BPC January 14 update

Key Phase 1/2 catalysts for the first quarter; Biotech week in review

Weekly watchlist

With 2022 in full swing, we now highlight Phase 1/2 catalysts likely due to read out in the first quarter. Next week we will focus on regulatory events to expect with PDUFA dates, regulatory filings, and Adcom meetings.

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

REGULATORY

The FDA placed a clinical hold on Denali Therapeutics Inc’s (NASDAQ: DNLI) Investigational New Drug (IND) application of DNL919. Shares closed the week down 14% at $34.84.

The FDA cleared Black Diamond Therapeutics, Inc’s (NASDAQ: BDTX) Investigational New Drug (IND) application for BDTX-1535 to treat glioblastoma multiforme (GBM) and non-small cell lung cancer (NSCLC). Shares closed the week down 16% at $4.24.

CLINICAL UPDATES:

Molecular Partners AG (NASDAQ: MOLN) and Novartis (NYSE: NVO) announced that Part A of their EMPATHY Phase 2 trial of ensovibep to treat COVID-19 met the primary endpoint. Molecular Partners will receive a milestone payment of 150M CHF and be entitled to a 22% royalty on sales. Shares closed the week up 57% at $31.89.

OTHERS:

Immuron Ltd. (NASDAQ: IMRN) was awarded AUD 6.2 million ($4.5 million) in funding from the U.S Department of Defense to evaluate a military strength dosing regimen for Travelan. Shares closed the week up 32% at $3.39.

BioCryst Pharmaceuticals, Inc. (NASDAQ: BCRX) announced that ORLADEYO net revenue is expected to more than double in 2022 to no less than $250 million with peak sales of $1 billion. Shares closed the week up 26% at $15.48.

Aclaris Therapeutics (NASDAQ: ACRS) announced late Tuesday that David Gordon stepped down as chief medical officer and left the company on January 7th but has yet to name a replacement. Shares closed the week down 15% at $10.81.

It was disclosed in a 13D filing that FiveT Investment Management has a 6.03% stake in Inhibikase Therapeutics, Inc. (NASDAQ: IKT). Shares closed the week up 14% at $1.67.

Drug Price Stage Catalyst Market Cap

ALDX – Aldeyra Therapeutics Inc.
ADX-629
Psoriasis / atopic asthma / COVID-19

$3.25
-0.22  -6%
Phase 2 Phase 2 top-line data due by 1Q 2022.
$188.8 million

AMTI – Applied Molecular Transport Inc.
AMT-101 combo with with anti-TNFα - (MARKET)
Ulcerative colitis

$8.84
-0.47  -5%
Phase 2 Phase 2 top-line data due 1H 2022.
$340.2 million

ANNX – Annexon Inc.
ANX005
Huntington’s disease

$7.50
-0.38  -5%
Phase 2 Phase 2 data reported that treatment was generally well-tolerated, noted January 4, 2022. Full Phase 2 data due in 1H 2022.
$287.8 million

BCAB – BioAtla Inc.
BA3011 (Mecbotamab Vedotin)
Solid Tumors

$10.07
-0.02  0%
Phase 2 Phase 2 interim data due in 1H 2022. Registration data due 2022.
$370.9 million

CNTB – Connect Biopharma Holdings Limited
CBP-201
Atopic dermatitis

$4.90
-0.13  -3%
Phase 2b Phase 2b data met primary endpoint all three doses achieving significant improvements. Also reported for key secondary endpoints including other measures of skin clearance and itch with a 300mg dose every two weeks (Q2W) arm, noted November 18, 2021. Additional Phase 2b analyses demonstrated a potential competitive therapeutic profile for 300mg administered every two weeks (Q2W) or every four weeks (Q4W), noted January 5, 2022. Phase 3 trial to be initiated 2H 2022. Additional data to be presented at the 18th Annual Maui Derm meeting, January 24-28, 2022.
$281.7 million

CNTB – Connect Biopharma Holdings Limited
CBP-307
Ulcerative Colitis

$4.90
-0.13  -3%
Phase 2b Phase 2b trial completed full enrollment noted November 19, 2021. Phase 2b top-line data expected 1Q 2022.
$281.7 million

CRBP – Corbus Pharmaceuticals Holdings Inc.
Lenabasum (Resunab)
Systemic lupus erythematosus (SLE)

$0.47
-0.04  -9%
Phase 2 Phase 2 data expected in 1Q 2022.
$58.5 million

CRDF – Cardiff Oncology Inc.
Onvansertib, FOLFIRI and AVASTIN (bevacizumab)
KRAS-Mutated Colorectal Cancer

$3.69
-0.17  -5%
Phase 1/2 Phase 1b/2 data demonstrated that from patients treated per protocol at the recommended dose 34% achieved an initial CR and 29% PR (awaiting confirmatory scan for 1 patient), 94% had a best response of disease control (CR + PR + SD). ORR of 5-13% observed in historical control trials. Patients evaluable for response treated at all dose levels 35% achieved an initial CR or PR, 27% have achieved a confirmed CR or PR (awaiting confirmatory scan for 1 patient and 92% had a best response of disease control (CR + PR + SD). 4 unconfirmed PRs pending and 10% evaluable patients discontinued therapy to pursue potentially curative metastasis-directed therapy, including 2 patients with SD, noted January 18, 2022.
$155.1 million

ETNB – 89bio Inc.
BIO89-100
Nonalcoholic steatohepatitis (NASH)

$10.02
-0.73  -7%
Phase 1/2 Phase 1b/2a data reported that treatment reduced spleen volume by an average of 11.8% in NASH patients, noted November 14, 2021. Phase 1b/2a additional top-line data due mid-January 2022.
$203.2 million

KDNY – Chinook Therapeutics Inc.
Atrasentan - (AFFINITY)
Chronic kidney disease

$12.72
-0.36  -3%
Phase 2 Phase 2 initial data due 1H 2022, with data from one or more additional cohorts due 2H 2022.
$694.7 million

NMTR – 9 Meters Biopharma Inc.
Vurolenatide - (VIBRANT)
Short bowel syndrome

$0.75
-0.04  -5%
Phase 2 Phase 2 top-line results expected by 1Q 2022. Phase 1b/2a pharmacokinetic data released at ACG on October 26, 2021 indicating half-life is consistent with twice monthly dosing.
$191.4 million

OPNT – Opiant Pharmaceuticals Inc.
OPNT003
Opioid Overdose

$25.64
+0.42  +2%
Phase 1 Phase 1 PD data due 1Q 2022.
$121 million

PDSB – PDS Biotechnology Corporation
PDS0101 - (VERSATILE-002)
Head and neck cancer

$5.71
-0.36  -6%
Phase 2 Phase 2 achieved its preliminary safety benchmark in its first 12 patients September 20, 2021. Phase 2 completion of enrollment announced October 4, 2021. Phase 2 initial data to be presented at the Multidisciplinary Head and Neck Cancers Symposium February 24-26, 2022.
$162.4 million

TSHA – Taysha Gene Therapies Inc.
TSHA-101
GM2 gangliosidosis

$7.93
-0.24  -3%
Phase 1/2 Phase 1/2 biomarker and preliminary clinical data to be presented in January 2022.
$305.1 million

VXRT – Vaxart Inc.
VXA-CoV2-1
COVID-19 Vaccine (oral)

$4.80
-0.26  -5%
Phase 2 Phase 2 trial data expected 1Q 2022, dosing commenced October 26, 2021.
$601.8 million