On November 12, 2019 SCYNEXIS, Inc. (NASDAQ: SCYX), a biotechnology company delivering innovative therapies for difficult-to-treat and often life-threatening infections, reported financial results for the quarter ended September 30, 2019, and provided an update on recent clinical developments (Press release, Scynexis, NOV 12, 2019, View Source [SID1234550997]). Ibrexafungerp (formerly SCY-078), the first representative of a novel family of compounds referred to as triterpenoids, is being developed for oral and intravenous administration and is in clinical development for the treatment of several serious fungal infections, including vulvovaginal candidiasis (VVC), invasive candidiasis (IC), invasive aspergillosis (IA) and refractory invasive fungal infections. If approved, ibrexafungerp would be the first agent approved for the treatment of VVC and prevention of recurrent VVC and the only oral alternative to azoles.

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"We are thrilled with the positive top-line results from the VANISH-303 study and are increasingly confident in an anticipated NDA submission for the treatment of VVC next year as we work to bring ibrexafungerp, potentially the first new class of antifungals approved in over 20 years, to patients in need," said Marco Taglietti, M.D., President and Chief Executive Officer of SCYNEXIS. "We believe the VVC market is quite large and underserved by existing agents, with only one FDA-approved oral drug in the market, which has lacked innovation since the introduction of fluconazole in the 1990s. We’re confident in the commercial potential of ibrexafungerp given its differentiated attributes as a new oral, non-azole antifungal and the eagerness of women with VVC and their treating physicians to try new therapies."

Dr. Taglietti continued: "We are also enthusiastically advancing our hospital-based development programs of ibrexafungerp as a novel treatment to fight invasive, increasingly drug-resistant and often life-threatening fungal infections, such as Candida auris. As part of this effort, we recently announced the expansion of our FURI protocol to a broader range of refractory, serious fungal infections that are potentially eligible for a limited-population FDA approval under the LPAD regulatory pathway. Given the robust evidence collected to date across all of our programs, we believe ibrexafungerp can have the same positive impact fluconazole had when it was first introduced nearly 30 years ago, leveraging ibrexafungerp’s versatility and strong attributes in many different treatment settings – from women’s health with VVC to deadly, hospital-based infections."

Ibrexafungerp Update

Positive top-line data reported for the first of two studies in the VANISH Phase 3 program evaluating the safety and efficacy of oral ibrexafungerp (300mg BID for one day) versus placebo as a treatment for women with VVC
SCYNEXIS recently announced positive results from the VANISH-303 study, a Phase 3, randomized, double-blind, placebo-controlled, study that enrolled 376 patients at 28 U.S. centers. These results were reported earlier than originally anticipated due to rapid enrollment in the study.
The primary endpoint required for registration is clinical cure, defined as complete resolution (score of 0) of all signs and symptoms (S&S) at the Day-10 test-of-cure (TOC) visit. The observed clinical cure for ibrexafungerp was 50.5%, showing highly statistically significant superiority to placebo (p=0.001). Mycological eradication (secondary endpoint) at TOC in ibrexafungerp patients was 49.5%, also showing superiority to placebo (p<0.001). Clinical improvement (S&S score of 0 or 1) at TOC, another secondary endpoint that is a clinically relevant assessment of treatment response, was achieved in 64.4% of ibrexafungerp patients (p<0.001 against placebo). The VANISH-303 ibrexafungerp efficacy results confirm results observed in the Phase 2b DOVE study and achieve the superiority versus placebo required for regulatory approval.
Oral ibrexafungerp was generally safe and well tolerated. Severe and serious adverse events (AEs) were rare, with more cases reported in the placebo group than the ibrexafungerp group, and there were no drug-related serious AEs. The majority of Treatment-Emergent AEs (TEAEs) observed at a higher frequency in the ibrexafungerp group were gastrointestinal in nature, with the three most common GI events (diarrhea/loose stool, nausea and abdominal pain) occurring at rates of 25.5%, 16.6% and 7.3%, respectively, similar to the rates seen in the Phase 2b DOVE study. These events were predominantly regarded as mild, of short duration and did not lead to discontinuation.
A second global Phase 3 study (VANISH-306), with identical design, is being conducted in the U.S. and Europe. Enrollment continues to progress rapidly, and the Company anticipates top-line data early in the second quarter of 2020. The combined results from the VANISH-303 and VANISH-306 pivotal studies are expected to provide the safety and efficacy data to support a New Drug Application (NDA) for ibrexafungerp for the treatment of VVC, with submission to the U.S. Food and Drug Administration (FDA) planned in the second half of 2020.

Ongoing enrollment in Phase 3 CANDLE study evaluating the safety and efficacy of oral ibrexafungerp versus placebo for the prevention of recurrent VVC
The CANDLE study is a global Phase 3, randomized, double-blind, placebo-controlled trial designed to evaluate the safety and efficacy of oral ibrexafungerp (300mg BID for one day, given once per month for a total of six treatment days) compared to placebo in female patients with recurrent VVC (defined as three or more episodes of VVC in the past 12 months, including the episode at screening). The study is being conducted at approximately 50 sites and is expected to enroll approximately 320 patients. Pending a positive outcome, the Company anticipates filing a supplemental NDA for the prevention of recurrent VVC with the FDA in 2021.

