BioShares Biotechnology Clinical Trials (BBC): $20.09, -$0.73, -30.8% YTD
BioShares Biotechnology Products (BBP): $30.63, -$0.95, -0.6% YTD
U.S. stock index futures were in the red, tracking movement in global markets ahead of a meeting of the European Central Bank on expectations that officials are beginning to consider reining in the volume of extra emergency aid they give to the euro zone economy next year. Investors keenly await industrial production data and NY Fed manufacturing figures. Gold edged lower and the dollar receded from seven-month highs. Crude prices slipped due to a rising rig count in the United States and record OPEC output.
Eiger BioPharmaceuticals announced today that the European Medicines Agency (EMA) has granted orphan designation to exendin 9-39 for the treatment of non-insulinoma pancreatogenous hypoglycemia syndrome (NIPHS). NIPHS describes a spectrum of acquired metabolic disorders characterized by inappropriately high insulin levels (hyperinsulinemia) and low blood glucose levels (hypoglycemia), which includes post-bariatric hypoglycemia (PBH). Eiger is developing a novel subcutaneously administered form of exendin 9-39 for patients who experience dangerously low postprandial (postmeal) blood glucose levels due to PBH, a rare and debilitating disorder with no approved treatment.
Gemphire Therapeutics is a sponsor of the FH Global Summit being held in Dallas, TX this year and the Advocates for Awareness program, an educational campaign by the FH Foundation that focuses on community outreach and strives to raise awareness of this vastly under diagnosed and untreated disease. The FH Foundation is a patient-centered non-profit organization dedicated to research, advocacy, and education of all forms of familial hypercholesterolemia including HoFH and HeFH.
TiGenix NV announced that Cx601, its lead product candidate being developed for the treatment of complex perianal fistulas in Crohn’s disease patients, has been granted Orphan Drug Designation status in Switzerland. This is the second orphan drug designation granted to Cx601.
Addex Therapeutics will conduct Phase IIa Proof of Concept Study of dipraglurant in focal cervical dystonia. Addex Therapeutics expects to initiate the trial in the fourth quarter of 2016. Addex’s Phase IIa Proof of Concept study will include 18 focal CD patients who are currently sub-optimally treated with BoNT. The single center study will be double-blinded and placebo-controlled. A single dose of dipraglurant will be administered in a crossover design. The TWSTR scale, a well-established clinical rating scale designed to detect drug induced changes, will serve as the primary endpoint of the trial. Key secondary endpoints will include an evaluation of the Cervical Dystonia Impact Profile, a patient-reported outcome for quality of life, pharmacokinetics and safety and tolerability.
DelMar Pharmaceuticals presented additional data regarding VAL-083′s unique anti-cancer mechanism on Saturday October 15, 2016 at the 12th Meeting of the European Association of Neuro-Oncology (EANO) in Mannheim, Germany.
PTC Therapeutics provided a regulatory update on Translarna (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD). PTC Therapeutics announced that at the end of last week, the Office of Drug Evaluation I (ODE-I) of the FDA denied the company’s first appeal of the refuse to file letter issued by the FDA’s Division of Neurological Products (DNP) on February 22, 2016 regarding PTC’s NDA for Translarna for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD).
Mast Therapeutics announced that its wholly-owned subsidiary, Aires Pharmaceuticals, has entered into a collaborative agreement with Philips Respironics, the sleep and respiratory care business of Royal Philips related to the supply of Philips’ I-neb Adaptive Aersol Delivery (AAD) devices and related consumables for the nebulized delivery of the Company’s lead product candidate, AIR001, a sodium nitrite solution for intermittent inhalation. The I-neb handheld AAD system is a small, portable and virtually silent vibrating mesh technology nebulizer system designed to deliver more precise, reproducible doses of medication faster than conventional jet or ultrasonic nebulizers.
