BPC February 26 update

Clinical and regulatory biotech events to watch for the remaining weeks of 1Q 2021

Weekly watchlist

With just four full weeks remaining in the first quarter of 2021, we count down remaining clinical and regulatory catalysts slated for the quarter. During the following three weeks we will publish an updated list, replacing completed catalysts with new entries of interest.

First, lets review the week that was with a selection of price-moving events and notable news.

The FDA Vaccines and Related Biological Products Advisory Committee (VRBPAC) met Friday to discuss the request for emergency use authorization (EUA) of a COVID-19 vaccine from (Janssen Biotech) Johnson & Johnson (NYSE:JNJ). The committee voted unanimously 22-0 that the benefits outweighed the risks in people over the age of 18 years. The vaccine becomes the third to be given a positive vote by the committee, following in the steps of Moderna and Pfizer. The FDA is likely to issue a EUA within the next few days. The company expects to deliver the U.S. 20 million doses of its single-dose vaccine candidate by the end of March and 100 million doses by the end of June.

In clinical trials, the vaccine was shown to be 66% effective in preventing moderate to severe COVID-19. Broken down into regions, the efficacy rate was 72% in the U.S. and 57% in South Africa. The vaccine was 85% effective in preventing severe disease. The vaccine is estimated to remain stable for two years at -4°F (-20°C), and a maximum of three months at routine refrigeration at temperatures of 36-46°F (2 to 8°C).

Otonomy, Inc. (NASDAQ:OTIC) announced its Phase 3 clinical trial of Otividex in patients with Ménière's disease did not achieve the primary endpoint, which was the count of definitive vertigo days (DVD) in Month 3 for vs. placebo for the intent-to-treat (ITT) population (n = 148; p value = 0.312) using the Negative Binomial Model. Shares slumped to close the week down 46% to $2.92.

BrainStorm Cell Therapeutics Inc. (NASDAQ:BCLI) shares closed the week down 41% to $4.06 on news the FDA has advised the company that data from its Phase 3 trial of NurOwn in patients with Amyotrophic lateral sclerosis does not provide the threshold of substantial evidence to support a Biologics License Application (BLA). The FDA advised that this recommendation does not preclude Brainstorm from proceeding with a BLA submission. The company announced in November 2020 that the trial did not meet the primary endpoint.

Merck (NYSE:MRK) announced the acquisition of Pandion Therapeutics, Inc. (NASDAQ:PAND) for $60 per share in cash or an approximate total equity value of $1.85 billion. Shares of Pandion closed Thursday up 133% to $59.81.

Tricida, Inc. (Nasdaq: TCDA) shares closed Friday down 31% to $5.11 following news it received an Appeal Denied Letter from the FDA in response to its Formal Dispute Resolution Request (FDRR) submitted in December 2020. The company received a Complete Response Letter in August last year for TRC101 in patients with chronic kidney disease (CKD) and filed the FDRR hoping that certain data from previous trials could be used to accelerate their resubmission.

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Selection of biotech earnings next week (15) – full list for premium members

M: BHVN CCXI FGEN FOLD INO NVAX OMER RETA SRPT TVTX

T: EGRX RIGL

Th: ANIK EVFM WVE

Drug Stage Catalyst Market Cap

ANAB – AnaptysBio Inc.
ANB019 - POPLAR
Palmo-plantar pustular psoriasis

Phase 2 Phase 2 trial did not meet primary endpoint - March 8, 2021.
$571.1 million

ANVS – Annovis Bio Inc.
ANVS401
Alzheimer’s disease (AD) and Parkinson’s disease (PD)

Phase 2 Phase 2 initial data released March 16, 2021. PD interim data (reversal of the toxic cascade) due April; AD data in May. Full data June / July 2021.
$184.4 million

ASLN – ASLAN Pharmaceuticals Limited
ASLAN004
Atopic dermatitis

Phase 1 Phase 1 interim data released March 1, 2021. Full data due mid-2021.
$209.9 million

AZN – AstraZeneca PLC
AZD1222
COVID-19 vaccine

Phase 3 Phase 3 data exhibited 70% efficacy (90% and 60% across two dosing regimens) - UK and Brazil trial. Data from U.S. trial noted 76% efficacy rate - March 24, 2021.
$131.1 billion

AZRX – AzurRx BioPharma Inc.
MS1819 - OPTION 2
Cystic fibrosis

Phase 2b Phase 2b top line data did not meet primary endpoint - March 31, 2021.
$69.2 million

BCLI – Brainstorm Cell Therapeutics Inc.
NurOwn
Progressive Multiple Sclerosis

Phase 2 Phase 2 top-line data released March 24, 2021 - safety primary endpoint met.
$130.4 million

CRMD – CorMedix Inc.
Defencath (Neutrolin)
Hemodialysis patients with central venous catheters

CRL CRL announced March 1, 2021.
$361.2 million

DCTH – Delcath Systems Inc.
Melphalan/HDS - FOCUS
Liver-dominant ocular melanoma

Phase 3 Phase 3 top-line data released March 31, 2021. Overall Response Rate (ORR) of 29.2%.
$86.2 million

FGEN – FibroGen Inc
Roxadustat
Anaemia in Chronic Kidney Disease

PDUFA PDUFA date TBC. Advisory Committee meeting July 15, 2021.
$1.7 billion

GSK – GlaxoSmithKline PLC
VIR-7831 / GSK4182136 (COMET-ICE)
COVID-19 antibody

Phase 3 Phase 3 data released March 10, 2021. 85% reduction in hospitalization or death. Trial to be stopped. FDA Emergency Use Authorization filing announced March 26, 2021.
$92.2 billion

IDRA – Idera Pharmaceuticals Inc.
Tilsotolimod (IMO-2125) + ipilimumab - ILLUMINATE 301
Melanoma

Phase 3 Phase 3 trial did not meet primary endpoint - March 18, 2021.
$56.6 million

KMPH – KemPharm Inc
KP415
ADHD

Approved FDA approval announced March 2, 2021.
$259.6 million

SAGE – Sage Therapeutics Inc.
SAGE-324 KINETIC (324-ETD-201)
Essential tremor

Phase 2 Phase 2 data due early-2021.
$4.5 billion

WVE – Wave Life Sciences Ltd.
WVE-120101 (PRECISION-HD1)
Huntington’s disease

Phase 1/2 Development to be stopped - March 29, 2021.
$305.3 million