BioShares Biotechnology Clinical Trials (BBC): $22.97, +$0.18, +24.8% YTD

BioShares Biotechnology Products (BBP): $37.37, +$0.27, +14.1% YTD

 

 

MARKET COMMENTARY

 

U.S. stock index futures slumped, and global market sentiment dampened, as U.S. President Donald Trump’s defeat over healthcare reform raised concerns over his ability to push through tax cuts and fiscal spending to boost the economy. The dollar weakened, while gold rose as investors rushed to the safety of safe-haven assets. Oil prices inched lower, pressured by uncertainty over whether an OPEC-led supply cut will be extended beyond June in an effort to counter record crude inventories.

 

 

MARKET HIGHLIGHTS

 

Advaxis published online a poster previously presented at the National Cancer Research Institute (NCRI) Cancer Conference in Liverpool that showed axalimogene filolisbac achieved durable response in a patient with persistent or recurrent metastatic (squamous or non-squamous cell) carcinoma of the cervix (PRmCC).  Nine patients who had documented disease progression after they had received curative treatments of chemotherapy and/or radiation with or without bevacizumab were enrolled in this phase 1, open-label, dose-determining study.  Axalimogene filolisbac was well-tolerated across two dose levels.  The study also established a recommended phase 2 dose of 1×1010 CFU and demonstrated antitumor activity at that dose. Axalimogene filolisbac was safely administered at 5 and 10 times the dose levels previously studied, without any significant toxicity. One patient experienced an ongoing and durable partial response.  This patient was recently featured in the Augusta Chronicle, as she is being treated at the Georgia Cancer Center at Augusta University.

 

BIOPHYTIS announced that the first clinical centers leading the observational study SARA-OBS have opened in Europe and started the recruitment of patients. These patients, diagnosed with sarcopenia, could later be treated with drug candidate Sarconeos in the phase 2b clinical trial SARA-INT.  The first clinical centers have opened and started the screening of patients with sarcopenia in Belgium, (Liège, Prof. Bruyère, Liège University), in France (Toulouse, Profs. Vellas & Rolland, Gérontopole). It will soon start in Italy (Roma, Profs. Donini & Gnessi, La Sapienza University). Regulatory approvals to start SARA-OBS study in these countries have been granted in 2016.

 

Bellerophon Therapeutics announced a poster presentation at the 37th Annual Meeting for the International Society for Heart and Lung Transplantation (ISHLT) which will take place in San Diego, CA, from April 5 to 8.  The poster will feature data from an investigator-led study of INOpulse® in patients with pulmonary arterial hypertension (PAH).

 

Foamix Pharmaceuticals announced the topline results of its two Phase 3 clinical trials investigating FMX101, Trial 04 and Trial 05, in patients with moderate-to-severe acne.  In the intent-to-treat analysis, FMX101 demonstrated statistical significance compared to vehicle on both co-primary endpoints in Trial 05, but did not demonstrate statistical significance on one of the two co-primary endpoints in Trial 04, specifically IGA success.

 

Allergan and Paratek Pharmaceuticals announced that two (2) Phase 3 trials of sarecycline for the treatment of moderate to severe acne met their 12 week primary efficacy endpoints. Sarecycline is a once-daily, oral, narrow spectrum tetracycline-derived antibiotic with anti-inflammatory properties for the potential treatment of moderate to severe acne in the community setting.  Based on these data, Allergan plans to file a New Drug Application (NDA) to the U.S. Food & Drug Administration (FDA) in the second half of this year.

 

Aurinia Pharmaceuticals announced the 48-week results from the “Aurinia Early Urinary Protein Reduction Predicts Response Study” (AURION) open-label study of voclosporin for the treatment of lupus nephritis (LN) at the 12th International Congress on Systemic Lupus Erythematosus and 7th Asian Congress on Autoimmunity jointly in Melbourne, Australia. The data were presented by Robert B. Huizinga, Vice President of Clinical Affairs at Aurinia.  The study successfully achieved its primary objective by demonstrating that early biomarker response in active LN patients can be a significant predictor of renal response at 24 and 48 weeks.

 

Kitov Pharmaceuticals announced that TyrNovo Ltd., a company majority-owned by Kitov, will present preclinical data on TyrNovo’s anti-tumor resistance drug candidate NT219 in a poster session at the American Association for Cancer Research (AACR) Annual Meeting 2017, to be held April 1-5, at the Walter E. Washington Convention Center in Washington, D.C.

 

Cellect Biotechnology announced today that the first stem cell transplant procedure has been successfully performed using its ApoGraft™ technology in the Company’s Phase I/II clinical trial in a blood cancer patient.

 

VBL Therapeutics announced financial results for the 3- and 12-months ended December 31, 2016.  At December 31, 2016, the Company had cash, cash equivalents and short-term bank deposits of $45.3 million and working capital of $41.8 million. The Company expects that its cash and cash equivalents and short-term bank deposits will enable it to fund operating expenses and capital expenditure requirements into 2019.  The Company reported a net loss for the year ended December 31, 2016 of $16.0 million, or ($0.64) per share, compared to a net loss of $14.9 million, or ($0.73) per share in the year ended December 31, 2015.

