Healthcare News

  • Today - Thursday, October 18, 2018
    • Thinly traded Apellis Pharmaceuticals (APLS -16.9%) is down on almost double normal volume, albeit on turnover of only 434K shares, in reaction to its disclosure that it has paused dosing in its Phase 3 clinical trial, OAKS, evaluating APL-2 in patients with geographic atrophy due to instances of non-infectious inflammation in patients receiving a single lot of product.
    • A total of eight participants developed inflammation, four in the Phase 3 program and four in the Phase 2 wet AMD study. Seven of the eight completely resolved and the other is expected to resolve.
    • The company has started a series of non-human studies to confirm that the problem is confined to the single lot, expected to be completed by late November. Phase 3 dosing will resume by year-end if the isolation of the problem is verified.
    • Dosing will continue in the Phase 2 study with a different lot.
    • Previously: Dosing underway in Apellis Pharma's late-stage program of APL-2 in geographic atrophy (Sept. 12)
    • Andrew Left (Citron) takes note of a Form 483 from the FDA regarding PolarityTE (NASDAQ:PTE) in late September. Polarity, says Left, tried to hide this from investors, but a FOIA request to the FDA has made it available.
    • The contents, says Left, are "damning." Among them: "Your firm has not performed and documented quantitative data that validates your process for SkinTE product to show consistent aseptic processing, aspetic tissue washing, and other relevant characteristics such as tissue size, cell counts, or relevant skin type identification."
    • The stock is a zero, says Left.
    • Shares were halted, but are now trading, down 4.4%.
    • Updated long-term data from a Spain-based Phase 1/2 clinical trial evaluating Rocket Pharmaceuticals' (RCKT -2.9%) gene therapy RP-L102 in patients with a rare inherited blood disorder called Fanconi Anemia (FA) continued to show a treatment benefit. The results were presented at the ESGCT Congress in Switzerland.
    • 12-month follow-up data on four patients showed durable engraftment, ranging from 18 - 30 months, since gene therapy administration as well as improvements in two key measures of functional and phenotypic correction in FA.
    • Dosing in U.S. patients should commence in H1 2019.
    • FA is a rare pediatric disorder characterized by bone marrow failure that is usually fatal within 10 years.
    • Opiant Pharmaceuticals (NASDAQ:OPNT) announces that Walter Reed Army Institute of Research (WRAIR) and SUNY Upstate Medical University in Syracuse, NY, have been awarded a grant by the National Institutes of Health (NIH) to advance OPNT005, a heroin vaccine candidate, through Phase 1/2a clinical trials to assess its safety and efficacy.
    • This new NIH grant of ~$3.7M for the budget period through August 31, 2019, will fund pilot production of OPNT005 and preliminary safety testing.
    • Additional funds will be available from the NIH, subject to availability and the successful execution of these tasks. If successful, it will progress to a clinical trial evaluating the efficacy of OPNT005 in healthy volunteers.
    • LabCorp (NYSE:LH) and Baptist Health have entered into a multi-year, comprehensive laboratory collaboration.
    • LabCorp will provide technical services for Baptist Health’s eight hospital-based labs and access to enhanced information technology and data analytics.
    • The new agreement builds upon and deepens an established relationship of more than five years. LabCorp has provided reference testing services as the primary laboratory for Baptist Health.
    • AbbVie (NYSE:ABBV) has resolved all IP-related litigation with Fresenius Kabi related to its proposed biosimilar to top seller HUMIRA (adalimumab), granting it a non-exclusive license beginning on September 30, 2023. ABBV will earn undisclosed royalties on net sales of Kabi's offering.
    • The timing will not be accelerated by the entry of other biosimilar makers who are already licensed. In the EU, Kabi may launch its product upon EMA approval.
    • Previously: AbbVie settles Humira patent dispute with Sandoz; shares up 1% after hours (Oct. 11)
    • DexCom (NASDAQ:DXCM) announces the new Dexcom G6 CGM System will be covered for Medicare beneficiaries, having met the category requirements for therapeutic CGM systems by the U.S. Centers for Medicare & Medicaid Services (CMS).
    • Coverage for therapeutic CGM includes certain beneficiaries who have either Type 1 or Type 2 diabetes and intensively manage their insulin.
    • The company expects to begin shipping product to Medicare customers in the first part of 2019.
    • Abeona Therapeutics (NASDAQ:ABEO) appoints of João Siffert, M.D. as Head of Research and Development and Chief Medical Officer (CMO). As a result, former CMO Juan Ruiz, M.D., Ph.D. will assume the role of Head of European Medical Affairs.
    • The Company also appointed Neena Patil, J.D. as General Counsel and Corporate Secretary. Both Dr. Siffert and Ms. Patil report to CEO, Carsten Thiel, Ph.D.
