BPC August 13 update

Phase 3 data readouts left for 3Q 2021; Biotech week in Review

Weekly watchlist

With the second-quarter earnings season reaching its close this latest week with just a handful of companies set to report early next week. With to a close our focus turns back to clinical data events slated for release this quarter.

First let’s review the week that was with price-moving events.

REGULATORY:

Sesen Bio (Nasdaq: SESN) shares closed Friday down 57% to $2.11 on news that it received a Complete Response Letter (CRL) from the FDA regarding its Biologics License Application (BLA) for Vicineum for the treatment of BCG-unresponsive non-muscle invasive bladder cancer (NMIBC). The FDA noted clinical/statistical data and analyses in addition to Chemistry, Manufacturing and Controls (CMC) issues following a recent pre-approval inspection and product quality for the CRL.

Axsome Therapeutics, Inc. (NASDAQ: AXSM) announced that it received notice from the FDA that it has identified deficiencies that preclude discussion of labeling and post-marketing requirements/commitments of its New Drug Application (NDA) of AXS-05. The letter did not state the nature of the deficiencies. The assigned PDUFA date for the NDA is August 22, 2021. Shares closed the week down 55% to $22.82.

Reata Pharmaceuticals, Inc. (NASDAQ: RETA) shares closed the week down 14% to $106.55 following an update regarding its New Drug Application for bardoxolone for the treatment of patients with chronic kidney disease caused by Alport syndrome. Reata noted the FDA has identified four significant clinical and statistical review issues for the company to address. An Advisory Committee meeting is tentatively scheduled for December 8, 2021, ahead of its PDUFA date of February 25, 2022.

FibroGen, Inc. (NASDAQ: FGEN) announced that the FDA has issued a Complete Response Letter (CRL) regarding its NDA for roxadustat for the treatment of anemia of chronic kidney disease (CKD). The FDA has requested an additional clinical study of roxadustat be conducted, prior to resubmission. Shares closed the week down 10% to $12.42

CLINICAL UPDATES:

Cortexyme, Inc. (NASDAQ: CRTX) shares closed the week up 49% to $100.31 following its second-quarter earnings release which noted that data from its Phase 3 GAIN trial for disease modification in Alzheimer's is expected by mid-November 2021, narrowing its previous timeline guidance of data due 4Q 2021.

bluebird bio, Inc. (NASDAQ: BLUE) reported a Suspected Unexpected Serious Adverse Reaction (SUSAR) of myelodysplastic syndrome (MDS) in a patient who was treated with eli-cel (Lenti-D) in its Phase 3 ALD-104 trial for cerebral adrenoleukodystrophy (CALD). As a result the FDA has placed the eli-cel program on a clinical hold. The company also announced that it will wind down operations in Europe for its severe genetic disease business and instead focus on the U.S. market. Shares closed the week down 26% to $18.44.

Fulcrum Therapeutics, Inc. (NASDAQ:FULC) announced positive interim results of its Phase 1 trial of FTX-6058, its pipeline candidate for sickle cell disease. The data showed promising signs of benefit for patients with sickle cell disease, demonstrating proof of biology and proof of mechanism. Shares surged to close the week up 184% to $22.99.

Longeveron Inc. (NASDAQ: LGVN) announced data from its Phase 2b trial of Lomecel-B in patients with Aging Frailty. At Day 180 patients on the treatment did not show a statistically significant improvement in the 6MWT distance walk assessment compared with placebo. The company noted significant differences from placebo were observed at Day 270. However, this was not the primary endpoint of the trial. Shares closed Friday down 28% to $3.90.

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Phase 3 data readouts left for 3Q 2021. (Note: RDHL 3Q estimate only - no firm timeline guidance provided by company):

Drug Price Stage Catalyst Market Cap

APLS – Apellis Pharmaceuticals Inc.
EMPAVELI (pegcetacoplan) - DERBY
Geographic atrophy (GA)

$34.21
+0.21  +1%
Phase 3 Phase 3 data did not meet primary endpoints September 9, 2021.
$3 billion

APLS – Apellis Pharmaceuticals Inc.
EMPAVELI (pegcetacoplan) - OAKS
Geographic atrophy (GA) associated with age-related macular degeneration (AMD)

$34.21
+0.21  +1%
NDA Filing Phase 3 data met primary endpoint September 9, 2021. NDA expected to be filed in the first half of 2022.
$3 billion

BHVN – Biohaven Pharmaceutical Holding Company Ltd.
Verdiperstat
Multiple system atrophy (MSA)

$135.00
-1.93  -1%
Phase 3 Phase 3 top-line data due 3Q 2021.
$8.8 billion

CTIC – CTI BioPharma Corp.
Pacritinib (PRE-VENT)
COVID-19

$3.05
+0.02  +1%
Phase 3 Phase 3 interim data due 3Q 2021.
$284.6 million

DVAX – Dynavax Technologies Corporation
S-Trimer COVID-19 Vaccine + CpG 1018 adjuvant plus alum (SPECTRA)
COVID-19 vaccine

$14.89
+0.45  +3%
Phase 2/3 Phase 2/3 interim analysis due 3Q 2021.
$1.7 billion

NVCR – NovoCure Limited
Tumor Treating Fields (TTFields) - INNOVATE-3
Ovarian cancer

$131.03
+3.94  +3%
Phase 3 Phase 3 interim analysis 3Q 2021 with final data due 2023.
$13.6 billion

RDHL – Redhill Biopharma Ltd.
YELIVA (Opaganib)
COVID-19

$4.89
+0.07  +1%
Phase 2/3 Phase 2/3 preliminary top-line data results did not meet its primary endpoint September 14, 2021.
$228.5 million

TBPH – Theravance Biopharma Inc.
Ampreloxetine
nOH

$7.11
+0.25  +4%
Phase 3 Phase 3 study did not meet its primary endpoint September 15, 2021.
$522.4 million

TVTX – Travere Therapeutics Inc.
Sparsentan (PROTECT)
IgA nephropathy

$22.51
+0.07  +0%
Phase 3 Phase 3 initial data released August 16, 2021. Primary efficacy endpoint met. Topline results from the confirmatory endpoint analysis are expected 2H 2023.
$1.4 billion