BPC September 17 update

Remaining 3Q biotech catalysts to watch; Biotech week in review

Weekly watchlist

This week’s abstract releases and presentations from ESMO have shed light on highly anticipated drug developments. As ESMO will come to a close on Tuesday, our watchlist is updated to focus on upcoming catalysts before quarter-end.

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

REGULATORY UPDATES:

Pfizer, Inc. (NYSE: PFE) and BioNTech SE (NASDAQ: BNTX)’s booster shot was discussed during the FDA Advisory committee meeting Friday. The committee voted in favor of emergency use for those 65 years and older, at high risk, or healthcare professionals. The committee voted against general use for those 16 years and older.

Protagonist Therapeutics, Inc. (NASDAQ: PTGX) announced that they will halt their trials with the drug rusferide, after the FDA issued a clinical hold. The hold is due to findings from a non-clinical 26-week rasH2 transgenic mouse model study. Shares closed the week down 61% at $17.53.

CLINICAL UPDATES:

Leap Therapeutics (NASDAQ: LPTX) presented data at the ESMO Congress for its Phase 2a trial of DKN-01 plus tislelizumab to treat gastric or gastroesophageal junction cancer (G/GEJ). The overall response rate (ORR) was 68.2%, with 90% ORR in DKK1-high patients and 56% in DKK1-low patients. Of the 25 patients, 15 displayed a partial response (PR), and 6 noted stable disease (SD). The presentation also stated that four Grade 5 adverse events [patient deaths] occurred. Shares closed the week up 81% at $2.64.

TCR2 Therapeutics, Inc. (NASDAQ: TCRR) presented data at the ESMO Congress for its Phase 1/2 trial of gavo-cel to treat mesothelin-expressing solid tumors. Data displayed an overall response rate (ORR) of 31%, with 6 patients achieving a partial response (PR) and 1 achieved a complete response (CR). The presentation also reported 1 Grade 3 event and 1 Grade 5 [patient death]. Shares closed the week down 45% at $10.00.

RedHill Biopharma Ltd. (NASDAQ: RDHL) released top-line data from its Phase 2/3 trial of opaganib to treat severe COVID-19 pneumonia. The data revealed that the study did not meet its primary endpoint. Shares closed the week down 40% at $4.89.

Aerie Pharmaceuticals, Inc. (NASDAQ: AERI) released that its Phase 2b study of AR-15512, an ophthalmic solution to treat dry eye disease, did not meet the primary endpoints. Aerie will hold an end of Phase 2 meeting with the FDA in the first quarter of 2022. Shares closed the week down 23% at $12.88.

MacroGenics, Inc. (NASDAQ: MGNX) presented two data sets at the ESMO Congress for its Phase 1 trial of MGC018 and Phase 2/3 MAHOGANY trial. MacroGenics’ Phase 1 trial presentation of MGC018 to treat solid tumors noted 8 patients achieved a partial response (PR), and 2 Grade 5 [patient deaths] occurred. MacroGenics’ Phase 2/3 MAHOGANY trial of margetuximab had 4 patients achieve complete responses and 17 confirmed partial responses (PR). Shares closed the week down 18% at $20.48.

Corvus Pharmaceuticals (NASDAQ: CRVS) traded on heavy volume Friday, following the data released regarding Innate Pharma and AstraZeneca’s anti-CD73 monoclonal antibody to treat non-small cell lung cancer (NSCLC). Corvus’ lead drug candidate Mupadolimab, is also an anti-CD73 antibody. Shares closed the week up 109% at $5.32.

Drug Price Stage Catalyst Market Cap

ALT – Altimmune Inc.
ALT-801
Non-alcoholic steatohepatitis (NASH) - Healthy Overweight and Obese Volunteers

$11.49
-0.31  -3%
Phase 1b Phase 1b trial initiated, first patient has been enrolled October 4, 2021. Phase 1b top-line data readout expected 1H 2022. Phase 2 obesity and NASH trials expected to initiate in 1H 2022.
$456.2 million

ASLN – ASLAN Pharmaceuticals Limited
ASLAN004
Atopic dermatitis

$1.47
-0.12  -8%
Phase 1 Phase 1 top-line data demonstrated to be well tolerated across all doses September 27, 2021. Phase 2b trial to be initiated 4Q 2021.
$102.3 million

BHVN – Biohaven Pharmaceutical Holding Company Ltd.
Verdiperstat
Multiple system atrophy (MSA)

$138.21
+1.33  +1%
Phase 3 Phase 3 trial did not met primary or secondary endpoints, noted September 27, 2021.
$9 billion

EDIT – Editas Medicine Inc.
AGN-151587 (EDIT-101) - BRILLIANCE
Leber Congenital Amaurosis type 10 (LCA10).

$37.69
-0.78  -2%
Phase 1/2 Phase 1/2 initial data displayed 2 (66%) subjects in the mid-dose cohort showed improvements in BCVA, FST, and/or mobility navigation. However, retinal tears were reported by 2 (50%) subjects, 1 report of hypotony in the low dose cohort, as well as 1 retinal hemorrhage. Treatment in the adult high-dose cohort continues and pediatric mid-dose cohort is commencing, noted September 29, 2021.
$2.6 billion

EVLO – Evelo Biosciences Inc.
EDP1815
Mild to moderate psoriasis

$7.93
-0.35  -4%
Phase 2 Phase 2 data displayed a statistically significant reduction in the PASI score of at least 50% improvement from baseline. 25% to 32% of patients across the three cohorts achieved a PASI-50 at week 16 compared to 12% on placebo, noted September 27, 2021.
$423.5 million

INCY – Incyte Corporation
JAKAFI (Ruxolitinib)
Steroid-refractory chronic GVHD (Graft versus host disease)

$66.00
+0.13  +0%
Approved Approved September 22, 2021.
$14.6 billion

INCY – Incyte Corporation
Opzelura (Ruxolitinib)
Atopic dermatitis

$66.00
+0.13  +0%
Approved Approved September 21, 2021.
$14.6 billion

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

$15.18
-0.57  -4%
Approved Approved September 29, 2021.
$463.2 million

NERV – Minerva Neurosciences Inc
Roluperidone (MIN-101)
Schizophrenia

$1.25
-0.05  -4%
Phase 3 Phase 3 results met all key pharmacokinetic (PK) objectives and the data demonstrate bioequivalence across the various formulations - noted September 30, 2021.
$53.4 million

PTCT – PTC Therapeutics Inc.
PTC518
Healthy volunteers

$35.77
-0.20  -1%
Phase 1 Phase 1 trial data to be released September 23, 2021 noted completion with achievement of all study objectives including protein reduction and projected CSF exposure. They also noted that dose-dependent reduction of HTT mRNA and Protein. Phase 2 trial to be initiated YE 2021.
$2.5 billion

VRCA – Verrica Pharmaceuticals Inc.
VP-102
Molluscum contagiosum

$12.71
+0.11  +1%
CRL CRL announced July 14, 2020. NDA resubmitted. CRL announced September 20, 2021.
$349.7 million

XENE – Xenon Pharmaceuticals Inc.
XEN1101 (X-TOLE)
Adult Focal Epilepsy

$30.58
-0.05  0%
Phase 2 Phase 2b trial met its primary efficacy endpoint demonstrating a statistically significant and dose-dependent reduction from baseline in monthly focal seizure frequency when compared to placebo. Additional primary and secondary seizure reduction endpoints statistically significant across all dose groups, noted October 4, 2021.
$1.6 billion