    • Dr. Siffert, most recently was Chief Scientific and Medical Officer for Nestle Health Science.
    • Ms. Patil, most recently served as Associate General Counsel and Vice President of Legal Affairs at Novo Nordisk.
    • Nano cap Matinas BioPharma (NYSEMKT:MTNB) is up 37% premarket on robust volume in reaction to the issuance of a new U.S. patent covering methods of use of its omega-3 fatty acid MAT9001, in development for treating cardiovascular and metabolic conditions. The patent will be in effect through 2033.
    • Acorda Therapeutics (NASDAQ:ACOR) is up 8% premarket on light volume on the heels of its disclosure that the FDA's pre-approval inspections of its Chelsea, MA manufacturing facility and the Inbrija inhaler device maker's facility have been completed with no further action required.
    • The company's marketing application, seeking approval for Inbrija (levodopa inhalation powder) for the treatment of OFF periods in Parkinson's patients, is currently under FDA review with an action date of January 5, 2019.
    • Previously: FDA extends action date for Acorda's Inbrija to January 5, 2019; shares down 7% premarket (Sept. 13)
    • The Connecticut Insurance Commissioner Katherine Wade has signed off on CVS Health's (NYSE:CVS) planned takeover of Aetna (NYSE:AET), contingent on the latter divesting its Medicare prescription drug businesses.
    • Connecticut is the primary regulator since Aetna is based in Harford. CVS has committed to keeping Aetna there for at least 10 years and to maintain the number of employees for at least four years.
    • Previously: CVS and Aetna up premarket on looming DOJ signoff on merger (Oct. 10)
    • Biocept (NASDAQ:BIOC) has been granted Japanese Patent No. 6404118, entitled METHODS FOR DETECTING NUCLEIC ACID SEQUENCE VARIANTS.
    • The patent provides intellectual property protection for the Company's Switch-Blocker technology which is core to Biocept's Target Selector assays for analysis using real-time PCR, Sanger sequencing and next-generation sequencing.
    • Shares are up 13% premarket.
    • Spectrum Pharmaceuticals (NASDAQ:SPPI) appoints Francois Lebel, M.D., F.R.C.P.C. as Chief Medical Officer, effective November 5.
    • Most recently, Dr. Lebel served as the Executive VP of Research & Development, Chief Medical Officer at ZIOPHARM Oncology.
    • A Phase 3 clinical trial, KEYNOTE-426, evaluating the combination of Merck's (NYSE:MRK) KEYTRUDA (pembrolizumab) and Pfizer's (NYSE:PFE) INLYTA (axitinib) for the first-line treatment of patients with advanced/metastatic renal cell carcinoma met both primary endpoints.
    • The combination produced statistically significant improvements in overall survival (OS) and progression-free survival (PFS) compared to Pfizer's SUTENT (sunitinib malate) as monotherapy. The study also met the key secondary endpoint of objective response rate (ORR).
    • The data will be filed with global regulatory authorities.
    • Iovance Biotherapeutics (NASDAQ:IOVA) and MaSTherCell S.A., a subsidiary of Orgenesis (NASDAQ:ORGS) have entered into a new three-year Manufacturing Services Agreement.
    • MaSTherCell will manufacture TIL for Iovance’s European late-stage clinical trials in its commercial-ready cGMP manufacturing suites.
    • As part of global development plan for TIL product, IOVA is initiating the IOV-COM-202 trial in Europe and have received MHRA approval.
    • Financial terms are not disclosed.
    • Hutchison China MediTech Limited (NASDAQ:HCM) has initiated a Phase I study of HMPL-523, its novel spleen tyrosine kinase inhibitor, in combination with azacitidine, an approved nucleoside metabolic inhibitor, in elderly patients with acute myeloid leukemia (“AML”) in China.
    • The study is to evaluate the safety, pharmacokinetics and preliminary efficacy of the combination in previously untreated elderly patients with AML who are not eligible for standard induction therapy.  The primary outcome measures are overall response rate and adverse events.
    • The two-stage study will have a dose escalation and dose expansion stage.
    • AbbVie (NYSE:ABBV) inks an R&D collaboration agreement with Morphic Therapeutic aimed at advancing the latter's oral integrin therapies for fibrosis-related disorders.
    • Under the terms of the partnership, ABBV will pay Morphic $100M upfront for exclusive license options on selected candidates, milestones and tiered royalties on net sales. Morphic will conduct R&D through IND-enabling studies at which point ABBV may elect to pay a license fee to assume clinical development and commercialization. Morphic will retain cost-sharing rights for liver fibrosis indications and may elect to pay a portion of ABBV's development costs in exchange for higher royalties.
