BPC March 02 update

Key biotech stock catalysts to watch for March 2019; Biotech stock week in review

Weekly watchlist

A huge week of price moving biotech stock news was dominated by a number of buyouts, clinical readouts, capped off by a plateful of companies reporting 4Q earnings.

On the earnings front, Puma Biotechnology, Inc. (NASDAQ: PBYI) was a major mover, closing Friday up 40% to $38.93 on the back of improved 4Q earnings, with sales of Nerlynx, its primary revenue source, reaching $61.1m compared with $20.1m in 4Q 2017.

In regulatory news, Lexicon Pharmaceuticals, Inc. (NASDAQ: LXRX) announced the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion on the Marketing Authorization of sotagliflozin. Shares of Lexicon closed up 28% to $6.81 on the news, with investors seeing the news as a positive leading to the FDA PDUFA date of March 22, 2019. As a reminder, an FDA Advisory Committee panel split their vote 8-8 in recommending approval, leaving uncertainty of the final approval decision.

ImmunoGen, Inc., (Nasdaq: IMGN) shares closed Friday down 47% to $2.51 following news its Phase 3 trial evaluating mirvetuximab soravtansine compared to chemotherapy in patients with ovarian cancer did not meet its primary endpoint of progression-free survival (PFS).

TG Therapeutics, Inc. (NASDAQ: TGTX) shares closed the week up 51% following news the Marginal Zone Lymphoma (MZL) cohort of the UNITY-NHL Phase 2b pivotal trial evaluating umbralisib (TGR-1202), met the primary endpoint of Overall Response Rate (ORR).

Horizon Pharma plc (Nasdaq: HZNP) shares closed the week up 37% to $28.18 on news its Phase 3 trial evaluating teprotumumab for the treatment of active thyroid eye disease (TED) met its primary endpoint.

Novavax, Inc. (Nasdaq: NVAX) shares plunged to close Thursday down 67% to $0.71 following news its Phase 3 Prepare trial of ResVax, being developed to protect infants via maternal immunization against respiratory syncytial virus (RSV) disease, did not meet the primary endpoint.

Celgene Corp (Nasdaq: CELG) shares closed Thursday down 9% to $83.12 on news that the top shareholder of Bristol-Myers Squibb Co (NYSE: BMY), Wellington Management, does not support the takeover of Celgene Corp (Nasdaq: CELG), announced last month.

Karyopharm Therapeutics Inc. (Nasdaq:KPTI) announced that the FDA Oncologic Drugs Advisory Committee (ODAC) voted 8 to 5 recommending that the FDA wait for the results from its Phase 3 BOSTON trial, evaluating selinexor in patients with relapsed or refractory multiple myeloma, before making a final decision regarding approval. Top-line data from the BOSTON trial are expected by the end of 2019 at the earliest, or into 2020. Shares closed the week down 17% to $4.18, adding to last Friday’s loss of 43% when briefing notes were released.

Clementia Pharmaceuticals (NASDAQ: CMTA) announced that it will be acquired by Ipsen (Euronext: IPN; ADR: IPSEY) for $25 per share,or $1.04b, plus a contingent value right (CVR) of $6 per share upon FDA acceptance of the NDA filing for palovarotene for the treatment of Multiple Osteochondroma, representing an additional potential payment of $263m. Shares closed Monday up 74% to $26.06.

Spark Therapeutics (NASDAQ: ONCE) announced that it will be acquired by Roche (RHHBY) for $114.50 per share, or $4.8b. Shares soared to close Monday up 120% to $113.48. Shares of other gene-therapy companies reacted positively to the news with Uniqure NV (NASDAQ: QURE) closing up 34% to $55.87.


