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

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 (NYSE: JNJ) kickstarting a Phase 3 trial of its coronavirus vaccine candidate.

A few clinical readouts released during the week triggered strong moves. Vaccinex Inc (NASDAQ: VCNX) and AC Immune SA (NASDAQ: 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.


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


The FDA is set to rule on Aquestive Therapeutics Inc's (NASDAQ: 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 (NASDAQ: 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 (NASDAQ: 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 (OTC: 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 (NASDAQ: 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 (NASDAQ: 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 (NASDAQ: SBPH) is dure to present at the ITOC7, Phase 1a/1b data for intravenous SB 11285 in solid tumors

MeiraGTx Holdings PLC (NASDAQ: 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 (NASDAQ: 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. (NASDAQ: 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.

See Also: Illumina Analysts Question Grail Deal's Commercial Opportunity, Hefty Price Tag

Q3 Releases

Ovid Therapeutics Inc (NASDAQ: 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 (NASDAQ: 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 (NYSE: SYN): interim futility analysis of Phase 2b investigator-sponsored clinical trial of SYN-010, intended to treat irritable bowel syndrome-constipation

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

Atara Biotherapeutics Inc (NASDAQ: 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. (NASDAQ: 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. (NASDAQ: MNTA): results from Part B of the Phase 1/2 study of M254 in idiopathic thrombocytopenic purpura

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

Ascendis Pharma A/S (NASDAQ: ASND): 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 (NASDAQ: AERI): Mercury 3 topline readout, highlighting Roclanda's intraocular pressure reducing performance compared to Ganfort

BioCryst Pharmaceuticals, Inc. (NASDAQ: 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 (NASDAQ: TNXP): interim analysis of results for Phase 3 RELIEF trial of TNX-102 SL for the management of Fibromyalgia

Coronavirus Vaccine Updates

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

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

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


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

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


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 (NASDAQ: PRFX)


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