    • The companies are no strangers. AbbVie Ventures was an investor in its Series A and Series B financings.
    • RepliCel Life Sciences (OTCQB:REPCF) has signed a collaborative research project agreement with the University of Victoria ("UVic") in Canada. The project will be co-funded through a grant from the National Science and Engineering Research Council of Canada ("NSERC"). The collaboration will focus on approaches to optimize cell culture platforms and manufacturing technologies.
    • The two-year NSCERC CRD funding agreement may result in publications, patents.
    • New intellectual property arising from the project will be the sole and exclusive property of UVic with a fully paid, non-exclusive, royalty-free license to RepliCel for any commercial purposes.
    • RepliCel has the option to negotiate an exclusive license to such intellectual property from UVic for commercial purposes important to the Company.
    • According to the report, KKR and TPG Global are among the private-equity outfits sniffing around Acadia Healthcare (NASDAQ:ACHC).
    • Shares are higher by 10% premarket.
    • Innovus Pharmaceuticals (OTCQB:INNV) plans to expand its product line into the hemp-derived CBD (“Cannabinoid”) oil-based products market beginning in Q4 with the introduction of MZS Sleeping Aid.
    • The product is intended to be sold as a supplement in the U.S. and eventually, pending regulatory approval, into the Canadian market.
    • MZS Sleeping Aid to be launched in U.S. will not contain any THC (Tetrahydrocannabinol) and is designed to be compliant with applicable U.S. state and federal laws.
    • Exact Sciences (NASDAQ:EXAS) has acquired privately held San Diego-based Biomatrica, a developer of sample preservation technology, including blood and saliva collection tubes, for an undisclosed sum.
    • Exact says Biomatica's collection tubes contain proprietary sample-stabilizing chemistry enabling quality preservation of circulating tumor DNA, cells and other biomarkers in specimens throughout the shipping and storage process.
    • Biomatrica will operate as a wholly owned subsidiary of EXAS and will continue to supply tubes and sample preservation technologies to other companies.
    • Avivagen (OTCPK:CHEXF) has entered into a distribution agreement for Vivamune Health Chews in the Vietnamese market with Viphavet Co. Ltd.
    • Under the terms of agreement, Viphavet will undertake the registration and trademark process of Vivamune Health Chews within Vietnam, which is anticipated to be completed by the end of this year.
    • Upon completion, Viphavet will be granted exclusivity for Vivamune Health Chews for one year which may be extended based on reaching specific volume targets.
    • Aurora Cannabis (OTCQX:ACBFF) will start trading on the New York Stock Exchange (NYSE) effective October 23 under the symbol "ACB."
    • Evercore's Ross Muken  says dental equipment distributors may be on the soft side after Danaher's (NYSE:DHR) Q3 results that showed continued weak demand in the space.
    • Peers Henry Schein (NASDAQ:HSIC), Patterson Companies (NASDAQ:PDCO) and DENTSPLY SIRONA (NASDAQ:XRAY) are not showing any appreciable premarket action thus far this morning.
    • Previously: Danaher Corporation beats by $0.02, beats on revenue (Oct. 18)
    • Co-Diagnostics (NASDAQ:CODX) announces that its Logix Smart Zika Test technical file has obtained CE mark approval, the principle regulatory clearance allowing the test to be sold as an in vitro diagnostic (“IVD”) in European Union states and other markets.
    • In a note, RBC's Brian Abrahams says any dip in Vertex Pharmaceuticals (NASDAQ:VRTX) following Proteostasis Therapeutics' (NYSE:PTI) positive early-stage data on its cystic fibrosis (CF) candidates will be a buying opportunity. He believes that PTI could be an "incremental negative" for VRTX but could emerge as a more serious threat to its CF franchise if the early-stage data are confirmed or if a larger company steps in to partner or acquire PTI.
    • Mr. Abrahams has upgraded PTI to Outperform with a $9 (376% upside) price target.
    • PTI is up 63% premarket on robust volume. No premarket action has been reported for VRTX.
    • Previously: Proteostasis up 32% after hours on positive early-stage data on CF candidates (Oct. 17)
    • Selecta Biosciences (NASDAQ:SELB) reports that the National Cancer Institute informed it of a patient death from pneumonitis in a Phase 1 clinical trial evaluating combination product SEL-403 in patients with solid tumors. The investigator believes the fatality was probably related to SVP-Rapamycin, one of the components of SEL-403 (the other is LMB-100).
    • A total of four patients have been dosed in the study. In addition to the death, one participant experienced a serious adverse event, pericardial effusion, a potential side effect of immunotoxin therapies targeting mesothelin. No patients are active in the study and enrollment has been stopped.
