Attention Biotech Investors: September Ushers In Another Slew Of PDUFA Catalysts

Biotech stocks had a somber performance in August, as they reacted to earnings news flow, FDA decisions and M&A news, among other catalysts. The iShares NASDAQ Biotechnology Index (ETF) IBB is nearly flat, clocking in a loss of a 0.69 percent thus far this month.

Meanwhile, the NYSE ARCA BIOTECH INDEX has fared better with a 1.72 percent gain.

As recently as this week, Gilead Sciences, Inc. GILD announced a deal to buy Kite Pharma Inc KITE for $11.9 billion, as it strives to diversify away from its core area and into the immune-oncology space.

August's Torrent Of Events Now Past

• Bristol-Myers Squibb Co BMY opened the scorecard for the month, with the FDA granting accelerated approval for its Opdivo in dMMR metastatic colorectal cancer that has progressed following treatment with fluoropyrimidine, Oxaliplatin and Irinotecan. The approval was granted based on findings from a phase 2 trial.
• Celgene Corporation CELG and Agios Pharmaceuticals Inc AGIO's Idhifa (Enasidenib) was approved on Aug. 1 for treating adult patients with relapsed or refractory acute myeloid leukemia who have a specific genetic mutation.
• On Aug. 3, the FDA approved AbbVie Inc ABBV's Mavyret (Glecaprevi and Pibrentasvir ) for treating adults with hepatitis C virus genotypes 1-6 without cirrhosis or with mild cirrhosis. The treatment regimen lasts for eight weeks as opposed to the previous standard treatment length of at least 12 weeks.
• Meanwhile, ahead of the Aug.10 PDUFA action date, Dynavax Technologies Corporation DVAX announced that the FDA has requested more detailed information on its proposed post-marketing study of Heplisav-B, a Hepatitis B vaccine, based on a feedback received from FDA's Vaccines and Related Biological Products Advisory Committee. The committee had voted 12 to 1 to support licensure. The company now sees the new PDUFA action date to be no later than Nov. 10, 2017, as the response to the information request will trigger a major amendment to the BLA. Despite the setback, the company said it continues to target an early 2018 U.S. commercial launch.
• Pfizer Inc. PFE received approval for Besponsa (inotuzumab ozogamicin), the first and only CD22-directed antibody drug conjugate meant to treat adults with relapsed or refractory B-cell precursor acute lymphoblastic leukemia. The treatment option was reviewed under the FDA's Breakthrough Therapy designation and Priority Review programs.
• Meanwhile, Valeant Pharmaceuticals Intl Inc VRX received a complete response letter from the FDA, regarding its NDA for latanoprostene bunod, an intraocular-pressure lowering single-agent eye drop meant to treat open angle glaucoma or ocular hypertension. The CRL was issued on Aug. 7 and followed an inspection at the company's Bausch + Lomb manufacturing facility in Tampa, Florida.
• Adamas Pharmaceuticals Inc ADMS's GOCOVRI was approved for treating dyskinesia in patients with Parkinson's disease receiving levodopa-based therapy, with or without dopaminergic medications.
• Novo Nordisk A/S (ADR) NVO received FDA approval for Victoza (liraglutide) as the only type 2 diabetes treatment, which reduces the risk of cardiovascular death.

See also: Playing The US Pharma Space: Buy Portola, Hold Corvus, Sell Teva

2017 Seeing Strong Pace Of NME Approvals

New Molecular Entities approvals thus far this year have numbered 29, with three NME approvals coming through in August alone. In comparison, the year 2016 saw only 22 NME approvals. NMEs contain active ingredients, which have not been previously approved by the FDA.

AbbVie's Mavyret, Pfizer's Besponsa, and Celgene and Agios' Idhifa were the NME's approved in August.

A Peek Into September FDA Decisions

Mylan's Race To Bring Out First Breast Cancer Drug Biosimilar

Company name(s): Mylan N.V. MYL And India's Biocon

• Type of Application: BLA, filing through the 351(k) pathway.
• Drug/Treatment Candidate: Trastuzumab Biosimilar.
• Indication: HER2-positive breast cancers.
• Date: Sept. 3

Trastuzumab, sold under the brand name Herceptin, is a monoclonal antibody meant to treat breast cancer that is HER2 positive. Developed by Roche Holding Ltd. (ADR) RHHBY's Genentech unit, it is administered as a slow injection into a vein.

