BPC August 07 update

​Rhythm RYTM rises 15% on late-stage data; Avedro AVDR acquired - shares +20% AH

Price and Volume Movers

Rhythm Pharmaceuticals, Inc. (Nasdaq:RYTM) announced that data from two Phase 3 trials evaluating setmelanotide, for the treatment of pro-opiomelanocortin (POMC) and leptin receptor (LEPR) deficiency obesities, met their primary and all key secondary endpoints. Eight of 10 patients with POMC deficiency obesity achieved the primary endpoint of greater than 10% weight loss over approximately one year (p<0.0001). Five of 11 patients with LEPR deficiency obesity achieved the primary endpoint of greater than 10% weight loss over one year (p=0.0001). Shares closed up 15% to $21.84.

Glaukos Corporation (NYSE: GKOS) announced it intends to acquire Avedro, Inc. (Nasdaq: AVDR) in an all-stock transaction. Under the terms of the merger agreement, for each share of Avedro common stock they own, Avedro shareholders will receive an exchange ratio equivalent of 0.365 shares of Glaukos stock. Upon closing, Glaukos shareholders are expected to own approximately 85% of the combined company, with Avedro shareholders expected to own the remaining 15%. Shares of Avedro are trading up 20% after hours to $20.50.

Paratek Pharmaceuticals, Inc. (NASDAQ: PRTK) shares closed up 35% to $4.17 following the release of second quarter earnings, which saw net sales of its chief revenue earner, Nuzrya, increase 26% to $1.7m.

Rexahn Pharmaceuticals, Inc. (NasdaqCM: REXN) shares closed down 47% to $2.61 on disappointing data from its Phase 2a trial of RX-3117 in combination with Abraxane in patients newly diagnosed with metastatic pancreatic cancer. An overall response rate of 23% was reported, compared with a rate of 38% in January 2019. Preliminary data indicates that the median progression free survival will be 5.4 months.

Titan Pharmaceuticals, Inc. (NASDAQ: TTNP) announced a registered direct offering with a single institutional investor to purchase approximately $2.1m of its common stock with attached warrants to purchase shares at a combined purchase price for one share and warrant of $0.75. Shares closed down 27% to $0.77.

Athenex, Inc. (Nasdaq: ATNX) announced that its Phase 3 trial of oral paclitaxel and encequidar met the primary efficacy endpoint with statistically significant improvement over IV paclitaxel in patients with breast cancer. The data displayed an overall response rate (ORR) of 36% compared to 24% for IV paclitaxel patients (p = 0.01). Shares, however, sold off to close down 20% to $15.14, likely due to initial data from its secondary endpoints, progression-free survival (p = 0.077) and overall survival (p = 0.11). Athenex noted that it expects both measures to improve upon follow-up.

AstraZeneca PLC (NYSE: AZN) and and Merck & Co., Inc. announced that their Phase 3 PROfound trial of Lynparza (olaparib) in men with metastatic castration-resistant prostate cancer (mCRPC) showed a statistically-significant and clinically-meaningful improvement in the primary endpoint of radiographic progression-free survival (rPFS). Shares of Clovis Oncology, Inc. (NASDAQ: CLVS), a competitor with its product Rubraca, closed down 5% to $8.97 on the news, before falling a further 11% after hours on news of a convertible bond offering.

Dynavax Technologies Corporation (Nasdaq: DVAX) shares are trading down 17% after hours following news of public offering of $60m of common stock, preferred stock and warrants.

Stemline Therapeutics, Inc. (Nasdaq:STML) also announced after hours a public offering of 5m shares.

MeiraGTx Holdingsplc (Nasdaq:MGTX) another company to announce an offering, noted it will offer $75m of its ordinary shares.

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

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

ADVANCERS:

Morphic Holding, Inc. (MORF): $29.05; +24%.

Neos Therapeutics, Inc. (NEOS): $1.46; +22%.

TherapeuticsMD, Inc. (TXMD): $2.26; +21%.

Assembly Biosciences, Inc. (ASMB): $14.25; +20%.

Miragen Therapeutics, Inc. (MGEN): $1.57; +18%.

DECLINERS:

AxoGen, Inc. (AXGN): $12.44; -29%.

Curis, Inc. (CRIS): $1.81; -22%.

Xencor, Inc. (XNCR): $37.00; -20%.

Codexis, Inc. (CDXS): $14.26; -16%.

Supernus Pharmaceuticals, Inc. (SUPN): $28.31; -15%.

Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

ATNX – Athenex Inc.
Oraxol
Metastatic breast cancer

Phase 3 Phase 3 top-line data met primary endpoint (ORR), secondary endpoints (PFS/OS) yet to hit. Presentation due at SABCS December 13, 2019.
$1.4 billion

AZN – Astrazeneca PLC
Lynparza
Castration-Resistant Prostate Cancer

Phase 3 Phase 3 trial met primary endpoint. Presentation at ESMO 2019 noted PFS 7.4 months vs. 3.6 months - standard of care.
$125.1 billion

BHC – Bausch Health Companies Inc.
IDP-123
Psoriasis

PDUFA PDUFA date December 22, 2019.
$10.3 billion

BPMC – Blueprint Medicines Corporation
Avapritinib BLU-285
PDGFRα driven Gastrointestinal stromal tumors (GIST) - 4th line

PDUFA PDUFA date under priority review February 14, 2020. Likely to be extended due to requested data from Voyager trial (2Q 2020).
$3.9 billion

MDWD – MediWound Ltd.
NexoBrid
Severe burns

BLA Filing BLA filing due 2Q 2020.
$77.7 million

MGTX – MeiraGTx Holdings plc
AAV-CNGA3
Achromatopsia

Phase 1/2 Phase 1/2 trial initiation announced August 7, 2019.
$776.8 million

PRQR – ProQR Therapeutics N.V.
Sepofarsen (QR-110 ) ILLUMINATE
Leber's Congenital Amaurosis (LCA) - Genetic blindness

Phase 2/3 Phase 2/3 data due 1H 2021.
$323.4 million

PTGX – Protagonist Therapeutics Inc.
PTG-300
Beta-thalassemia

Phase 2 Phase 2 preliminary data released December 3, 2019. Further data due 2020.
$209.2 million

RYTM – Rhythm Pharmaceuticals Inc.
Setmelanotide
POMC deficiency obesity

Phase 3 NDA filing due late 4Q 2019 / early 1Q 2020.
$1.1 billion

RYTM – Rhythm Pharmaceuticals Inc.
Setmelanotide
Leptin Receptor Deficiency Obesity

NDA Filing NDA filing due late 4Q 2019 / early 1Q 2020.
$1.1 billion

TRVN – Trevena Inc.
Oliceridine (TRV130)
Moderate to severe acute pain

NDA Filing CRL issued November 2, 2018. NDA to be resubmitted 1Q 2020.
$65.5 million