    • SVP-Rapamycin is also a component of SEL-212 (together with pegsiticase), currently being investigated in a Phase 2 study in symptomatic gout. There have been no cases of pneumonitis among the 150 participants dosed to date. Interim data will be presented at ACR/ARHP next week.
    • Getinge Industrier AB (OTCPK:GNGBF): Q3 Non-GAAP EPS of SEK0.78; GAAP EPS of -SEK5.34.
    • Revenue of SEK5.68B (+15.0% Y/Y)
    • Press Release
    • Osmotica Pharmaceuticals (NASDAQ:OSMT) prices its IPO of 6.65M shares of common stock (down from 8.3M) at $7 per share. Trading commences today.
    • OpGen (NASDAQ:OPGN) commenced an underwritten public offering of its common stock.
    • 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.
    • Net proceeds will be used for research and development, including funding the continued development and manufacturing of its Acuitas AMR Gene Panel tests, including its Acuitas AMR Gene Panel u5.47 that was released for Research Use Only in February 2018 and its Acuitas Lighthouse software, to support acquisitions of products and technologies, capital expenditures, working Shares -5.9%
    • Press Release
    • PhaseBio Pharmaceuticals (NASDAQ:PHAS) prices its IPO of 9.2M shares of common stock at $5 per share. Trading commences today.
    • The original plan was 5M shares at $12 - 14.
    • Previously: PhaseBio sets terms for IPO (Oct. 6)
    • Update: Shares opened at $5.
    • Evolent Health (NYSE:EVHannounces the pricing of $150M aggregate principal amount of 1.50% convertible senior notes due 2025.
    • The size of the offering was increased from the previously announced $125M of notes. Evolent Health has also granted the initial purchasers a 30 day option to purchase up to an additional $22.5M of notes.
    • The notes are convertible into cash, shares of Evolent Health's Class A common stock, or a combination of cash and shares, , based on an initial conversion rate of 29.9135 shares of Class A common stock per $1,000 of notes, which is equivalent to an initial conversion price of ~$33.43 per share of Class A common stock..
    • Evolent Health may not redeem the notes prior to October 20, 2022.
    • Net proceeds will be used for working capital and other general corporate purposes.
    • Settlement is expected to occur on October 22, 2018.
    • Luminex (NASDAQ:LMNX) has agreed to acquire MilliporeSigma's flow cytometry portfolio for $75M, ~$69.9M under a Stock and Asset Purchase Agreement and ~$5.1M in committed inventory purchases. The transaction should close by year-end.
    • The company expects the business to contribute $40M - 50M in revenue in 2019.
    • Aclaris Therapeutics (NASDAQ:ACRS) prices its public offering of 8.645M common shares at $10.75 per share. Underwriters over-allotment is an additional ~1.297M shares. Closing date is October 22.
    • Yesterday's close was $12.17.
    • Shares are down 12% premarket on light volume.
    • Novartis (NVS) Q3 results: Revenues: $12,779M (+2.9%); Innovative Medicines: $8,802M (+6.2%); Sandoz: $2,469M (-5.2%); Alcon: $1,763M (+2.9%).
    • Net Income: $1,624M (-22.0%); Non-GAAP Net Income: $3,063M (+1.6%); EPS: $0.70 (-21.3%); Non-GAAP EPS: $1.32 (+2.3%); CF Flow Ops: $4,050M (+12.9%).
    • Key growth drivers: Cosentyx: $750M (+35%); Entresto: $271M (+112%); Promacta/Revolade: $295M (+30%); Tafinlar + Mekinist: $291M (+30%); Kisqali: $72M; Jakavi: $248M (+23%); Lutathera: $56M; Kymriah: $20M.
    • 2018 Guidance: Total sales growth: mid-single digit; Innovative Medicines: grow mid to high single-digit; Sandoz: low-single digit decline; Alcon: grow mid-single digit.
    • Shares are up 1% premarket.
    • Previously: Novartis beats by $0.02, misses on revenue (Oct. 18)
    • Novartis (NYSE:NVS): Q3 Non-GAAP EPS of $1.32 beats by $0.02; GAAP EPS of $0.70 misses by $0.27.
    • Revenue of $12.78B (+3.0% Y/Y) misses by $210M.
    • Shares +1.04% PM.
    • Press Release
    • Bringing it closer to becoming a global top 10 drugmaker, Takeda Pharmaceutical (OTCPK:TKPYY) has received approval from Japan's Fair Trade Commission for its $62B acquisition of Shire (NASDAQ:SHPG).
    • The deal, which will be the largest overseas purchase by a Japanese company, has already received unconditional clearance from regulators in the U.S., China and Brazil, and just needs one more green light from the EU.
    • Expanding its radiopharmaceuticals business, Novartis (NYSE:NVS) is acquiring Endocyte (NASDAQ:ECYT) for $2.1B, or $24-per-share, representing a premium of 54% to Endocyte's Wednesday closing price.
