BPC September 04 update

Phase 1/2 clinical trial readouts to watch in September; Biotech week in Review

Weekly watchlist

Following on from last week’s list of Phase 3 events to watch, this week we turn our attention to notable Phase 1/2 key catalysts expected during September. We also add one Phase 3 catalyst from Myovant (MYOV) which was included late in last week’s list.

First let’s review the week that was with notable price-moving events in the healthcare sector.

BUYOUTS

Aimmune Therapeutics Inc. (NASDAQ:AIMT) shares closed Monday up 172% to $34.22 following news that it will be acquired by Nestlé for $34.50 per share. Aimmune received FDA approval for the first treatment for peanut allergy earlier this year in January.

CLINICAL DATA

Akebia Therapeutics, Inc. (Nasdaq: AKBA) shares plunged to close Thursday down 74% to $2.65 on news the Phase 3 PRO2TECT trial, its cardiovascular trial evaluating vadadustat for the treatment of anemia due to chronic kidney disease (CKD), did not meet the primary safety endpoint.

Acasti Pharma Inc. (NASDAQ:ACST) announced Monday that its second Phase 3 trial, Trilogy 2, evaluating the efficacy of CaPre in patients with severe hypertriglyceridemia, did not meet the primary endpoint (p=0.19) of a statistically significant reduction in triglyceride levels. Shares closed Monday down 65% to $0.24

DEALS

Gilead Sciences, Inc. (NASDAQ:GILD) announced an agreement with Jounce Therapeutics, Inc. (NASDAQ:JNCE) to license JTX-1811, a monoclonal antibody designed to deplete immunosuppressive tumor-infiltrating T regulatory (TITR) cells. Gilead will make a $85m upfront payment and a $35m equity investment. Jounce is also eligible for up to an additional $685m in milestone payments. Jounce shares closed Tuesday up 56% to $7.53.

Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) announced Monday that it will acquire all of the outstanding shares of Akcea Therapeutics, Inc. (NASDAQ:AKCA) it does not already own, approximately 24%, for $18.15 per share in cash. Akcea shares closed Monday up 61% to $18.28.

LEGAL

Amarin Corporation plc (NASDAQ:AMRN) announced the U.S. Court of Appeals upheld a previous court ruling that its patents for its heart drug Vascepa were invalid, allowing drugmakers to move ahead with their generic versions. Shares closed the week down 42% to $4.30.


Phase 1/2 events to watch in September (also includes MYOV survival data which was added late to last week’s Phase 3 list).

Drug Stage Catalyst Market Cap

AGTC – Applied Genetic Technologies Corporation
AGTC-501
X-linked Retinitis Pigmentosa (XLRP)

Phase 1/2 Phase 1/2 12-month data due 4Q 2020.
$141.6 million

ASND – Ascendis Pharma A/S
TransCon
Hypoparathyroidism

Phase 2 Phase 2 six-month data from the open-label extension portion due 3Q 2020. Phase 3 trial to commence 4Q 2020.
$7.8 billion

BCRX – BioCryst Pharmaceuticals Inc.
Galidesivir
COVID-19

Phase 1 Information from Part 1 of the trial are due 3Q 2020.
$708 million

CYCN – Cyclerion Therapeutics Inc.
Olinciguat (IW-1701) - STRONG-SCD
Sickle Cell disease

Phase 2 Phase 2 data due late-3Q 2020.
$284.6 million

DVAX – Dynavax Technologies Corporation
SCB-2019 + adjuvant system (Clover)
COVID-19 vaccine

Phase 1 Phase 1 initiation announced June 19, 2020 with preliminary safety and immunogenicity data due September 2020.
$594.6 million

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

Phase 1/2 Phase 1/2 update due 3Q/4Q 2020.
$521.1 million

IMGN – ImmunoGen Inc.
Mirvetuximab soravtansine - FORWARD II
Ovarian cancer and relapsed endometrial cancer

Phase 1/2 Phase 1b/2 data from platinum-sensitive triplet cohort at ESMO September 2020 noted overall response rate (ORR) of 83%, with a median duration of response (DOR) of 10.9 months and median progression free survival (PFS) of 12.8 months.
$710.4 million

JNJ – Johnson & Johnson
Ad26.COV2-S
COVID-19 vaccine

Phase 1/2 Phase 1/2 trial has been initiated. Initial Phase I data due second half of September, with a Phase 3 trial to start mid to end September.
$392.8 billion

MRNA – Moderna Inc.
mRNA-1273
COVID-19 vaccine

Phase 2/3 Phase 2 interim efficacy data due 3Q 2020. Phase 3 data likely due November 2020.
$27.6 billion

MRSN – Mersana Therapeutics Inc.
XMT-1536
Ovarian Cancer, Non Small Cell Lung Cancer

Phase 1 Phase 1 interim update September 17, 2020 noted 34% objective response rate and 79% disease control rate.
$1.5 billion

MYOV – Myovant Sciences Ltd.
Relugolix
Advanced prostate cancer

Phase 3 PDUFA date under priority review December 20, 2020. Castration Resistance Free Survival data due 3Q 2020.
$2 billion

PRTA – Prothena Corporation plc
PRX002 (PASADENA)
Parkinson's disease

Phase 2 Phase 2 (Part 1) trial did not meet primary endpoint - April 22, 2020. Data presented September 11, 2020.
$425.5 million

SELB – Selecta Biosciences Inc.
SEL-212 (COMPARE) - Head to head trial vs Krystexxa
Tophaceous gout

Phase 2 Phase 2 top-line data due 3Q 2020.
$278.6 million