BPC January 10 update

Key Phase 3 readouts slated for the first quarter of 2020; Biotech week in Review

Weekly watchlist

The focus for the upcoming week will be on the annual J.P.Morgan Healthcare Conference (January 13-16), regarded as the premier healthcare investor conference of the year. Three Advisory Committee meetings also feature, which were noted in last week’s regulatory watch list for the quarter.

This week we look ahead at key Phase 3 readouts slated for this quarter. We list companies that have specifically provided a first quarter timeline. Not included are readouts slated for the first half of 2020, which might include readouts in either the first or second quarter.

Next week we summarise upcoming key Phase 1 and 2 data readouts.

First, let’s review notable events from the week that was.

Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) announced that its Phase 3 Pegasus trial of pegcetacoplan (APL-2) in adults with paroxysmal nocturnal hemoglobinuria (PNH) met the primary efficacy endpoint, demonstrating superiority to eculizumab (Soliris) with a statistically significant improvement in adjusted means of 3.8 g/dL of hemoglobin at week 16 (p<0.0001). Shares closed the week up 22% to $38.43.

Applied Therapeutics Inc. (Nasdaq:APLT) shares closed the week up 36% to $40.56 following the release of top-line data from its pivotal Phase 2 ACTION-Galactosemia trial of AT-007, in adult Galactosemia patients. AT-007 treatment resulted in a statistically significant reduction in plasma galactitol vs placebo.

Applied Genetic Technologies Corporation (Nasdaq: AGTC) shares surged to close the week up 88% to $7.84 following the release of positive interim six-month data from its ongoing Phase 1/2 trial in X-linked retinitis pigmentosa (XLRP).

Blueprint Medicines Corporation (NASDAQ:BPMC) announced that the FDA approved Ayvakit (avapritinib) for the treatment of adults with unresectable or metastatic gastrointestinal stromal tumor (GIST) harboring a platelet-derived growth factor receptor alpha (PDGFRA) exon 18 mutation.

Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) announced positive data from its Phase 1/2 trial of DTX301, for the treatment of ornithine transcarbamylase (OTC) deficiency. The company noted up to six responders were evident across all nine patients dosed in the trial. Shares closed Friday up 23% to $53.96.

Portola Pharmaceuticals, Inc. (NASDAQ:PTLA) shares closed Friday down 40% to $14.76 following its report after hours on Thursday of Andexxa net revenues of $28m for the quarter, well down on expectations.

Nektar Therapeutics (Nasdaq:NKTR) shares closed Friday up 27% to $26.92. The company announced a revision to its agreement with Bristol-Myers Squibb Company (NYSE:BMY) regarding the development of bempegaldesleukin (bempeg) plus Opdivo (nivolumab). Development will be expanded from three ongoing trials in first-line metastatic melanoma, urothelial cancer and renal cell carcinoma (RCC) to include two additional trials in adjuvant melanoma and in muscle-invasive bladder cancer.

Sorrento Therapeutics, Inc. (NASDAQ: SRNE) shares closed Friday up 40% to $4.76 following its announcement it has received a non-binding proposal from a private equity fund to acquire a majority or all of the issued and outstanding shares for up to $7 per share.

Key Phase 3 readouts slated for 1Q 2020:

Drug Stage Catalyst Market Cap

ACST – Acasti Pharma Inc.
CaPre (TRILOGY 1 and 2)

Phase 3 Phase 3 TRILOGY 1 data did not meet primary endpoint - January 13, 2020. TRILOGY 2 data due on OR about August 31, 2020,
$65.6 million

AXSM – Axsome Therapeutics Inc.
Treatment resistant depression

Phase 3 Phase 3 data did not meet primary endpoint. Secondary endpoints met. Second Phase 3 trial to commence 3Q 2020.
$2.9 billion

BHVN – Biohaven Pharmaceutical Holding Company Ltd.
Nurtec (Rimegepant)
Migraine - preventative

sNDA Filing sNDA filing due 4Q 2020.
$3.6 billion

EXEL – Exelixis Inc.
CABOMETYX (cabozantinib) with Opdivo and Yervoy - CheckMate 9ER
First-line renal cell carcinoma

Phase 3 Phase 3 trial met PFS and OS endpoints - April 20, 2020.
$6.8 billion

Elafibranor - RESOLVE-IT
Non-alcoholic steatohepatitis (NASH)

Phase 3 Phase 3 data did not meet primary endpoint - May 11, 2020.
$194.6 million

ITRM – Iterum Therapeutics plc
Complicated urinary tract infections (cUTI)

Phase 3 Phase 3 data did not meet primary endpoint - June 1, 2020.
$19.1 million

KALA – Kala Pharmaceuticals Inc.
KPI‑121 0.25% STRIDE 3
Dry eye disease

Phase 3 Phase 3 data met primary endpoint - March 9, 2020.
$491.6 million

KPTI – Karyopharm Therapeutics Inc.
Multiple myeloma

PDUFA PDUFA date estimate March 19, 2021.
$1.1 billion

MIST – Milestone Pharmaceuticals Inc.
Etripamil - NODE-301
Paroxysmal supraventricular tachycardia (PSVT)

Phase 3 Phase 3 top-line data released March 23, 2020 did not meet primary endpoint.
$210.5 million

MNLO – Menlo Therapeutics Inc.
Pruritus associated with prurigo nodularis

Phase 3 Phase 3 data did not meet primary endpoint - April 6, 2020.
$270 million

SNGX – Soligenix Inc.
SGX301 (synthetic hypericin)
Cutaneous T-Cell Lymphoma (CTCL) cancer

Phase 3 Phase 3 top-line data met primary endpoint - March 19,2020. Results from Cycle 3 and the subsequent 6-month follow-up due 4Q 2020.
$58.8 million

TGTX – TG Therapeutics Inc.
TG-1101 and TGR-1202 - UNITY-CLL study
Chronic Lymphocytic Leukemia (CLL) and non-Hodgkin's Lymphoma (NHL)

Phase 3 Phase 3 PFS primary endpoint met. Trial to be stopped due to superior efficacy - May 5, 2020. Full data to be presented 2H 2020.
$2.7 billion