The Week Ahead In Biotech: FDA Decisions For Mesoblast And Eton, Coronavirus Vaccine Updates

Biotech stock reversed course last week, dragged lower by the broader market weakness.

The week's news flow was headlined by Johnson & Johnson JNJ kickstarting a Phase 3 trial of its coronavirus vaccine candidate.

A few clinical readouts released during the week triggered strong moves. Vaccinex Inc VCNX and AC Immune SA ACIU were among the worst decliners of the week, with the former reacting to a failed mid-stage trial of lead drug in Huntington's disease, while the latter plunged after an out-licensed Alzheimer's drug flunked a mid-stage trial.

Here are the key catalysts for the unfolding week.

Conferences

World Muscle Society, or WMS, 25: Virtual Congress: Sept. 28-Oct. 2
Annual Northeast Amyotrophic Lateral Sclerosis, or NEALS, meeting: Sept. 30- Oct. 1
Heart Failure Society of America, or HFSA, Virtual Annual Scientific Meeting 2020: Sept. 30-Oct. 6
virtual Jefferies Cell Therapy Summit: Oct. 5-6
Virtual Jefferies Gene Therapy/Editing Summit: Oct. 1-2
North American Neuroendocrine Tumor Society, or NANETS', Multidisciplinary NET Medical Virtual Symposium: Oct. 2-3
Virtual 7th Immunotherapy of Cancer Conference, or ITOC7: Oct. 2-3
EURETINA 2020 Virtual Meeting: Oct. 2-4

PDUFA Dates

The FDA is set to rule on Aquestive Therapeutics Inc's AQST NDA for libervant (AQST-203) in seizure clusters. (Sunday)

The regulatory agency is also scheduled to give its verdict on Eton Pharmaceuticals Inc's ETON NDA for Alkindi Sprinkle, which is being evaluated as a replacement therapy for pediatric adrenal insufficiency, including congenital adrenal hyperplasia in patients from birth to less than 17 years of age. (Tuesday)

Mesoblast limited MESO awaits FDA decision on its BLA for remestemcel-L (MSC-100-IV) in steroid-refractory acute graft versus host disease in children. (Wednesday)

Roche Holdings AG's Basel RHHBY regulatory application seeking label expansion for Xolair to be used for chronic rhinosinusitis with nasal polyps has a PDUFA timeline of Q3

Clinical Readouts

Capricor Therapeutics Inc CAPR is scheduled to present at the WMS Congress, novel data from its Phase 2 HOPE-2 clinical trial of lead asset CAP-1002, in boys and young men with Duchenne muscular dystrophy. (Thursday afternoon)

Xencor Inc XNCR will present at the NANETS symposium initial data from its Phase 1 dose-escalation study of tidutamab in patients with neuroendocrine tumors. (Friday)

Spring Bank Pharmaceuticals Inc SBPH is dure to present at the ITOC7, Phase 1a/1b data for intravenous SB 11285 in solid tumors

MeiraGTx Holdings PLC MGTX will present at the EURETINA meeting nine-month results from the ongoing Phase 1/2 clinical trial of AAV-RPGR, an investigational gene therapy for the treatment of X-linked retinitis pigmentosa. (Saturday)

At the EURETINA meeting, Apellis Pharmaceuticals Inc APLS will make a late-breaker oral presentation, of post hoc analysis of the Phase 2 FILLY study investigating intravitreal pegcetacoplan for the treatment of geographic atrophy secondary to age-related macular degeneration. (Saturday)

Cytokinetics, Inc. CYTK is due to present at the HFSA meeting new data from the Phase 3 event driven cardiovascular outcomes clinical trial of omecamtiv mecarbil and additional results thee completed Phase 2 clinical trial which evaluated omecamtiv mecarbil in patients with heart failure with reduced ejection fraction.

Q3 Releases

Ovid Therapeutics Inc OVID: results from the Phase 2 ARCADE trial in CDKL5 deficiency disorder and Dup15q syndrome, results from Phase 2 ELEKTRA trial in patients with Dravet syndrome and Lennox-Gastaut syndrome, and updated data from the ENDYMION open-label extension study in developmental and epileptic encephalopathies

Selecta Biosciences Inc SELB: topline data from the head-to-head COMPARE trial of once-monthly dose of SEL-212 (ImmTOR + pegadricase) compared to biweekly doses of pegloticase in tophaceous gout

Synthetic Biologics Inc SYN: interim futility analysis of Phase 2b investigator-sponsored clinical trial of SYN-010, intended to treat irritable bowel syndrome-constipation

Cyclerion Therapeutics Inc CYCN: top-line results from the Phase 2 STRONG-SCD study of olinciguat, in sickle cell disease (late Q3)

Atara Biotherapeutics Inc ATRA: interim analysis of the tab-cel Phase 3 study in Epstein-Barr virus and post-transplant lymphoproliferative disease after solid organ transplant

Alnylam Pharmaceuticals, Inc. ALNY: topline results from the ILLUMINATE-B Phase 3 study of lumasiran in primary hyperoxaluria type 1 patients less than six years of age with preserved renal function

Momenta Pharmaceuticals, Inc. MNTA: results from Part B of the Phase 1/2 study of M254 in idiopathic thrombocytopenic purpura

Myovant Sciences Ltd MYOV: Castration-resistance free survival data for relugolix in prostate cancer

Ascendis Pharma A/S ASND: six-month data from the open-label extension portion of the PaTH Forward Trial evaluating TransCon for hypoparathyroidism

Corbus Pharmaceuticals Holdings Inc CRBP: topline results from the Phase 2b study of lenabasum for cystic fibrosis

Aerie Pharmaceuticals Inc AERI: Mercury 3 topline readout, highlighting Roclanda's intraocular pressure reducing performance compared to Ganfort

BioCryst Pharmaceuticals, Inc. BCRX: Part 1 of galidesivir trial in COVID-19 patients (end of Q3) and data from the Phase 1 study of BCX9930 in treatment-naïve Paroxysmal nocturnal hemoglobinuria in patients

September Releases

Tonix Pharmaceuticals Holding Corp TNXP: interim analysis of results for Phase 3 RELIEF trial of TNX-102 SL for the management of Fibromyalgia

Coronavirus Vaccine Updates

Moderna Inc MRNA: interim Phase 2 efficacy data of RNA-m1273 (Q3)

Inovio Pharmaceuticals Inc INO: commencement of Phase 2/3 study of INO-4800 DNA vaccine candidate (September)

Regeneron Pharmaceuticals Inc REGN: initial virology and biomarker data Phase 2/3 trial REGN-COV2, an antiviral antibody, in COVID-19 (September)

Earnings

AngioDynamics, Inc. ANGO (Tuesday, before the market open)

IPOs

Exton, Pennsylvania-based Immunome, Inc. has filed with the SEC a preliminary prospectus to offer 2.5 million shares of its common stock in an initial public offering, or IPO. The biopharma, which uses its proprietary human memory B cell platform to discover and develop first-in-class antibody therapeutics, said it expects to price the offering between $11 and $13. The company has applied for listing its shares on the Nasdaq under the ticker symbol IMNM.

Pulmonx Corporation, a commercial-stage MedTech company that provides a minimally invasive treatment for patients with severe emphysema, a form of chronic obstructive pulmonary disease, has filed for a 6.67-million IPO at an estimated price range of $14-$16. The Redwood City, California-based company has applied for listing its shares on the Nasdaq under the ticker symbol LUNG.