Herceptin fetched Roche revenues of $6.79 billion in 2015, although sales are likely to dwindle to $3.98 billion in 2022, given the likelihood of generics entering the fray. The U.S. patent on it is set to expire by 2019.

On July 13, FDA's Oncologic Drugs Advisory Committee recommended approval of the biosimilar, voting 16-0 in support of eligible indications of the reference product.

Allergan And Amgen's Quest For Multiple-indication Cancer Biosimilar

Company name(s): Allergan plc Ordinary Shares AGN and Amgen, Inc. AMGN

• Type of Application: BLA.
• Drug/Treatment Candidate: ABP 215.
• Indication: Oncology.
• Date: Sept. 14.

The Allergan-Amgen ABP 215 is a biosimilar candidate to bevacizumab sold under the brand name Avastin. It is developed by Genentech. Avastin is used to treat a host of cancers, including unresectable, locally advanced, recurrent or metastatic non-squamous NSCLC, as well as metastatic carcinoma of the colon or rectum, metastatic renal cell carcinoma, and other region-specific indications.

Bevacizumab is a recombinant immunoglobulin G1 monoclonal antibody that binds to vascular endothelial growth factor, or VEGF, and inhibits the interaction of VEGF with its receptors, VEGF receptor-1 and VEGF receptor-2, thus inhibiting the establishment of new blood vessels necessary for the maintenance and growth of solid tumors.

Neos Looking To Bulk Up ADHD Portfolio

Company name(s): Neos Therapeutics Inc NEOS

• Type of Application: NDA.
• Drug/Treatment Candidate: NT-0201.
• Indication: Attention Deficit Hyperactivity Disorder.
• Date: Sept. 15.

Neos' NT-0201 is an amphetamine XR liquid suspension used to treat ADHD.

Glaxo, Innoviva's 3-as-1 Combo Awaits FDA Scrutiny

Company name(s): GlaxoSmithKline plc (ADR) GSK and Innoviva Inc INVA

• Type of Application: NDA.
• Drug/Treatment Candidate: Closed triple combination therapy fluticasone furoate/umeclidinium/vilanterol.
• Indication: Chronic obstructive pulmonary disease.
• Date: Sept. 21.

The regulatory filing for the combo therapy was filed in November 2016, much ahead of the planned first-half 2018 filing.

J&J Unit Expects FDA To Overrule Panel Recommendation

Company name(s): Johnson & Johnson JNJ Unit Janssen Pharma

• Type of Application: BLA.
• Drug/Treatment Candidate: Sirukumab.
• Indication: Rheumatoid Arthritis.
• Date: Sept. 23.

The BLA for this human monoclonal antibody was filed with the FDA on Sept. 23, 2016. However, in a major setback, FDA's Arthritis Advisory Committee, which met on Aug. 2, voted against recommending it to the FDA.

Bristol-Myers Squibb Seeks Versatility For Opdivo

Company name(s): Bristol-Myers Squibb

• Type of Application: sBLA.
• Drug/Treatment Candidate: Opdivo.
• Indication: treating hepatocellular carcinoma after prior sorafenib therapy.
• Date: Sept. 24

IntelliPharmaCeutics Bets Against FDA Panel Verdict

Company name(s): IntelliPharmaCeutics Intl Inc (USA) IPCI

• Type of Application: sBLA.
• Drug/Treatment Candidate: Rexista.
• Indication: treating: Abuse and alcohol-deterrent treatment for pain.
• Date: Sept. 25.

Rexista is an abuse-deterrent oxycodone hydrochloride extended release tablet meant to treat pain. However, the Anesthetic and Analgesic Drug Products Advisory Committee and Drug Safety and Risk Management Advisory Committees of the FDA, which met on July 26, voted 22 to 1 against approval of the drug.