BPC June 10 update

Bluebird BLUE +31% AH Second AdCom Green Light; Cogent COGT +59% EHA Phase 2 Data

Price and Volume Movers

Bluebird Bio, Inc. (NASDAQ:BLUE) announced that its second item for review by the FDA’s Advisory committee meeting received positive feedback. The committee voted 13-0 in favor of approving betibeglogene autotemcel (beti-cel) for the treatment of people with beta-thalassemia who require regular red blood cell (RBC) transfusions. Shares are trading up 32% at $4.94% during after hours.

Cogent Biosciences, Inc. (NASDAQ: COGT) presented at the European Hematology Association (EHA) Congress. The Phase 2 APEX trial of bezuclastinib to treat advanced systemic mastocytosis (AdvSM) had 100% of patients achieving ≥50% reduction in serum tryptase levels. Shares closed up 59% at $7.87.

Caribou Biosciences (NASDAQ: CRBU) shares closed down 20% at $6.94 after it released EHA data from its Phase 1 ANTLER trial of CB-010 to treat relapsed or refractory B cell non-Hodgkin lymphoma. 100% complete response (CR) rate was reported after the first dose, however, after 6 months it dropped to a 40% CR rate.

Fulcrum Therapeutics, Inc (NASDAQ: FULC) shares closed down 39% at $4.92 after it reported data from its Phase 1b trial of FTX-6058 to treat sickle cell disease (SCD). Initial data reported HbF increases within 5-10% range, however 3 of the 6 patients were not included in evaluation due to being non-adherent or discontinued due to protocol deviation.

Cyclerion Therapeutics, Inc. (NASDAQ: CYCN) released topline data from its Phase 2 MELAS trial of CY6463 noting that treatment was well tolerated with no adverse events leading to treatment discontinuation, and improvements in biomarkers associated with mitochondrial function including lactate and GDF-15 were noted. Shares closed down 20% at $0.66.

Advancers

CompanyPriceChange
OMER
Omeros Corporation
$1.01 +1.01  +100%
CLVS
Clovis Oncology Inc.
$1.01 +1.01  +100%
MREO
Mereo BioPharma Group plc
$1.01 +1.01  +100%
BLTE
Belite Bio Inc
$1.01 +1.01  +100%
INDP
Indaptus Therapeutics Inc.
$1.01 +1.01  +100%
TYRA
Tyra Biosciences Inc.
$1.01 +1.01  +100%
BLCM
Bellicum Pharmaceuticals Inc.
$1.01 +1.01  +100%
CRTX
Cortexyme Inc.
$1.01 +1.01  +100%
AUPH
Aurinia Pharmaceuticals Inc
$1.01 +1.01  +100%
ELYM
Eliem Therapeutics Inc
$1.01 +1.01  +100%

Decliners

CompanyPriceChange
TALS
Talaris Therapeutics Inc.
$1.01 -1.01  -100%
AKUS
Akouos Inc.
$1.01 -1.01  -100%
FEMY
Femasys Inc.
$1.01 -1.01  -100%
CLDX
Celldex Therapeutics Inc.
$1.01 -1.01  -100%
PRVB
Provention Bio Inc.
$1.01 -1.01  -100%
EPIX
ESSA Pharma Inc.
$1.01 -1.01  -100%
ADIL
Adial Pharmaceuticals Inc
$1.01 -1.01  -100%
CDAK
Codiak BioSciences Inc.
$1.01 -1.01  -100%
LABD
Direxion Daily S&P Biotech Bear 3X Shares
$1.01 -1.01  -100%
PPBT
Purple Biotech Ltd.
$1.01 -1.01  -100%

Pipeline Database Updates

Drug Price Stage Catalyst Market Cap

ADCT – ADC Therapeutics SA
Camidanlumab tesirine - (Cami)
Hodgkin lymphoma (HL)

$8.54
+0.59  +7%
Phase 2 Phase 2 updated efficacy and safety data presented at EHA showed an overall response rate of 70.1% and complete response rate of 33.3% in heavily pretreated patients; median duration of response of 13.7 months, noted June 10, 2022. Meeting with FDA for pre-BLA meeting planned for 2H 2022.
$656 million

AGIO – Agios Pharmaceuticals Inc.
Mitapivat (AG-348) - (ACTIVATE)
Pyruvate Kinase Deficiency

$23.36
+1.19  +5%
Phase 3 Phase 3 long-term extension study showed that treatment improved markers of ineffective erythropoiesis and iron metabolism in adults with PK deficiency, regardless of transfusion status, noted December 14, 2021. Phase 3 new patient outcome reported that 28/78 (35.9%) of all patients achieved a normal hemoglobin level at least once during treatment, noted June 10, 2022.
$1.3 billion

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

$45.43
+0.21  +0%
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. Phase 3 results to be presented at EHA demonstrated that treatment resulted in meaningful improvements in quality of life for treatment-naïve patients and suggested the incidence of thrombosis was comparable to eculizumab, a C5 inhibitor, noted June 10, 2022.
$4.8 billion

