BPC January 08 update

​Chimerix CMRX adds oncology target - shares rally 69%; Neoleukin NLTX IND clinical hold -11%

Price and Volume Movers

Chimerix (NASDAQ:CMRX) shares rallied to close up 69% to $8.42 on news it has acquired Oncoceutics, Inc., a privately-held, clinical-stage biotechnology company. Oncoceutics’ lead product candidate, ONC201, is currently in a pivotal Phase 2 trial for recurrent H3 K27M-mutant glioma with data due later this year. Chimerix will pay Oncoceutics shareholders $78m, of which $39m is payable in Chimerix stock and $39m in cash.

Sarepta Therapeutics, Inc. (NASDAQ:SRPT) shares closed Friday down 51% to $82.29. Investors sold down shares in response to data released late-Thursday from its Phase 2 trial of SRP-9001 (Study 102) in patients with Duchenne muscular dystrophy (DMD). The trial did not meet the primary functional endpoint of a significant increase in NSAA total score (a muscle function test score) compared to placebo (P=0.37).

Solid Biosciences Inc. (NASDAQ:SLDB), which is also developing treatments for DMD, saw its shares also sell off on the news, closing down 21% to $6.70.

Aldeyra Therapeutics, Inc. (Nasdaq: ALDX) shares closed up 22% to $10.31, adding to Thursday’s gain of 26%. The company released data from the run-in cohort of the Phase 3 TRANQUILITY clinical trial in patients with dry eye disease. The main cohort of the Tranquility trial is expected to commence enrolment next month with data from the trial due in the second half of 2021.

Neoleukin Therapeutics, Inc., (NASDAQ:NLTX) announced that the FDA issued a clinical hold for its Phase 1 Investigational New Drug (IND) Application of NL-201. The FDA noted that the company needs to develop a new assay that measures the amount of protein being administered and to accurately deliver the intended dose of NL-201. Shares fell 11% to $14.05.

KemPharm, Inc. (NASDAQ:KMPH) announced a public offering of 7,692,307 shares of its common stock with attached warrants to purchase a further 7,692,307 shares at a combined public offering price of $6.50 for gross proceeds of $50m. Shares closed down 32% to $5.60.

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Major price movers (stocks priced > $1.00, volume > 50k listed on NASDAQ/NYSE):

Refer to the BioPharmCatalyst Biotech Stocks page for Top 10 Gainers/Losers/Unusual Volume:

ADVANCERS:

Jaguar Health, Inc. (JAGX): $4.00; +49%.

Merus N.V. (MRUS): $20.73; +28%.

Dyne Therapeutics, Inc. (DYN): $27.13; +17%.

Seneca Biopharma, Inc. (SNCA): $1.24; +16%.

Alimera Sciences, Inc. (ALIM): $5.80; +16%.

DECLINERS:

Cerecor Inc. (CERC): $2.50; -14%.

Endo International plc (ENDP): $7.06; -11%.

Larimar Therapeutics, Inc. (LRMR): $19.15; -10%.

BioDelivery Sciences International, Inc. (BDSI): $4.49; -8%.

Anika Therapeutics, Inc. (ANIK): $42.86; -7%.


Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

ARGX – argenx SE
Cusatuzumab and Azacitidine (CULMINATE)
Acute myeloid leukemia (AML)

Phase 2 Phase 2 interim data released January 8, 2021. Complete remission (CR) rate 27% (14/52) and composite complete remission (CRc), including CRs with incomplete hematologic recovery 40% (21/52).
$14.9 billion

AZRX – AzurRx BioPharma Inc.
MS1819 - OPTION 2
Cystic fibrosis

Phase 2b Phase 2b top line data due in 1Q 2021.
$42.6 million

DNLI – Denali Therapeutics Inc.
DNL343
Healthy Volunteers

Phase 1 Phase 1 data due 1H 2021. Phase 1b study in ALS patients planned to begin in 2H 2021​.
$9 billion

DNLI – Denali Therapeutics Inc.
DNL788 (SAR443820)
Healthy volunteers

Phase 1 Phase 1 data due 2H 2021.
$9 billion

ENTA – Enanta Pharmaceuticals Inc.
EDP-305 ARGON-2
Non-alcoholic steatohepatitis (NASH)

Phase 2b Phase 2b interim analysis due mid-2021.
$939.9 million

ENTA – Enanta Pharmaceuticals Inc.
EDP-938 (RSVP)
Respiratory Syncytial Virus (community-acquired)

Phase 2b Phase 2b trial ongoing. Data delayed due to RSV season not beginning in the Northern Hemisphere due to COVID-19 mitigation measures.
$939.9 million

ENTA – Enanta Pharmaceuticals Inc.
EDP-514
Hepatitis B - NUC-Suppressed

Phase 1b Phase 1b top-line data due 2Q 2021.
$939.9 million

NBIX – Neurocrine Biosciences Inc.
Valbenazine
Huntington's disease

Phase 3 Phase 3 data due 4Q 2021.
$10.7 billion

NBIX – Neurocrine Biosciences Inc.
TAK-831 / NBI-1065844
Negative symptoms of schizophrenia

Phase 2 Phase 2 top-line data due 1H 2021.
$10.7 billion

ONCS – OncoSec Medical Incorporated
Tavo + Opdivo
Neoadjuvant Therapy for Melanoma

Phase 2 Phase 2 initiation of dosing announced January 8, 2021.
$198.5 million

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

Phase 1/2 Phase 1 additional safety, efficacy and translational data due 1H 2021.
$969.9 million

TCRR – TCR2 Therapeutics Inc.
TC-110
CD19+ non-Hodgkin lymphoma or adult acute lymphoblastic leukemia

Phase 1/2 Phase 1 interim update due 2021.
$969.9 million
AOC 1001
Myotonic dystrophy type 1 (DM1)

Phase 1/2 Phase 1/2 trial to commence 2H 2021.

CMRX – Chimerix Inc.
ONC201
H3 K27M-mutant Glioma

Phase 2 Phase 2 pivotal data due 2021.
$666.1 million