Omeros announced positive data from the company’s Phase II clinical trial of OMS721 for the treatment of kidney disorders, none of which currently have an approved treatment and all of which frequently lead to end-stage renal disease and dialysis. Statistical significance was achieved on key endpoints of improvement in renal function. Omeros also reported the outcome of a recent FDA meeting regarding breakthrough therapy designation for OMS721 in immunoglobulin A (IgA) nephropathy (also known as Berger’s disease). Based on that meeting, Omeros is pursuing FDA breakthrough therapy designation. In addition to the Phase II program in renal diseases, OMS721 is being evaluated in a Phase III program for patients with atypical hemolytic uremic syndrome (aHUS) and in a Phase II program for patients with thrombotic microangiopathies (TMAs), including hematopoietic stem cell transplant-associated TMAs and thrombotic thrombocytopenic purpura. OMS721 is Omeros’ lead human monoclonal antibody targeting mannan-binding lectin-associated serine protease-2 (MASP-2), the effector enzyme of the lectin pathway of the complement system.
Lipocine announced the completion of a Post Action meeting with the FDA regarding its NDA for LPCN 1021, an oral testosterone product candidate for testosterone replacement therapy ("TRT") in adult males for conditions associated with a deficiency or absence of endogenous testosterone, also known as hypogonadism. The purpose of the meeting was to review the CRL and to determine actions needed to achieve approval of LPCN 1021.
Regeneron Pharmaceuticals and Teva Pharmaceutical Industries provided an update on fasinumab, triggered by a recent development in a Phase IIb fasinumab study in patients with chronic low back pain. Fasinumab is an investigational Nerve Growth Factor (NGF) antibody in clinical development for osteoarthritis pain and chronic low back pain.
Shire announced an update to the Vyvanse (lisdexamfetamine dimesylate) labeling to include information regarding the approval of a sNDA by the FDA. The labeling will now include maintenance of efficacy data from SPD489-346, the first-ever longer-term pharmacologic study (38 weeks) in adults with moderate to severe binge eating disorder (B.E.D.). Results from SPD489-346, which were announced in July 2015, indicated that Vyvanse (n=136) demonstrated significant maintenance of efficacy compared to placebo (n=131) based upon the primary endpoint of time to relapse. Vyvanse is approved in the U.S. for adults with moderate to severe B.E.D. Vyvanse is not for weight loss. It is not known if Vyvanse is safe and effective for the treatment of obesity.
Synergy Pharmaceuticals presented new long-term safety data of plecanatide, its investigational, orally-administered compound currently being evaluated by the FDA for the treatment of chronic idiopathic constipation (CIC) and irritable bowel syndrome with constipation (IBS-C). These data, which were presented at the American College of Gastroenterology (ACG) annual scientific meeting, showed that plecanatide was associated with low adverse events and low discontinuation rates in patients with CIC who received plecanatide (3 mg or 6 mg) once-daily for up to 72 weeks.
Advanced Accelerator Applications S.A. announced that the EMA has issued a positive opinion on the MAA for SomaKit TOC 40 ug, a kit for radiopharmaceutical preparation of gallium (Ga 68) edotreotide solution for injection, for Positron Emission Tomography (PET) imaging of somatostatin receptor overexpression in adult patients with confirmed or suspected well-differentiated gastroenteropancreatic neuroendocrine tumors (GEP-NET) for localizing primary tumors and their metastases.
Nuvo Pharmaceuticals announced it has received approval from the German Federal Institute for Drugs and Medical Devices and Ethical Review Committee to conduct a placebo controlled Phase III trial of Pennsaid 2% for the treatment of acute ankle sprains (the Trial). The 130 patient Trial will be conducted in Germany commencing this month. Topline results are expected to be available in Q2 2017. The Trial is intended to support regulatory applications for marketing approval of Pennsaid 2% in the E.U., Canada and Australia.
Allergan and Vitae Pharmaceuticals announced that the waiting period under the Hart-Scott Rodino Antitrust Improvements Act of 1976, as amended ("HSR"), has expired with respect to Allergan’s pending acquisition of Vitae.
