BPC July 15 update

Phase 1/2 biotech stock events to watch during 3Q 2021: Biotech week in Review

Weekly watchlist

We conclude our preview of biotech stock events to watch this quarter with notable Phase 1 and 2 trial readouts of interest. This follows from previous previews of regulatory catalysts (PDUFA and Advisory Committee meetings) and Phase 3 readouts due this quarter.

First, let’s review the week that was with price moving events that hit the wires.

REGULATORY:

FibroGen (NASDAQ:FGEN) shares closed Friday down 42% to $14.35 following news an FDA Advisory Committee delivered negative votes for its regulatory application for roxadustat for the treatment of anemia of chronic kidney disease. The committee voted 12-2 against recommending approval in patients on dialysis and 13-1 against approval for patients not on dialysis.

CLINICAL UPDATES:

Galapagos NV (NASDAQ:GLPG) shares closed down 13% to $57.23 following the release of negative data from two of three readouts late-Wednesday of its pipeline candidate GLPG3970. While promising signs were noted from its trial in patients with psoriasis, both rheumatoid arthritis and ulcerative colitis patients saw no differentiation from placebo in their respective trials. Shares closed the week down 14% to $50.30.

Celldex Therapeutics, Inc. (NASDAQ:CLDX) shares closed the week up 25% to $40.64. following the release of updated data the previous week from its Phase 1b trial of CDX-0159 in patients with antihistamine refractory cold urticaria and symptomatic dermographism. 18/19 (95%) experienced a complete response and 1/19 (5%) experienced a marked partial response.

CohBar, Inc. (NASDAQ:CWBR) announced the release of data from its Phase 1a/1b trial of CB4211 for the potential treatment of nonalcoholic steatohepatitis and obesity has been delayed. Initially slated for release during early-July 2021, the company now expects data to be available sometime prior to Labor Day (September 6, 2021). Shares dropped 28% for the week to close down at $1.05.

OTHER:

PLx Pharma Inc. (NASDAQ:PLXP) announced a deal with Walmart that will see its product VAZALORE, an approved liquid-filled aspirin capsule, available in over 4,500 stores across the United States in mid-August. Stock closed the week up 30% to $16.70.


Phase 1/2 events to watch during 3Q 2021:

Drug Price Stage Catalyst Market Cap

ALEC – Alector Inc.
AL001 (INFRONT-2)
Frontotemporal Dementia

$19.92
-1.36  -6%
Phase 2 Phase 2 12-month biomarker data reported a double from baseline in progranulin levels when measured in plasma or CSF. Changes in biomarkers suggest that AL001 restores normal lysosome and complement function in FTD-GRN patients, noted November 12, 2021.
$1.6 billion

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

$25.76
-0.10  0%
Phase 2a Phase 2a data showed improvements in cognition in AD patients by 4.4 points compared to placebo at 3.3. WAIS coding in AD patients show a statistically-significant 23% improvement and PD patients a statistically-significant 24% improvement compared with placebo, noted September 2021. Phase 2a of PD patients noted statistically significant improvements in WAIS coding scores, in the UPDRS 2, 3, 4, and in total MDS-UPDRS test compared to baseline, noted October 5, 2021. Additional Phase 2a data reported reduced neurotoxic proteins in both AD and PD patients, with decreased inflammation and improved axonal integrity and synaptic functions in both AD and PD patients, noted November 12, 2021. Phase 3 trials to be initiated 1Q 2022.
$208.7 million

APRE – Aprea Therapeutics Inc.
Eprenetapopt (APR-246) and Vidaza (azacitidine)
TP53 mutated myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML) - post transplant

$4.15
-0.15  -3%
Phase 2 Phase 2 additional data to be presented at ASH December 12, 2021. Phase 2 data released July 21, 2021. De58% relapse free survival at 1 year post-transplant; 79% overall survival at 1 year post-transplant. Partial clinical hold announced August 5, 2021.
$89.1 million

