BPC July 02 update

Biotech regulatory events to watch this quarter for small and mid-cap companies

Weekly watchlist

With the first half of 2021 done and dusted, it is time to look forward to key catalysts slated for the third quarter. We start this week with small and large cap regulatory events, followed by a series of clinical data readout previews from next week.

First, let’s review the week that was with price-moving events.


MediWound Ltd. (NASDAQ:MDWD) and Vericel Corporation (NASDAQ: VCEL) announced they received a Complete Response Letter (CRL) from the FDA regarding the Biologics License Application (BLA) seeking approval of NexoBrid for eschar removal in adults with thermal burns. The FDA identified Chemistry, Manufacturing and Controls (CMC) issues for the CRL. Shares of MediWound closed the week down 31% to $3.95, while Vericel shares closed down 23% to $51.77.

Iterum Therapeutics plc (NASDAQ:ITRM) announced the FDA has identified deficiencies that preclude the discussion of labeling and post marketing requirements/commitments for its NDA for sulopenem. The assigned PDUFA date is July 25, 2021. Shares closed Friday down 38% to $1.42.


Intellia Therapeutics, Inc. (NASDAQ:NTLA) and Regeneron Pharmaceuticals, Inc. (NASDAQ:REGN) announced positive interim data from their Phase 1 trial of their CRISPR gene editing candidate, NTLA-2001, for treatment for transthyretin (ATTR) amyloidosis. Treatment with NTLA-2001 led to dose-dependent reductions in serum TTR, with mean reductions of 87% among the three patients in the high dose group. Shares closed the week up 84% to $163.71. Other companies developing candidates using CRSPR technology also rose in sympathy, with Editas Medicine, Inc. (NASDAQ:EDIT) shares closing the week up 31% to $54.90 while Beam Therapeutics (NASDAQ: BEAM) added 36% to $130.21.

Cerevel Therapeutics (NASDAQ:CERE) shares surged to close the week up 78% to $23.13 following the release of positive data from its Phase 1b trial of CVL-231 in patients with schizophrenia. In the 30 mg cohort, scores in the Positive and Negative Syndrome Scale (PANSS) were a statistically significant and clinically meaningful 19.5 points lower than baseline and 12.7 points lower than the placebo group (p=0.023).

Altimmune, Inc. (NASDAQ:ALT) announced that it will discontinue development of AdCOVID, its investigational COVID-19 vaccine, following the release of Phase 1 data which showed the vaccine did not stimulate an adequate immune response in subjects. It also noted it has terminated enrollment in its Phase 1/2 T-COVID vaccine trial. Shares closed the week down 42% to $9.34.

CEL-SCI Corporation (NYSE:CVM) announced data from its Phase 3 trial of Multikine in the treatment of advanced previously untreated squamous cell carcinoma of the head and neck (SCCHN) did not meet the primary endpoint. Shares slumped to close the week down 68% to $8.05.

Exelixis, Inc. (NASDAQ: EXEL) announced data from its Phase 3 COSMIC-312 trial evaluating cabozantinib (CABOMETYX) in combination with atezolizumab versus sorafenib in patients with previously untreated advanced hepatocellular carcinoma (HCC). While the trial met one of the primary endpoints, demonstrating significant improvement in progression-free survival (PFS), interim analysis for the second primary endpoint of overall survival (OS), suggested the probability of meeting the endpoint at the time of the final analysis in early 2022 is low. Shares of Exelixis fell 22% to close the week down at $18.26.

PDUFA and Advisory Committee meeting dates for small/mid-cap biotech companies - 3Q 2021

Drug Price Stage Catalyst Market Cap

ALBO – Albireo Pharma Inc.
BYLVAY (odevixibat)
Progressive familial intrahepatic cholestasis (PFIC)

-1.41  -6%
Approved FDA approval announced July 20, 2021.
$458.4 million

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

-0.03  -3%
CRL FDA issued CRL on July 29 2021. The FDA held a Type A meeting with Ardelyx, but it did not provide enough clarity on what constitutes "clinical relevance of the magnitude of treatment effect" and continues to await additional information regarding the path forward for the company's NDA, noted October 13, 2021. Company to issue a formal dispute resolution request (FDRR), noted November 4, 2021.
$100.6 million

AXSM – Axsome Therapeutics Inc.
Dextromethorphan-bupropion (AXS-05)
Major Depressive Disorder

-1.01  -3%
PDUFA priority review PDUFA date under priority review August 22, 2021. Noted August 9, 2021 that the FDA has identified deficiencies that preclude discussion of labeling and post-marketing requirements/commitments. Updated August 22, 2021 that the FDA informed the company that its decision has been delayed. No further details provided.
$1.4 billion

CALT – Calliditas Therapeutics AB
Nefecon (NefIgArd)
IgA nephropathy

-1.36  -7%
PDUFA priority review PDUFA priority review date extended to December 15, 2021.
$467.1 million

CARA – Cara Therapeutics Inc.
KORSUVA (CR845/difelikefalin) injection
Chronic kidney disease (CKD) on hemodialysis (HD) with Moderate-to Severe Pruritus

-0.54  -4%
Approved FDA approval announced August 23, 2021.
$684.3 million

CCXI – ChemoCentryx Inc.
TAVNEOS (avacopan)
Associated vasculitis (AAV)

-2.60  -7%
Approved FDA approval announced October 8, 2021.
$2.5 billion

ETON – Eton Pharmaceuticals Inc.
EPRONTIA (topiramate) - Oral Solution

-0.10  -2%
Approved FDA Approval November 8, 2021.
$103.8 million

FGEN – FibroGen Inc
Anaemia in Chronic Kidney Disease

-0.73  -6%
CRL CRL announced August 11, 2021.
$1.1 billion

KDMN – Kadmon Holdings Inc.
REZUROCK (Belumosudil)
Chronic graft-versus-host disease (cGVHD)

0.00  0%
Approved FDA approval announced July 16, 2021.
$1.7 billion

MIRM – Mirum Pharmaceuticals Inc.
LIVMARLI (Maralixibat)
Alagille Syndrome (ALGS)

-0.91  -6%
Approved Approved September 29, 2021.
$410.9 million

PRVB – Provention Bio Inc.
Teplizumab (PRV-031)
Type 1 diabetes

-0.37  -5%
CRL CRL issued on July 2, 2021. Additionally at the FDA Type A meeting on PK Model held in November 18, 2021, the FDA expressed its concurrence with the Company proceeding to schedule a Type B, pre-BLA re-submission meeting, noted November 22, 2021.
$444.9 million

SESN – Sesen Bio Inc.
Non-muscle invasive bladder cancer (NMIBC)

-0.01  -1%
CRL CRL announced August 13, 2021. Type A meeting on November 1, 2021, with additional meeting later 2021.
$215.4 million

SLGL – Sol-Gel Technologies Ltd.

-0.67  -8%
Approved FDA approval announced July 27, 2021.
$178.2 million

VRCA – Verrica Pharmaceuticals Inc.
Molluscum contagiosum

-0.65  -6%
CRL CRL announced July 14, 2020. NDA resubmitted. CRL announced September 20, 2021.
$287.8 million