BPC December 17 update

​Adamas ADMS and Proteostasis PTI shares fall on respective data releases

Price and Volume Movers

Adamas Pharmaceuticals, Inc. (Nasdaq: ADMS) announced that its Phase 3 trial of ADS-5102 in multiple sclerosis (MS) patients with walking impairment met its primary endpoint at the high dose but missed at the lower dose. ADS-5102 did not demonstrate a significant effect on the secondary walking measures at either dose. As a result of the data it will not initiate the originally planned replicate second Phase 3 placebo-controlled trial. Shares closed down 43% to $4.14.

Proteostasis Therapeutics, Inc. (NASDAQ: PTI) shares slid to close down 64% to $1.58 following the release of data from its Phase 2 trial in F508del homozygous and heterozygous cystic fibrosis patients. The company released only a subset of data and noted that mean changes in ppFEV1, a measure of lung function, were not statistically significant in the heterozygous population.

Achieve Life Sciences, Inc. (NASDAQ:ACHV) shares closed down 36% to $0.4875 following the pricing of its public offering of shares and attached warrants at a price of $0.60 per share and warrant for gross proceeds of $12m.

Homology Medicines, Inc. (Nasdaq: FIXX) announced initial clinical data from the first gene therapy clinical trial in phenylketonuria (PKU). As of December 2, 2019, two patients had received HMI-102 gene therapy in Cohort 1 (low-dose) and one patient in Cohort 2 (mid-dose). The patient dosed on Cohort 2 saw Phe (phenylalanine) levels, an amino acid in PKU, decline by 48% after four weeks from baseline. Tyrosine levels rose by 85%. The two patients in the low dose cohort did not see a reduction in Phe. Shares are trading down 22% to $17.00 after hours in reaction to the data.

Aduro Biotech, Inc. (NASDAQ: ADRO) shares are trading down 10% after hours following a SEC filing which noted its partner Novartis has removed ADU-S100 from its portfolio and enrolment will be discontinued. Novartis is also terminating enrollment of the ongoing Phase 1 trial evaluating ADU-S100 in relapsed and refractory melanoma in combination with Yervoy (ipilimumab).

Capricor Therapeutics, Inc. (NASDAQ:CAPR) announced a securities purchase agreement with a single institutional investor for the purchase of 4,078,304 shares at a purchase price of $1.226 per share and associated warrants for gross proceeds of $5.1m. Shares, which closed normal trading down 5%, are trading up 10% after hours to $1.14.

Neoleukin Therapeutics, Inc. (Nasdaq:NLTX) announced that it intends to offer and sell $50m of shares of its common stock in an underwritten public offering. Shares are trading down 2% to $9.00 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:

Oncolytics Biotech Inc. (ONCY): $1.80; +34%.

Viela Bio, Inc. (VIE): $28.66; +20%.

NantKwest, Inc. (NK): $3.82; +20%.

Satsuma Pharmaceuticals, Inc. (STSA): $15.82; +19%.

Acasti Pharma Inc. (ACST): $2.40; +19%.

DECLINERS:

MEI Pharma, Inc. (MEIP): $1.69; -26%.

Adial Pharmaceuticals, Inc. (ADIL): $2.16; -15%.

Kala Pharmaceuticals, Inc. (KALA): $4.08; -13%.

ObsEva SA (OBSV): $2.88; -13%.

Iterum Therapeutics plc (ITRM): $2.36; -11%.

Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

ADMS – Adamas Pharmaceuticals Inc.
GOCOVRI (amantadine)
Multiple sclerosis (MS)

Phase 3 Phase 3 data met primary endpoint at one of two doses. Secondary endpoint in walking impairment not met. Second trial will not be initiated as a result of data - December 17, 2019.
$146.3 million

ADRO – Aduro Biotech Inc.
ADU-S100 and Spartalizumab
Solid tumors or lymphomas

Phase 1b Phase 1 trial to be discontinued - noted December 17, 2019.
$299.5 million

ADRO – Aduro Biotech Inc.
ADU-S100 and pembrolizumab
Squamous Cell Carcinoma of the Head and Neck (SCCHN)

Phase 2 Phase 2 data due 2H 2020.
$299.5 million

ALNY – Alnylam Pharmaceuticals Inc.
Lumasiran (ALN-GO1) ILLUMINATE-A
Primary Hyperoxaluria Type 1 (PH1)

NDA Filing Rolling NDA filing has been initiated - January 10, 2020. To be completed early-2020. Full data from Phase 3 trial to be presented at the OxalEurope International Congress on March 31, 2020 in Amsterdam.
$13.8 billion

AVDL – Avadel Pharmaceuticals plc
FT 218
Excessive daytime sleepiness and cataplexy in patients suffering from narcolepsy

Phase 3 Phase 3 data due 2Q 2020.
$399.8 million

BHVN – Biohaven Pharmaceutical Holding Company Ltd.
Vazegepant
Acute treatment of migraine

Phase 2/3 Phase 2/3 top-line data met primary endpoint - December 17, 2019.
$2.5 billion

DMAC – DiaMedica Therapeutics Inc.
DM199
Chronic Kidney Disease (African Americans)

Phase 2 Phase 2 initiation announced December 17, 2019 with interim analysis 1Q 2020.
$76.3 million

FIXX – Homology Medicines Inc.
HMI-102
Phenylketonuria (PKU)

Phase 1/2 Phase 1/2 initial data released December 17, 2019.
$752.1 million

IMGN – ImmunoGen Inc.
Mirvetuximab - SORAYA
Ovarian cancer

Phase 3 Phase 3 trial to commence enrolment 1Q 2020 with data due mid-2021.
$886.8 million

LLY – Eli Lilly and Company
Solanezumab
Inherited Alzheimer's disease

Phase 2/3 Phase 3 trial did not meet primary endpoint - February 10, 2020.
$132.1 billion

MESO – Mesoblast Limited
MPC-150-IM - Class 2/3
Heart failure

Phase 3 Phase 3 data due mid-2020.
$843.6 million

MRK – Merck & Company Inc. (new)
Keytruda KN-057
NMIBC Bladder cancer

Approved FDA Approval announced January 8, 2020.
$207.1 billion

PTI – Proteostasis Therapeutics Inc.
PTI-428 + PTI-801 + PTI-808
Cystic fibrosis

Phase 2 Phase 2 negative data released December 17, 2019. Mean changes in ppFEV1 were not statistically significant in the heterozygous population. Phase 3 trial to be initiated in 2020.
$98.7 million

SMMT – Summit Therapeutics plc
Ridinilazole
C. difficile infection (CDI)

Phase 3 Phase 3 data due 2H 2021.
$96.8 million