Q3 Biotech Catalyst Watch: Key PDUFAs (Part 3)

by: Slingshot Insights


2017’s third quarter holds important data and regulatory releases with significant potential impacts on pharmaceutical share prices.

This week covers the upcoming PDUFAs investors should have on their radar. In my last two articles, I covered Phase 2 and Phase 3 data releases (linked below).

Investors informed on major market events can capitalize on their diligence. This article covers upcoming PDUFA dates for ABBV/ENTA, ADMS, CELG, DVAX, EGRX, and IPCI.

By Killian McKee, Slingshot Contributor

The third quarter of 2017 packs an abundance of Phase 2, Phase 3, and PDUFAs about which diligent pharma investors should know. If you need a PDUFA refresher, a crash course of this important pharma event occupies the next section. Over the last 2 weeks, we have been outlining some of the most prominent upcoming Phase 2 and Phase 3 catalysts to help investors develop a concise summary of the Q3 landscape. This week similarly examines upcoming PDUFA meeting dates for 7 pharma companies: AbbVie Inc. (ABBV), Enanta Pharmaceuticals (ENTA), Adamas Pharmaceuticals (ADMS), Celgene (CELG), Dynavax Technologies (DVAX), Eagle Pharmaceuticals (EGRX), and IntelliPharmaCeutics International Inc (IPCI). Additionally, you can check out the free spreadsheet I made overviewing many upcoming Q3 Phase 2, Phase 3, and PDUFA pharma catalysts here. Investors should care about these events because they will significantly impact stock prices and could provide profitable investment opportunities.

What is a PDUFA?

The Prescription Drug User Fee Act (PDUFA) allows the FDA to collect fees from drug makers to fund and expedite the approval process for pharmaceutical drugs. Investors care about PDUFA meeting dates because they represent the target day of the FDA decision to approve or not approve a new product candidate.

Before a PDUFA decision can take place, drug manufacturers must successfully clear clinical trials, submit a New Drug Application (NDA) to the FDA and consider an optional third party expert advisory committee (AdComm). Time-wise, NDA filings typically come 2-3 months after data readout, and AdComm meetings occur about 1 month before the PDUFA. PDUFA decision dates are around 10 months after the NDA submission completes, but drugs given FDA priority review designations are examined in 6 months. This is because they could address a large unmet medical need or mark a major improvement over current treatments.

The results from the clinical trial topline readout, NDA, and AdComm often snowball. If Phase 3 results are strong, the NDA submitted tends to be approved. Robust NDAs usually result in AdComm drug approval recommendations, which are mostly upheld by the FDA. Ultimately, the PDUFA outcome dramatically impacts share prices for better or worse.

PDUFAs Coming Q3

1) EGRX: Ryanodex for Exertional Heat Stroke (EHS) - 7/23/17 PDUFA

EGRX’s Ryanodex priority review PDUFA meeting happens 7/23/17. Ryanodex targets exertional heat stroke, a rare and unpredictable medical emergency characterized by excessive body heat and consequent neurological dysfunction. EHS deserves serious attention; left untreated it can be fatal, about 30% of those who suffer an episode have lasting neurological complications, and it can lead to nervous system/internal organ damage. EHS occurs most frequently in athletes, military personnel, and those active during very hot weather. About 75,000 people in the U.S. each year suffer from EHS. Currently, EHS treatments involve rapid cooling of the body with things like water, ice, or moving indoors. Although usually effective, these treatments are not always readily available and do not always cool the body quickly enough to avoid permanent damage.

Ryanodex was granted Orphan Drug Status by the FDA, so if approved it will not only be the only drug for EHS on the market, it will also have seven years of exclusive protection. Test results for Ryanodex were positive, but it is important to note the primary basis for their NDA submission relies on animal results due to the inability to test for EHS in a controlled environment reliably. Trial results indicated 58.3% of animals given Ryanodex in addition to traditional cooling methods reversed the induced exertional heat stroke compared to 9.1% of animals given traditional cooling treatments exclusively.