    • The deal will help develop a broader role for radioligand therapies in cancer treatment and enable Novartis to investigate the potential development of a drug candidate for use in earlier lines of prostate cancer therapy.
    • Update: Shares are up 50% premarket.
  • Wednesday, October 17, 2018
    • Thinly traded micro cap OncoMed Pharmaceuticals (NASDAQ:OMED) is down 8% after hours following its disclosure that Celgene (NASDAQ:CELG) will not exercise its option to license rosmantuzumab and has terminated their collaboration agreement effective February 12, 2019. OncoMed will retain global rights thereafter.
    • Celgene's decision was motivated by less-than-expected results from a Phase 1a/1b clinical trial in patients with solid tumors.
    • Celgene maintains its option to in-license etigilimab.
    • Previously: Celgene backs away from OncoMed's navicixizumab (Sept. 20)
    • Thinly traded micro cap Eiger BioPharmaceuticals (NASDAQ:EIGR) is up 4% after hours following its announcement of positive results from a Phase 2 clinical trial, LIMT HDV, evaluating pegylated interferon lambda in patients with hepatitis D virus (HDV) infection.
    • At week 48, 40% (n=4/10) treated patients were HDV-RNA-negative while 60% (n=6/10) experienced at least a 2 log (10) decrease (100-fold decrease) in HDV-RNA.
    • Detailed results will be presented at the Liver Meeting in early November.
    • End-of-treatment data from the Phase 2 LIFT study of lambda interferon and AbbVie's Norvir should be available in 2019.
    • Eiger in-licensed global rights to lambda interferon from Bristol-Myers Squibb in April 2016.
    • Previously: Enrollment underway in Eiger's mid-stage study of pegylated interferon lambda in HDV (Aug. 6)
    • Thinly traded nano cap Proteostasis Therapeutics (NYSE:PTI) is up 32% after hours following its announcement of positive preliminary data from a Phase 1 clinical trial evaluating its doublet and triplet therapy candidates in cystic fibrosis (CF) patients.
    • The three candidates are: PTI-801, a third-generation cystic fibrosis transmembrane conductance regulator (CFTR) corrector, PTI-808, a CFTR potentiator and PTI-428, a CFTR amplifier.
    • CF patients receiving the doublet therapy of PTI-801/PTI-808 (300 mg of each) experienced a statistically significant increase in lung function (ppFEV1) at day 7 and day 14 from baseline and compared to placebo. There was also a statistically significant decrease in sweat chloride concentration from baseline at days 7 and 14.
    • Data from the PTI-801 (400 mg)/PTI-808 (300 mg) cohort should be available in Q1 2019.
    • Preliminary data on the triplet therapy should be available later this quarter. Complete data for all doublet and triplet groups should be available next quarter.
    • Management will host a conference call at 5:00 pm ET to discuss the results.
    • Update: Shares are up almost four-fold on Thursday, October 18, on a whopping 313x surge in volume.
    • Aclaris Therapeutics (NASDAQ:ACRS) slips 3% after hours in response to its planned stock offering. Price, volume and terms have yet to be announced.
    • Krystal Biotech (NASDAQ:KRYS) is down 5% after hours in response to its $60M public offering of common stock. Price, volume and terms have yet to be announced.
    • Ultra-thinly traded micro cap ASLAN Pharmaceuticals (ASLN +2.7%) is up on double normal volume, albeit on turnover of only 11K shares, on the heels of its announcement that the first subject has been enrolled in a Phase 1 clinical trial evaluating ASLAN004 for the treatment of atopic dermatitis (AD) (eczema).
    • The single ascending dose portion will involve healthy volunteers, followed by the multiple ascending dose portion with moderate-to-severe AD patients. The first phase should wind up in H1 2019.
    • ASLAN004 is a fully human monoclonal antibody that binds to the interleukin-13 (IL-13) receptor α1 subunit (IL-13Rα1). The company says it has demonstrated the ability to inhibit IL-4 and IL-13, two cytokines that trigger AD symptoms like redness and itchy skin.
    • Speaking earlier with Jim Cramer, Tilray (NASDAQ:TLRY) CEO Brendan Kennedy, says he expects the number of countries legalizing medical cannabis should rise from 35 to about 60 over the next 2-3 years. He also expects a third country to legalize cannabis for recreational use sometime in the next year.
    • Pot, he says, can disrupt $150B-$200B worth of other industries - pharmaceutical, alcohol, food&beverage.
    • Speaking on Bloomberg in the last few minutes, Kennedy says the current black market for pot in Canada is C$6B. Pot, he says, will be stealing sales from alcohol.
