BPC September 09 update

Acadia ACAD surges +63% on Phase 3 data; Neurotrope NTRP slides 77% on failed trial

Price and Volume Movers

ACADIA Pharmaceuticals Inc. (Nasdaq: ACAD) shares surged to close up 63% to $38.85 following news that its Phase 3 HARMONY trial evaluating pimavanserin for the treatment of dementia-related psychosis, met its primary endpoint, demonstrating a highly statistically significant longer time to relapse of psychosis with pimavanserin compared to placebo in a planned interim efficacy analysis. The trial will now be stopped early based on pre-specified stopping criteria requiring a one-sided p-value less than 0.0033 on the study’s primary endpoint.

Amgen (NASDAQ:AMGN) released data from its ongoing Phase 1 study evaluating AMG 510 in patients with previously treated KRAS G12C-mutated solid tumors. The additional follow-up in a larger group of patients with non-small cell lung cancer (NSCLC) continued to show anti-tumor activity with no dose-limiting toxicities, with 23 of the patients being evaluable for efficacy. A disease control rate of 100% was noted which included seven (54%) patients who achieved a partial response and six (46%) who achieved stable disease. Despite the promising data, shares closed down 2.6% to $202.34. Shares of potential competitor Mirati Therapeutics, Inc. (NASDAQ: MRTX) also fell, closing down 5% to $82.70.

Neurotrope, Inc. (NASDAQ: NTRP) shares slumped to close down 77% to $1.00 following its announcement its Phase 2 study of Bryostatin-1 in moderate to severe Alzheimer’s Disease did not achieve statistical significance on the primary endpoint.

Spero Therapeutics (NASDAQ:SPRO) shares closed up 11% to $11.10 following the initiation of coverage by Janney Montgomery Scott with a Buy rating and a price target of $27.

Translate Bio (Nasdaq: TBIO) announced after hours that it will discontinue the development of MRT5201 for OTC deficiency. The IND had previously been placed on clinical hold in January 2019. Shares are trading down 3% to $9.65 after hours.

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Major price movers (stocks priced > $1.00, volume > 50k):

Refer to the BioPharmCatalyst Biotech Stocks page for Top 10 Gainers/Losers/Unusual Volume

ADVANCERS:

Eyenovia, Inc. (EYEN): $4.17; +22%.

Paratek Pharmaceuticals, Inc. (PRTK): $4.60; +18%.

Ardelyx, Inc. (ARDX): $5.62; +15%.

Idera Pharmaceuticals, Inc. (IDRA): $3.14; +13%.

Acorda Therapeutics, Inc. (ACOR): $3.15; +12%.

DECLINERS:

Akero Therapeutics, Inc. (AKRO): $22.37; -14%.

ArQule, Inc. (ARQL): $8.10; -13%.

Cassava Sciences, Inc. (SAVA): $1.22; -13%.

Endo International plc (ENDP): $3.07; -12%.

Repligen Corporation (RGEN): $82.18; -12%.

Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

ACAD – ACADIA Pharmaceuticals Inc.
Pimavanserin - Harmony
Alzheimer’s disease psychosis

Phase 3 Phase 3 trial stopped early after meeting primary endpoint - September 9, 2019.
$6 billion

AGN – Allergan plc
ABICIPAR
Age-related macular degeneration (AMD)

PDUFA priority review BLA acceptance of filing announced September 9, 2019. Estimated PDUFA date July 9, 2020.
$54.5 billion

ALEC – Alector Inc.
AL001
Frontotemporal Dementia

Phase 1 Phase 2 trial initiation announced September 9, 2019.
$1.3 billion

ATNX – Athenex Inc.
Oraxol and CYRAMZA (Ramucirumab)
Gastric cancer

Phase 1b Phase 1b enrolment to be completed 4Q 2019.
$1.1 billion

AZN – Astrazeneca PLC
Imfinzi + tremelimumab (CASPIAN)
Small cell lung cancer (SCLC)

Phase 3 Phase 3 interim analysis noted primary endpoint met - June 27, 2019. Data presented at WCLC September 9, 2019 noted hazard ratio of 0.73.
$116.9 billion

CYCC – Cyclacel Pharmaceuticals Inc.
CYC065
Advanced cancers

Phase 1 Phase 1 (Part 2/3) data due 2020.
$6.8 million

KALV – KalVista Pharmaceuticals Inc.
KVD900
Hereditary Angioedema (HAE)

Phase 2 Phase 2 data due late 2019.
$261.2 million

KTOV – Kitov Pharma Ltd.
NT-219
Squamous cell carcinoma of the head & neck (SCCHN)

Phase 1 Phase 1/2 trial to be initiated 1Q 2020.
$15.9 million

NTRP – Neurotrope Inc.
Bryostatin
Alzheimer's disease

Phase 2 Phase 2 trial did not meet primary endpoint - September 9, 2019.
$12.1 million

SAVA – Cassava Sciences Inc.
PTI-125
Alzheimer’s disease

Phase 2 Phase 2b trial initiation announced September 16, 2019.
$22.2 million

TBIO – Translate Bio Inc.
MRT5201
OTC deficiency

Phase 1/2 Phase 1/2 IND placed on clinical hold - January 22, 2019. Development to be discontinued - noted September 9, 2019.
$520.3 million

TBPH – Theravance Biopharma Inc.
TD-8236
Asthma

Phase 1 Phase 1 data released September 9, 2019 - well tolerated.
$1.3 billion

XLRN – Acceleron Pharma Inc.
Sotatercept - PULSAR
Pulmonary arterial hypertension (PAH)

Phase 2 Phase 2 data due 1Q 2020.
$2.3 billion