BPC May 14 update

Biotech week in Review; Upcoming readouts and ASCO abstracts to watch

Weekly watchlist

With the first-quarter earnings season drawing to a close and only a few stragglers left to report on Monday, we now turn our focus towards near term biotech catalysts, including the upcoming ASCO oncology conference to be held June 4-8, 2021. Abstracts for the conference will be price moving and are scheduled for release on Wednesday May 19 at 5pm EST, some of which are noted below. First, let’s review the week that was.

REGULATORY:

Apellis Pharmaceuticals, Inc. (NASDAQ:APLS) received approval from the FDA late-Friday for Empaveli (pegcetacoplan) injection to treat adults with paroxysmal nocturnal hemoglobinuria (PNH), a rare, life-threatening blood disease. PNH is a rare disease that is characterized by red blood cell destruction, anemia and blood clots. Empaveli will be made available through a restricted program under a risk evaluation and mitigation strategy (REMS).

Heron Therapeutics, Inc. (Nasdaq: HRTX) also received FDA approval for ZYNRELEF (bupivacaine and meloxicam) for use in adults for soft tissue or periarticular instillation to produce postsurgical analgesia after bunionectomy, open inguinal herniorrhaphy and total knee arthroplasty. Shares, however, closed the week down 17% to $13.85 with investors selling off on the news.

CorMedix Inc. (NASDAQ:CRMD) shares closed Friday down 20% to $6.05 following its note in its fourth quarter earnings call that additional work is required to address the deficiencies in response to the Complete Response Letter issued by the FDA for Defencath earlier this year. It intends to resubmit the New Drug Application in the fourth quarter of 2021.

CLINICAL DATA:

Curis, Inc. (NASDAQ: CRIS) shares rallied to close the week up 32% to $13.57 following data released in its abstract for the European Hematology Association (EHA) meeting scheduled for June 9-17, 2021 from its Phase 1/2 trial of CA-4948 in patients with acute myeloid leukemia (AML) or high-risk myelodysplastic syndromes (MDS). The data showed bone marrow blast reductions observed at all tested doses in 8 of 9 (89%) evaluable patients. Objective responses observed included 1 patient experiencing a full hematologic recovery complete response, 1 CRi (Complete Remission with Incomplete Hematologic Recovery) and 2 bone marrow CRs.

Autolus Therapeutics plc (AUTL: NASDAQ) shares closed the week up 15% to $6.17, also on the back of data released in its EHA abstract for its Phase 1 cell therapy candidate, AUTO1. The data exhibited a 100% remission rate in patients with relapsed/refractory indolent non-Hodgkin lymphoma. As of the data cut-off date of February 18, 2021, 10 patients had received AUTO1 and nine patients were evaluable.

OTHER:

Novavax Inc. (NASDAQ: NYSE) shares fell 25% to close the week down at $131.69. The downward move followed a report from the Washington Post that the company is not expected to seek emergency use authorization for its Covid-19 vaccine until June at the earliest, with data not expected until late-May, later than had been expected by investors. Moreover, the company announced in its first-quarter earnings release a revised timeline to reach manufacturing capacity. It expects capacity of 100 million doses per month by the end of the third quarter of 2021 before ramping up to 150 million does per month during the fourth quarter. The company had previously guided that full capacity would be reached in mid-2021.

Near term catalysts to watch (Including ASCO abstract release scheduled for Wednesday May 19 at 5pm):

Drug Price Stage Catalyst Market Cap

ADAP – Adaptimmune Therapeutics plc
Afami-cel - (SPEARHEAD‑1)
Synovial sarcoma or myxoid/round cell liposarcoma (MRCLS)

$3.86
-0.04  -1%
Phase 2 Phase 2 enrolment has been completed. Initial data at ASCO June 4-8, 2021. Abstract noted overall response rate was 39.3% (13/33), 41.4% (12/29) for synovial sarcoma; 25.0% (1/4) for MRCLS.
$600.2 million

ALPN – Alpine Immune Sciences Inc.
ALPN-202 (NEON-1)
Advanced Malignancies

$9.05
-0.26  -3%
Phase 1 Phase 1 data presented at ASCO June 4, 2021. 61% (14 of 23) showed clinical benefit (stable disease or better).
$216.4 million

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

$84.25
-11.85  -12%
Phase 2 Phase 2 full data July / August 2021. Biomarker data released May 21, 2021.
$585.3 million

BIIB – Biogen Inc.
ADUHELM (Aducanumab)
Alzheimer’s disease

$374.40
-6.51  -2%
Approved FDA approval announced June 7, 2021.
$56.4 billion

CGEM – Cullinan Oncology Inc.
CLN-081
Non-Small Cell Lung Cancer

$24.96
-1.07  -4%
Phase 1/2 Phase 1/2 updated data at ASCO June 4-8, 2021 noted objective partial responses (PR) were observed in 21 of 42 (50%) patients.
$1.1 billion

CLLS – Cellectis S.A.
ALLO-501A (ALPHA2)
Large B-cell lymphoma

$14.61
+0.10  +1%
Phase 1 Phase 1 data at ASCO June 4, 2021 noted overall response rate 56% (5/9).
$628.8 million

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

$194.37
+13.38  +7%
Phase 3 Phase 3 U.S. trial data released June 14, 2021. 90.4% efficacy overall, 100% protection against moderate and severe disease.
$14.4 billion

ONCT – Oncternal Therapeutics Inc.
TK216
Relapsed or refractory Ewing sarcoma

$5.19
-0.24  -4%
Phase 1/2 Phase 1/2 interim data presented at ASCO June 4, 2021.
$256.2 million

PDSB – PDS Biotechnology Corporation
PDS0101 and Bintrafusp alfa (M7824)/ NHS-IL12
Human papillomavirus (HPV) associated cancers

$10.69
-0.55  -5%
Phase 2 Phase 2 presentation at ASCO June 7, 2021. Abstract showed overall objective response rate of 71%; 56% during ASCO presentation.
$303.2 million

PRVB – Provention Bio Inc.
Teplizumab (PRV-031)
Type 1 diabetes

$8.37
-0.21  -2%
PDUFA priority review Advisory Committee meeting May 27, 2021 voted 10-7 in favor that benefits outweigh the risks. PDUFA date under priority review July 2, 2021. Noted April 8, 2021 that FDA has identified deficiencies that preclude discussion of labeling and post-marketing requirements/commitments.
$530.4 million

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

$55.69
-1.75  -3%
Phase 3 Phase 3 data released June 15, 2021. Primary endpoint met at Day 15 but not significant at Day 42.
$3.3 billion

SCYX – SCYNEXIS Inc.
BREXAFEMME (ibrexafungerp)
Vulvovaginal candidiasis (VVC)

$7.22
+0.10  +1%
Approved FDA approval announced June 2, 2021.
$149 million

SEEL – Seelos Therapeutics Inc.
SLS-002
Acute Suicidal Ideation and Behavior in patients with Major Depressive Disorder (ASIB in MDD)

$2.86
-0.10  -3%
Phase 2 Phase 2 Part 1 data released May 17, 2021. 76.5% response rate in the primary endpoint on MADRS 24 hours after dosing. Mean reduction from 39.4 to 14.5 points. Part 2 double-blind and placebo-controlled trial to be initiated.
$292.4 million

VRTX – Vertex Pharmaceuticals Incorporated
VX-864
Alpha-1 antitrypsin (AAT) deficiency

$188.87
+1.17  +1%
Phase 2 Phase 2 data released June 10, 2021. Primary endpoint met but insufficient clinical benefit to advance program.
$48.9 billion