Journey Medical, a Fortress Biotech company, launched Targadox (doxycycline hyclate USP) 50 mg film coated tablets indicated as adjunctive therapy for the treatment of severe acne. Targadox is the only 50 mg immediate-release doxycycline hyclate tablet currently available on the market. Targadox is available by prescription at major U.S. retail and specialty pharmacy chains. According to Symphony Health, doxycycline is the most-prescribed oral antibiotic in Dermatology with more than 3.7 million prescriptions written in 2015.
Ritter Pharmaceuticals announced that the last patient has completed dosing and all monitoring visits in its Phase IIb/III clinical trial of RP-G28 for the treatment of lactose intolerance. Topline results of the trial are expected to be announced in the first quarter of 2017.
ChemoCentryx announced that positive data from an ongoing Phase II proof-of-concept study of CCX168 for the treatment of Atypical Hemolytic Uremic Syndrome (aHUS) will be presented at the American Society of Nephrology (ASN) Kidney Week 2016 Annual Meeting. CCX168 (newly designated "avacopan") is an orally-administered small molecule that is a selective inhibitor of the complement C5a receptor, or C5aR. CCX168 (avacopan) is a lead drug candidate in the Company’s orphan and rare disease program.
Imprimis Pharmaceuticals announced the availability of a significantly lower-cost compounded alternative to Valeant‘s Calcium Disodium Versenate (edetate calcium disodium injection, USP), commonly used to stabilize and treat patients exposed to lead poisoning.
Amunix announced that Versartis has been granted approval in Japan by the PMDA to initiate enrollment for its Phase III study of somavaratan (XTENylated recombinant growth hormone) in pediatric patients with growth hormone deficiency (GHD).
Aequus Pharmaceuticals announced that is has filed a Clinical Trial Application (CTA) with Health Canada for its once-weekly transdermal aripiprazole patch. Aequus has developed this product to provide patients with sustained and controlled delivery of aripiprazole in a convenient, weekly dosage form. Upon approval of the CTA, the Company plans to initiate a repeat dose, 28-day safety and bioavailability study of aripiprazole transdermal patches in eight healthy volunteers in late 2016.
Osiris announced that it has commenced steps to terminate enrollment in its OTI-15-01 Phase III Clinical Trial. The trial was investigating safety and efficacy of OTI-15- 01, a cryopreserved human amniotic membrane, in patients suffering from chronic DFUs that have not responded to standard of care therapy. Patients currently enrolled in the trial will continue with their current course of treatment and scheduled visit assessments, as outlined in the protocol. The decision to terminate further enrollment in the OTI- 15-01 trial reflects the Company’s desire to allocate more of its research and development resources to other clinical programs, including its new cellular drug platform technology. The Company presently anticipates the new platform will have utility across multiple indications in wound care, orthopedics and sports medicine.
MorphoSys AG presented updated safety and efficacy data from an ongoing Phase I/IIa clinical study evaluating MOR202, an investigational antibody targeting CD38, alone and in combination with immunomodulatory drugs (IMiDs) lenalidomide (Len) and pomalidomide (Pom), plus dexamethasone (Dex) in heavily pre-treated patients with relapsed/refractory multiple myeloma (MM). Data were presented during a poster presentation at the 2016 annual meeting of the German, Austrian and Swiss Societies for Hematology and Medical Oncology in Leipzig/Germany.
Teva Pharmaceutical announced that the CHMP of the EMA has adopted a positive opinion recommending an indication extension of Trisenox (arsenic trioxide). The indication extension is for use in newly diagnosed low to intermediate risk Acute Promyelocytic Leukemia (APL) in combination with retinoic acid. Trisenox, in combination with retinoic acid, has shown a very high overall survival rate with almost no relapses after more than four years (50 months) of median follow-up. If the EC approves this label extension, it would mark the first time that a form of acute leukemia can be effectively treated with a regimen that is entirely chemotherapy-free.
