BPC April 18 update

12 Phase 3 small-cap biotech stock readouts due by June; Upcoming earnings; Biotech week in review

Weekly watchlist

Last week we looked ahead at upcoming PDUFA FDA approval dates for small and mid-cap stocks. This week’s list looks at Phase 3 data catalysts expected by the end of June for solely micro and small cap biotech stocks. We also look ahead at companies reporting first quarter earnings next week.

First, let’s review the week that was from the shortened four-day trading week.

Mustang Bio, Inc. (NASDAQ: MBIO) shares closed up Thursday 112% to $5.64. The gains followed news released late-Wednesday by Mustang that initial data from its Phase 1/2 gene therapy, MB-107 in patients with X-linked Severe Combined Immunodeficiency (XSCID) or bubble disease, were published in the New England Journal of Medicine. Patients with XSCID have no T-cells or natural killer cells, resulting in a severely compromised immune system. The data showed that in seven of the eight boys, normal cell numbers occurred within three to four months after treatment, while the eighth boy, who initially had low T-cell levels, saw his numbers return to normal following a boost of gene-corrected cells. Most patients were discharged within one month.

In May 2018, initial data noted that six of the eight boys achieved normal cell numbers within three to four months following treatment, which suggests the stock surge from Wednesday after hours was due to the fact it was published in the NEJM, rather than the actual release of slightly more updated data.

Wave Life Sciences Ltd. (NASDAQ: WVE) shares closed the week down 25% to $25.60. Just one month ago shares were trading over $46. The company announced data from its Phase 1 clinical trial of suvodirsen (WVE-210201) in boys with Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping. Patients were dosed ranging from 0.5 mg/kg to 10mg/kg. However, in its Phase 2/3 design also released, the company noted that patients will receive only 3 mg/kg or 4.5 mg/kg doses of suvodirsen, with investors concerned the low dosage level will be too low to show sufficient efficacy and higher doses not possible due to safety.

Achaogen, Inc. (Nasdaq: AKAO) shares slid to close Monday down 69% to $0.16 on news that it has filed a voluntary petition under Chapter 11 of the Bankruptcy Code.

Two nano-cap companies, Fibrocell Science, Inc. (Nasdaq: FCSC) and Rexahn Pharmaceuticals, Inc. (NYSE American: RNN) saw their shares spike to close Monday and Tuesday up 43% and 25%, respectively, following the release of separate license collaborations. However, shares faded to close the week up 26% and 16%, respectively, as the initial attraction of the total potential value of the respective deals lost its momentum.

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Selection of companies reporting earnings next week (expect a full week the following week):

W: BIIB NVS

T: ABBV ALXN BMY BMRN


12 Phase 3 Small-cap biotech stock readouts due by June:

Drug Stage Catalyst Market Cap

ATRA – Atara Biotherapeutics Inc.
ATA 129 (tab-cel) - ALLELE
Epstein-Barr virus (EBV-PTLD) after solid organ transplant (SOT)

Phase 3 BLA filing due 2H 2020. Phase 3 top-line data due prior to filing but will only release data until after acceptance of the BLA filing.
$657.9 million

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

Phase 3 BLA filing due 2H 2020. Phase 3 top-line data due prior to filing but will only release data until after acceptance of the BLA filing.
$657.9 million

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

Phase 3 Phase 3 data due 1Q 2020.
$2.9 billion

BCRX – BioCryst Pharmaceuticals Inc.
Berotralstat BCX7353
Reduce or eliminate attacks in HAE patients

PDUFA PDUFA date December 3, 2020
$462.2 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.
$701.6 million

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

Phase 3 Phase 3 data failed to meet endpoints - August 2, 2019.
$157 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.
$1.2 billion

ITCI – Intra-Cellular Therapies Inc.
ITI-007 - Monotherapy (Study 404)
Bipolar depression

Phase 3 Phase 3 trial met primary endpoint - July 8, 2019.
$1.2 billion

MYOV – Myovant Sciences Ltd.
Relugolix - LIBERTY 1 and LIBERTY 2
Menstrual bleeding associated with uterine fibroids

NDA Filing NDA filing due April 2020.
$924.8 million

OCUL – Ocular Therapeutix Inc.
OTX-TP
Glaucoma and ocular hypertension

Phase 3 Phase 3 data May 20, 2019 did not meet primary endpoint.
$226.2 million

SNDX – Syndax Pharmaceuticals Inc.
E2112
HR+, HER2- breast cancer

Phase 3 Phase 3 PFS date did not meet primary endpoint - October 25, 2018. OS final analysis due 2Q 2020.
$284 million

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

Phase 3 Phase 3 data June 12, 2019 did not meet primary endpoint. Noted October 2, 2019 that the FDA has recommended that a BLA not be filed. Open label data due 1Q 2020.
$94.4 million

TBPH – Theravance Biopharma Inc.
Closed Triple - CAPTAIN
Asthma

PDUFA sNDA filing announced October 2, 2019. PDUFA date 2Q 2020. Estimate August 2, 2020 using 10-month timeline.
$1.5 billion

TOCA – Tocagen Inc.
Toca 511 & Toca FC - Toca 5
Recurrent brain cancer

Phase 2/3 Phase 2/3 primary and secondary endpoints missed - September 12, 2019.
$33.9 million