BPC May 11 update

Biotech week in review; Week ahead with - Earnings, ASCO and EHA abstracts and other catalysts

Weekly watchlist

Heavy news flow from companies reporting first quarter earnings dominated the past week, with over 200 companies reporting during just a three-day window from Tuesday through Thursday. While many individual biotech traders tend to focus on clinical data readouts and FDA approval PDUFA date catalysts, earnings season provides just as worthy a catalyst, particularly for companies with FDA approved drugs generating revenue.

Most companies left to report will do so by Wednesday, of which some are noted below. However, most revenue earning companies have already reported so do not expect the large swings that we saw this latest week.

Head of the earnings list to the downside last week was Puma Biotechnology, Inc. (NASDAQ: PBYI), which saw its shares close Friday down 39% to $18.42 following an earnings miss. The company recorded net Nerlynx revenue for the quarter of $46m, compared to consensus of approximately $66m.

On the positive side of the ledger, Flexion Therapeutics, Inc. (NASDAQ: FLXN) shares put together a two-day gain of 30% to close Friday at $12.56. The company recorded first quarter revenue of $10.6m but did note that revenue just for April is estimated at $5.1m, suggesting for a much-improved performance for the second quarter. To put the gain in some perspective, however, shares were trading at around $28 this time last year.

In regulatory news, Catalyst Pharmaceuticals Inc (NASDAQ: CPRX) shares slumped to close the week down 49% to $3.14, following a decision by the FDA to approve Ruzurgi, to be marketed by Jacobus Pharmaceutical Company, Inc, for the treatment of Lambert-Eaton myasthenic syndrome (LEMS) in children. Catalyst currently markets Firdapse in adults with LEMS and had a monopoly on the market until the approval.

uniQure N.V. (NASDAQ: QURE) shares closed Friday up 7% to $61.10 following the release of updated data from its Phase 2b trial of AMT-061, for the treatment of patients with severe and moderately severe hemophilia B. Two of the three patients exhibited Factor IX (FIX) activity in the normal range, with mean FIX activity for the three patients at six months after administration of 47% (51%, 33% and 57%) of normal. An ongoing Phase 3 trial is slated to complete enrolment by the end of this year and has a current estimated primary completion date of March 2020.

FibroGen, Inc. (NASDAQ:FGEN) shares closed Friday down 20% to 36.39 after it, together with AstraZeneca (NYSE:AZN), announced top-line results from their safety trial of roxadustat, for the treatment of anemia in chronic kidney disease (CKD) patients. Major adverse cardiac events (MACE) were measured in patients taking roxadustat compared with placebo in non-dialysis-dependent (NDD) patients and roxadustat compared with epoetin alfa in dialysis-dependent (DD) patients. In order for roxadustat to be awarded approval, regulators need to be convinced that it is safer option compared with the current standard of care. However, the company noted "based on the MACE safety analyses of this population, we believe there is no clinically meaningful difference in risk of MACE between roxadustat and epoetin alfa". The lack of clarity in the press release and following conference call led some to consider that regulators might call into question the safety of roxadustat.

Biotech companies scheduled to report first quarter earnings:




With earnings season drawing to a close, much attention next week will be on the release of abstracts on Wednesday May 15 at 5pm ET for next month’s ASCO (American Society of Clinical Oncology) meeting. Official abstracts will be placed on the meeting website, with further data likely to be released by companies at the meeting itself. The cut-off date for inclusion of data in the abstracts was February 12, 2019. Once abstracts are released a more defined list of stocks to watch will be listed, but for the time being a selection of abstracts to look out for is listed below. On a related note, abstracts for the European Hematology Association (EHA) meeting will be released the following day at 15:00 CEST, a couple of such stocks are included below together with other events slated to occur next week.

Drug Stage Catalyst Market Cap

ACHN – Achillion Pharmaceuticals Inc.
Danicopan (ACH-4471)
Paroxysmal nocturnal hemoglobinuria (PNH)

Phase 2 Phase 2 full data due 4Q 2019.
$877.2 million

AMGN – Amgen Inc.
AMG 510
Solid tumors

Phase 1 Phase 1 update at ESMO noted 54% PR rate for NSCLC patients (960mg); 1/12 for CRC patients.
$121.9 billion

ARQL – ArQule Inc.
ARQ 531
B-cell malignancies

Phase 1 Phase 1 data at EHA June 14, 2019 noted 66% (4/6) ORR in CLL patients.
$1.1 billion

BPMC – Blueprint Medicines Corporation
RET-altered solid tumors

Phase 1 Phase 1 updated data at ASCO 2019 - 60% ORR in post-platinum RET-fusion NSCLC and 63% ORR in RET-mutant MTC patients. NDA filing due 1Q 2020.
$3.6 billion

CLDX – Celldex Therapeutics Inc.
Recurrent/metastatic head and neck squamous cell cancer

Phase 2 Phase 2 data ASCO June 1, 2019 noted 2/19 CRs + one PR.
$36 million

MDCO – The Medicines Company
Inclisiran - ORION 3
Cardiovascular disease (ASCVD)

Phase 2 Phase 2 open-label data presented May 18, 2019. 51% decrease in LDL-C levels.
$4.3 billion

MGNX – MacroGenics Inc.
Margetuximab SOPHIA study
Metastatic breast cancer

Phase 3 Second interim overall survival analysis and BLA filing due 4Q 2019.
$545.7 million

MRTX – Mirati Therapeutics Inc.
Solid tumors

Phase 1/2 Phase 1/2 update due 4Q 2019. Possibly at AACR-NCI-EORTC October 26-29, 2019.
$3.2 billion

REGN – Regeneron Pharmaceuticals Inc.
Nonproliferative Diabetic Retinopathy

Approved FDA Approval announced May 13, 2019.
$32.9 billion

REGN – Regeneron Pharmaceuticals Inc.
Non-Hodgkin Lymphoma (NHL)

Phase 1 Phase 2 trial to commence June 2019. Updated Phase 1 data EHA June 14, 2019 noted 93% ORR in follicular lymphoma, 57% ORR in DLBCL.
$32.9 billion

XOMA – XOMA Corporation
Non-muscle invasive bladder cancer (NMIBC)

BLA Filing BLA filing due 4Q 2019.
$159.8 million