    • As for his own portfolio, Kennedy says he's sold no shares of Tilray.
    • Tilray is down 7.6% on today's session.
    • VIVO Cannabis’s (OTCQB:VVCIF -5.1%) subsidiary Harvest Medicine Inc. has completed the acquisition of Trauma Healing Centers (THC) from Organigram Holdings Inc.
    • Harvest Medicine will acquire 100% of the issued and outstanding shares of THC from Organigram for a total purchase price of $1.2M.
    • THC is a multi-disciplinary clinic network that has been providing medical cannabis care and education, it specializes in patient assessment and medical cannabis prescribing and also offers patients individualized, multi-disciplinary wellness plans.
    • With this acquisition Harvest Medicine will add locations in Nova Scotia, New Brunswick, and Ontario to its existing network of medical cannabis clinics in Edmontonand Calgary, and will bring the total number of active patients to above 22,000.
    • Aurora Cannabis (OTCQX:ACBFF -1.1%) says that its Aurora Sky production plant was granted a sales license by Canada's health regulator as the country becomes the second in the world to legalize recreational marijuana.
    • Press Release 
    • Thinly traded micro cap OncoMed Pharmaceuticals (OMED +25.3%) is up on a healthy 21x surge in volume following the release of an abstract for the ESMO congress in Munich detailing preliminary data from a Phase 1b study of navicixizumab + paclitaxel in platinum-resistant ovarian cancer.
    • 18 patients with a median of four prior therapies were treated. All had received prior paclitaxel and 13 had received bevacizumab (Roche's Avastin). The disease control rate (responders + stable cancer) was 78% (n=14/18), including eight partial responders. 71% (n=10/14) of patients with elevated CA-125, a cancer biomarker, responded according to GCIG (Gynecologic Cancer Intergroup) criteria.
    • Last month, Celgene decided against in-licensing the anti-DLL4/VEGF bispecific antibody citing "strategic product portfolio considerations."
    • Previously: Celgene backs away from OncoMed's navicixizumab (Sept. 20)
    • BioCentury reports that Johnson & Johnson (JNJ +2.2%) has suspended all clinical trials investigating antiviral lumicitabine citing the need for "additional" preclinical data. Three mid-stage studies, in respiratory syncytial virus (RSV) and human metapneumovirus (hMPV), have been closed.
    • The action precipitated a $630M impairment charge in Q3 to reflect the diminished value of its original $1.75B cash outlay for discoverer Alios BioPharma. The company says it will decide on conducting further studies after analyzing the data and will monitor the remaining $900M intangible asset for further impairment.
    • RSV therapy-related tickers: (ARDS +0.5%)(ENTA -0.8%)(ASNS +1.2%)(ADMA -1.3%)(OTCPK:ABLYF)(VXRT +7.2%)(SNY +0.9%)(AZN +0.3%)
    • Previously: J & J to buy viral disease-focused biotech (Sept. 30, 2014)
    • Eisai (OTCPK:ESALF) and development partner Purdue Pharma L.P. announce positive topline results from a Phase 3 clinical trial, SUNRISE 2, evaluating lemborexant in patients with sleep-wake disorders.
    • At month 6, the study met the primary and key secondary endpoints. Specifically, patients receiving either 5 mg or 10 mg of lemborexant experienced a statistically valid improvement in subjective sleep onset latency compared to control, the primary endpoint.
    • Both doses also produced statistically significant improvements in sleep maintenance measures of subjective sleep efficiency and subjective wake after sleep onset compared to placebo.
    • On the safety front, the discontinuation rates were comparable between lemborexant 5 mg and placebo, but higher for the 10 mg dose.
    • Complete results will be submitted for presentation at future medical conferences.
    • The company says lemborexant, a dual orexin receptor antagonist, acts on the orexin neurotransmitter system and dampens wakefulness without compromising the ability to wake up in response to external stimuli. Orexin is a hormone excreted by the brain's hypothalamus that plays a key role in regulating sleep and appetite.
    • Previously: Eisai's lemborexant matches Sanofi's extended-release Ambien in late-stage sleep disorder study (March 7)
    • Preliminary results from a Phase 1/2 clinical trial evaluating Aeglea BioTherapeutics' (AGLE -4.2%) lead candidate pegzilarginase in patients with rare inherited disorder called Arginase 1 Deficiency (ARG1-D) showed a treatment effect. The data were presented at the ASHG Conference in San Diego.
    • 100% of treated patients (n=6/6) who completed the repeat-dose portion of the study showed consistent levels of reduced arginine. 67% (n=4/6) showed clinically meaningful improvements in mobility and/or adaptive behavior after eight weeks of repeat dosing.
