BPC June 11 update

Biotech stock events to watch for remainder of June; Biotech week in Review

Weekly watchlist

The controversial decision by the FDA to award accelerated approval to Biogen’s (BIIB) aducanumab in patients with Alzheimer’s disease, led headlines for the week. The call by the FDA was despite an expert panel overwhelmingly recommending against awarding approval. In light of the decision, 3 of the 11 experts on the panel have handed in their resignations. In order to verify the clinical benefit the FDA have instructed Biogen to conduct a further trial to confirm efficacy of the drug but the company has until August 2029 to complete the trial and 2030 to submit final data. The company noted that the annual cost at the maintenance dose (10 mg/kg) would be $56,000. Shares of Biogen closed the week up 39% to $396.64.

Other price-moving events are noted below, while our watchlists for the remaining weeks of June will focus on key catalysts still left on the calendar scheduled for this month.

REGULATORY:

Ocugen, Inc. (NASDAQ: OCGN) shares closed the week down 27% to $6.34 following news that it will no longer file a COVID-19 vaccine Emergency Use Authorization (EUA) for COVAXIN, partnered with Bharat Biotech. Instead, on the recommendation from the FDA, it will go through the longer and traditional approval process by submitting a Biologics License Application (BLA).

Lyra Therapeutics, Inc. (NASDAQ: LYRA) announced the FDA has agreed on the design of its Phase 3 trial of LYR-210, its pipeline candidate for the treatment of chronic rhinosinusitis (CRS). The single primary endpoint will evaluate improvement at week 24 using a composite score of three cardinal symptoms (3CS) of CRS: nasal blockage, nasal discharge, and facial pain. Initiation of the trial is due towards the end of 2021. Shares closed the week up 17% to $9.19.

CLINICAL DATA:

Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) closed Friday down 11% to $193.02 following the release of data from its much-awaited Phase 2 trial of VX-864 in people with alpha-1 antitrypsin deficiency (AATD). The company said the treatment effect is unlikely to translate into substantial clinical benefit. As a result, Vertex will not advance VX-864 into late-stage development.

Novan, Inc. (NASDAQ:NOVN) shares surged to close Friday up 62% to $14.68 following its announcement its Phase 3 B-SIMPLE4 trial of SB206, a topical antiviral gel, for the treatment of molluscum contagiosum, met the primary endpoint. The company noted no adverse effects with the treatment and intends to file a New Drug Application by 3Q 2022.

The European Hematology Association Virtual Congress (EHA) commenced Friday, with a number of stocks moving on updated data. Major movers were:

Protagonist Therapeutics, Inc. (PTGX): $39.64; +17%.

Autolus Therapeutics plc (AUTL): $7.73; +13%.

Curis, Inc. (CRIS): $8.01; -37%.

Aptose Biosciences Inc. (APTO): $3.76; -32%.

Drug Price Stage Catalyst Market Cap

ABUS – Arbutus Biopharma Corporation
AB-729
Hepatitis B (HBV)

$3.13
-0.06  -2%
Phase 1 Phase 1a/b data at EASL meeting June 26, 2021 - HBsAg declines below 100 IU/ml in 75% of treated subjects. Favorable safety and tolerability profile. Noted that HBsAg suppression at levels <100 IU/mL is maintained in some patients up to 20 weeks following the last dose of treatment, November 1, 2021. Phase 1a/b data noted suppression of HBsAg to levels <100 IU/mL were maintained up to 24 weeks off-treatment in 3 of 7 patients in cohort E and 1 of 3 patients in cohort F, noted November 10, 2021.
$427 million

ALT – Altimmune Inc.
AdCOVID
COVID-19 vaccine

$10.42
-0.01  0%
Phase 1 Phase 1 data released June 29, 2021 did not stimulate an adequate immune response. Development to be discontinued.
$414.1 million

CRBP – Corbus Pharmaceuticals Holdings Inc.
Lenabasum (Resunab) - DETERMINE
Dermatomyositis

$0.88
-0.03  -3%
Phase 3 Phase 3 trial did not meet primary endpoint - June 24, 2021. Working on gaining clarity from m FDA on potential path forward, noted November 12, 2021.
$110.7 million

GMTX – Gemini Therapeutics Inc.
GEM103 (ReGAtta)
Dry Age-related Macular Degeneration / Geographic Atrophy

$2.92
-0.18  -6%
Phase 2a Phase 2a data released June 22, 2021. Phase 2a 6-month data due by year-end 2021.
$125.9 million

HEPA – Hepion Pharmaceuticals Inc.
CRV431 (ASCEND-NASH)
Non-alcoholic steatohepatitis

$1.23
-0.06  -5%
Phase 2a Phase 2a data released July 13, 2021. Primary endpoints met. Further Phase 2a data presented on September 13, 2021. Pro-C3 and ALT reductions point to anti-inflammatory and antifibrotic effects of CRV431, PK-PD models successfully predict early reductions in Pro-C3 and ALT. Phase 2a data reported ALT decreased in 50%, 67%, and 87% of the subjects in the placebo, 75 mg, and 225 mg cohorts, respectively, noted November 15, 2021. Phase 2b activities initiated September 13, 2021.
$93.8 million

MDWD – MediWound Ltd.
NexoBrid
Severe burns

$2.59
-0.16  -6%
CRL CRL announced June 29, 2021.
$70.6 million

NTLA – Intellia Therapeutics Inc.
NTLA-2001
Transthyretin amyloidosis (ATTR)

$113.13
-2.39  -2%
Phase 1 Phase 1 interim data due 1Q 2022. Phase 1 initial data presented June 26, 2021 at Peripheral Nerve Society Annual Meeting. Mean reductions of 52% among the three patients in the low-dose group, and 87% among the three patients in the high dose group.
$8.4 billion

NVAX – Novavax Inc.
NVX-CoV2373
COVID-19 vaccine

$217.97
+17.90  +9%
BLA Filing BLA due in US by year-end 2021.
$16.5 billion

ORPH – Orphazyme A/S
Arimoclomol
Niemann-Pick disease Type C (NPC)

$3.30
-0.10  -3%
CRL CRL issued June 18, 2021. Type A meeting announced October 31, 2021, to discuss pathway to resubmission.
$115.3 million

QURE – uniQure N.V.
Etranacogene dezaparvovec (AMT-061) - HOPE-B
Hemophilia B

$28.82
-0.27  -1%
Phase 3 Phase 3 top-line data released June 22, 2021. FIX levels sustained at a mean of 41.5% at 52 weeks. Phase 3 top-line 78-week follow-up data expected in 4Q 2021. BLA filing due 1H 2022.
$1.3 billion

RCUS – Arcus Biosciences Inc.
Domvanalimab (AB154) + Zimberelimab (AB122) - (ARC-7)
Non-small cell lung cancer (NSCLC)

$46.36
-2.10  -4%
Phase 2 Phase 2 interim analysis noted "encouraging clinical activity" - no specific data released - June 23, 2021. Results will be submitted for presentation in 2022.
$3.3 billion

SAGE – Sage Therapeutics Inc.
Zuranolone (SAGE-217) - WATERFALL Study (MDD-301B)
Major depressive disorder (MDD)

$39.32
-0.69  -2%
NDA Filing Phase 3 study primary endpoint met at Day 15 but not significant at Day 42 - June 15 2021. New data showed improvement in measurements of overall quality of life, functioning and general well-being achieved at Day 15 and sustained at Day 42 - October 4, 2021. NDA filing 2H 2022 with rolling submission in early 2022.
$2.3 billion