BPC December 06 update

Key biotech stock catalysts left for December; Biotech week in Review

Weekly watchlist

The biotech sector continues to ride a wave of anticipation with major price-moving news seemingly released on a daily basis, something not seen for a sustained period for many years. Despite only three weeks left in the trading year, a number of major catalysts still remain on the calendar, which suggests the wild price swings over the last month are set to continue for at least a further 2-3 weeks. One would expect things to settle down come the turn of the new year.

Through to the end of the year we will be updating our list of key clinical and regulatory catalysts to watch. First, let’s review the week that was with a summary of key price moving events.

Audentes Therapeutics, Inc (NASDAQ: BOLD) announced it will be acquired by Astellas Pharma Inc. for $60 per share in cash. Shares closed the week up 104% to $59.25.

Kodiak Sciences Inc. (Nasdaq: KOD) shares closed the week up 118% to $64.47 on news of a funding agreement to sell a capped 4.5% royalty right on global net sales of KSI-301 to Baker Bros. Advisors (BBA) for $225m and then followed up with a public offering of $317.4m.

Aurinia Pharmaceuticals Inc. (NASDAQ: AUPH) shares closed the week up 94% to $15.44 following news Wednesday that its Phase 3 AURORA trial of voclosporin, for the treatment of lupus nephritis, met its primary endpoint of renal response rates.

Allakos Inc. (NASDAQ: ALLK) shares closed Wednesday up 44% to $137.73 following a report by Bloomberg that the company is exploring strategic options including a potential sale. Shares pulled back to close the week up 24% to $117.36.

Axsome Therapeutics, Inc. (NASDAQ: AXSM) announced that its Phase 2 trial of AXS-12 (reboxetine) met the primary endpoint and significantly reduced the number of cataplexy attacks as compared to placebo in patients with narcolepsy. Shares closed the week up 15% to $45.43.

Sage Therapeutics (NASDAQ: SAGE) announced Thursday that it did not meet the primary endpoint of a statistically significant reduction from baseline compared to placebo in the 17-item Hamilton Rating Scale for Depression (HAM-D) in its Phase 3 Mountain trial of SAGE-217 in adults with major depressive disorder (MDD). Shares closed the week down 58% to $64.46.

Cara Therapeutics, Inc. (Nasdaq: CARA) announced that it met the primary endpoint in its Phase 2 trial of Oral Korsuva for the treatment of pruritus in patients with stage III-V (moderate-to-severe) chronic kidney disease (CKD). However, key secondary endpoints were not met, sending shares down 35% for the week to $16.76.

Correvio Pharma Corp (NASDAQ: CORV) shares closed down 39% to $1.32 following the release of briefing documents for its upcoming Advisory Committee meeting to be held on Tuesday December 10, where an expert panel will discuss the regulatory application to market Brinavess (vernakalant) for the rapid conversion of recent onset atrial fibrillation (AFib) to sinus rhythm (SR). While sufficient evidence of benefit of the treatment was noted, safety concerns led the FDA to ‘not believe that the benefit proffered by vernakalant, conversion of AFib to SR, outweighs the serious risks associated with its use.’

14 key remaining catalysts to watch for December:

Drug Stage Catalyst Market Cap

ACST – Acasti Pharma Inc.
CaPre (TRILOGY 1 and 2)

Phase 3 Phase 3 TRILOGY 1 data did not meet primary endpoint - January 13, 2020. TRILOGY 2 data due 3Q 2020.
$79.8 million

AMRN – Amarin Corporation plc
High Triglycerides With Mixed Dyslipidemia

Approved FDA Approval announced December 13, 2019.
$2.6 billion

ATNX – Athenex Inc.
Metastatic breast cancer

NDA Filing NDA to be filed in 2020.
$928.9 million

AXSM – Axsome Therapeutics Inc.
Major Depressive Disorder

NDA Filing NDA filing due 4Q 2020.
$2.8 billion

BGNE – BeiGene Ltd.
Zanubrutinib (BGB-3111) - ASPEN
Waldenström’s macroglobulinemia

Phase 3 Phase 3 top-line data did not meet primary endpoint - December 16, 2019.
$13.1 billion

BMRN – BioMarin Pharmaceutical Inc.

NDA Filing Phase 3 top-line data met primary endpoint - December 16, 2019. Regulatory filing due 3Q 2020.
$20.1 billion

CORV – Correvio Pharma Corp.
Atrial fibrillation (AF)

CRL CRL announced December 24, 2019.
$27.8 million

FIXX – Homology Medicines Inc.
Phenylketonuria (PKU)

Phase 1/2 Phase 1/2 update due mid-2020.
$719.8 million

KALV – KalVista Pharmaceuticals Inc.
Diabetic macular edema (DME)

Phase 2 Phase 2 data did not meet primary endpoint - December 9, 2019.
$207 million

OBSV – ObsEva SA
Linzagolix OBE2109 - PRIMROSE 2
Uterine fibroids

Phase 3 Phase 3 data met primary endpoint - December 9, 2019. 12 month data due 2Q 2020.
$236.3 million

PTI – Proteostasis Therapeutics Inc.
PTI-428 + PTI-801 + PTI-808
Cystic fibrosis

Phase 2 Phase 2 negative data released December 17, 2019. Mean changes in ppFEV1 were not statistically significant in the heterozygous population. Phase 3 trial to be initiated in 2020.
$72 million

SLGL – Sol-Gel Technologies Ltd.

NDA Filing NDA filing due 2H 2020.
$184.9 million

SPPI – Spectrum Pharmaceuticals Inc.
Poziotinib - Zenith20
Non-small cell lung cancer (NSCLC) with exon 20 insertion mutation in EGFR or HER2

Phase 2 Phase 2 primary endpoint from Cohort 1 was not met - December 26, 2019. Cohort 2 data due mid-2020 with Cohort 3 data due year-end 2020.
$340.1 million

WVE – Wave Life Sciences Ltd.
Huntington’s disease

Phase 1/2 Phase 1b/2a top line data released December 30, 2019 noted statistically significant reduction of 12.4% (p<0.05) in mutant huntingtin (mHTT) protein but no difference in total HTT (tHTT) protein. 32 mg data due 2H 2020.
$336 million