August FDA dates

Biotech stocks had a fairly decent run up in July after a nearly flat performance in June. The iShares NASDAQ Biotechnology Index (ETF) IBB 0.51% is up about 5 percent.

As the earnings season kicks into high gear and amid several other catalysts, August is likely to be an action-packed month. Here are some key PDUFA action dates a biotech investor should stay focused on.

Pain Therapeutics Looks For Alleviation Of Pain Inflicted By Negative FDA Panel Vote

• Company: DURECT Corporation DRRX 0.68% and Pain Therapeutics, Inc. PTIE 10.27%
• Type of Application: NDA
• Candidate: Remoxy
• Indication: severe pain
• Date: August 7

Pain Therapeutics has licensed Remoxy, an abuse-deterrent extended release capsule formulation of oxycodone, from DURECT. Pain said in mid-February it resubmitted the NDA for FDA review. The previous application was issued a complete response letter in Sept. 2016. Remoxy is indicated to treat severe pain.

Meanwhile, in an adverse development, a FDA panel that met in late June to discuss the NDA voted 14 to 3 against approval of Remoxy for managing pain severe enough to require daily, around-the-clock, long-term opioid treatment and for which alternative treatment options are inadequate.

The stock, which shed about 71 percent following the FDA panel vote, hasn’t recovered.

Alnylam Awaits Approval Of Drug To Treat Hereditary ATTR Amyloidosis

• Company: Alnylam Pharmaceuticals, Inc. ALNY 0.62%
• Type of Application: NDA
• Candidate: Patisiran
• Indication: Hereditary ATTR, or hATTR, amyloidosis
• Date: August 11

Patisiran is an investigational RNAi therapeutic targeting transthyretin, or TTR, for treating hATTR amyloidosis. It has been accorded priority review status and also has a Breakthrough Therapy designation.

hATTR amyloidosis results from mutations in the TTR gene, which causes abnormal amyloid protein to accumulate and damage body organs and tissues such as peripheral nerves and heart.

Alnylam uses Arbutus Biopharma Corp ABUS 4.05%‘s intellectual property in RNAi therapeutic products using LNP technology.

Regeneron’s Twin FDA Review Events

• Company: Regeneron Pharmaceuticals Inc REGN 0.66%
• Type of Application: sBLA/sBLA
• Candidate: Eylea / Praluent with apheresis
• Indication: Wet Age-related Macular Degeneration, or AMD/treating elevated low-density lipoprotein cholesterol
• Date: August 11/ August 24

The FDA had accepted Regeneron’s aBLA for a 12-week dosing interval of Eylea in patients with wet AMD last December. Eylea is recommended in a 2mg dosage, administered as injection in the eye, every eight weeks following three initial monthly (four-week) injections.

Regeneron and partner Sanofi SA (ADR) SNY 1.22% are also knocking the FDA altar for using Praluent injection in patients with heterozygous familial hypercholesterolemia with apheresis, an invasive procedure to remove LDL cholesterol from the blood.

Praluent, chemically Alirocumab and a PCSK9 inhibitor, is used along with diet and maximally tolerated statin drugs to lower LDL cholesterol in blood.

Amicus’ Fabry’s Disease Drug Awaits FDA Approval

• Company: Amicus Therapeutics, Inc. FOLD 0.21%
• Type of Application: NDA
• Candidate: Migalastat
• Indication: Treating patients 16 years and older with Fabry disease who have amenable mutations
• Date: August 13

The FDA accepted the NDA for Migalastat, an oral precision medicine, according it priority review status. The candidate had previously received Orphan Drug as well as Fast Track designations. It has already been approved in the European Union.

Fabry’s disease is a type of lysosomal storage disorder and is an inherited disorder that results from the build up of a type of fat called globotriaosylceramide in body’s cells.

Bristol-Myers Seeks FDA Nod For Opdivo To Treat Small-Cell Lung Cancer

• Company: Bristol-Myers Squibb Co BMY 1.93%
• Type of Application: sBLA
• Candidate: Opdivo
• Indication: Small-cell lung cancer, or SCLC
• Date: August 16

The FDA accepted the sBLA for Opdivo, indicated to treat patients with SCLC, whose disease has progressed after two or more lines of therapy. The application was supported by safety and efficacy data from the SCLC cohort of the Phase 1/2 CheckMate-032 trial evaluating Opdivo monotherapy following platinum-based chemotherapy.

Mallinckrodt’s Infantile Jaundice Drug Up Before FDA

• Company: Mallinckrodt PLC MNK 0.72%
• Type of Application: NDA
• Candidate: Stannsoporfin
• Indication: Treating neonates at risk for developing severe hyperbilirubinemia, or severe jaundice
• Date: August 22

Mallinckrodt has added Stannsoporfin to its pipeline through its acquisition of privately-held InfaCare.

KALA Awaits FDA Clearance For Post-ocular Surgery Pain Treatment

• Company: Kala Pharmaceuticals Inc KALA 1.02%
• Type of Application: NDA
• Candidate: Inveltys
• Indication: inflammation and pain in patients who have undergone ocular surgery
• Date: August 24

Valeant’s Acne Treatment Candidate Under FDA Scanner

• Company: Valeant Pharmaceuticals Intl Inc NYSEVRX
• Type of Application: NDA
• Candidate: IDP-121 lotion
• Indication: For treating acne
• Date: August 27

Valeant’s Ortho Dermatologics unit announced FDA acceptance of the NDA for IDP-121 lotion, chemically tretinoin 0.05 percent, on Jan. 12. If approved, the company said it would be the first tretinoin product in lotion form rather than a gel or cream.

Tetraphase Seeks Approval For Antibiotic to Treat Abdominal Infections

• Company: Tetraphase Pharmaceuticals Inc TTPH 0.35%
• Type of Application: NDA
• Candidate: Eravacycline
• Indication: Complicated intra-abdominal infections
• Date: August 28

The NDA submission for Eravacycline, a fully-synthetic fluorocycline, was supported by data from the IGNITE1 and IGNITE 4 Phase 3 clinical trials.

Can Akcea’s Volanesorsen Pass The FDA Muster?

• Company: Akcea Therapeutics Inc AKCA 0.17%
• Type of Application: NDA
• Candidate: Volanesorsen, a RNAi drug
• Indication: Treating patients with familial chylomicronemia syndrome, or FCS
• Date: August 30

A FDA panel discussed the NDA of Akcea, a unit of Ionis Pharmaceuticals Inc IONS 0.18%, in May and voted 12-8 in favor of approving the drug.

FCS, a liposome disorder, is characterized by the buildup of fats in the body due to a deficiency in the enzyme lipoprotein lipase.

Can Second Time Be Charm For RedHill Biopharma?

• Company: REDHILL BIOPHAR/S ADR RDHL 2.32%
• Type of Application: NDA
• Candidate: RHB-104
• Indication: Acute migraines
• Date: August (estimated)

RedHill’s initial application filed in 2013 was met with a FDA complete response letter issued in Feb. 2014, citing concerns regarding third-party chemistry, manufacturing and control, and about the labeling and packaging. The company made a resubmission last November, and the resubmitted application was deemed complete.

The PDUFA date was set for the first half of 2018.

Allergan’s Uterine Fibroid Drug Awaits FDA Approval

• Company: Allergan plc AGN 0.53%
• Type of Application: NDA
• Candidate: Esmya
• Indication: Treating abnormal uterine bleeding in women with uterine fibroids
• Date: August (estimated)

The FDA extended the review period of Esmya, chemically ulipristal acetate, to August 2018 to provide time for a full review of the file.

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