BPC July 09 update

Phase 3 clinical updates to watch this quarter

Weekly watchlist

Following on from our preview last week of regulatory catalysts (PDUFA and Advisory Committee meetings), we turn our attention to clinical trial updates scheduled for this quarter, looking at Phase 3 readouts this week before looking at Phase 1 and 2 events during the following weeks.

First, let’s review the week that was with price moving events in what was a relatively quiet week of news flow.

REGULATORY:

Provention Bio, Inc. (Nasdaq: PRVB) announced Tuesday that the FDA issued a Complete Response Letter (CRL) for its Biologics License Application (BLA) for teplizumab for the delay of clinical type 1 diabetes (T1D) in at-risk individuals. The CRL noted that the planned commercial product failed to show pharmacokinetic (PK) comparability with drug product originating from drug substance manufactured for historic clinical trials. The company expects relevant additional PK/PD will be available later this quarter and following review of the data a decision will be made whether to submit the data to the FDA or justify why PK comparability is not necessary. Shares closed the week down 22% to $6.47.

ChemoCentryx, Inc., (Nasdaq: CCXI) shares closed the week up 27% to $16.77 on news it filed an amendment to its New Drug Application (NDA) for avacopan for the treatment of Anti-neutrophil Cytoplasmic Autoantibody (ANCA)-associated vasculitis, addressing points raised during the FDA Advisory Committee meeting on May 6, 2021. The FDA has indicated that the filing constitutes a major amendment to the NDA and will result in the setting of a new PDUFA goal date of October 7, 2021.

CLINICAL UPDATES:

Celldex Therapeutics, Inc. (NASDAQ:CLDX) announced late-Friday updated data from its open label Phase 1b clinical trial of CDX-0159 in patients with antihistamine refractory cold urticaria and symptomatic dermographism. 18/19 (95%) experienced a complete response and 1/19 (5%) experienced a marked partial response. Shares closed the after-hours session Friday up 35% to $43.88.

Sigilon Therapeutics, Inc. (NASDAQ:SGTX) shares dropped Friday 25% to $6.90 following its announcement the FDA placed a clinical hold on its Phase 1/2 trial of SIG-001 in patients with severe or moderately severe hemophilia A. The clinical hold was initiated following a serious adverse event (SAE). One of the three patients on the high dose developed inhibitors to Factor VIII (FVIII).

Phase 3 events to watch during 3Q 2021:

Drug Price Stage Catalyst Market Cap

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

$43.54
-1.47  -3%
NDA Filing NDA submission due in 1H 2022. NDA will be submitted in conjunction with OAKS trial.
$4.2 billion

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

$2.48
+0.14  +6%
Phase 3 Phase 3 trial did not demonstrate a clinical benefit, trial will no longer continue.
$239.8 million

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

$16.32
-0.07  0%
Phase 2/3 Phase 2/3 interim analysis showed primary and secondary endpoints met. 100% efficacy against severe COVID-19 & hospitalization and 84% efficacy against moderate-to-severe COVID-19 , and 79% efficacy against COVID-19 of any severity caused by the Delta variant, noted September 22, 2021.
$2 billion

OCUP – Ocuphire Pharma Inc.
Nyxol Eye Drops (LYNX-1)
Dim Light or Night Vision Disturbances (DLD)

$4.35
+0.21  +5%
Phase 3 Phase 3 data due early-2022.
$75.3 million

OTLK – Outlook Therapeutics Inc.
ONS-5010
Wet age-related macular degeneration (wet AMD)

$1.37
+0.10  +8%
BLA Filing BLA filing due 1Q 2022.
$244 million

QURE – uniQure N.V.
Etranacogene dezaparvovec (AMT-061) - HOPE-B
Hemophilia B

$28.82
-0.27  -1%
Phase 3 Phase 3 top-line data released June 22, 2021. FIX levels sustained at a mean of 41.5% at 52 weeks. Phase 3 top-line 78-week follow-up data expected in 4Q 2021. BLA filing due 1H 2022.
$1.3 billion

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

$3.00
+0.15  +5%
Phase 2/3 Phase 2/3 preliminary top-line data results did not meet its primary endpoint September 14, 2021. Phase 2/3 data showed 62% statistically significant reduction in mortality shown for moderately severe COVID-19 patients, noted October 4, 2021.
$140.2 million

TBPH – Theravance Biopharma Inc.
Ampreloxetine
Neurogenic Orthostatic Hypotension (nOH)

$8.01
-0.44  -5%
Phase 3 Phase 3 study did not meet its primary endpoint September 15, 2021. Phase 3 top-line results due in 1Q 2022.
$590.3 million

TNXP – Tonix Pharmaceuticals Holding Corp.
Tonmya (TNX-102 SL)
Fibromyalgia

$0.50
+0.03  +6%
Phase 3 Phase 3 top-line met primary endpoint - December 7, 2020. Enrollment in second Phase 3 trial stopped due to futility - July 23, 2021. Phase 3 results noted that treatment significantly reduced daily pain compared to placebo, with similar trial discontinuation rates to placebo and no severe adverse events, noted November 8, 2021.
$222 million

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

$29.03
-1.27  -4%
NDA Filing NDA filing expected 1Q 2022, topline results from the confirmatory endpoint analysis are expected 2H 2023. Phase 3 initial data released August 16, 2021. Primary efficacy endpoint met.
$1.8 billion