BPC October 30 update

Agile AGRX receives positive vote from Advisory Committee; Cyclerion CYCN slumps on double failure of mid-stage trials

Price and Volume Movers

Agile Therapeutics, Inc. (NASDAQ:AGRX) announced that the FDA Bone, Reproductive and Urologic Drugs Advisory Committee voted 14-1, with one abstention, that the benefits of Twirla in the prevention of pregnancy outweigh the risks to support approval. The PDUFA date for a final decision by the FDA is November 16, 2019. Shares initially slumped following the release of briefing documents on Monday for the meeting, with shares sliding 67% from $1.11 to $0.37 in the two days leading up to the panel. Trading of the stock has yet to resume after hours since the start of the trading session.

Cyclerion Therapeutics, Inc. (Nasdaq: CYCN) shares slumped to close down 80% to $2.75 following news that it did not meet the primary endpoints in two Phase 2 trials, of praliciguat, in heart failure with preserved ejection fraction (HFpEF) and in diabetic nephropathy, respectively.

Novartis (NYSE: NVS) announced the FDA placed a partial hold on clinical trials for intrathecal administration of Zolgensma (AVXS-101) following findings from a pre-clinical trial in which animal findings showed dorsal root ganglia (DRG) mononuclear cell inflammation, sometimes accompanied by neuronal cell body degeneration or loss. This partial hold by the FDA does not impact marketed Zolgensma its intravenous (IV) clinical trials. Regenxbio Inc. (NASDAQ: RGNX), which is entitled to milestone payments and royalties for Zolgensma , saw its shares close down 12% to $34.54 on the news.

AMAG Pharmaceuticals, Inc. (NASDAQ: AMAG) shares closed down 22% to $10.35 following a negative vote from an Advisory Committee reviewing its marketed treatment of Makena for pre-term births. The panel voted 9-7 in favor of recommending the withdrawal of Makena from the market, following a post-approval trial that showed the treatment not to be effective. Makena was originally approved in 2011. The FDA generally follows an advisory committee's recommendation but is not bound to do so.

IVERIC bio, Inc. (NASDAQ: ISEE) shares closed up a further 66% to $3.56 for a 3-day gain of 283%. The company announced Monday its Phase 2b trial of Zimura met the primary endpoint in reducing the rate of geographic atrophy (GA) growth in patients with dry age-related macular degeneration (AMD).

Catalyst Pharmaceuticals, Inc. (Nasdaq:CPRX) announced that its Phase 3 CMS-001 trial evaluating amifampridine phosphate for the symptomatic treatment of genetically confirmed Congenital Myasthenic Syndromes (CMS) in adults and children aged 2 years and above, did not meet its primary endpoint of subject global impression (SGI). Shares closed down 13% to $4.96.

COCRYSTAL PHARMA, INC. (NASDAQ: COCP) announced it intends to offer shares of its common stock for sale in an underwritten public offering. Shares are trading down 23% to $0.88 after hours.

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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:

Seneca Biopharma, Inc / Neuralstem, Inc. (CUR): $1.83; +40%.

Oncolytics Biotech Inc. (ONCY): $1.16; +29%.

Clearside Biomedical, Inc. (CLSD): $1.20; +18%.

Fortress Biotech, Inc. (FBIO): $1.47; +10%.

Epizyme, Inc. (EPZM): $11.56; +10%.

DECLINERS:

Iterum Therapeutics plc (ITRM): $2.89; -17%.

Cardiovascular Systems, Inc. (CSII): $43.43; -17%.

Sesen Bio, Inc. (SESN): $1.20; -15%.

Verastem, Inc. (VSTM): $1.08; -14%.

Y-mAbs Therapeutics, Inc. (YMAB): $28.74; -10%.

Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

ACAD – ACADIA Pharmaceuticals Inc.
Trofinetide
Rett Syndrome

Phase 3 Phase 3 trial initiation announced October 30, 2019.
$7.3 billion

AGRX – Agile Therapeutics Inc.
Twirla
Contraceptive patch

PDUFA CRLs issued 2013 and 2017. New PDUFA extended by three months to February 16, 2020. Advisory Committee Meeting October 30, 2019 voted 14 to 1 in favor.
$103.2 million

ALKS – Alkermes plc
Diroximel fumarate (BIIB098)
Multiple sclerosis (MS)

Approved FDA Approval announced October 30, 2019.
$3.2 billion

ALPN – Alpine Immune Sciences Inc.
ALPN-101
Healthy volunteers

Phase 1 Phase 1 presentation at ASH December 8, 2019: 11:00 AM ET.
$56.7 million

CPRX – Catalyst Pharmaceuticals Inc.
Firdapse
Congenital Myasthenic Syndromes (CMS)

Phase 3 Phase 3 trial did not meet primary endpoint - October 30, 2019.
$448.3 million

CYCN – Cyclerion Therapeutics Inc.
IW-1973 (Praliciguat)
Diabetic nephropathy

Phase 2 Phase 2 trial did not meet primary endpoint - October 30, 2019.
$58.4 million

CYCN – Cyclerion Therapeutics Inc.
IW-1973 (Praliciguat)
Heart failure HFpEF

Phase 2 Phase 2 trial did not meet primary endpoint - October 30, 2019.
$58.4 million

EPZM – Epizyme Inc.
Tazemetostat
Follicular lymphoma

Phase 2 NDA filing due December 2019. Updated Phase 2 data due at ASH 2019.
$1.5 billion

EXEL – Exelixis Inc.
CABOMETYX (cabozantinib) with Opdivo and Yervoy - CheckMate 9ER
First-line renal cell carcinoma

Phase 3 Phase 3 data due early 2020.
$5 billion

KLDO – Kaleido Biosciences Inc.
KB195
Urea cycle disorder (UCD)

Phase 2 Phase 2 data due mid-2020.
$220.4 million

PTGX – Protagonist Therapeutics Inc.
PTG-300
Polycythemia vera

Phase 2 Phase 2 initiation announced October 30, 2019.
$275.6 million

RTRX – Retrophin Inc.
Sparsentan - DUPLEX
Focal segmental glomerulosclerosis (FSGS)

Phase 3 Phase 3 top-line data due 1H 2021.
$579.5 million

VTVT – vTv Therapeutics Inc.
Azeliragon
Mild Alzheimer’s disease with type 2 diabetes

Phase 2/3 Phase 2/3 top-line data due late 2020 or early 2021.
$91.7 million