BPC August 24 update

Biotech stock events to watch for the remainder of 3Q 2019; Biotech week in Review

Weekly watchlist

The latest biotech trading week was dominated with Complete Response Letters (non-approval letters) issued by the FDA to two companies while another gave notice it also anticipates being on the receiving end by its PDUFA date in October.

We review these decisions and other price moving news in what was largely a week of negative news for the sector.

Sarepta Therapeutics, Inc. (NASDAQ:SRPT) announced Monday it was issued a Complete Response Letter (CRL) from the FDA regarding its New Drug Application (NDA) of golodirsen injection for the treatment of Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation amenable to exon 53 skipping. The CRL noted concerns regarding the risk of infections related to intravenous infusion ports and renal toxicity seen in preclinical trials. Shares closed the week down 25% to $93.61.

Vanda Pharmaceuticals Inc. (Nasdaq: VNDA) also announced that the FDA issued a Complete Response Letter (CRL) for the supplemental New Drug Application (sNDA) for Hetlioz (tasimelteon) for the treatment of Jet Lag Disorder (JLD). In the CRL, the FDA asserted that measures demonstrating improved sleep in its previous trials were unclear clinical significance.

Clearside Biomedical, Inc. (Nasdaq:CLSD) announced that it expects to receive a Complete Response Letter from the FDA for its New Drug Application (NDA) for Xipere (triamcinolone acetonide suprachoroidal injectable suspension) by its PDUFA date of October 19, 2019. Clearside noted the FDA has requested stability data for its new manufacturing process, which it plans to provide with its resubmitted NDA in 1Q 2020. Shares closed the week down 40% to $0.71.

Endo International plc (NASDAQ: ENDP) announced that it has reached a settlement with Cuyahoga and Summit counties to pay $10m and will provide up to $1m of its Vasostrict and Adrenalin products free of charge. The settlement will avoid the company from going to trial, after being accused of contributing to the recent opioid epidemic. Shares closed the week up 28% to $2.90.

Retrophin, Inc. (NASDAQ: RTRX) shares closed the week down 27% to $13.07 following news its Phase 3 FORT trial evaluating fosmetpantotenate compared to placebo in patients with pantothenate kinase-associated neurodegeneration (PKAN) did not meet its primary endpoint and did not demonstrate a difference between treatment groups.

Synlogic, Inc., (Nasdaq: SYBX) shares closed the week down 29% to $3.27 following news that it will discontinue development of SYNB1020, for the treatment of hyperammonemia. The decision follows interim analysis from its Phase 1b/2a trial which showed there was no evidence of blood ammonia lowering or changes in other endpoints relative to placebo.

Biotech stock events to watch for remainder of 3Q 2019:

Drug Stage Catalyst Market Cap

ADVM – Adverum Biotechnologies Inc.
Wet age-related macular degeneration (Wet-AMD)

Phase 1 Phase 1 presentation of 24-week data from the second cohort and an update from first cohort of patients in the OPTIC trial due at the Angiogenesis Meeting, February 8, 2010 at 2:28 pm EST.
$701.4 million

AIMT – Aimmune Therapeutics Inc.
Peanut Allergy

PDUFA Advisory Committee meeting September 13, 2019 voted in favor of supporting approval. PDUFA date late-January 2020.
$2.1 billion

ALNY – Alnylam Pharmaceuticals Inc.
Inclisiran - ORION 11
Cardiovascular disease (ASCVD)

Phase 3 Phase 3 trial met all primary and secondary efficacy endpoints. Noted September 2, 2019 a 54% LDL-C lowering after 11 months and 50% over 18 months.
$13.2 billion

ANAB – AnaptysBio Inc.
Generalized pustular psoriasis

Phase 2 Phase 2 interim data on first TWO patients announced September 30, 2019 with patients reaching primary endpoint. Further data due 2020.
$387.7 million

AVEO – AVEO Pharmaceuticals Inc.
TIVO-3 - tivozanib
Third line treatment of patients with renal cell cancer

NDA Filing NDA to be filed in 1Q 2020. Will withdraw if overall survival data in 2Q 2020 yields an OS HR above 1.00.
$83.6 million

AZRX – AzurRx BioPharma Inc.
Cystic fibrosis

Phase 2 Phase 2 higher dose trial to be initiated early 2020 with data due mid-2020.
$26.5 million

ENTA – Enanta Pharmaceuticals Inc.
Non-alcoholic steatohepatitis (NASH)

Phase 2 Phase 2b trial to be initiated 2Q 2020.
$1 billion

MRNS – Marinus Pharmaceuticals Inc.
Refractory status epilepticus (RSE)

Phase 2 Phase 3 trial to be initiated mid-2020.
$179.5 million

NERV – Minerva Neurosciences Inc
MIN-202 (seltorexant) vs quetiapine
Major Depressive Disorder

Phase 2 Phase 2b data noted quantitative advantage but no statistical separation between two arms. Company noted trial was not designed to detect statistical significance.
$316.5 million

NTRP – Neurotrope Inc.
Alzheimer's disease

Phase 2 Phase 2 trial did not meet primary endpoint - September 9, 2019.
$16.9 million

SNGX – Soligenix Inc.
SGX942 (dusquetide)
Oral Mucositis in Patients with Head & Neck Cancer

Phase 3 Phase 3 top-line data due 2Q 2020.
$55.6 million

URGN – UroGen Pharma Ltd.
VesiGel UGN-102
Low grade non-muscle invasive bladder cancer (LG-NMIBC)

Phase 2b Phase 3 trial planned for 2020.
$658.4 million

XERS – Xeris Pharmaceuticals Inc.
Glucagon Rescue Pen
Severe hypoglycemia

Approved FDA Approval announced September 10, 2019.
$163.3 million

ZEAL – Zealand Pharma A/S
Severe hypoglycemia in diabetes - children

NDA Filing NDA filing due early 2020.
$1.3 billion