Ferring Pharmaceuticals announced that the CHMP has adopted a positive opinion for REKOVELLE (follitropin delta), recommending that the European Commission grants marketing authorisation for use in controlled ovarian stimulation for the development of multiple follicles in women undergoing assisted reproductive technologies (ART), such as an in vitro fertilisation (IVF) or intracytoplasmic sperm injection (ICSI) cycle.
Apitope announced it has regained global rights to its compound, ATX-MS-1467, a potentially disease-modifying therapy for the treatment of Multiple Sclerosis (MS). Under the agreement with Merck KGaA, Apitope will regain all rights and clinical data associated with its compound ATX-MS-1467, which just completed a Phase II clinical trial.
Synapse Biomedical announced that the results of a small French trial (RespiStimALS), designed to assess the NeuRx Diaphragm Pacing System (DPS) in newly diagnosed ALS patients, were published in Lancet Neurology. The French trial, which consisted of 74 patients, studied the use of DPS to delay the need for noninvasive ventilation (NIV) (e.g. BiPaP, Trilogy). All patients were implanted earlier than usual, close to their initial diagnosis. Half the patients had their device activated at surgery. The other half, the control patients, had their device activated much later, at the usual time, once they met criteria for NIV. The trial results reported by the authors showed that early implantation was ineffective. However, the trial does nothing to contradict the previous conclusion that on-label DPS use, at the usual later time, concomitant with NIV use, improves survival.
Globavir Biosciences announced a partnership with Bio-Rad Laboratories. Under the terms of the agreement, Bio-Rad will license Globavir’s PanGlob dengue detection technology for use in an assay that includes dengue virus detection. Financial terms of the agreement were not disclosed.
Enterprise Therapeutics announced that it has appointed Dr John Ford as CEO with immediate effect.
ContraVir Pharmaceuticals announced that following a positive recommendation from an independent Data Safety Monitoring Board (DSMB), the company has begun enrolling the next dosing group in its head-to-head Phase IIa dose-escalation study comparing the safety and efficacy of ContraVir’s CMX157 to tenofovir disoproxil fumarate (TDF, marketed by Gilead Sciences as Viread) in patients with chronic hepatitis B (HBV). ContraVir expects to complete the remaining two escalation cohorts and report top-line results by year-end 2016.
Antibe Therapeutics provided an update on it second pipeline drug, ATB-352, a potent pain-killer intended for the relief of acute, severe pain. Recently completed pre-clinical studies have now demonstrated that ATB-352, unlike opiate drugs, does not bind to opioid receptors and therefore does not pose a risk of addiction.
Zynerba Pharmaceuticals provided a development update for ZYN001 and ZYN002. Zynerba and its development partner, LTS LOHMANN Therapie-Systeme AG (LTS), a market leader in transdermal therapeutic solutions, are working together to optimize the formulation of ZYN001 into a state of the art drug-adhesive matrix transdermal patch. ZYN001 is a patent-protected pro-drug of THC being developed for the treatment of fibromyalgia and peripheral neuropathic pain. Based on the additional optimization work in progress, Zynerba now expects to begin Phase I clinical trials for ZYN001 in the first half of 2017 and Phase II clinical trials are now planned to begin during the second half of 2017.
Soligenix announced that it has received preliminary approval for an approximate $570,000 tax credit from the New Jersey Economic Development Authority’s (NJEDA) New Jersey Technology Business Tax Certificate Transfer (NOL) program. As a result, the Company anticipates being able to transfer this credit and receive approximately $530,000 in cash by year end.
Jefferies analyst Matthew Andrews increased his price target of Gemphire to $17 from $15, citing Phase II gemcabene program is on track with dosing of first HoFH patient, two other Phase II’s to start by late Q4 2016/Q1 2017, and interim HoFH data in early 2017.
The following analysts initiated coverage of Novan: JMP analyst Donald Ellis with a “market outperform” rating and $31 price target; Wedbush analyst David Nierengarten with an “outperform” rating and $27 price target; Piper Jaffray analyst David Amsellem with an “overweight” rating and $25 price target.
Jefferies analyst Gena Wang increased her price target of Progenics to $10 from $9, citing increased launch assumptions.