BPC October 01 update

Key Phase 3 catalysts for the quarter; Biotech week in review

Weekly watchlist

With the third quarter of 2021 coming to a close, we now look forward to a fresh batch of catalysts for the new quarter. Over the next few weeks, we highlight catalysts commencing with notable Phase 3 data readouts this week, followed by Phase 1/2 catalysts the week after.

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

REGULATORY UPDATES:

Omeros Corporation (NASDAQ: OMER) announced the FDA identified deficiencies in its Biologics License Application (BLA) for narsoplimab to treat hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA). Omeros does not expect resolution to occur before its set PDUFA date of October 17, 2021, however FDA stated that the notification does not reflect a final decision. Shares closed the week down 46% at $8.54.

CLINICAL UPDATES:

Merck (NYSE: MRK) released interim data from its Phase 3 MOVe-OUT trial of oral molnupiravir to treat COVID-19. Merck reported molnupiravir reduced the risk of hospitalization or death by approximately 50%. Shares closed the week up 11% at $81.40.

Editas Medicine, Inc. (NASDAQ: EDIT) released data from its Phase 1/2 BRILLIANCE trial of EDIT-101 to treat Leber congenital amaurosis type 10. Initial clinical data displayed two (66%) subjects in the mid-dose cohort showed improvements in BCVA, FST, and mobility navigation, while the low dose cohort (2) was ineffective. Shares closed the week down 32% at $39.91.

OTHER

Acceleron Pharma Inc. (NASDAQ: XLRN) announced its acquisition by a subsidiary of Merck for a total value of $11.5 billion. Acceleron’s lead drug candidate, Sotatercept to treat pulmonary arterial hypertension (PAH), is currently in a Phase 3 trial.

Keros Therapeutics Inc. (NASDAQ: KROS) traded off rumors of the Acceleron acquisition due to its preclinical candidate to treat PAH. Keros shares gained as much as 25% Monday and closed the week up 11% at $39.91.

Drug Price Stage Catalyst Market Cap

AGLE – Aeglea BioTherapeutics Inc.
Pegzilarginase (AEB1102) - PEACE
Arginase I deficiency

$7.30
+0.01  +0%
Phase 3 Phase 3 data due 4Q 2021.
$358.2 million

ALDX – Aldeyra Therapeutics Inc.
Reproxalap (ADX-102) - (TRANQUILITY)
Dry eye syndrome

$7.89
-0.25  -3%
Phase 3 Phase 3 data due 4Q 2021.
$456.5 million

ALLK – Allakos Inc.
Lirentelimab (ENIGMA 2)
Eosinophilic Gastritis and/or eosinophilic duodenitis (EoD)

$107.52
+3.90  +4%
Phase 3 Phase 3 top-line data due 4Q 2021.
$5.8 billion

ANGN – Angion Biomedica Corp.
ANG-3777
Acute kidney injury (AKI) / Delayed Graft Function

$8.55
-0.14  -2%
Phase 3 Phase 3 top-line data early-4Q 2021.
$254.8 million

AVIR – Atea Pharmaceuticals Inc.
AT-527 (MORNINGSKY)
COVID-19

$12.57
-0.18  -1%
Phase 3 Phase 3 data due 2H 2022.
$1 billion

BBIO – BridgeBio Pharma Inc.
Acoramidis (AG10) - ATTRibute-CM
ATTR-CM

$46.54
+0.41  +1%
Phase 3 Phase 3 top-line Part A data due late-2021. Part B data due 2023. NDA filing expected in 2022.
$7 billion

CRTX – Cortexyme Inc.
Atuzaginstat - GAIN
Alzheimer’s disease

$59.50
+1.65  +3%
Phase 2/3 Phase 2/3 top-line data to be announced in advance of its data presentation at CTAD November 9-12, 2021. Results from Phase 2 portion of trial to be announced in mid-November. Open label extension trial has been stopped following partial clinical hold - noted February 15, 2021.
$1.8 billion

DCPH – Deciphera Pharmaceuticals Inc.
QINLOCK (ripretinib) - INTRIGUE
Gastrointestinal Stromal Tumors (GIST) - second-line

$34.02
-0.03  0%
Phase 3 Phase 3 top-line data due 4Q 2021.
$2 billion

ERYP – Erytech Pharma S.A.
GRASPA (Eryaspase) - (Trybeca-1)
Pancreatic cancer

$5.83
-0.09  -2%
Phase 3 Phase 3 final data due 4Q 2021.
$162.8 million

IMGN – ImmunoGen Inc.
Mirvetuximab - SORAYA
Ovarian cancer

$5.85
+0.02  +0%
Phase 3 Phase 3 trial data due 4Q 2021.
$1.2 billion

MDGL – Madrigal Pharmaceuticals Inc.
Resmetirom (MGL-3196) - MAESTRO-NAFLD-1
Non-Alcoholic Fatty Liver Disease

$77.39
-0.02  0%
Phase 3 Phase 3 52-week top-line data due by end of 2021.
$1.3 billion

PHAS – PhaseBio Pharmaceuticals Inc.
Bentracimab (PB2452) - (REVERSE-IT)
Reversal agent for ticagrelor

$4.00
+0.03  +1%
Phase 3 Phase 3 completion of enrollment announced August 12, 2021. Phase 3 results to be presented at AHA Scientific Sessions November 15, 2021. BLA to be filed in mid-2022.
$191.9 million

PHAT – Phathom Pharmaceuticals Inc.
Vonoprazan PHALCON-EE
Erosive esophagitis / Gastroesophageal reflux disease (GERD)

$29.28
-0.22  -1%
NDA Filing NDA expected by the end of 1H 2022.
$918.1 million

RFL – Rafael Holdings Inc. Class B
CPI-613 (devimistat) + mFOLFIRINOX - (AVENGER 500)
Pancreatic Cancer

$30.80
-0.51  -2%
Phase 3 Phase 3 data readout due 4Q 2021, NDA filing due 1H 2022.
$636.3 million

SWTX – SpringWorks Therapeutics Inc.
Nirogacestat (DeFi)
Desmoid tumors

$60.34
+3.18  +6%
Phase 3 Phase 3 top-line data due 2H 2021.
$3 billion