    • ARG1-D is characterized by the buildup of arginine and ammonia in the body due to the lack of an enzyme called arginase. Untreated children experience seizures, spasticity, short stature and intellectual disability.
    • Pegzilarginase, an enzyme replacement therapy, is an enhanced human arginase that breaks down arginine.
    • In an interview with CNBC, Tilray (TLRY -5.9%) CEO Brendan Kennedy says he expects growth in medical cannabis and recreational cannabis to continue. At present, 35 countries have OK'd medical use and two recreational (Canada and Uruguay).
    • He believes that the total available global market will be $150B - 200B considering medical-use cannabis and the potential in food, drink and consumer packaged goods.
    • Shares are down as investors "sell on the news" on the first day of legal recreational use in Canada.
    • Results from a preclinical study evaluating Adverum Biotechnologies' (ADVM -0.4%) gene therapy ADVM-022 in a non-human primate model of wet age-related macular degeneration (wet AMD) showed an encouraging treatment effect. The data were presented at the ESGCT Congress in Switzerland.
    • A single intravitreal injection of ADVM-022 produced robust expression of aflibercept (Regeneron's EYLEA) that was sustained for ~two years. It also provided stable intraocular expression of aflibercept at levels comparable with 3-4 weeks post-dose of EYLEA in vitreous humor, aqueous humor, retina and choroid.
    • Systemic treatment with prophylactic prednisone to preempt a potential inflammatory response slightly lowered intraocular levels of aflibercept but the levels remained within the therapeutic range.
    • A Phase 1 study will be launched this quarter in wet AMD patients who are responding to anti-vascular endothelial growth factor (VEGF) therapy (e.g., EYLEA).
    • NeuroMetrix (NURO -1.5%) Q3 results: Revenues: $3.7M (+5.7%); Net Income: $1.1M (-131.4%); EPS: $0.15 (-104.8%).
    • U.S. launch of Quell 2.0 commenced in September 2018.
    • Thinly traded nano cap Rexahn Pharmaceuticals (RNN) slumps 29% on almost a 4x surge in volume, albeit on turnover of only 444K shares, in reaction to its direct sale of 5.77M units to institutional investors at $1.30 per unit.
    • Each unit consists of one share of common stock and one five-year warrant to purchase one common share at $1.67.
    • Closing date is October 19.
    • Yesterday's close was $1.67.
    • Oragenics (OGEN +16.7%) has received clearance to enroll patients residing in Belgium, into its Phase 2 clinical trial of AG013, a live biotherapeutic product for the potential prevention and treatment of oral mucositis (OM).
    • The Phase 2 trial is a double-blind, placebo-controlled, trial, enrolling approx. 200 patients. The purpose of the study is to evaluate the safety, tolerability and efficacy of topically administered AG013 compared to placebo for reducing the incidence and severity of OM in patients undergoing traditional chemoradiation for the treatment of head and neck cancer.
    • Key measures include duration, time to development, and overall incidence of OM.
    • The FDA designates Albireo Pharma's (ALBO) lead candidate A4250 for Fast Track review for the treatment of progressive familial intrahepatic cholestasis (PFIC), a rare life-threatening inherited liver disorder that typically leads to liver failure.
    • A4250, an ileal bile acid transporter inhibitor, is in Phase 3 development for the indication. It also has Orphan Drug and Rare Pediatric Disease status for PFIC.
    • Sonoma Pharmaceuticals (NASDAQ:SNOA) announces the launch of a new Ceramax Skin Barrier Lotion. This new product complements Sonoma’s current Ceramax Skin Barrier Cream.
    • Both Ceramax products contain skin-enriching technology that helps manage dry itchy skin, minor skin irritations, rashes and inflammation.
    • Ceramax lotion will be available via prescription throughout the U.S. beginning in late October.
    • PAVmed (NASDAQ:PAVM) provides an update on its business.
    • CarpX Update: The Company plans to provide public update shortly after its concludes the meeting with FDA and will then resubmit the 510(k) after completing additional testing, if any, that the FDA requests. PAVM plans to complete a first-in-human clinical series in New Zealand later this quarter.
    • Local IRB approval and initiation of patient recruitment is expected in the coming weeks. CE Mark submission for European commercialization is also on target for later this quarter.
    • EsoCheck Update: EsoCheck is progressing on schedule through two-phase regulatory and commercialization strategy. Dr. Aklog explained. “Phase I seeks to launch initial commercial EsoCheck products in late Q1-2019. The EsoCheck cell sampling device will be submitted for 510(k) clearance later this quarter." Finalizing of Phase II regulatory and commercialization strategy is anticipated to culminate in a PMA pre-submission meeting with FDA in early 2019.
    • FDA 510(k) submission for PAVmed’s PortIO, DisappEAR and NextFlo is planned in 2019.
