This section provides a list of FAQs that SCYNEXIS has accumulated from investors, as a quick reference. The information provided in this page is also available publicly on our web site, our SEC filings or in the public domain and it is subject to our Forward Looking Statement Disclaimer. If you have additional questions, please, submit them to investorrelations@scynexis.com. (last updated 04/10/2019)

Main Index

Regulatory and Exclusivity Clinical Development – Vulvovaginal Candidiasis (VVC) Clinical Development – Invasive Aspergillosis Clinical Development – Refractory Fungal Infections Miscellaneous Financial

Regulatory and Exclusivity

What is the GAIN Act?

Generating Antibiotic Incentives Now (GAIN) was passed in 2012 as part of the Food and Drug Administration Safety and Innovation Act (FDASIA) to address the public health threat of antimicrobial drug resistance by stimulating the development and approval of new antibacterial and antifungal drugs. The GAIN Act provides potential for an extended exclusivity period of five years, fast track designation and eligibility for priority review to novel antibiotics designated as Qualified Infectious Disease Product (QIDP) by FDA.

For more information: https://www.fda.gov/downloads/AboutFDA/CentersOffices/OfficeofMedicalProductsandTobacco/CDER/UCM595188.pdf

Is ibrexafungerp a QIDP product eligible for 5 years of exclusivity, fast track status and priority review?

Yes. Ibrexafungerp has received QIDP status for invasive candidiasis and aspergillosis (oral 2014 and IV 2016) and for vulvovaginal candidiasis in 2018. Therefore, ibrexafungerp is eligible to receive five extra years of exclusivity, fast track status and priority review (6 months review rather than a standard review of 10 months) for these indications. Additionally, the Prescription Drug User Fee Act (PDUFA) payment ($2.6 million for 2019) will be waived for the first NDA filing as a small company.

What is the Limited Population Pathway for Antibacterial and Antifungal Drugs (LPAD)?

LPAD (Limited Population Pathway for Antibacterial and Antifungal Drugs) is intended as an accelerated regulatory path for products treating serious or life-threatening infections in a limited patient population with unmet needs. FDA will accept greater uncertainty and higher risk for products that qualify for the pathway, but the labelling will include statements to convey to the health care community that the drug has been shown to be safe and effective only for use in a limited population.

For more information: https://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM610498.pdf

Are the FURI and CARES studies eligible for LPAD?

The FURI and CARES protocols have been designed to treat patients suffering from fungal infections with organisms resistant to other products or who cannot tolerate other products. Success against these life-threatening infections would address an unmet medical need and we believe could qualify for approval under the LPAD regulatory pathway.

Is ibrexafungerp eligible for benefits of Orphan Drug Designation?

The Orphan Drug Act (ODA) provides for granting special status to a drug or biological product (“drug”) to treat a rare disease or condition upon request of a sponsor and it provides waiver of PDUFA fees and 7 additional years of exclusivity for the orphan indication. Ibrexafungerp has been granted orphan drug status for invasive candidiasis in 2016 and for invasive aspergillosis 2017. Approval under QIDP and approval for an Orphan indication could result in a total of 12 years of marketing exclusivity for the indication.

When does SCYNEXIS expect to file the first NDA for ibrexafungerp?

SCYNEXIS plans to submit an initial NDA for oral ibrexafungerp for the treatment of VVC in the second half of 2020 utilizing the QIDP designation of ibrexafungerp.

When will patent protection for ibrexafungerp expire?

Ibrexafungerp is covered by a composition of matter patent (i.e., a patent covering the ibrexafungerp compound itself). The 2030 expiration date for this patent is expected to be extended until 2035, pursuant to the patent term extension provisions of U.S. law.

We are also pursuing patent coverage beyond the composition of matter, including formulations and methods of treatment, which may provide limited patent protection beyond 2035.

Clinical Development – Vulvovaginal Candidiasis (VVC)

What is the development plan to obtain regulatory approval for VVC?

SCYNEXIS will perform 3 studies: 2 studies in acute VVC (VANISH) and 1 study in recurrent VVC. The 2 studies in acute VVC have been initiated while the study in recurrent VVC is planned to start in the second quarter of 2019.

The VANISH Phase 3 program comprises 2 Phase 3 trials (approximately 350 patients each) designed to evaluate the safety and efficacy of one-day oral ibrexafungerp versus placebo for the treatment of acute VVC in female patients 12 years and older. The study will randomize in a 2:1 randomization, oral ibrexafungerp 300 mg BID for one day vs. placebo BID for one day. The primary efficacy endpoint of the study is the percentage of subjects with clinical cure (complete resolution of signs and symptoms) at the Test-of-Cure Visit (Day10).

Pending successful completion of the VANISH program, SCYNEXIS plans to submit an initial NDA for oral ibrexafungerp for the treatment of VVC in the second half of 2020 utilizing the QIDP designation of ibrexafungerp.

