BPC April 04 update

Biotech stock catalysts likely to occur in April; Biotech week in Review

Weekly watchlist

With a tumultuous first quarter behind us, all eyes will be on how both the markets and the world as a whole grapple with and get on top of the COVID-19 Coronavirus pandemic.

April is stacked with biotech stock catalysts and most attention likely will be on the Phase 3 data readout from Gilead for its Phase 3 trial of Remdesivir, in patients with COVID-19. This is a key event, not only for investors, but more importantly in our battle to assist in turning the tide of the pandemic.

We list other key catalysts likely to occur in April below. First, let’s review the week that was with price-moving events.

Johnson & Johnson (NYSE:JNJ) shares closed the week up 9% to $134.17 on news of an expansion of the existing partnership with Biomedical Advanced Research and Development Authority (BARDA) in its development of a vaccine for COVID-19. Human trials will commence by September 2020 and it anticipates the first batches of the vaccine could be available for emergency use authorization in early 2021.

Kiniksa Pharmaceuticals, Ltd. (NASDAQ:KNSA) announced that six COVID-19 patients treated with mavrilimumab in Italy all responded to treatment, and 3 patients were discharged within 5 days. A follow-on controlled study in Italy is planned. Shares closed the week up 43% to $17.91.

Akero Therapeutics, Inc. (Nasdaq: AKRO) shares closed the week up 24% to $20.00. The company announced that all three AKR-001 dose groups in the BALANCED study met the primary endpoint of absolute change from baseline in liver fat as measured by magnetic resonance imaging – proton density fat fraction (MRI-PDFF) – at week 12.

Fate Therapeutics, Inc. (NASDAQ: FATE) shares closed Friday up 9% to $22.94 on news of a collaboration with Janssen Biotech, Inc. (Johnson & Johnson) to develop novel off-the-shelf CAR NK and T-cell cancer immunotherapies. Fate will receive $50m in cash and $50m from the purchase of newly issued shares at a price per share of $31.00. Fate is also eligible to receive payments of up to $1.8b upon the achievement of development and regulatory milestones and up to $1.2b upon the achievement of commercial milestones.

Amarin Corporation plc (NASDAQ:AMRN) shares slid to close the week down 64% to $4.78 following news released of a negative patent decision which ruled in favor of the generic companies in the company's litigation against filers of abbreviated new drug applications for Amarin's Vascepa. Following the news, in a conference call with Jefferies analyst Michael Yee, a patent lawyer suggested the company has an even-money chance of winning an appeal.

Reata Pharmaceuticals, Inc. (Nasdaq: RETA) announced it will stop its Phase 3 CATALYST trial of bardoxolone methyl (bardoxolone) in patients with connective tissue disease-associated pulmonary arterial hypertension (CTD-PAH) following the recommendation of the trial’s Data Safety Monitoring Board (DSMB) which noted that the trial is unlikely to meet the primary endpoint. Shares closed the week down 14% to $128.86.

Axsome Therapeutics, Inc. (NASDAQ: AXSM) announced that its Phase 3 STRIDE-1 trial of AXS-05 for treatment resistant depression (TRD) did not meet the primary endpoint. Shares closed Monday down 9% to $57.58.

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Biotech stock catalysts likely to occur in April:

Drug Stage Catalyst Market Cap

ASND – Ascendis Pharma A/S
TransCon
Hypoparathyroidism

Phase 2 Phase 2 six-month data from the open-label extension portion due 3Q 2020. Phase 3 trial to commence 4Q 2020.
$7 billion

AXSM – Axsome Therapeutics Inc.
AXS-05 ADVANCE-1
Agitation in patients with Alzheimer’s disease (AD)

Phase 2/3 Phase 2/3 top-line met primary endpoint - April 27, 2020.
$2.9 billion

AXSM – Axsome Therapeutics Inc.
AXS-07 - INTERCEPT
Migraine

Phase 3 Phase 3 data met primary endpoints - April 6, 2020.
$2.9 billion

BPMC – Blueprint Medicines Corporation
Avapritinib - Voyager
Gastrointestinal stromal tumors (GIST)

Phase 3 Phase 3 data did not meet primary endpoint - April 28, 2020.
$3.5 billion

CANF – Can-Fite Biopharma Ltd Sponsored ADR (Israel)
Namodenoson (CF102)
Non-alcoholic steatohepatitis (NASH)

Phase 2 Phase 2 endpoints met - April 7, 2020.
$21.1 million

GILD – Gilead Sciences Inc.
Remdesivir
Coronavirus / COVID-19 - severe patients

Phase 3 Phase 3 data from open-label trial released April 29, 2020 - 10-day course achieved similar improvement compared to 5-day course.
$97.6 billion

GNFT – GENFIT S.A.
Elafibranor - RESOLVE-IT
Non-alcoholic steatohepatitis (NASH)

Phase 3 Phase 3 data did not meet primary endpoint - May 11, 2020.
$202.7 million

ITRM – Iterum Therapeutics plc
Sulopenem
Uncomplicated urinary tract infections (uUTI)

Phase 3 Phase 3 data due May 2020.
$62.6 million

KNSA – Kiniksa Pharmaceuticals Ltd.
KPL-716
Prurigo nodularis

Phase 2a Phase 2a trial met primary endpoint - April 22, 2020.
$1.2 billion

MLND – Millendo Therapeutics Inc.
Livoletide
Prader-Willi syndrome (PWS)

Phase 2b Phase 2b data did not meet primary endpoint - April 6, 2020.
$50.5 million

MNLO – Menlo Therapeutics Inc.
Serlopitant
Pruritus associated with prurigo nodularis

Phase 3 Phase 3 data did not meet primary endpoint - April 6, 2020.
$325.7 million

MYOV – Myovant Sciences Ltd.
Relugolix - SPIRIT 2
Endometriosis-associated pain

Phase 3 Phase 3 data met co-primary endpoints - April 22, 2020.
$1.1 billion

NBIX – Neurocrine Biosciences Inc.
Opicapone
Parkinson's disease

Approved FDA Approval announced April 27, 2020.
$11.6 billion

URGN – UroGen Pharma Ltd.
MitoGel - UGN-101
Urothelial Carcinoma

Approved FDA Approval announced April 15, 2020.
$514.8 million