BPC June 18 update

Biotech stock events left on the calendar for June; Biotech week in review

Weekly watchlist

Just two weeks remain in the first half of 2021, yet a number of catalysts still remain scheduled for June. We continue to highlight readouts still left on the calendar, with a caveat that it is not uncommon for companies to miss timeline guidance.

First, let’s review the week that was with price-moving clinical data readouts and FDA decisions.

REGULATORY:

Avenue Therapeutics, Inc. (NASDAQ: ATXI) announced the FDA issued a Complete Response Letter (CRL) regarding the New Drug Application (NDA) for IV tramadol. Shares closed the week down 43% to $2.41 while Fortress Biotech, Inc. (NASDAQ:FBIO), which founded Avenue, saw its shares close down 22% to $3.50.

Orphazyme A/S (NASDAQ: ORPH) also announced it received a CRL from the FDA for its NDA for arimoclomol, intended for the treatment of Niemann-Pick disease type C (NPC). Shares fell Friday 50% to $7.33, while CytRx Corporation (CYTR), which would have been eligible for milestone payments upon approval, saw its shares fall 62% to $1.08.

CLINICAL DATA:

Clearside Biomedical, Inc. (NASDAQ:CLSD) shares closed the week up 61% to $4.84 following the release of data from Cohort 1 of its Phase 1/2a open-label clinical trial of CLS-AX (axitinib injectable suspension) in six patients with neovascular age-related macular degeneration (wet AMD). One month after receiving CLS-AX, five of six patients exhibited improvement in the best corrected visual acuity (BCVA) score, each gaining four or more letters, with mean score of all patients increasing by +4.7 letters (p=0.029).

Sage Therapeutics, Inc. (Nasdaq: SAGE) and Biogen Inc. (Nasdaq: BIIB) announced that the WATERFALL Study in patients with major depressive disorder met its primary endpoint with zuranolone (SAGE-217/BIIB125) 50 mg showing statistically significant improvement in depressive symptoms compared with placebo at Day 15. However, questions remained regarding its durability. Shares of Sage closed the week down 28% to $56.88.

RAPT Therapeutics, Inc. (Nasdaq: RAPT) shares surged to close the week up 86% to $34.57 following the release of positive topline results from its Phase 1b clinical trial of RPT193 as monotherapy in patients with moderate-to-severe atopic dermatitis (AD). After four weeks of treatment, patients with moderate-to-severe AD showed a 36.3% improvement from baseline in the Eczema Area and Severity Index (EASI) score, a standard measure of disease severity, compared to 17.0% in the placebo group.

CureVac N.V. (Nasdaq: CVAC) announced data from its Phase 2b/3 trial of its COVID-19 vaccine candidate, CVnCoV, demonstrated an interim vaccine efficacy of only 47% against COVID-19 disease of any severity and did not meet the prespecified statistical success criteria. Shares closed the week 37% lower at $61.94.

Novavax, Inc. (Nasdaq: NVAX) announced much-awaited Phase 3 data of their COVID-19 vaccine, NVX-CoV2373. The trial met its primary endpoint, exhibiting 100% protection against moderate and severe disease, 90.4% efficacy overall. 73 cases in total were seen, 63 in the placebo group and 14 in the vaccine group. All cases observed in the vaccine group were mild as defined by the trial protocol, with all hospitalizations/deaths occurring in the placebo group. Against variants of concern/interest, which represented 82% of the cases, vaccine efficacy was 93.2%. Shares, however, closed the week down 17% to $174.41.

Biotech stock events left on the calendar for June:

Drug Price Stage Catalyst Market Cap

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

$2.69
-0.07  -3%
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.
$262.4 million

ALT – Altimmune Inc.
AdCOVID
COVID-19 vaccine

$9.07
+0.01  +0%
Phase 1 Phase 1 data released June 29, 2021 did not stimulate an adequate immune response. Development to be discontinued.
$348.3 million

CORT – Corcept Therapeutics Incorporated
Relacorilant plus ABRAXANE (nab-paclitaxel) - (RELIANT)
Pancreatic cancer

$20.77
+0.48  +2%
Phase 3 Phase 3 initial data released June 22, 2021. 2/31 (6%) partial responses. Enrollment to be stopped. Level of benefit does not justify further study.
$2.4 billion

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

$1.37
-0.03  -2%
Phase 3 Phase 3 trial did not meet primary endpoint - June 24, 2021.
$171.3 million

DARE – Dare Bioscience Inc.
DARE-HRT1
Hormone replacement therapy

$1.54
+0.02  +1%
Phase 1 Phase 1 data released June 28, 2021 - successfully delivered estradiol and the progesterone over the 28-day evaluation period.
$76 million

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

$4.07
-0.31  -7%
Phase 2a Phase 2a data released June 22, 2021. GA progression at three months and six months in the study eye compared to fellow eyes was statistically indistinguishable.
$175.2 million

HEPA – Hepion Pharmaceuticals Inc.
CRV431 - AMBITION
Non-alcoholic steatohepatitis

$1.54
0.00  0%
Phase 2a Phase 2a data released July 13, 2021. Primary endpoints met.
$117.4 million

KRTX – Karuna Therapeutics Inc.
KarXT
Healthy elderly volunteers / dementia-related psychosis

$114.22
+0.81  +1%
Phase 1b Phase 2 trial to commence 1H 2022.
$3.4 billion

MDWD – MediWound Ltd.
NexoBrid
Severe burns

$3.80
-0.28  -7%
CRL CRL announced June 29, 2021.
$103.5 million

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

$141.85
+4.83  +4%
Phase 1 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.
$10.3 billion

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

$29.01
-0.15  -1%
Phase 3 Phase 3 top-line data released June 22, 2021. FIX levels sustained at a mean of 41.5% at 52 weeks. 78-week follow-up visits to be completed 3Q 2021 at which point the non-inferiority analysis of annualized bleeding rates (ABR) for the primary endpoint will be conducted. BLA filing due 1Q 2022.
$1.3 billion

RCKT – Rocket Pharmaceuticals Inc.
RP-L102
Fanconi Anemia (FA)

$35.82
-2.22  -6%
Phase 2 Phase 1/2 further data due 3Q 2021.
$2.3 billion

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

$31.41
-1.56  -5%
Phase 2 Phase 2 interim analysis noted "encouraging clinical activity" - no specific data released - June 23, 2021. Results will be submitted later this year for presentation at a medical meeting
$2.2 billion

TARS – Tarsus Pharmaceuticals Inc.
TP-03 - Saturn-1
Demodex Blepharitis

$22.57
-0.20  -1%
Phase 2/3 Phase 2b/3 top-line data released June 21, 2021. Endpoints met.
$463.2 million