BPC December 10 update

American Society of Hematology (ASH) catalysts; Biotech week in review

Weekly watchlist

This week we will highlight notable presentations from the American Society of Hematology (ASH) conference being held December 11-14, 2021. Following this watchlist, we will begin our countdown to the end of the quarter and focus on the remaining small and mid-cap catalysts remaining for the year.

First, however, let’s review the week that was with price-moving events.

REGULATORY UPDATES:

Reata Pharmaceuticals, Inc. (NASDAQ: RETA) shares closed the week down 60% at $29.32, after the FDA Advisory Committee voted unanimously to denounce that evidence demonstrated that bardoxolone is effective in slowing the progression of CKD in patients with Alport syndrome and that its benefits outweigh its risks. Reata’s application has a PDUFA date of February 25, 2022.

CLINICAL UPDATES:

Radius Health, Inc’s (NASDAQ: RDUS) Phase 3 wearABLe study of abaloparatide transdermal system (abalo-TDS) to treat postmenopausal women with osteoporosis did not meet its primary endpoint. Shares closed the week down 52% at $7.09.

Infinity Pharmaceuticals, Inc. (NASDAQ: INFI) presented updated data from the ongoing Phase 2 MARIO-3 study of eganelisib in combination to treat metastatic triple-negative breast cancer (TNBC). Safety and efficacy data reported 88.6% of patients achieved tumor reduction, with a 92.8% disease control rate, and median PFS improvement of 47%. Shares closed the week up 24% at $2.31.

Bolt Biotherapeutics, Inc. (NASDAQ: BOLT) released data from its Phase 1/2 study of BDC-1001 to treat HER2-expressing cancers. There was one report of a durable partial response (PR) and multiple reports of stable disease. Shares closed the week down 44% at $4.65.

GH Research PLC (NASDAQ: GHRS) announced that its Phase 2 trial of GH001 to treat depression met its primary endpoint with 87.5% of patients in remission at day 7 after dosing. Shares closed the week up 24% at $23.96.

OTHERS:

Vyne Therapeutics (NASDAQ: VYNE) Shares closed the week up 43% at $1.50 after HC Wainwright & Co announced a price target of $7.

Drug Price Stage Catalyst Market Cap

AGIO – Agios Pharmaceuticals Inc.
Mitapivat (AG-348) - ESTIMATE open label
Sickle Cell Disease

$29.80
-0.56  -2%
Phase 2 Phase 2 data demonstrated the potential to provide clinically meaningful outcomes for patients, including improvements in anemia, hemolysis and red blood cell sickling. No SAEs occurred, and all AEs were mild and mostly transient. One vaso-occlusive crisis (VOC) occurred without hospital admission and did not require dose reduction or discontinuation, noted December 13, 2021.
$1.6 billion

AGIO – Agios Pharmaceuticals Inc.
AG-946
Myelodysplastic syndrome (MDS)

$29.80
-0.56  -2%
Phase 2a Phase 1 data reported 6 early discontinuations unrelated to study treatment. 4 of 39 (10.3%) subjects experienced ≥ 1 treatment-emergent AE (TEAE); all TEAEs were assessed as mild (Grade [Gr] 1). In the multiple ascending oral doses 4 of 17 (23.5%) subjects experienced ≥ 1 TEAE; the majority of the TEAEs were mild (Gr 1), with 1 subject experiencing a serious AE (Gr 2) of exercise-induced rhabdomyolysis 14 days after last dose, noted December 13, 2021. Phase 2a study in adults with low- to intermediate-risk myelodysplastic syndrome (MDS) to initiate by year-end, noted January 10, 2021.
$1.6 billion

ALLO – Allogene Therapeutics Inc.
ALLO-501 - (ALPHA)
Non-Hodgkin lymphoma (NHL)

$11.36
-0.22  -2%
Phase 1 Phase 1 data at ASCO June 4, 2021. Overall Response Rate (ORR) of 75% and CR Rate of 50% across histologies in CAR T Naïve patients. Phase 1 data reported that 21 FL patients and 11 LBCL patients who were autologous CAR T naïve, achieved a complete response of 33% and 36%, respectively, noted December 13, 2021.
$1.6 billion

APLS – Apellis Pharmaceuticals Inc.
EMPAVELI (pegcetacoplan) - (PRINCE)
Paroxysmal Nocturnal Hemoglobinuria (PNH)

