BPC June 13 update

Day One DAWN +125% Phase 2 success; Soleno SLNO +30% AH PWS data

Price and Volume Movers

Soleno Therapeutics, Inc. (NASDAQ: SLNO) presented clinical data of diazoxide choline extended-release (DCCR) to treat Prader-Willi syndrome (PWS) from a Phase 3 and open-label trial. Data reported statistically significant changes in lean body mass, ratio of lean body mass to fat mass, levels of leptin, fasting insulin and adiponectin. Shares are currently trading up 30% at $0.22 after hours.

Day One Biopharmaceuticals (NASDAQ: DAWN) shares closed up 125% at $14.96 after it released initial data from its Phase 2 FIREFLY-1 trial of tovorafenib to treat low-grade glioma. Data show an overall response rate (ORR) of 64% and clinical benefit rate (CBR) of 91% in the first 22 evaluable patients treated.

Hillstream BioPharma, Inc’s (NASDAQ: HILS) authorized a share repurchase program for up to $1 million of stock. Shares closed up 75% at $1.35.

Optinose (NASDAQ: OPTN) released data from its Phase 3 ReOpen2 trial of XHANCE to treat chronic sinusitis, noting that the trial met both co-primary endpoints. Shares closed up 41% at $2.61.

Cogent Biosciences, Inc. (NASDAQ: COGT) commenced a public offering of $125 million of shares of its stock. SVB Securities also set a $15 price target on Cogent shares and kept an Outperform rating. Shares closed up 11% at $8.74.

Mallinckrodt plc (OTC: MNKKQ) resubmitted its New Drug Application (NDA) for terlipressin to treat hepatorenal syndrome (HRS). Mallinckrodt has previously received two complete response letters (CRL) for its application. Shares closed down 75% at $0.02.

Valneva SE (NASDAQ: VALN) shares closed down 19% at $16.12 after it proposed a remediation plan for its advance purchase agreement with the European Commission (EC) for its COVID-19 vaccine candidate. The EC announced a notice of intent to terminate the deal on Friday.

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

ADGI – Adagio Therapeutics Inc.
ADG20 - (STAMP)
COVID-19 (treatment)

$3.50
+0.22  +7%
NDA Filing Phase 2/3 trial primary endpoints were met with statistical significance, noted March 30, 2022. FDA Emergency Use Authorization (EUA) submission planned.
$384.1 million

AKRO – Akero Therapeutics Inc.
Efruxifermin - (BALANCED)
Nonalcoholic steatohepatitis (NASH)

$10.15
+0.70  +7%
Phase 2a Phase 2a trial data demonstrated that most EFX-treated patients with end-of-treatment biopsies showed improvements in features of steatohepatitis (35 of 40; 87%) and/or fibrosis (32 of 40; 80%), after only 16 weeks. Histological improvements were evident across all types of patients, including those at higher risk of progressing to advanced stages of NASH, noted November 12, 2021. Phase 3 to be initiated in 2022. Phase 2a data from 58/80 patients with biopsy confirmed showed that treatment improved histopathology and noninvasive markers of liver injury across PNPLA3 genotypes, including in patients carrying the I148M variant, noted June 27, 2022.
$381.1 million

BBIO – BridgeBio Pharma Inc.
Encaleret
Autosomal Dominant Hypocalcemia Type 1 (ADH1)

$9.92
+0.84  +9%
Phase 3 Phase 2b treatment data presented at ENDO resulted in rapid and sustained restoration of normal mineral homeostasis, with mean values of blood calcium, urinary calcium, and blood PTH within the normal range by day 5 of therapy and sustained at 24 weeks, and was well-tolerated without any reported serious adverse events, noted June 13, 2022. Phase 3 trial planned to initiate in 2022, with top-line data due in 2023.
$1.5 billion

BGNE – BeiGene Ltd.
BRUKINSA (zanubrutinib) - (SEQUOIA)
First-line chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL)

$166.95
+5.10  +3%
PDUFA PDUFA date January 20, 2023.
$17.2 billion

DAWN – Day One Biopharmaceuticals Inc.
DAY101 - (FIREFLY-1)
Low-grade Glioma

$19.95
+2.05  +11%
Phase 2 Phase 2 initial data showed an overall response rate (ORR) of 64% and clinical benefit rate (CBR) of 91% in the first 22 evaluable patients treated with monotherapy, noted June 13, 2022. Phase 2 top-line results due in 1Q 2023. Phase 3 trial expected to commence in 3Q 2022. NDA expected to be filed in 2023.
$1.4 billion

