BPC August 15 update

Biotech events slated for the next two weeks; Biotech week in Review

Weekly watchlist

Over recent weeks we have highlighted key regulatory (PDUFA) and clinical data readouts slated for the next few months.

A number of companies have guided for release of data during the third quarter without narrowing down timelines so effectively such catalysts might read out during either August or September. Only a few locked-in August catalysts remain on the calendar, which we note in this week’s list, assuming that “mid-2019” refers to August and not September.

First, let’s review news from the week that was, a much quieter one compared with the hustle of the week before.

Deciphera Pharmaceuticals, Inc. (NASDAQ:DCPH) shares surged to close up the week up 58% to $37.00 on the release of data from its trials of ripretinib in patients with gastrointestinal stromal tumors (GIST). Its Phase 3 Invictus trial in patients with fourth-line and fourth-line plus GIST achieved its primary endpoint of improved progression free survival (PFS), exhibiting a median PFS of 6.3 months compared to 1.0 month in the placebo arm and significantly reduced the risk of disease progression or death by 85% (HR of 0.15, p<0.0001) compared to placebo. The trial did not meet the key secondary endpoint of objective response rate (ORR), with an ORR of 9.4% shown compared with 0% for placebo (p-value=0.0504). The company also released data from its ongoing Phase 1 trial of ripretinib, in patients with second-line through fourth-line plus GIST. The data showed ORRs of 22% (second-line), 13% (third-line) and 8% (fourth-line). Later in the week, it announced that it would be raising $400m in a registered underwritten public offering.

Blueprint Medicines Corporation (NASDAQ: BPMC), which is developing Avapritnib also for GIST, saw its shares close the week down 9% to $84.06, in reaction to the data released by Deciphera.

Trovagene, Inc. (Nasdaq: TROV) shares closed the week at $2.09, recording a two-day gain of 47% across Thursday and Friday. The company announced Thursday that new clinical data from its Phase 2 trial of onvansertib, in combination with Zytiga (abiraterone acetate)/prednisone, in metastatic Castration-Resistant Prostate Cancer (mCRPC), will be presented at the Asia-Pacific Prostate Cancer Conference, on August 24 in Australia.

Solid Biosciences Inc (NASDAQ: SLDB) shares closed Friday up 43% to $8.81. Analysts at Chardan upgraded their rating of the company from Neutral to Buy, raising their price target from $7.50 to $10.

A number of large cap companies announced approval of their regulatory applications by the FDA. While not price moving events due to their large market cap, the events were newsworthy, nonetheless.

Roche Holding AG (OTCQX: RHHBY) announced Thursday that the FDA approved Rozlytrek (entrectinib) for the treatment of patients with any type of solid tumor that has a neurotrophic tyrosine receptor kinase (NTRK) gene fusion. This is just the third time the FDA has approved a cancer treatment based on a common biomarker across different types of tumors rather than the location in the body where the tumor originated.

AbbVie (NYSE: ABBV) announced Friday that the FDA has approved Rinvoq (upadacitinib), a once-daily oral Janus kinase (JAK) inhibitor, for the treatment of adults with moderately to severely active rheumatoid arthritis (RA).

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Likely biotech events for the next two weeks:

ANAB AVEO NBRV SRPT TROV + (NKTR* IPCIF*)

*likely delayed – see notes below:

Drug Stage Catalyst Market Cap

ANAB – AnaptysBio Inc.
ANB019
Generalized pustular psoriasis

Phase 2 Phase 2 data due mid-2019.
$1.1 billion

AVEO – AVEO Pharmaceuticals Inc.
TIVO-3 - tivozanib
Third line treatment of patients with renal cell cancer

Phase 3 Phase 3 interim data noted overall survival of 16.4 months vs 19.7 months for sorafenib.
$156.2 million

IPCIF – Intellipharmaceutics International Inc - Ordinary Shares
Rexista
Pain relief

PDUFA CRL issued September 25, 2017. NDA resubmitted with new PDUFA date of August 28, 2019. Noted July 26, 2019 that PDUFA date will be delayed. No updated date provided.
$3.9 million

NBRV – Nabriva Therapeutics plc
Lefamulin - Intravenous and Oral
Moderate to severe Community Acquired Bacterial Pneumonia (CABP)

Approved FDA approval announced August 19, 2019.
$167.3 million

NKTR – Nektar Therapeutics
NKTR-181
Lower back pain

PDUFA PDUFA date August 29, 2019. Company noted the date will not be met due to the postponement of an advisory committee meeting.
$3.5 billion

SRPT – Sarepta Therapeutics Inc.
Golodirsen - Exon 53
Duchenne muscular dystrophy

CRL CRL issued August 19, 2019.
$6.2 billion

TROV – TrovaGene Inc.
Onvansertib (PCM-075) and Zytiga
Prostate cancer

Phase 2 Phase 2 data released August 26, 2019.
$10.9 million