Biotech week ahead, Sept. 28

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

The week's news flow was headlined by Johnson & Johnson JNJ 0.72% kickstarting a Phase 3 trial of its coronavirus vaccine candidate.

A few clinical readouts released during the week triggered strong moves. Vaccinex Inc VCNX 1.1% and AC Immune SA ACIU 1.34% 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 2.31% 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 1.22% 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 2.1% 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 0.27% 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 6.43% 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 1.72% 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 5.19% is dure to present at the ITOC7, Phase 1a/1b data for intravenous SB 11285 in solid tumors

MeiraGTx Holdings PLC MGTX 1.82% 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 6.73% 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 6.61% 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 4.02%: 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 2.54%: 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 0.66%: interim futility analysis of Phase 2b investigator-sponsored clinical trial of SYN-010, intended to treat irritable bowel syndrome-constipation

Cyclerion Therapeutics Inc CYCN 1.88%: top-line results from the Phase 2 STRONG-SCD study of olinciguat, in sickle cell disease (late Q3)

Atara Biotherapeutics Inc ATRA 3.35%: 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 3.73%: 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 0.02%: results from Part B of the Phase 1/2 study of M254 in idiopathic thrombocytopenic purpura

Myovant Sciences Ltd MYOV 6.21%: Castration-resistance free survival data for relugolix in prostate cancer

Ascendis Pharma A/S ASND 3.22%: six-month data from the open-label extension portion of the PaTH Forward Trial evaluating TransCon for hypoparathyroidism

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

Aerie Pharmaceuticals Inc AERI 3.69%: Mercury 3 topline readout, highlighting Roclanda's intraocular pressure reducing performance compared to Ganfort

BioCryst Pharmaceuticals, Inc. BCRX 6.12%: 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 1.97%: interim analysis of results for Phase 3 RELIEF trial of TNX-102 SL for the management of Fibromyalgia

Coronavirus Vaccine Updates

Moderna Inc MRNA 6.6%: interim Phase 2 efficacy data of RNA-m1273 (Q3)

Inovio Pharmaceuticals Inc INO 10.21%: commencement of Phase 2/3 study of INO-4800 DNA vaccine candidate (September)

Regeneron Pharmaceuticals Inc REGN 2.98%: initial virology and biomarker data Phase 2/3 trial REGN-COV2, an antiviral antibody, in COVID-19 (September)

Earnings

AngioDynamics, Inc. ANGO 2.34% (Tuesday, before the market open)

Related Link: AstraZeneca Has Lost The Lead In Race For Coronavirus Vaccine, Says SVB Leerink

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.

IPO Quiet Period Expiry

PainReform Ltd PRFX 3.07%

https://www.benzinga.com/general/biotech/20/09/17647004/the-week-ahead-in-biotech-fda-decisions-for-mesoblast-and-eton-coronavirus-vaccine-updates