BPC May 24 update

Key small-cap biotech stock catalysts left for 2Q 2019; Biotech week in Review

Weekly watchlist

The week ahead will see most eyes on the upcoming ASCO conference, which is set to commence Friday May 31 through to Tuesday June 4. Last week we highlighted several stocks to watch leading up to the conference.

Post ASCO, a number of key clinical readouts and regulatory catalyst dates remain on the calendar. In mid-April we listed key Phase 3 small-cap catalysts to watch this quarter. This week we update the list, given that some companies have already delivered data (Tickers: BCRX, MYOV, OCUL and TOCA), while others have delayed their readouts following updates from the first quarter earnings season (Tickers: AXSM, GLYC and SNDX). We also add a few regulatory and other Phase 2 readouts, focusing on small cap stocks.

First, let’s review the week that was.

Novartis (NYSE: NVS) shares closed Friday up 4% to $87.52 following its announcement the FDA approved Zolgensma, the first gene therapy approved for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA). Novartis will price Zolgensma at $2.125m, or an annualized cost of $425,000 per year for five years.

Iovance Biotherapeutics, Inc. (NASDAQ:IOVA), one of the standouts following the release of its ASCO abstract, announced that LN-145, currently in trials for cervical cancer, has been granted Breakthrough Therapy designation, which potentially will see it on an expedited track towards FDA approval. The company will be presenting at ASCO on June 1. Shares, which closed the week up 15% to $17.25, have risen 54% since the release of its abstract.

Array BioPharma Inc. (Nasdaq: ARRY) announced that its Phase 3 BEACON CRC trial evaluating Braftovi (encorafenib), Mektovi (binimetinib) and Erbitux (cetuximab) in patients with colorectal cancer, met both primary endpoints of confirmed objective response rate (ORR), and overall survival (OS). Patients treated with the triplet combo demonstrated a statistically significant improvement in ORR (26.1% vs. 1.9%), and OS (9.0 months vs. 5.4 months, [HR 0.52]) compared to the control arm. Shares closed the week up 27% to $27.09.

BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) shares slumped to close the week down 55% to $3.32 following the release of data from its Phase 3 APeX-2 trial of oral BCX7353 for the prevention of hereditary angioedema (HAE) attacks. While the trial met its primary endpoint, reducing attack rates by 30% and 44% across two dose levels (110 mg and 150 mg), rates were well below expectations, with analysts and investors doubting, even if it were granted approval, how it would compete with currently approved treatments on the market.

Ocular Therapeutix, Inc. (NASDAQ: OCUL) shares closed the week down 24% to $2.64 following news that top-line data from its Phase 3 clinical trial of OTX-TP, for the reduction of intraocular pressure (IOP) in patients with primary open-angle glaucoma or ocular hypertension, did not achieve its primary endpoint.

Tocagen Inc. (Nasdaq: TOCA) announced an Independent Data Monitoring Committee (IDMC) recommended that its Phase 3 Toca 5 trial evaluating Toca 511 and Toca FC in patients with recurrent high-grade glioma (HGG), to continue through to final analysis. Investors had hoped that the trial would be stopped due to sufficient efficacy. Shares closed the week down 48% to $4.59 on the basis the lack of efficacy in the interim analysis will also be reflected in the final analysis due towards the end of this year.


Selection of key small-cap biotech stock catalysts left for 2Q 2019:

Drug Stage Catalyst Market Cap

ACHV – Achieve Life Sciences Inc.
Cytisine - ORCA-2
Smoking cessation

Phase 3 Phase 3 trial to be initiated 2H 2020.
$15.6 million

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

Approved FDA approval announced June 21, 2019.
$271.8 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.
$770.3 million

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

Phase 2b Clinical development of Seladelpar discontinued - November 25, 2019. Noted May 11, 2020 that seladelpar did not cause drug-induced liver injury as noted in November 2019.
$265.9 million

IFRX – InflaRx N.V.
Hidradenitis Suppurativa

Phase 2b Phase 2b data June 5, 2019 did not meet primary endpoint.
$215.4 million

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

Phase 3 Phase 3 trial did not meet primary endpoint - July 8, 2019.
$1.5 billion

KRYS – Krystal Biotech Inc.
B-VEC (Beremagene Geperpavec) - KB103
Epidermolysis Bullosa

Phase 3 Phase 3 trial to commence 1H 2020.
$921.4 million

KZR – Kezar Life Sciences Inc.

Phase 1/2 Phase 1b next data due by the end of 2Q 2020.
$210.4 million

NERV – Minerva Neurosciences Inc
MIN-202 (seltorexant)
Primary insomnia

Phase 2b Phase 2b data June 24, 2019 met primary endpoint.
$560 million

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

Phase 3 Phase 3 data June 12, 2019 did not meet primary endpoint. Second Phase 3 trial planned.
$127.9 million