    • DaVita (NYSE:DVA) is feeling Fresenius Medical Care's pain (-18% premarket) after the latter softened its 2018 guidance due to headwinds in the North American dialysis market. Shares are down 4% premarket on light volume.
    • DVA is scheduled to release Q3 results after the close on November 7. Consensus view is EPS of $0.87 on revenues of $2.93B.
    • Previously: Fresenius down 18% premarket on softer earnings guidance (Oct. 17)
    • Kadmon Holdings (NYSE:KDMN) is up 7% premarket on light volume following its announcement that the FDA has designated KD025 a Breakthrough Therapy for the treatment of patients with chronic graft-versus-host disease (cGVHD) who have failed to respond to two or more prior lines of systemic therapy.
    • A Phase 2 study is currently recruiting patients.
    • Breakthrough Therapy status provides for more intensive guidance from the FDA on development, the involvement of more senior agency personnel and a rolling review of the marketing application.
    • Small molecule KD025 inhibits an pro-inflammatory enzyme called rho-associated coiled-coil kinase 2 (ROCK2). Inhibiting the enzyme down-regulates the ability of the immune system's T cells to secrete IL-21 and IL-17, thereby dampening the immune response.
    • Previously: Kadmon to launch registration study for KD025 in GvHD; shares up 3% (April 10)
    • AzurRx BioPharma (NASDAQ:AZRX) initiated with Outperform rating and $6 (140% upside) price target at Oppenheimer. Shares up 8% premarket.
    • Bio-Techne (NASDAQ:TECH) initiated with Neutral rating and $190 (3% upside) price target at Goldman Sachs.
    • Bio-Rad Laboratories (NYSE:BIO) initiated with Buy rating and $350 (21% upside) price target at Goldman.
    • Akorn (NASDAQ:AKRX) upgraded to Overweight with a $9 (34% upside) price target at Piper Jaffray. Shares up 5% premarket.
    • Kadmon Holdings (NYSE:KDMN) upgraded to Hold with a $2.90 (flat) price target at WBB Securities. Shares up 11% premarket.
    • Acadia Healthcare (NASDAQ:ACHC) downgraded to Hold at Craig Hallum citing valuation.
    • NantKwest (NASDAQ:NK) downgraded to Sell at Citigroup citing "unwarranted" recent rally.
    • Merck (NYSE:MRKappoints Jim Scholefield as Chief Information and Digital Officer, effective October 29.
    • Scholefield joins Merck from NIKE, Inc., where he is currently the company’s chief information officer.
    • The FDA has granted Orphan Drug Designation to Axsome Therapeutics' (NASDAQ:AXSM) AXS-12 for the treatment of narcolepsy.
    • AXS-12 is an oral, highly selective and potent norepinephrine reuptake inhibitor. The Company plans to initiate a Phase 2 trial of AXS-12 in Q4 with top-line results anticipated in H1 2019.
    • Among the benefits of Orphan Drug status in the U.S. is a seven-year period of market exclusivity for the indication, if approved.
    • Shares are up 5% premarket.
    • Sinovac Biotech (NASDAQ:SVAannounces that the United States District Court of Massachusetts denied 1Globe Capital LLC's August 2018 motion for a preliminary injunction seeking to enjoin Sinovac from issuing common shares.
    • The Court found, among other things, that 1Globe failed to demonstrate that it has a reasonable likelihood of success on the merits of its direct and derivative federal securities law claims challenging the PIPE transaction because it lacked standing to bring those claims.
    • Under Priority Review status, the FDA accepts for review AbbVie's (NYSE:ABBV) marketing application seeking approval for IMBRUVICA (ibrutinib), combined with Roche's (OTCQX:RHHBY) GAZYVA (obinutuzumab), in treatment-naive adult patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL).
    • If approved, it will be the first chemo-free regimen for CLL/SLL in a first-line setting.
    • Related ticker: (NYSE:JNJ)
    • Previously: AbbVie's Imbruvica successful in late-stage leukemia study (May 24)
    • Achaogen (NASDAQ:AKAO) is up 5% premarket on light volume on the heels of its announcement that is has submitted a marketing application in Europe seeking approval for plazomicin for the treatment of complicated tract infections, bloodstream infections due to certain Enterobacteriaceae and infections due to Enterobacteriaceae in adult patients with limited treatment options.
    • The FDA approved the antibiotic, branded as ZEMDRI, in June.
    • Allergan (NYSE:AGN) has completed two safety and tolerability studies for acute migraine candidat ubrogepant, completing the necessary data to support a U.S. marketing application, expected to be submitted to the FDA by Q1 2019.
    • The company says the NDA is the first in the U.S. for an oral CGRP receptor antagonist for acute migraine in adults with or without aura.