BPC March 29 update

13 Biotech Small/Mid cap catalysts slated for 2Q; Biotech week in review

Weekly watchlist

The last week of March saw all of the events noted in last week’s BioPharmCatalyst watch list read out data. Undoubtedly, however, such biotech catalysts are easier to predict when a company notes that data is slated for release in March or by the end of the first quarter and there is only one week left in the quarter.

It’s certainly more challenging at the beginning of a quarter to predict exactly when trials will read out data slated for that quarter. This week we look ahead at what’s due for 2Q 2019. It is a snapshot only with over 150 events on the calendar.

First, let’s review the week that was.

Proteon Therapeutics, Inc. (Nasdaq: PRTO) shares plunged to close Thursday down 86% to $0.50 on news its PATENCY-2 Phase 3 clinical trial of vonapanitase in patients with chronic kidney disease (CKD), did not meet its co-primary endpoints.

Tricida, Inc. (NASDAQ: TCDA) shares closed Thursday up 57% to $37.80 following news its Phase 3 extension trial of TRC101 (veverimer), in patients with chronic kidney disease (CKD), met its primary and all secondary endpoints.

Gilead Sciences, Inc. (NASDAQ: GILD) and Galapagos NV (NASDAQ: GLPG) announced after hours Thursday that their FINCH 1 and 3 trials of filgotinib, in adults with moderately-to-severely active rheumatoid arthritis, met their respective primary endpoints. Shares of Galapagos closed Friday up 23% to $117.78

Fibrocell Science, Inc. (Nasdaq: FCSC) shares closed Wednesday down 26% to $1.85. The company announced promising wound closure rates of 63% from its ongoing Phase 1/2 clinical trial of FCX-007. However, shares closed down as we saw investors appear to “sell-on-the-news”, not uncommon when companies provide a set date for a data release.

Osmotica Pharmaceuticals plc (Nasdaq: OSMT) announced that it met just one of two primary endpoints in its second Phase 3 trial of arbaclofen for the treatment of spasticity in Multiple Sclerosis (MS). Shares closed Thursday down 41% to $4.00.

Aldeyra Therapeutics, Inc. (Nasdaq: ALDX) closed Tuesday up 36% to $9.71 before retracing some of its gains to close the week at $9.03. The company announced that its Phase 3 ALLEVIATE Trial of 0.25% and 0.5% reproxalap topical ophthalmic solution in patients with allergic conjunctivitis met the primary endpoint.

Can-Fite BioPharma Ltd. (NYSE MKT: CANF) shares closed Tuesday down 28% to $0.93 following news its Phase 2 advanced liver cancer study did not achieve its primary endpoint of overall survival.

Proteostasis Therapeutics, Inc. (NASDAQ: PTI) shares closed the week down 69% to $1.26 following disappointing data from its triple combination trial of PTI-428, PTI-801 and PTI-808, in patients with cystic fibrosis. The company also announced it will not be proceeding with its Symdeko trials combined with PTI-428 and PTI-801, due to insufficient efficacy.


2Q small/mid cap biotech catlaysts: ATRA AXSM BCRX CARA CBAY FGEN GLYC HRTX ITCI NBRV PTN SVRA UTHR

Drug Stage Catalyst Market Cap

AMAG – AMAG Pharmaceuticals Inc.
Vyleesi (Bremelanotide)
Female sexual dysfunction (FSD)

PDUFA PDUFA date extended to June 23, 2019.
$303.6 million

ATRA – Atara Biotherapeutics Inc.
ATA 129 (tab-cel) - MATCH
Epstein-Barr virus (EBV-PTLD) after after hematopoietic cell transplant (HCT)

Phase 3 Phase 3 data due 2020.
$1 billion

AXSM – Axsome Therapeutics Inc.
AXS-05 STRIDE-1
Treatment resistant depression

Phase 3 Phase 3 data due 2H 2019.
$836.6 million

BCRX – BioCryst Pharmaceuticals Inc.
BCX7353 - APeX-2
Reduce or eliminate attacks in HAE patients

Phase 3 Phase 3 data May 21, 2019 met primary endpoint but lower than expectations.
$424.7 million

CARA – Cara Therapeutics Inc.
KORSUVA (CR845/difelikefalin) injection - KALM-1
Chronic kidney disease (CKD) on hemodialysis (HD) with Moderate-to Severe Pruritus

Phase 3 Phase 3 data met primary endpoint - May 29, 2019.
$804.6 million

CBAY – CymaBay Therapeutics Inc.
Seladelpar
Non-alcoholic steatohepatitis (NASH)

Phase 2b Phase 2b top-line data noted reductions in liver fat that were not significant compared with placebo - June 11, 2019. 52-week histology results likely due 1H 2020.
$497.3 million

FGEN – FibroGen Inc
Roxadustat
Anaemia in Chronic Kidney Disease

NDA Filing Phase 3 MACE safety data released May 9, 2019. Non-inferior in non-dialysis, no worse risk than placebo. NDA filing due September/October 2019.
$3.9 billion

GLYC – GlycoMimetics Inc.
GMI-1070 (rivipansel)
Vaso-occlusive crisis of sickle cell disease

Phase 3 Phase 3 data due 2H 2019.
$513.4 million

HRTX – Heron Therapeutics Inc.
HTX-011
Post operative pain

CRL CRL announced May 1, 2019.
$1.5 billion

ITCI – Intra-Cellular Therapies Inc.
ITI-007 - Monotherapy (Study 401)
Bipolar depression

Phase 3 Phase 3 top-line data due 2Q 2019.
$758.1 million

NBRV – Nabriva Therapeutics plc
Contepo
Complicated urinary tract infections (cUTI)

CRL CRL issued April 30, 2019.
$162.3 million

SVRA – Savara Inc.
Molgradex - IMPALA
Autoimmune pulmonary alveolar proteinosis (PAP)

Phase 3 Phase 3 data June 12, 2019 did not meet primary endpoint.
$102.6 million

UTHR – United Therapeutics Corporation
Esuberaprost - BEAT
Pulmonary arterial hypertension (PAH)

Phase 3 Phase 3 data released April 8, 2019 did not meet primary endpoint.
$3.5 billion