BPC November 19 update

Myovant MYOV shares surge on positive late-stage data; CRISPR releases initial gene-editing data

Price and Volume Movers

Myovant Sciences (NYSE: MYOV) shares closed up 113% to $12.92 following news its Phase 3 HERO trial of oral relugolix met its primary efficacy endpoint and all six key secondary endpoints in men with advanced prostate cancer. A New Drug Application (NDA) is expected to be filed with the FDA in 2Q 2020. In the primary endpoint responder analysis, 96.7% of men receiving once-daily, oral relugolix achieved sustained testosterone suppression to castrate levels, demonstrating non-inferiority to leuprolide acetate, which showed a 88.8% response rate.

CRISPR Therapeutics (NASDAQ: CRSP) and Vertex Pharmaceuticals Incorporated (NASDAQ: VRTX) announced interim data from the first two patients in a Phase 1/2 trial with severe hemoglobinopathies treated with their CRISPR/Cas9 gene-editing therapy CTX001. One patient with transfusion-dependent beta thalassemia (TDT) required 16.5 transfusions per year before enrolling in the trial, with the patient transfusion-independent nine months following the therapy. The second patient with sickle cell disease experienced seven vaso-occlusive crises (VOCs) per year prior to enrolling and after four months of CTX001 infusion, the patient was free of VOCs. Shares of CRISPR closed up 17% to $68.46. The company announced after hours a public offering of 4.25m common shares, with shares now trading down 5% to $65.01.

Other gene-editing stocks also rallied on the news. Editas (EDIT) shares climbed 8% to $24.96 while Intellia (NTLA) closed up 10% to $14.98.

Athenex, Inc. (NASDAQ:ATNX) shares closed up 36% to $15.52 following its announcement that its abstract for the Phase 3 trial of oral paclitaxel and encequidar (oral paclitaxel) has been selected for the press program of the 2019 San Antonio Breast Cancer Symposium (SABCS), with a presentation on Friday, December 13, 2019. The company had previously indicated that it would be presenting at the meeting, so investors appeared to react to the positive nature of the abstract title released today noted below. Abstracts will be posted online on the day of the presentation.

"Oral Paclitaxel with Encequidar: The first orally administered paclitaxel shown to be superior to IV paclitaxel on confirmed response and survival with less neuropathy: a Phase III clinical study in metastatic breast cancer."

Agile Therapeutics Inc (NASDAQ: AGRX) shares closed up 29% to $1.77. In a SEC filing released Monday, it was noted Perceptive Advisors has increased its ownership of the company to 18.5%.

The Medicines Company (NASDAQ: MDCO) shares closed up 20% to $70.21 following a report by Bloomberg that Novartis has expressed an interest in purchasing the company. Neither company has yet to comment on the report.

Karuna Therapeutics, Inc. (Nasdaq: KRTX) shares closed up 29% to $123.99, adding to Monday’s gain of 443% following the release of positive data from its Phase 2 clinical trial of KarXT for the treatment of acute psychosis in patients with schizophrenia.

Checkpoint Therapeutics, Inc. (NASDAQ:CKPT) announced it intends to sell shares of its common stock in an underwritten public offering. Shares are trading down 14% to $1.32 after hours.

Endo International plc (NASDAQ:ENDP) announced the FDA has accepted for review the original Biologics License Application (BLA) for collagenase clostridium histolyticum (CCH) for the treatment of cellulite in the buttocks. The PDUFA date is July 6, 2020.

-

Major price movers (stocks priced > $1.00, volume > 50k):

Refer to the BioPharmCatalyst Biotech Stocks page for Top 10 Gainers/Losers/Unusual Volume

ADVANCERS:

Cabaletta Bio Inc (NASDAQ: CABA): $17.71; +28%.

Fulcrum Therapeutics Inc (NASDAQ: FULC): $10.09; +25%.

CELYAD (NASDAQ: CYAD): $11.00; +20%.

BioCryst Pharmaceuticals, Inc. (NASDAQ: BCRX): $2.02; +19%.

DECLINERS:

Plus Therapeutics Inc (NASDAQ: PSTV): $2.10; -12%.

Surface Oncology Inc (NASDAQ: SURF): $1.44; -11%.

Melinta Therapeutics Inc (NASDAQ: MLNT): $1.42; -11%.

Evoke Pharma Inc (NASDAQ: EVOK): $1.33; -10%.

Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

AFMD – Affimed N.V.
AFM24
EGFR-expressing solid tumors

Phase 1/2 Phase 1/2a trial to commence 1Q 2020.
$173.8 million

AFMD – Affimed N.V.
AFM13
CD30-positive peripheral T cell lymphoma (PTCL)

Phase 2 Phase 2 dosing has been initiated - noted November 19, 2019.
$173.8 million

ALKS – Alkermes plc
ALKS 3831 - ENLIGHTEN-2
Schizophrenia, bipolar

NDA Filing NDA filing announced November 19, 2019.
$3.3 billion

ATRS – Antares Pharma Inc.
Selatogrel and QuickShot
Acute myocardial infarction (AMI)

Phase 1 Phase 2 trial planned for 2020.
$798.2 million

BSTC – BioSpecifics Technologies Corp
XIAFLEX (collagenase clostridium histolyticum) - RELEASE
Edematous fibrosclerotic panniculopathy (“cellulite”)

PDUFA PDUFA date July 6, 2020.
$406.3 million

CYAD – Celyad SA
CYAD-101 - alloSHRINK
Colorectal cancer

Phase 1 Phase 1 further data due 1H 2020.
$176.8 million

MYOV – Myovant Sciences Ltd.
Relugolix - HERO
Advanced prostate cancer

Phase 3 Phase 3 data meet primary endpoint - November 19, 2019. NDA filing due 2Q 2020.
$1.5 billion

SGMO – Sangamo Therapeutics Inc.
TX200
Kidney transplantation

Phase 1/2 Phase 1/2 trial to be initiated in 2020.
$1.4 billion

SRRK – Scholar Rock Holding Corporation
SRK-015 - TOPAZ
Spinal muscular atrophy (SMA

Phase 2 Phase 2 initial PK/PD data confirmed presence of latent myostatin in patients with SMA - November 19, 2019. Interim efficacy data 1H 2020 with top-line data 4Q 2020.
$251.7 million

VIR – Vir Biotechnology Inc - Ordinary Shares
VIR-2482
Influenza

Phase 1/2 Phase 1/2 additional data due 2H 2020.
$1.4 billion

VKTX – Viking Therapeutics Inc.
VK2809
Hypercholesterolemia and Non-Alcoholic Fatty Liver Disease

Phase 2 Phase 2b trial initiation announced November 19, 2019.
$530.4 million