Ryanodex represents EGRX’s most advanced product candidate (their two others are Bendamustine RTD and Pemetrexed). Investors should have EGRX on their horizon for two reasons: firstly, Ryanodex constitutes a third of EGRX’s pipeline and the results of their PDUFA will majorly impact share price. Secondly, Ryanodex could be an orphan drug in a relatively large market and help those suddenly stricken by life threatening EHS.

2) ABBV/ENTA: Glecaprevir/Pibrentasvir (G/P) for Hepatitis C (HCV) - Estimated 8/2/2017 PDUFA

ABBV/ENTA’s codeveloped Hepatitis C drug Glecaprevir/Pibrentasvir (G/P) received priority review designation on 2/2/2017 but doesn’t yet have a publicly released PDUFA date. An 8/2/2017 PDUFA is a safe estimate for G/P based on the standard 6 month track for drugs with priority review status. G/P combines two protease inhibitors codeveloped via the ongoing collaboration between Abbvie and Enanta to cure HCV. ABBV is responsible for all worldwide commercialization of paritaprevir containing treatments for HCV, but both companies’ share price will see meaningful movement based on the results (ENTA will receive royalties).

Phase 3 topline data showed promise; G/P averaged 97.5% SVR12 (12 weeks without detectable HCV) over the 6 major HCV genotypes in patients new to treatment and without cirrhosis. Hepatitis C chronically damages the liver and afflicts about 2.7 million Americans. The disease spreads via contact with infected blood and is most commonly transmitted by drug abusers sharing needles, but can also be spread sexually or via broken skin to skin contact. This represents the majority of people with HCV. These numbers were similar to GILD’s recently approved EPCLUSA, which achieved a 98% cure rate for the same patient cohort. HCV patients with decompensated cirrhosis achieved a 98% SVR12 rate while on G/P, 4% higher than EPCLUSA. ABBV/ENTA would benefit heavily if G/P can become a primary treatment for the massively profitable HCV market.

Pipeline wise, G/P represents ENTA’s most developed product. ENTA also has a Phase 1 NASH trial in progress. More info on ABBV’s colossal pipeline available here.

3) DVAX: HEPLISAV-B Vaccine for Hepatitis B (HBV) - 8/10/17 PDUFA

DVAX’s HEPLISAV-B PDUFA takes place 8/10/17 with an AdComm set for 7/28/17. Like HBC, HBV is a serious transmissible liver infection capable of causing fatal complications if left untreated (cirrhosis and liver cancer being the most common). Unlike HBC, there are vaccines for HBV, but no cure for those who already have the disease. There are effective, albeit very expensive treatments for the approximately 1.2 million Americans (often those from Asia or Africa where vaccination is not common) who have HBV.

Phase 3 topline data for HEPLISAV-B met its primary endpoints and proved to be a faster acting, more protective vaccine than GlaxoSmithKline’s (GSK) popular HBV vaccine Engerix-B. Becoming a first line vaccination would benefit both DVAX’s trading price and those afflicted by HBV, but it remains to be seen if HEPLISAV-B will be able to overcome the issues that led to its initial FDA rejection relating to some specific adverse results and some cardiac issues during trials among other issues. Slingshot will be getting an expert’s view on this before the PDUFA here where you can add your questions for an infectious disease expert.

In terms of other products DVAX’s pipeline includes AZD1419, a Phase 2 drug being developed with AstraZeneca (AZN) for asthma. Several additional early stage products target cancers like melanoma.