 

Onconova Therapeutics reported financial results for the year ended December 31, 2016.  Net loss was $19.7 million for the year ended December 31, 2016, compared to $24.0 million for the year ended December 31, 2015.  Cash and cash equivalents as of December 31, 2016, totaled $21.4 million, compared to $19.8 million as of December 31, 2015.  Onconova believes that its current cash and cash equivalents will be sufficient to fund its ongoing trials and operations into the fourth quarter of 2017. 

 

Arena Pharmaceuticals will present a corporate update at the 16th Annual Needham Healthcare Conference on Tuesday, April 4 at 3:40pm ET.

 

SciClone Pharmaceuticals will present a corporate overview and business update at the 16th Annual Needham Healthcare Conference at 5:00 pm ET on Tuesday, April 4, 2017. 

 

AstraZeneca announced that the China Food and Drug Administration (CFDA) has granted marketing authorization for Tagrisso (osimertinib) 40 mg and 80mg once-daily oral tablets for the treatment of adult patients with locally-advanced or metastatic epidermal growth factor receptor (EGFR) T790M mutation-positive NSCLC whose disease has progressed on or after EGFR tyrosine kinase inhibitor (TKI) therapy.

 

ArQule reported that its partner, Kyowa Hakko Kirin, announced top-line results of the JET-HCC Phase III trial of tivantinib in Japan, and that the trial did not meet its primary endpoint of progression free survival (PFS).

 

Innovus Pharmaceuticals received a Notice of Intention to Grant from the European Patent Office (“EPO”) to issue a European-wide patent entitled “Sensitization composition and method of use” for its Sensum+® product, for reduced penile sensitivity. On March 8, 2017, Innovus Pharma announced that it received the CPNP notification number required for commercialization of Sensum+® in all 28 member countries of the European Union.

 

Synergy Pharmaceuticals announced that the company has submitted a sNDA for TRULANCE (plecanatide) for the treatment of adults with irritable bowel syndrome with constipation (IBS-C).

 

Matinas BioPharma Holdings reported positive topline data from its Phase I single-ascending dose study of MAT2501 in healthy volunteers.

 

Newron Pharmaceuticals presented detailed results of a Phase IIa study with its unique sodium channel blocker, Evenamide (NW-3509), in patients with schizophrenia. The new chemical entity is orally available and specifically targets voltage-gated sodium channels by a unique mechanism of action.

 

Nektar Therapeutics announced that it has begun dosing in a Phase I clinical study evaluating NKTR-358, the company’s new biologic therapy, which is being developed to treat a wide range of auto-immune diseases and inflammatory disorders. NKTR-358 selectively stimulates the growth and activation of regulatory T cells in the body in order to restore the body’s self-tolerance mechanisms.  Unlike immunosuppressant medicines that treat the symptoms of auto-immune disease by inhibiting the entire immune system which can cause unwanted side effects, NKTR-358 is designed to correct the underlying immune system dysfunction found in patients with immune disorders. The Phase I study will evaluate single-ascending doses of NKTR-358 in approximately 50 healthy subjects.

 

Novo Nordisk announced that Fiasp, a new, fast-acting mealtime insulin for the treatment of diabetes in adults, has been launched in Canada, following the recent marketing authorization from Health Canada on 6 January 2017.

 

EUSA Pharma and Apeiron Biologics announced that the CHMP of the EMA has adopted a positive opinion recommending the approval of dinutuximab beta for use in the treatment of high-risk neuroblastoma.

 

APEIRON Biologics AG announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending the approval of dinutuximab beta for immunotherapy of high risk neuroblastoma.

 

Pfizer announced that the European Commission has approved XELJANZ (tofacitinib citrate) 5 mg twice daily (BID) oral tablets in combination with methotrexate (MTX) for the treatment of moderate to severe active rheumatoid arthritis (RA) in adult patients who have responded inadequately to, or who are intolerant to one or more disease-modifying antirheumatic drugs (DMARDs). XELJANZ can be given as monotherapy in case of intolerance to MTX or when treatment with MTX is inappropriate. XELJANZ belongs to a new class of therapies called Janus kinase (JAK) inhibitors.

 

Infectex announced positive results of a Phase IIb-III clinical study of SQ109 added to the standard drug therapy regimen in patients with multidrug-resistant pulmonary tuberculosis (MDR-TB). SQ109 is a new small molecule drug discovered by scientists at Sequella (USA) and the US National Institutes of Health.

 

Dr. Reddy’s Laboratories and Integra LifeSciences announced today that they have entered into an exclusive distribution agreement. Under the agreement, Dr. Reddy’s will market and distribute DuraGen Plus and Suturable DuraGen Dural Regeneration Matrices for use in patients in India.

 

Eagle Pharmaceuticals announced that their 505(b)(2) NDA for Ryanodex (dantrolene sodium) for the treatment of exertional heat stroke (EHS) has been accepted for filing and granted a priority review designation by the FDA. The FDA grants priority review to medicines that may offer major advances in care or provide a treatment option where no adequate therapy exists. Under the PDUFA, the FDA will aim to complete its review within six months of the NDA submission; the PDUFA date for the NDA has been set for July 23, 2017.