AUTL – Autolus Therapeutics plc
AUTO4 - (LibrA T1)
T-cell lymphoma

$2.75
-0.08  -3%
Phase 1/2 Phase 1 data presented at EHA demonstrated a tolerable safety profile, with no patient experiencing any dose limiting toxicities, and no neurotoxicity/immune effector cell-associated neurotoxicity (ICANS) and no Grade 3 or higher infections. CRS was only seen at the highest dose level of 450 x 106 CAR T cells (Grade 3 in 1 patient; Grade 1-2 in 3 patients), 9 patients were evaluable for efficacy. At the highest dose level (3/3) patients dosed achieved a complete metabolic remission (CMR) at 1 month. 2 of these patients remain in ongoing CMR by PET-CT at Month 3 and 6 respectively, whilst the 3rd relapsed at 3 months, noted June 10, 2022.
$250 million

COGT – Cogent Biosciences Inc.
Bezuclastinib - (APEX)
Advanced Systemic Mastocytosis

$9.39
+0.37  +4%
Phase 2 Phase 2 initial data reported that 11/11 patients achieved ≥50% reduction in serum tryptase levels by central assessment, with an 89% median reduction in serum tryptase and six of these patients achieved reduction to <20 ng/mL, noted June 10, 2022.
$598.3 million

CRBU – Caribou Biosciences Inc.
CB-010 - (ANTLER)
B-Cell Non-Hodgkin Lymphoma

$5.34
-0.09  -2%
Phase 1 Phase 1 initial data reported a 100% (6/6) ORR with 40% (2/5) complete response (CR) rate at 6 months, achieved as best response following 1 dose at the initial dose level. First patient treated in the trial remained in CR at 12 months, based on initial safety profile and clinical activity, noted June 10, 2022. Phase 1 trial is currently enrolling patients at dose level 2. Additional data expected by year-end 2022.
$324.2 million

CYCN – Cyclerion Therapeutics Inc.
CY6463 - (MELAS)
Mitochondrial Encephalomyopathy, Lactic Acidosis and Stroke-like Episodes (MELAS)

$0.58
+0.03  +6%
Phase 2 Phase 2 clinical trial update reported that from its eight-patient, open-label study, improvements were noted across multiple biomarkers of mitochondrial function, inflammation, cerebral blood flow, and functional connectivity, noted June 10, 2022. Additional Phase 2 data reported that treatment was well tolerated, with no reports of serious adverse events (SAEs) or treatment discontinuation due to adverse events (AEs), and showed improvements across multiple mitochondrial disease-associated biomarkers, inflammatory biomarkers, cerebral blood flow, and functional connectivity between neural networks, noted June 28, 2022.
$25.2 million

FULC – Fulcrum Therapeutics Inc.
FTX-6058
Sickle Cell Disease

$4.92
+0.02  +0%
Phase 1b Phase 1b trial initiated, noted January 10, 2022. Phase 1b initial data reported a rapid and robust induction of HbF, and subjects achieved increases of up to 6.3% over baseline, noted June 10, 2022.
$201 million

IMGO – Imago BioSciences Inc.
Bomedemstat
Myelofibrosis

$14.15
+0.76  +6%
Phase 2 Phase 2 data presented at EHA demonstrated that 52% had reductions in mutant allele frequencies, including ASXL1, 85% of evaluable patients demonstrated improved or stable fibrosis scores and 90% of transfusion independent patients had stable or improved hemoglobin, noted June 10, 2022.
$477.4 million

ISEE – IVERIC bio Inc.
Zimura - (GATHER2)
Geographic atrophy

$9.40
-0.22  -2%
Phase 3 First Phase 3 trial additional data reported a decrease in GA growth observed with ACP 2 mg, noted February 11, 2022. Phase 3 GATHER1 data reported a 30% reduction was observed in OCT-measured GA growth with Zimura at 12 months, noted June 10, 2022. Phase 3 second trial top-line data due 3Q 2022.
$1.1 billion

KROS – Keros Therapeutics Inc.
KER-050
Myelodysplastic syndromes (MDS)

$28.09
+0.46  +2%
Phase 2 Phase 2 initial data released June 22, 2021. Phase 2 data reported that 50% of the evaluable patients achieved an overall erythroid response, noted December 13, 2021. Additional Phase 2 ascending dose data reported that 51.9% of the evaluable patients achieved an overall erythroid response, noted June 10, 2022.
$675.1 million

MEIP – MEI Pharma Inc.
Zandelisib (ME-401) - (TIDAL)
Relapsed/refractory Follicular Lymphoma (FL) Non Hodgkin Lymphoma

$0.58
-0.03  -4%
Phase 2 Phase 2 top-line data presented at ASCO 22 reported a 70.3% objective response rate and that 35.2% achieved a complete response. 87.5% of responses achieved in first two cycles of therapy, 75% of CRs in first four cycles, 9.9% of patients discontinued the trial due to drug related adverse events, follow-up time for response was 8.4 months, and 83% reported grade 3 AE of special interest in cycles 1-3, noted June 4, 2022.
$77.2 million

RHHBY – Roche Holding AG ADR
Polivy (polatuzumab vedotin) - (POLARIX)
Diffuse Large B-Cell Lymphoma

$42.01
+0.30  +1%
NDA Filing Phase 3 trial met primary endpoint - August 9, 2021. Phase 3 subgroup analysis reported that the 2-year PFS rate was 74.2% and grade 3-4 adverse events were 72.9%, noted May 26, 2022. Phase 3 subgroup analysis of people with previously untreated DLBCL reported significant improvement in PFS, noted June 10, 2022. Regulatory submission expected in 2022.
$269 billion