The Week Ahead In Biotech (March 28-April 3): Acadia's Dementia Drug Update, Clinical Readouts, Few Earnings In Holiday-Shortened Week

Biotech stocks reversed course and ended lower in the week ended March 26, drawing inspiration from the broader market weakness.

The earnings news flow was confined to micro- and small-cap companies. Five biopharma companies listed their shares on the Nasdaq through initial public offerings. These companies raised about $666.5 million in gross proceeds.

Several Food and Drug Administration approvals came through during the week. Merck's MRK Keytruda was cleared for easophageal cancer, while Pacira BioSciences, Inc.'s PCRX non-opioid pain drug and Zealand Pharma A/S' ZEAL ready-to-use glucagon pen to treat low blood sugar levels also received clearance from the FDA.

A T-cell therapy co-developed by bluebird bio Inc BLUE and Bristol-Myers Squibb Co BMY won the nod for a type of blood cancer.

Odonate Therapeutics, Inc. ODT was among the worst performers of the week after it shelved the study of its oral chemotherapy medication in breast cancer and also decided to wind down operations.
Frequency Therapeutics, Inc. FREQ was another big loser after its shares shed about 80% of their value following the release of an adverse clinical readout.

Here are the key catalysts for the unfolding week:

Conferences

Stifel 3rd Annual CNS Day: March 31
Guggenheim Healthcare Talks: 2021 Genomic Medicines & Rare Disease: April 1

PDUFA Dates

ACADIA Pharmaceuticals Inc.'s ACAD regulatory application seeking expanded use of pimavanserin has a decision date of Saturday, April 3. The company is hoping to get approval for the additional indication of hallucinations and delusions associated with dementia-related psychosis.

But it looks likely there will be either a delay in approval or an outright rejection. The company announced on March 8 that the FDA has identified deficiencies that preclude discussion of labeling and post-marketing requirements or commitments.

Clinical Readouts/Presentations

Pending Readouts For March/ Q1/Early 2021

Sage Therapeutics, Inc. SAGE expects to release sometime soon topline data from a Phase 2 KINETIC study that is evaluating its SAGE-324 in patients with a nervous disorder called essential tremor. It also expects to release topline data from a Phase 2a study of SAGE-718 in patients with Parkinson's disease cognitive dysfunction.

Merck & Co., Inc. MRK will release in the first quarter full results from a Phase 3 study of MK-7110, a therapeutic candidate for the treatment of patients with severe and critical COVID-19. MK-7110 came into Merck's stable through its acquisition of OncoImmune.

Delcath Systems, Inc. DCTH is scheduled to release topline results from the Phase 3 study of Hepzato kit in liver dominant ocular melanoma. Hepzato kit consists of melphalan hydrochloride for injection or hepatic delivery, and it is meant to deliver high-dose chemotherapy to the liver while minimizing systemic exposure and associated side effects.

Yumanity Therapeutics, Inc. YMTX is due to release results from the Phase 1 multiple-ascending dose study of its investigational therapy YTX-7739 in healthy volunteers. YTX-7739 is being evaluated for the treatment of Parkinson's disease.

BioXcel Therapeutics, Inc. BTAI will release in the first quarter topline results from the Phase 1b/2 study of BXCL501 for the treatment of opioid withdrawal symptoms.

Related Link: Inovio Has 'Bigger Value Drivers' Outside Of COVID-19 Vaccine Program: BofA Analyst

Wave Life Sciences Ltd. WVE is due to release at the end of the first quarter, data from ongoing PRECISION-HD and OLE clinical trials evaluating WVE-120101 in Huntington's disease.

Forma Therapeutics Holdings, Inc. FMTX is expected to release in the first quarter, data from the 600mg cohort of the multiple-ascending dose 2 part of the Phase 1 study of FT-4202 in sickle cell disease patients.

Celldex Therapeutics, Inc. CLDX is scheduled to release in the first quarter Phase 1b study of CDX-0159 in chronic inducible urticaria – an allergic skin reaction.

AzurRx BioPharma, Inc. AZRX will release by the end of the first quarter topline data from the Phase 2b OPTION 2 study of MS1819 in cystic fibrosis patients with exocrine pancreatic insufficiency.

Humanigen, Inc. HGEN is scheduled to release at the end of March topline data from the Phase 3 study of lenzilumab in COVID-19.

New Drug Application Filings

Alnylam Pharmaceuticals, Inc. ALNY is scheduled to file a new drug application for vutrisiran in early 2021. The drug is a subcutaneously administered investigational RNAi therapeutic in development for the treatment of ATTR amyloidosis. ATTR amyloidosis is related to the abnormal production and buildup of a type of a protein called transthyretin, which is used to transport thyroid hormones and vitamin A throughout the body.

CTI BioPharma Corp. CTIC expects to complete a rolling submission of its NDA for pacritinib, for use in myelofibrosis patients with severe thrombocytopenia before the end of March.

Earnings

Monday

Matinas BioPharma Holdings, Inc. MTNB (before the market open)
I-Mab IMAB (before the market open)
F-star Therapeutics, Inc. FSTX (before the market open)
Biocept, Inc. BIOC (after the close)

Tuesday

PolarityTE, Inc. PTE (before the market open)
Pieris Pharmaceuticals, Inc. PIRS (before the market open)
Forma Therapeutics (before the market open)
Protalix BioTherapeutics, Inc. PLX (before the market open)
AngioDynamics, Inc. ANGO (before the market close)
Bellicum Pharmaceuticals, Inc. BLCM (after the close)
Daré Bioscience, Inc. DARE (after the close)
IGM Biosciences, Inc. IGMS (after the close)
Osmotica Pharmaceuticals plc OSMT (after the close)
EDAP TMS S.A. EDAP (after the close)
Dyadic International, Inc. DYAI (after the close)
Crinetics Pharmaceuticals, Inc. CRNX (after the close)

Wednesday

Apyx Medical Corporation APYX (before the market open)
Delcath Systems (before the market open)
BiomX Inc. PHGE (before the market open)
Precipio, Inc. PRPO (after the close)
Rockwell Medical, Inc. RMTI (after the close)

IPOs

IPO Pricing

U.K.-based Achilles Therapeutics plc has filed with the SEC to sell 9.75 million American depositary shares in an initial public offering, with each ADS representing one ordinary share of the company. The clinical stage immuno-oncology company is developing precision T-cell therapies to treat multiple types of solid tumors. The company plans to price the offering between $17 and $19. It has applied for listing its shares on the Nasdaq under the ticker symbol ACHL.

Related Link: A Spotlight On Israel's Rollout Of Pfizer's COVID-19 Vaccine, Why Some Have Privacy Concerns

Photo by Anna Shvets from Pexels.

Story has been updated to reflect the type of cancer targeted by the drug under development by bluebird bio Inc and Bristol-Myers Squibb.