 

Opiant Pharmaceuticals announced the completion of a study evaluating two doses of a naloxone nasal spray on the occupation of brain opiate receptors using PET imaging.  The study was commissioned by the National Institute for Health and Welfare of Finland and was carried out by researchers at Clinical Research Services Turku (CRST) and Turku PET Centre, a leading international PET center.

 

Summit Therapeutics announced that it will proceed with the planned extension phase of PhaseOut DMD, a Phase II clinical trial evaluating the utrophin modulator ezutromid, subject to regulatory approval. This follows an interim review of the safety and tolerability data from the ongoing trial by an independent Data Monitoring Committee and its support of Summit’s plans to extend the clinical trial.

 

ZIOPHARM Oncology announced the receipt of positive guidance from an End-of-Phase II meeting with the FDA for its lead gene therapy product candidate, Ad-RTS-hIL-12 plus orally administered veledimex (V), to harness and control IL-12 production for the investigational treatment of recurrent glioblastoma.

 

Mesoblast Limited announced it has successfully completed a fully underwritten institutional placement of 26.25 million new shares (approx. 6% of issued capital) and has raised approximately $40 million. The placement price of A$2.00 per share represents a 4.8% discount to the 15 day VWAP of A$2.10. Existing global institutional investors, together with new institutional and sophisticated investors, have strongly supported and participated in the placement. The proceeds will be used for Mesoblast’s ongoing Phase III clinical programs including chronic heart failure, as well as for manufacturing requirements associated with product commercialization. The Company expects to report during CY2017 multiple clinical and regulatory outcomes related to its Tier 1 product candidates, which may facilitate strategic alliances with partners who share our corporate vision. Bell Potter Securities Limited acted as Lead Manager and Underwriter to the placement.

 

Akcea Therapeutics announced that it has filed a registration statement on Form S-1 with the SEC relating to a proposed initial public offering of its common stock. The number of shares to be offered and the price range for the proposed offering have not been determined. Akcea has applied to list its common stock on the Nasdaq Global Market under the trading symbol "AKCA." Cowen and Company, Stifel and Wells Fargo Securities are acting as joint book running managers for the offering.

 

Impax Laboratories announced that its Board of Directors has appointed Paul M. Bisaro as Impax’s President and CEO and a member of the Company’s Board, effective March 27, 2017. Mr. Bisaro will succeed J. Kevin Buchi, who has served as Interim President and CEO since December of 2016.

 

Aptose Biosciences announced that Mr. Avanish Vellanki, Senior Vice President and Chief Business Officer, tendered his resignation with the Company. The resignation is effective immediately, and the Board of Directors does not contemplate replacing Mr. Vellanki at this time.

 

Lannett Company announced that its former chairman and CEO William (Bill) Farber, R. Ph., has passed away from natural causes.  Mr. Farber served as the company’s CEO from 1991 until 2006, chairman from 1991 to 2011 and chairman emeritus from 2011 until 2014.

 

Insys Therapeutics announced that, effective April 17, 2017, Saeed Motahari, will become the Company’s President and CEO and be appointed to the Company’s Board of Directors.

 

ProQR Therapeutics N.V. announced that it appointed David M. Rodman, MD as Chief Development Strategy Officer.

 

Alexion Pharmaceuticals announced that its Board of Directors has appointed Ludwig N. Hantson, Ph.D., as CEO and member of the Board of Directors, effective immediately. Dr. Hantson most recently served as President and CEO of Baxalta, a successful spin-off as a public company from Baxter.

 

 

ANALYST RECOMMENDATIONS

 

HC Wainwright analyst Ed Arce revised his price target of the following companies: CymaBay Therapeutics increased to $7 from $6; Galmed decreased to $12 from $13.

 

Janney analyst Paul Knight revised his price target and rating of the following companies: Bio-Techne downgraded to “neutral” from “buy” and decreased his price target to $105 from $115; PerkinElmer upgraded to “buy” from “neutral” and increased his price target to $75 from $55; Bruker downgraded to “neutral” from “buy” and decreased his price target to $23 from $28.

 

Wedbush analyst Liana Moussatos decreased her price target of Xoma to $9 from $14, citing Xoma’s new strategy of focusing on out-licensing all internal assets while maintaining a low cash burn could take some time to develop and drive shareholder value.

 

HC Wainwright analyst Shaunak Deepak initiated coverage of CytomX with a “buy” rating and $24 price target, citing CytomX’s proprietary Probody technology offers enhanced specificity for cell killing agents, allowing a drug to be selectively activated in the tumor microenvironment, sparing healthy tissue.

 

Cowen analyst Doug Schenkel initiated coverage of Exact Sciences with an “outperform” rating and $30 price target, citing Exact Science’s Cologuard is a less invasive screening test for colon cancer and it is FDA approved.

 

Jefferies analyst Eun Yang increased her price target of Tesaro to $124 from $80, citing near-term takeout possibility.

 

Stifel analyst Thomas Shrader increased his price target of Bioverativ to $57 from $54, citing less 2016 SG&A spend than expected.