$35.46
-2.17  -6%
Phase 3 Phase 3 demonstrated clinically meaningful improvements in key markers of disease, noted November 4, 2021. Phase 3 results reported 46% of patients achieved hemoglobin normalization in the absence of transfusions vs. 0% for standard of care and 91% achieved transfusion avoidance vs. 6% for standard of care, noted December 13, 2021.
$3.5 billion

APTO – Aptose Biosciences Inc.
HM43239
Acute myeloid leukemia (AML)

$1.21
-0.03  -2%
Phase 1/2 Phase 1/2 data reported 3 of 8 (37.5%) patients achieved a durable composite complete response, noted December 13, 2021.
$107.6 million

ATNM – Actinium Pharmaceuticals Inc. (Delaware)
Iomab-B - (SIERRA)
Acute myeloid leukemia (AML)

$5.35
+0.04  +1%
Phase 3 Phase 3 data reported a 100% engraftment rate in the lomab-B crossover with 33% of patients reported a Grade 3 adverse event of Febrile neutropenia, noted December 13, 2021. Phase 3 top-line data in 3Q 2022.
$117.9 million

ATRA – Atara Biotherapeutics Inc.
Tabelecleucel (ATA 129)
Epstein-Barr virus (EBV-PTLD) after solid organ transplant (SOT)

$13.99
-0.32  -2%
BLA Filing Rolling BLA filing to be completed 2Q 2022.
$1.3 billion

CLLS – Cellectis S.A.
ALLO-715 - (UNIVERSAL)
Multiple myeloma

$6.62
-0.11  -2%
Phase 1/2 Additional Phase 1/2 data noted a 71% overall response rate (ORR) and that 46% of patients achieved a very good partial response or better (VGPR+) including 25% complete response or stringent complete response (CR/sCR), noted December 13, 2021.
$301 million

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

$6.62
-0.11  -2%
Phase 1 Phase 1 data reported a 44% overall response rate and complete response rate, noted December 13, 2021. Phase 2 trial planned for mid-2022.
$301 million

GERN – Geron Corporation
Imetelstat - (IMerge Study)
Myelodysplastic syndromes

$1.05
-0.03  -3%
Phase 2 Phase 3 enrollment completed, noted October 18, 2021. Phase 3 data due in early January 2023.
$338 million

GMAB – Genmab A/S
Epcoritamab
B-cell Lymphoma

$33.50
-0.89  -3%
Phase 3 Phase 3 trial to be initiated.
$21.9 billion

GMAB – Genmab A/S
EPCORE
Non-Hodgkin Lymphoma

$33.50
-0.89  -3%
Phase 1/2 Phase 1/2 data reported that 25% of patients reported Grade 4 Neutropenia, with a 73% CMR and a 27% PMR, noted December 14, 2021.
$21.9 billion

GMDA – Gamida Cell Ltd.
NiCord (omidubicel)
Hematologic Malignancies

$3.24
+0.28  +9%
Phase 3 Phase 3 data demonstrated rapid and sustained T cell and B cell recovery following transplantation with omidubicel in a subset of patients, noted December 11, 2021. Phase 3 data to be presented at the Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR Tandem Meetings (TCT) February 2-6, 2022. BLA filing due 1H 2022.
$192.1 million

KPTI – Karyopharm Therapeutics Inc.
Selinexor - (ESSENTIAL)
Myelofibrosis

$8.28
+0.55  +7%
Phase 2 Phase 2 data reported median duration of treatment was 36 weeks. In the nine patients who were on treatment for over 24 weeks, SVR of ≥25% and 35% occurred in four (44%) and three (33%) patients, respectively, noted November 4, 2021. Additional Phase 2 results reported that of the ten patients who were on treatment for at least 24 weeks, four (40%) patients achieved SVR of ≥35% and six (60%) patients achieved SVR of ≥25%, noted December 11, 2021.
$625.5 million

MOR – MorphoSys AG
Pelabresib (CPI-0610) - (MANIFEST)
Myelofibrosis

$7.90
-0.24  -3%
Phase 2 Phase 2 data reported that pelabresib significantly reduced plasma levels of several cytokines in RUX naïve or experienced patients, and treatment was generally well tolerated, noted December 13, 2021.
$1.1 billion

MOR – MorphoSys AG
MONJUVI (tafasitamab) with REVLIMID (lenalidomide) - (RE-MIND2)
Diffuse Large B-Cell Lymphoma

$7.90
-0.24  -3%
Phase 3 Phase 3 data reported that trial met primary endpoint, with overall survival (OS) observed for tafasitamab plus lenalidomide at 20.1 months. The objective response rate (ORR) was 63.6% with a CR at 39.4%, noted December 11, 2021.
$1.1 billion