For more information: https://clinicaltrials.gov/ct2/show/NCT03734991

SCYNEXIS plans to initiate a third Phase 3 trial (approximately 350 patients) evaluating oral ibrexafungerp vs. placebo in recurrent VVC in the second quarter of 2019, an indication with no product currently approved. Pending successful completion of this Phase 3 study, SCYNEXIS plans to submit a supplemental NDA for oral ibrexafungerp for the prevention of recurrent VVC in 2021.

When does SCYNEXIS plan to file the NEW Drug Applications (NDAs) for acute VVC and recurrent VVC?

Pending successful completion of the VANISH program, SCYNEXIS plans to submit an initial NDA for oral ibrexafungerp for the treatment of VVC in the second half of 2020, utilizing the QIDP designation of ibrexafungerp with potential for priority review. A supplemental NDA for the prevention of recurrent VVC is anticipated for 2021.

Clinical Development – Invasive Aspergillosis

What is the evidence suggesting that ibrexafungerp in combination may be superior to azoles alone (current standard of care) in Invasive Aspergillosis?

Invasive aspergillosis is a severe, hospital-based, infection with high mortality rates despite the latest available agents (azole class).

A neutropenic rabbit model has shown that ibrexafungerp given in combination with isavuconazole provided significantly better outcomes and improved survival rates versus isavuconazole alone. Moreover, a large clinical study (published by Marr et al. in 2015) showed that glucan synthase inhibitors (like ibrexafungerp) when given in combination with azoles may provide clinical benefits over standard of care alone.

For more information: Combination Therapy with SCY-078 and Isavuconazole for Treatment of Experimental Invasive Pulmonary Aspergillosis

What are the key aspects of the Phase 2 Study in Invasive Aspergillosis (SCYNERGIA Study)?

The Phase 2 trial (SCYNERGIA) designed to evaluate the safety and efficacy of oral ibrexafungerp in combination with standard-of-care voriconazole in patients with invasive pulmonary aspergillosis. An animal model of pulmonary aspergillosis demonstrated improved outcomes and survival rates, supporting the potential superiority of ibrexafungerp in combination with azole therapy versus standard of care alone in this high-mortality indication.

The SCYNERGIA study is a multicenter, randomized, double-blind, two-arm study to evaluate the safety, tolerability, efficacy and PK of the combination therapy of ibrexafungerp plus voriconazole compared to those of voriconazole monotherapy in subjects 18 years of age and older with a hematological malignancy (HM) or a myelodysplastic syndrome or aplastic anemia or hematopoietic cell transplantation (HCT) and a probable or proven invasive pulmonary aspergillosis. The study will accrue approximately 60 patients and is expected to take 2 years to complete.

For more information: https://clinicaltrials.gov/ct2/show/NCT03672292

Clinical Development – Refractory Fungal Infections

What is the FURI study?

The FURI study is a multicenter, open-label, non-comparator, single arm study to evaluate the safety and efficacy of ibrexafungerp in patients > 18 years of age with a documented invasive and/or severe mucocutaneous fungal disease that has been intolerant or refractory (rIFI) to standard of care (SoC) antifungal treatment or a continued IV antifungal therapy is not desirable or feasible.

Enrolled patients receive an initial loading dose of 750mg BID (twice a day) of oral ibrexafungerp during the first two days of treatment and subsequent oral doses of 750mg QD (once a day) for up to 90 days.

Data from the FURI study will be analyzed periodically.

For more information: https://clinicaltrials.gov/ct2/show/NCT03059992.

What is Candida auris?

Candida auris has been classified by the Centers for Disease Control and Prevention (CDC) as a serious public health threat, as it is multidrug-resistant, has resulted in high mortality rates (up to 60%) and can be spread from patients (and surfaces) to patients, resulting in hospital outbreaks. The CDC is concerned about C. auris for several reasons:

  • It causes serious infections. C. auris can cause bloodstream infections and even death, particularly in hospital and nursing home patients with serious medical problems. More than 1 in 3 patients with invasive C. auris infections (for example, an infection that affects the blood, heart, or brain) die.
  • It's often resistant to medicines. Antifungal medicines commonly used to treat Candida infections often don't work for Candida auris. Some C. auris infections have been resistant to all three types of antifungal medicines.
  • It's becoming more common. Although C. auris was just discovered in 2009, it has spread quickly and caused infections in more than a dozen countries.
  • It's difficult to identify. C. auris can be misidentified as other types of fungi unless specialized laboratory technology is used. This misidentification might lead to a patient getting the wrong treatment.
  • It can spread in hospitals and nursing homes. C. auris can spread through contact with affected patients and contaminated surfaces or equipment and, as a result, has caused outbreaks in healthcare facilities. Good hand hygiene and cleaning in healthcare facilities is important because C. auris can live on surfaces for several weeks.

For more information: https://www.cdc.gov/fungal/candida-auris/c-auris-drug-resistant.html

What is the CARES study?

The CARES study is a multi-center (U.S. and India), open-label, single-arm study designed to evaluate the efficacy, safety and tolerability of oral ibrexafungerp in subjects with documented C. auris infections. Similar to the FURI study, subjects receive an initial loading dose of oral ibrexafungerp 750 mg twice a day (BID) during the first 2 days and then subsequent oral ibrexafungerp 750 mg once a day (QD) for up to 90 days.

