BPC July 23 update

Phase 1/2 clinical events to watch this quarter; Biotech earnings calendar; Biotech week in Review

Weekly watchlist

Last week we previewed key companies undergoing COVID-19 vaccine trials and their respective development timelines. At the time of publication Novavax (NVAX) had guided for data release later this month. The company provided an update Thursday that data are now due during the first week of August.

This week we look ahead at Phase 1/2 readouts due this quarter. This follows our preview of Phase 3 events we noted two weeks ago. First, let's review the week that was with notable events.

COVID-19 VACCINE:

AstraZeneca PLC (NYSE:AZN) announced interim data from its Phase 1/2 trial of its COVID-19 vaccine, AZD1222. The vaccine was well tolerated and generated robust immune responses against the virus, with 91% of subjects exhibiting neutralising antibody responses one month following a single AZD1222 dose, and in 100% of participants who received a second dose. Data from non-U.S. Phase 3 trials to test the efficacy of the vaccine are due later this quarter. A U.S. Phase 3 trial is slated to commence soon.

OTHER CLINICAL:

BioXcel Therapeutics, Inc.(Nasdaq: BTAI) shares closed the week up 15% to $60.76 following its announcement that its two Phase 3 trials (SERENITY I and SERENITY II) of BXCL501 met the primary and secondary endpoints demonstrating a robust treatment effect in patients with schizophrenia (SERENITY I) and bipolar disorder (SERENITY II).

ACADIA Pharmaceuticals Inc. (Nasdaq: ACAD) announced top-line data from its Phase 3 trial of pimavanserin as an adjunctive treatment for major depressive disorder (MDD) failed to meet the primary endpoint. Shares closed the week down 21% to $43.46.

Pieris Pharmaceuticals, Inc. (NASDAQ:PIRS) shares closed the week down 11% to $2.91 after hours on news its Phase 1 trials of PRS-343 have been placed on partial clinical hold by the FDA while Pieris conducts an additional in-use and compatibility study requested by the agency.

REGULATORY:

Jazz Pharmaceuticals plc (Nasdaq: JAZZ) announced Thursday that the FDA approved Xywav oral solution for the treatment of cataplexy or excessive daytime sleepiness (EDS) in patients 7 years of age and older with narcolepsy. Shares closed Thursday up 4% to $110.26.

Milestone Pharmaceuticals Inc. (NASDAQ:MIST) announced an update on its Etripamil nasal spray program for patients with paroxysmal supraventricular tachycardia (PSVT) following meetings with the FDA. The company noted that the FDA indicated that its recently completed NODE-301 and ongoing NODE-301B trials could be used as two efficacy studies supporting a future New Drug Application (NDA). Data from the NODE-301B trial (renamed the RAPID study) are due late-2021/early 2022. The company also announced a $25m private placement. Shares closed the week up 133% to $8.28.
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Biotech earnings for the upcoming week (selection of 15):

Tue: PFE AMGN

Wed: ALKS GSK SNY

Thur: AZN ALXN EBS EXAS GILD LLY

Fri: ABBV MRK SGEN VRTX

Phase 1/2 events to watch this quarter:

Drug Price Stage Catalyst Market Cap

ALBO – Albireo Pharma Inc.
Elobixibat
Nonalcoholic steatohepatitis (NASH)

$34.74
-0.61  -2%
Phase 2 Phase 2 trial met primary endpoint. However, development in NASH will not continue - August 18, 2020.
$666.8 million

ASND – Ascendis Pharma A/S
TransCon - PaTHway
Hypoparathyroidism

$139.99
-0.93  -1%
Phase 3 Phase 3 top-line data due 4Q 2021.
$7.5 billion

BLRX – BioLineRx Ltd.
Motixafortide (BL-8040) and KEYTRUDA (pembrolizumab) - COMBAT trial
Pancreatic cancer

$2.67
-0.01  -0%
Phase 2 Phase 2 data released December 16, 2020. ORR 21%; PFS 4 months; OS 6.5 months.
$123 million

CCXI – ChemoCentryx Inc.
Avacopan (CCX168)
Hidradenitis Suppurativa (HS)

$12.89
-0.17  -1%
Phase 3 Phase 2b data released October 28, 2020. Primary endpoint across Hurley Stages 2 and 3 not met. Phase 3 trial planned in Stage 3 patients for 4Q 2021.
$899.1 million

CRBP – Corbus Pharmaceuticals Holdings Inc.
Lenabasum (Resunab)
Cystic Fibrosis

$1.94
-0.04  -2%
Phase 2b Phase 2b trial did not meet primary endpoint - October 6, 2020.
$242.6 million

CYCN – Cyclerion Therapeutics Inc.
Olinciguat - STRONG-SCD
Sickle Cell disease

$3.70
0.00  0%
Phase 2 Phase 2 data released October 14, 2020 - insufficient efficacy shown to support further development.
$149.6 million

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

$6.99
-0.19  -3%
Phase 2 Phase 1/2 dose expansion data due by end of 2021.
$399 million

FULC – Fulcrum Therapeutics Inc.
Losmapimod (ReDUX4)
Facioscapulohumeral muscular dystrophy (FSHD)

$8.91
+0.08  +1%
Phase 2b Phase 2b interim data did not show separation from placebo - August 11, 2020. Top-line data to be presented at the virtual FSHD International Research Congress taking place June 24-25, 2021.
$291.2 million

GNCA – Genocea Biosciences Inc.
GEN-009
Various cancers

$2.25
+0.05  +2%
Phase 1/2 Phase 1 long-term follow-up clinical and immunogenicity data presented at ASCO June 4, 2021. 4/9 checkpoint inhibitor-sensitive patients experienced responses post vaccination.
$122.5 million

IMVT – Immunovant Inc.
IMVT-1401
Myasthenia Gravis (MG)

$11.09
-0.16  -1%
Phase 2 Phase 2 data released August 25, 2020. Phase 3 trial to be initiated in late 2021 or early 2022.
$907.3 million

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

$4.55
-0.27  -6%
Phase 2 Phase 2 top-line data released September 30, 2020. Numerically higher response rate on the primary endpoint during months 3 and 6 combined, but did not meet the primary endpoint of statistical superiority.
$515 million

TCRR – TCR2 Therapeutics Inc.
Gavo-cel (TC-210)
Mesothelin-positive solid tumors

$17.24
-0.03  -0%
Phase 1/2 Phase 1 additional safety, efficacy and translational data due throughout 2021.
$657.9 million

TPTX – Turning Point Therapeutics Inc.
Repotrectinib (TPX-0005) - TRIDENT-1
Non-small-cell lung cancer (NSCLC) and solid tumors

$80.12
-0.42  -1%
Phase 2 Phase 2 top-line data due 1Q 2022.
$3.9 billion

TRIL – Trillium Therapeutics Inc.
TTI-621-01
Diffuse Large B-cell Lymphoma (DLBCL), Cutaneous T-cell lymphoma (CTCL)

$9.93
-0.24  -2%
Phase 1 Phase 1 TTI-621 monotherapy showed 18-29% ORR in R/R T- and B-cell lymphomas.
$880.6 million