EIGR – Eiger BioPharmaceuticals Inc.
Avexitide
Hyperinsulinemic hypoglycemia after bariatric or other gastrointestinal surgeries

$6.40
+0.10  +2%
Phase 2b Phase 2b data reported that trial met primary and secondary endpoints, noted June 13, 2022.
$276.6 million

FBIO – Fortress Biotech Inc.
MB-106
B-cell Non-Hodgkin Lymphoma & Chronic Lymphocytic Leukemia (CLL)

$0.80
-0.04  -5%
Phase 1/2 Phase 1/2 interim data presented at EHA meeting June 11, 2021. 93% overall response rate and 67% complete response rate. Additional Phase 1/2 data reported a 95% overall response rate, and 65% complete response rate, noted December 13, 2021. Phase 1/2 data showed very favorable safety profile suitable for outpatient treatment, high complete response rate and strong durability observed, dosing expected to initiate this quarter, noted April 25, 2022. Phase 1/2 data presented at EHA showed an 94% overall response rate and 78% complete response rate in patients with FL, including complete response in a patient previously treated. 100% overall response rate in other B-cell NHL, noted June 13, 2022.
$85.9 million

GRCL – Gracell Biotechnologies Inc.
TruUCAR-T (GC502)
B-Cell Lymphoma (B-ALL)

$5.10
-0.40  -7%
Phase 1 Phase 1 data reported that 3/4 patients achieved MRD- CR/CRi, noted June 13, 2022.
$344.8 million

KZIA – Kazia Therapeutics Limited
Paxalisib (PNOC022)
Diffuse intrinsic pontine glioma (DIPG) and diffuse midline gliomas (DMGs).

$4.19
-0.01  0%
Phase 2 Phase 2 study initiated, noted November 10, 2021. Phase 2 data due in 1H 2023.
$58.1 million

MRK – Merck & Company Inc.
KEYTRUDA (Pembrolizumab) - (KEYNOTE-091)
Stage IB, II or IIIA non-small cell lung cancer (NSCLC)

$92.42
+1.25  +1%
PDUFA Phase 3 trial results accepted to support sBLA submission, noted June 13, 2022. PDUFA date January 29, 2023.
$233.7 billion

NBIX – Neurocrine Biosciences Inc.
Crinecerfont - (adolescent open-label)
Classic congenital adrenal hyperplasia (CAH)

$99.25
+1.77  +2%
Phase 2 Phase 2 data reported that median percent reductions ranged from -57.1% to -76.2% and a greater than 50% reduction from baseline for ACTH, 17-OHP, and androstenedione (males and females), testosterone (females), and androstenedione/testosterone ratio (males) was achieved by 63%, 75%, 50%, 60%, and 67% of participants, respectively, noted June 13, 2022.
$9.5 billion

OPTN – OptiNose Inc.
XHANCE - (ReOpen2)
Chronic sinusitis

$3.64
-0.02  -1%
Phase 3 Phase 3 top-line data reported statistically significant benefits for both the symptoms co-primary endpoint and the CT scan co-primary endpoint, noted June 13, 2022.
$301 million

STSA – Satsuma Pharmaceuticals Inc.
STS101 - (ASCEND)
Migraine

$4.32
+0.18  +4%
Phase 3 Phase 3 initial data presented at AHA reported that when asked at the 3-month and 6-month assessments to compare STS101 to their usual migraine medication, patients reported that 68.9% and 75.5% of subjects agreed or strongly agreed that STS101 helped them return to normal faster than their usual medication, noted June 13, 2022. Phase 3 top-line data due in 2H 2022.
$136.3 million

VRTX – Vertex Pharmaceuticals Incorporated
CTX001 - (CLIMB-SCD-121)
Sickle cell disease

$287.32
+5.53  +2%
NDA Filing Data presented at EHA reported that all 31 patients with severe SCD were free of VOCs after exa-cel infusion through duration of follow-up, with follow-up ranging from 2.0 to 32.3 months, noted June 13, 2022. Regulatory submission planned for year-end 2022.
$73.5 billion