The study expects to enroll 30 patients.

For more information: https://clinicaltrials.gov/ct2/show/NCT03363841

When would SCYNEXIS file FURI or CARES data for a potential LPAD approval?

Both FURI and CARES studies have been designed to be eligible for the LPAD process. Since filing for an LPAD is dependent on the data that is collected from the studies, we have not provided any guidance when a filing will occur. We will continue to review data on a regular basis from the two studies and make a determination, in consultation with FDA, on the feasibility of a submission under the LPAD guidance using data from FURI and/or CARES studies.

Miscellaneous

What is the status of the ibrexafungerp IV formulation?

In January 2018, we announced encouraging pre-clinical results for the prototype liposomal IV formulation of ibrexafungerp, showing improved local tolerability profile at the infusion site in head-to-head pre-clinical evaluations with the cyclodextrin-based IV formulation. As part of our development plans, the process for the liposomal formulation was transferred for scale-up purposes at a manufacturing site intended to provide clinical supplies. Additional preclinical evaluations were performed with the scaled-up formulation, which unexpectedly revealed differences in tolerability at the injection site, delaying advancement of the IV product into human trials. As it is generally recognized that changes to manufacturing processes and/or scale-up can impact the characteristics of drug products, particularly for more technically complex formulations such as liposomal products, SCYNEXIS is currently working with our vendors and CMC experts to enable us to resume the IND-enabling pre-clinical activities for the IV formulation of ibrexafungerp. SCYNEXIS will provide updates on the IV formulation studies as they become available.

Where can I find scientific data and presentations on Ibrexafungerp?

Financial

What is convertible debt?

Convertible debt (or convertible bond / note) is a type of bond that the holder can convert into a predetermined number of shares of common stock in the issuing company during the bond's life, usually at the discretion of the bond holder. It is a hybrid security with debt- and equity-like features.

For more information: https://en.wikipedia.org/wiki/Convertible_bond

What is the benefit of the convertible debt for SCYNEXIS?

The current convertible debt offering completed by SCYNEXIS allowed the Company to retire in full its existing $15 million loan, that was set to be repaid by the end of the third quarter 2020. As a result, the Company was able to lower its interest expenses and to push out near-term principal payment obligations. In addition, upon conversion, stock will be issued at a premium price, resulting in less dilution for shareholders versus typical equity offerings.

For more information: https://ir.scynexis.com/press-releases/detail/171/scynexis-is-retiring-existing-term-loan-through-a

What is SCYNEXIS' current cash balance and how long will it last?

As of December 31, 2018, SCYNEXIS had cash, cash equivalents and short-term investments of approximately $44.2 million.

In January 2019, SCYNEXIS completed the sale of a portion of its NOLs and received a cash receipt of $6.7 million, resulting in a pro forma cash balance of $51 million as of January 3, 2019. In March 2019, SCYNEXIS also completed the sale of a $16.0 million convertible unsecured senior note in a private placement. SCYNEXIS used the proceeds of the convertible debt to retire in full the previous term loan, strengthening near-term cash flows and extending the Company's cash runway past an anticipated NDA submission in the second half of 2020.

What are SCYNEXIS's current numbers of shares outstanding (basic and diluted)?

As of Mach1, 2019 (per the last filed 10-K), the number of basic shares outstanding was 50.1 million and the number of fully diluted shares was 81.7 million.

Who are the top shareholders of SCYNEXIS?

What is the New Jersey NOL non-dilutive grant and are you eligible to receive the full $15MM NOL amount?

In January 2019, SCYNEXIS completed the sale of a portion of its Net Operating Losses (NOLs) and received a cash receipt of $6.7 million. This sale was structured through the New Jersey Technology Business Tax Certificate Transfer (NOL) Program, which allows eligible companies to sell their New Jersey NOLs and research and development tax credits up to a maximum lifetime benefit of $15 million per company.

For more information: https://www.njeda.com/technology_lifesciences/Technology-Business-Tax-Certificate-Transfer-(NOL)

Which Wall Street research analysts follow the SCYNEXIS stock?

Investment Bank

Analyst

Rating

Price Target

Brookline Capital Markets

Kumar Raj PhD

BUY

$10.00

Canaccord Genuity

Dewey Steadman, CFA

BUY

$4.00

Guggenheim Securities

Adnan Butt

BUY

$5.00

Ladenburg Thalmann Financial Services

Michael Higgins

BUY

$6.00

HC Wainwright

Oren Livnat, CFA

BUY

$4.50

Maxim Group

Jason McCarthy, PhD

BUY

$4.00

Needham & Co.

Alan Carr, PhD

BUY

$5.00

Opus National Capital Markets

Jonathan Aschoff, PhD

BUY

$6.00

Roth Capital Partners

in transition

BUY

---

WBB Securities

Steve Brozak

BUY

$8.00

For more information: https://ir.scynexis.com/analyst-coverage