4) ADMS: ADS-5102 for Levodopa-Induced Dyskinesia (LID) in Patients with Parkinson’s Disease - 8/24/2017 PDUFA

The PDUFA for ADMS’s LID drug ADS-5102 takes place 8/24/2017. Phase 3 topline data showed an approximate 30% improvement in LID patients compared to a placebo. LID is a condition associated with taking levodopa, the current gold standard treatment for Parkinson’s disease. Over time, Parkinson’s patients require greater quantities of levodopa to avoid underlying symptoms and most develop LID as a result. LID manifests itself via involuntary, nonrhythmic, and unpredictable movements. Approximately 150,000-200,000 people in the U.S. suffer from LID. A successful PDUFA would make ADS-5102 the only treatment for LID in Parkinson’s patients and represents a major positive catalyst for ADMS. Slingshot will be interviewing a neurologist with significant experience treating patients with LID before the PDUFA. You can join and add your questions for the expert here. ADS-5102 is also in 2 other trials for other indications, highlighting the drugs critical importance for ADMS’ small pipeline.

5) CELG: Enasidenib (AG-221) to Treat Relapsed/Refractory IDH2 Mutant-Positive Acute Myeloid Leukemia - 8/30/2017 PDUFA

The PDUFA for AG-221 will take place 8/30/2017. AG-221 was developed in conjunction with Agios (AGIO) to treat Acute Myelogenous Leukemia (AML) patients with the IDH2 mutation. AGIO stands to make a maximum of $120 million from the collaboration royalties. AML is a blood/bone cancer affecting over 20,000 people in the U.S. Of those 20,000, about 10-20% have the IDH2 mutation. AML’s five year survival is only about 20-25% due to the rapid progression of the disease and lack of available treatments (risky bone marrow transplants are common). AGIO presented positive Phase 3 for AG-221 early 2017 demonstrating a 40.3% progression free survival rate in patients. AG-221 would be a first in class drug and would likely become the standard of care for AML pending approval. Although CELG has a large pipeline tackling a range of diseases from cancer to inflammation, expect the outcome of AG-221’s PDUFA to move their share price meaningfully.

6) IPCI: Rexista, an Abuse Deterrent Oxycodone - 9/25/17 PDUFA

IPCI’s Rexista PDUFA meeting takes place 9/25/17. Rexista’s abuse deterrent oxycodone formulation targets the massive $2.1 billion oxycodone market. There are countless prescription painkillers available, but the potential for abuse has led drug makers to pursue abuse deterrent formulations, especially for oxycodone. When taken at higher doses oxycodones are very effective, but can also induce a habit forming euphoric state. Studies from 2013 estimate about 500,000 people abuse oxycodone a month. Clearly, an abuse resistant oxycodone could become a major feature in the pain management market.

The FDA provides a list of 10 abuse deterrent opioids we can view as direct competition to Rexista in the profitable painkiller market. Rexista stacks up strongly based on Phase 3 results. When crushed, Rexista coagulates into a viscous gel to prevent snorting or injecting and emits a stigmatizing blue dye. Additionally, Rexista is apparently difficult to inhale causing burning and trial results didn’t reveal any significant reduction in its ability to treat pain.

IPCI has approximately 12 drugs in development and 2 on the market treating schizophrenia and attention deficit disorder. IPCI partnered with Par Pharmaceutical and Mallinckrodt (MNK) for its schizophrenia and ADHD drugs respectively - Par was acquired by Endo Internat (ENDP) in 2015. IPCI’s new oxycodone formulation is an important catalyst warranting investor attention because of its potential to carve out a chunk of the oxycodone market while reducing rampant abuse.


2017’s third quarter holds a multitude of important trial results and regulatory news. Here we touched on 7 companies (ABBV, ENTA, ADMS, CELG, DVAX, EGRX, and IPCI, PRTK, RDHL, SAGE, and TPBH) with PDUFA meetings taking place in the third quarter. Although not fully comprehensive, this list serves as a starting point for investors looking to stay up to date on current events and make more informed investments. If you are interested in Phase 2 data coming this quarter, I covered that similarly here and Phase 3 here. You can check out the free spreadsheet I made overviewing many upcoming Q3 Phase 2, Phase 3, and PDUFA pharma catalysts here as well. If there are any other companies with upcoming PDUFAs this quarter you think we should know about, I’d love to hear about them in the comments!

Disclosure: I/we have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours.

I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it. I have no business relationship with any company whose stock is mentioned in this article.

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