Selection of Biotech Stock Earnings to watch next week (10):

Mon: BCRX LJPC

Tue: FATE ZIOP

Wed: JNCE MRNA

Thu: ACRX SNDX

Fri: ALLO CNAT

13 key biotech stock catalysts to watch in March:

Drug Stage Catalyst Market Cap

AERI – Aerie Pharmaceuticals Inc.
Roclatan
Glaucoma

Approved FDA Approval announced March 12, 2019.
$1.9 billion

ALDX – Aldeyra Therapeutics Inc.
Reproxalap (ADX-102)
Allergic conjunctivitis

Phase 3 Phase 3 data March 25, 2019 - primary endpoint met.
$201 million

ALNY – Alnylam Pharmaceuticals Inc.
Givosiran
Acute hepatic porphyrias

NDA Filing Rolling NDA filing to be completed mid-2019.
$7.2 billion

ARPO – Aerpio Pharmaceuticals Inc.
AKB-9778 - TIME 2B
Non-Proliferative Diabetic Retinopathy

Phase 2 Phase 2 data released March 18, 2019 did not meet primary endpoint.
$40.6 million

ASND – Ascendis Pharma A/S
TransCon hGH
Growth hormone deficiency in children

Phase 3 Phase 3 data released March 4, 2019. Primary endpoint met. Phase 3 fliGHt trial detailed data due June 26, 2019.
$5.9 billion

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

Phase 3 Phase 3 data due 2H 2019.
$786 million

DERM – Dermira Inc.
Lebrikizumab
Atopic Dermatitis

Phase 2b Phase 2b data released March 18, 2019 met primary endpoints. Phase 3 trial to be initiated later in 2019.
$550.1 million

GLPG – Galapagos NV
Filgotinib - FINCH 1
Rheumatoid arthritis (RA)

Phase 3 Phase 3 data released March 28, 2019 - primary endpoint met.
$6.4 billion

LXRX – Lexicon Pharmaceuticals Inc.
Sotagliflozin
Type 1 Diabetes

CRL CRL issued March 22, 2019.
$645.1 million

NBIX – Neurocrine Biosciences Inc.
NBI-74788
Congenital Adrenal Hyperplasia (CAH) - adults

Phase 2 Pivotal trial to be initiated 2H 2019 pending talks with FDA.
$7.2 billion

PRTO – Proteon Therapeutics Inc.
Vonapanitase (PRT-201) PATENCY-2
Chronic kidney disease (CKD) patients undergoing surgical placement of an arteriovenous fistula (AVF)

Phase 3 Phase 3 data released Match 28, 2019 did not meet primary endpoint.
$8.1 million

PTI – Proteostasis Therapeutics Inc.
PTI-428 + PTI-801 + PTI-808
Cystic fibrosis

Phase 2 Phase 2 trial planned with enrolment to be completed by YE 2019.
$58.3 million

REPH – Recro Pharma Inc.
Intravenous (IV) meloxicam
Acute pain following bunionectomy surgery

CRL First CRL announced May 24, 2018. Second CRL issued March 22, 2019.
$202.1 million

RYTM – Rhythm Pharmaceuticals Inc.
Setmelanotide
POMC Heterozygous Deficiency Obesity, Alstrom Syndrome, POMC Epigenetic Disorders

Phase 2 Phase 2 additional data due 2020.
$956.2 million

SAGE – Sage Therapeutics Inc.
Brexanolone - SAGE-547 (202C)
Postpartum Depression - moderate

Approved FDA approval announced March 19, 2018.
$8.7 billion

SGEN – Seattle Genetics Inc.
Enfortumab vedotin - EV201
Urothelial cancer

Phase 3 Phase 2 pivotal data March 28, 2019 noted 44% ORR. Full data due at ASCO June 3, 2019, 9:24am.
$10.8 billion

UROV – Urovant Sciences Ltd.
Vibegron
Overactive bladder (OAB)

Phase 3 Phase 3 data released March 18, 2019 - primary endpoints met.
$252.6 million

UTHR – United Therapeutics Corporation
Esuberaprost - BEAT
Pulmonary arterial hypertension (PAH)

Phase 3 Phase 3 data released April 8, 2019 did not meet primary endpoint.
$3.9 billion