ASLN – ASLAN Pharmaceuticals Limited
ASLAN004
Atopic dermatitis

$1.53
+0.04  +3%
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.
$106.5 million

BCEL – Atreca Inc.
ATRC-101
Solid tumors

$4.34
-0.30  -6%
Phase 1b Phase 1b first-in-human data suggest a manageable safety profile, with no dose-limiting toxicities observed. Pharmacokinetics appear to be dose proportional. Enrollment in the Q2W monotherapy dose-escalation cohort and at the 30 mg/kg dose level Q3W is continuing, noted November 9, 2021. Phase 1b monotherapy data due in mid-2022, and pembrolizumab combination data in 3Q 2022.
$161.2 million

DRNA – Dicerna Pharmaceuticals Inc.
Nedosiran - PHYOX2
Primary hyperoxaluria (PH) - Type 1

$37.99
-0.01  0%
Phase 3 Phase 2 results reported that treatment showed a reduction from baseline in urinary oxalate (Uox) excretion compared to placebo. Additionally, a higher proportion of patients achieved and sustained normal or near-normal Uox at two or more consecutive visits after Day 90, noted November 4, 2021.
$3 billion

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

$32.37
-0.93  -3%
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. Dosing of mid-dose cohorts to be completed 1H 2022.
$2.2 billion

EVLO – Evelo Biosciences Inc.
EDP1815
Mild to moderate psoriasis

$8.51
-0.31  -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. Part b data of the trial will be available 1Q 2022.
$454.9 million

FATE – Fate Therapeutics Inc.
FT596
B-Cell Chronic Lymphoma, chronic lymphocytic leukemia

$52.52
-1.90  -3%
Phase 1 Phase 1 interim data released August 19, 2021. 10/14 patients (71%) achieved an objective response; 7/14 (50%) complete responses. Phase 1 updated clinical data to be presented December 13, 2021.
$5 billion

INFI – Infinity Pharmaceuticals Inc.
Eganelisib (IPI-549) + TECENTRIQ (atezolizumab) and ABRAXANE (nab-paclitaxel) and IPI-549 + TECENTRIQ (atezolizumab) and AVASTIN (Bevacizumab) - (MARIO-3)
Front-line triple negative breast cancer (TNBC); Renal cell cancer (RCC)

$2.18
-0.09  -4%
Phase 2 Phase 2 updated data released July 27, 2021. Overall response rate 55% in patients. PFS was extended as compared to benchmark data for atezolizumab and nab-paclitaxel alone, increasing from 7.2 months to 7.4 months. Updated safety and efficacy data will be presented at the San Antonio Breast Cancer Symposium (SABCS) Annual Meeting, December 7-10, 2021.
$194.1 million

INMB – INmune Bio Inc.
XPro (pegipanermin)
Alzheimer’s disease

$13.33
-0.23  -2%
Phase 2 Phase 2 trial planned with data due 2H 2023.
$237.9 million

MCRB – Seres Therapeutics Inc.
SER-287
Mild-to-Moderate Ulcerative Colitis

$9.35
-0.32  -3%
Phase 2b Phase 2b trial did not meet primary endpoint - July 22, 2021. Microbiome analyses due 2H 2021.
$858.8 million

SAVA – Cassava Sciences Inc.
Simufilam (PTI-125) - (RETHINK-ALZ)
Alzheimer’s disease

$55.96
-2.09  -4%
Phase 3 First Phase 3 trial safety and efficacy 52-week study initiated in 750 Patients with AD - October 6, 2021. Second Phase 3 trial initiated November 18, 2021.
$2.2 billion

TBPH – Theravance Biopharma Inc.
Izencitinib
Ulcerative Colitis

$8.01
-0.44  -5%
Phase 2b Phase 2b data did not meet primary endpoint - August 23, 2021.
$590.3 million

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

$27.44
-0.75  -3%
Phase 2b Phase 2b data to be presented at AES December 3-7, 2021. Phase 3 be to initiated 2H 2022.
$1.4 billion