BPC September 15 update

Ardelyx ARDX PDUFA set for April 2021; CEL-SCI CVM Phase 3 data delay

Price and Volume Movers

Ardelyx, Inc. (Nasdaq: ARDX) announced that the FDA has accepted its New Drug Application (NDA) of tenapanor for the control of serum phosphorus in adult patients with chronic kidney disease (CKD) on dialysis. The PDUFA date is April 29, 2021.

Novus Therapeutics, Inc. (NASDAQ:NVUS) shares closed up 193% to $1.11 following news released after hours Monday that it has completed the acquisition of privately-held Anelixis Therapeutics, Inc, which is developing treatments for organ and cellular transplantation, autoimmune diseases, and neurodegenerative diseases. Novus also announced a private placement for gross proceeds of $108m.

Marinus Pharmaceuticals, Inc. (Nasdaq: MRNS) shares closed up 52% to $3.20. The company announced after hours yesterday that its Phase 3 Marigold trial evaluating oral ganaxolone in children and young adults with CDKL5 deficiency disorder (CDD) met the primary endpoint.

resTORbio, Inc (NASDAQ:TORC) announced that it has completed its reverse merger with Adicet Bio, Inc. and will commence trading under the ticker symbol "ACET" on September 16, 2020.

CEL-SCI Corporation (NYSE American: CVM) provided an update regarding its Phase 3 trial of Multikine in patients with head and neck cancer. Despite announcing on May 4 that the trial had reached the required number of events to evaluate the data, the company has yet to release data, noting today that the COVID-19 pandemic has “complicated and delayed the data lock process for our Phase 3 study and added to our workload.No other timeline guidance was provided.


Major price movers (stocks priced > $1.00, volume > 50k):

Refer to the BioPharmCatalyst Biotech Stocks page for Top 10 Gainers/Losers/Unusual Volume:


Plus Therapeutics, Inc. (PSTV): $3.03; +24%.

Jounce Therapeutics, Inc. (JNCE): $10.95; +23%.

Inovio Pharmaceuticals, Inc. (INO): $13.63; +20%.

Checkpoint Therapeutics, Inc. (CKPT): $3.91; +19%.

MediWound Ltd. (MDWD): $3.72; +16%.


89bio, Inc. (ETNB): $30.44; -17%.

Vaxart, Inc. (VXRT): $6.50; -16%.

Capricor Therapeutics, Inc. (CAPR): $4.68; -13%.

Harmony Biosciences Holdings, Inc. (HRMY): $35.60; -11%.

Cassava Sciences, Inc. (SAVA): $7.02; -9%.

Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

ABUS – Arbutus Biopharma Corporation
Hepatitis B (HBV)

Phase 1 Phase 1a/b data released September 15, 2020 noted favorable safety and tolerability profile.
$264 million

APLS – Apellis Pharmaceuticals Inc.
Pegcetacoplan (APL-2) PEGASUS
Paroxysmal Nocturnal Hemoglobinuria (PNH)

NDA Filing NDA filing announced September 15, 2020.
$2.5 billion

ARDX – Ardelyx Inc.
Serum phosphorus - chronic kidney disease (CKD) on dialysis

PDUFA PDUFA date April 29, 2021.
$540.2 million

ARPO – Aerpio Pharmaceuticals Inc.
Razuprotafib (AKB-9778)
Open-angle glaucoma (OAG)

Phase 2 Phase 2 enrollment completion announced September 15, 2020 with top-line data due 4Q 2020.
$63.1 million

CVM – Cel-Sci Corporation
Head and Neck cancer

Phase 3 Phase 3 trial has reached the required number of events to evaluate data - May 4, 2020. Noted delays in collecting data due to COVID-19 - September 15, 2020.
$553.9 million

EQ – Equillium Inc.

Phase 3 Phase 3 trial to be initiated in 4Q 2020.
$155.5 million

OPK – Opko Health Inc.
Mild-to-moderate COVID-19

Phase 2 Phase 2 trial initiation announced September 15, 2020.
$2.1 billion

PFE – Pfizer Inc.
Duchenne muscular dystrophy (DMD)

Phase 3 Phase 3 trial to be initiated 2H 2020 with interim data due in 2022.
$203.5 billion

PFE – Pfizer Inc.
Alopecia areata

Phase 3 Phase 3 data due 3Q 2021.
$203.5 billion

PFE – Pfizer Inc.
Atopic Dermatitis

NDA Filing Regulatory filing submitted August 2020.
$203.5 billion

PSTI – Pluristem Therapeutics Inc.
PLX-PAD (stem cells)
Critical limb ischemia (CLI)

Phase 3 Phase 3 data due 4Q 2020.
$257.8 million

PSTI – Pluristem Therapeutics Inc.
COVID-19 / Acute Respiratory Distress Syndrome (ARDS)

Phase 2 Phase 2 enrollment to be completed 1Q 2021.
$257.8 million

TFFP – TFF Pharmaceuticals Inc - Ordinary Shares
Voriconazole Inhalation Powder
Invasive Pulmonary Aspergillosis (IPA)

Phase 1 Phase 1 data released September 15, 2020. Phase 2 trial planned.
$359.3 million