price to the public of $1.10 per share. Gross proceeds, before underwriting discounts and commissions and estimated offering costs, are expected to be approximately $9.5 million. Cytori currently intends to use the net proceeds of the offering for working capital and general corporate purposes, including funding of the HABEO and ATI-0918 development programs. Cytori has granted the underwriter a 45-day option to purchase up to 15% of additional shares of its common stock. The offering is expected to close on or about April 17, 2017, subject to satisfaction of customary closing conditions. Maxim Group is acting as sole book-running manager for the offering.

 

 

ANALYST RECOMMENDATIONS

 

HC Wainwright analyst Ed Arce increased his price target of Paratek to $36 from $33, citing top-line results from its pivotal Phase 3 trial of omadacycline for the treatment of community-acquired bacterial pneumonia (CABP) that demonstrates the drug’s strong efficacy across the board, and better overall safety than active comparator, moxifloxacin.

 

Cantor analyst Elemer Piros initiated coverage of Aurinia Pharmaceuticals with an “overweight” rating and $14 price target, citing voclosporin, a drug analog of cyclosporine, has demonstrated compelling efficacy in its Phase 2b AURA-LV study for patients with active lupus nephritis (LN) and is well-positioned in late-stage development to succeed.

 

FBR analyst Vernon Bernardino increased his price target of Aurinia Pharmaceuticals to $11 from $9, citing AUPH is now in a strong position to independently advance voclosporin (VCS), its investigational therapy for patients with active lupus nephritis (LN), into Phase III testing.

 

Wedbush analyst Robert Driscoll assumed coverage of Cidara Therapeutics with an “outperform” rating and $13 price target, citing the upbeat tone at the analyst day, and see the re-focused development strategy on targeting life-threatening infections with IV therapies as risk-reduced.

 

Chardan analyst Gbola Amusa increased his price target of Regenxbio to $50 from $35, citing conviction on the landmark Danos appointment.

 

Following OncoMed’s announcement that demcizumab had missed its primary endpoint in the Phase 2 YOSEMITE trial in pancreatic cancer and its partner Bayer had decided not to opt-in to develop Wnt inhibitors vantictumab and ipafricept; HC Wainwright analyst Shaunak Deepak decreased his price target to $9 from $20; BMO analyst Ian Somaiya downgraded the stock to “market perform” from “outperform” and decreased his price target to $6 from $18; SunTrust analyst Peter Lawson decreased his price target to $9 from $16; Jefferies analyst Brian Abrahams decreased his price target to $10 from $15. 

 

Chardan analyst Gbola Amusa increased his price target of Avexis to $102.50 from $100, citing model updates.