Continued advancement of oral ibrexafungerp for hospital-based, invasive fungal infections with two Phase 3 studies (FURI in refractory infections and CARES in Candida auris infections) and one Phase 2 study in invasive aspergillosis (SCYNERGIA)
The protocol for the FURI study, investigating the safety and efficacy of oral ibrexafungerp as a salvage treatment for patients with resistant or refractory severe fungal infections, was expanded to include a broader range of infections. Under the amended study design, patients with aspergillosis, coccidioidomycosis, histoplasmosis, blastomycosis and infections caused by other emerging fungi, including yeasts and molds, are now eligible for enrollment along with those suffering from Candida infections. Additionally, the maximum allowed duration of treatment with ibrexafungerp has been extended from 90 days to up to 180 days, as needed, for chronic conditions. Ibrexafungerp will also be available as a combination therapy with standard of care for selected patients.
Continued and expanded patient enrollment is expected to support future NDA submissions and potential approval through the Limited Population Pathway for Antibacterial and Antifungal Drugs (LPAD). The Company anticipates reporting a second interim data review from the FURI study by an independent data review committee in the first quarter of 2020.
Enrollment is ongoing for the CARES and SCYNERGIA studies.

Data presented at 3rd International Society of Infectious Diseases in Obstetrics and Gynecology (ISIDOG), IDWeek 2019 and the 9th Congress on Trends in Medical Mycology (TIMM-9) further highlighting the scientific and clinical evidence of ibrexafungerp’s activity against multiple fungal pathogens
Post-hoc data from the Phase 2b DOVE study presented at ISIDOG 2019 highlighted ibrexafungerp’s improved trend in signs and symptoms versus fluconazole on a per patient basis.
Data presented at IDWeek 2019 highlighted a large body of evidence demonstrating the activity and efficacy of ibrexafungerp against Candida auris, both in vivo and in vitro.
In an oral presentation at TIMM-9, studies demonstrating ibrexafungerp’s broad spectrum of activity against Candida, Aspergillus, and Pneumocystis were presented, along with evidence of its activity against resistant organisms and ibrexafungerp’s potential utility for the treatment of hospital-based resistant and refractory fungal infections. Additional posters presented provided in vitro, in vivo and clinical evidence of ibrexafungerp’s broad utility against multiple fungal pathogens.
Corporate Highlight

SCYNEXIS received notification from the State of New Jersey of its available unused Net Operating Losses (NOLs) and R&D credits and expects to receive approximately $3.3 million in cash by the end of 2019.
The New Jersey Economic Development Authority’s (NJEDA) Technology Business Tax Certificate Transfer (NOL) Program 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. This may be the second non-dilutive NOL-related cash injection through the NJEDA for SCYNEXIS. In January 2019, SCYNEXIS announced the receipt of approximately $6.7 million of net cash proceeds through the sale of unused New Jersey NOLs.
Third Quarter 2019 Financial Results

Cash, cash equivalents and short-term investments totaled $28.1 million as of September 30, 2019, with net working capital of $20.9 million. Based upon its existing operating plan, SCYNEXIS believes its existing cash, cash equivalents, short-term investments, and the sale of a portion of its New Jersey NOLs, may enable SCYNEXIS to fund operating requirements past an anticipated NDA submission for acute VVC in the second half of 2020.

Research and development expenses increased to $9.3 million for the quarter ended September 30, 2019, compared to $3.9 million in the third quarter of 2018. The increase of $5.3 million, or 136%, was primarily driven by an increase of $4.1 million in clinical development costs, an increase of $0.7 million in chemistry, manufacturing, and controls (CMC) costs, and a net increase in other research and development costs of $0.5 million.

Selling, general and administrative expenses in the third quarter of 2019 increased to $2.5 million, compared with $2.4 million in the third quarter of 2018.

Total other income decreased to $3.8 million in the third quarter of 2019, compared to $6.7 million in the third quarter of 2018. The decrease in other income is attributable to a $6.9 million non-cash gain recorded on the fair value adjustment of the warrant liabilities during the third quarter of 2018.

Net loss for the third quarter of 2019 was $7.9 million. This compares with net income for the third quarter of 2018 of $0.4 million.

About Ibrexafungerp
Ibrexafungerp [pronounced eye-BREX-ah-FUN-jerp] is an investigational antifungal agent and the first representative of a novel class of structurally-distinct glucan synthase inhibitors, triterpenoids. This agent combines the well-established activity of glucan synthase inhibitors with the potential flexibility of having oral and intravenous (IV) formulations. Ibrexafungerp is currently in development for the treatment of fungal infections caused primarily by Candida (including C. auris) and Aspergillus species. It has demonstrated broad spectrum antifungal activity, in vitro and in vivo, against multidrug-resistant pathogens, including azole- and echinocandin-resistant strains. The FDA has granted Qualified Infectious Disease Product (QIDP) and Fast Track designations for the formulations of ibrexafungerp for the indications of invasive candidiasis (IC) (including candidemia), invasive aspergillosis (IA) and VVC (including prevention of recurrent VVC) and has granted Orphan Drug Designation for the IC and IA indications. Ibrexafungerp is formerly known as SCY-078.

On November 12, 2019 Exelixis, Inc. (NASDAQ:EXEL) reported that its partner Ipsen Biopharmaceuticals Canada Inc. received Health Canada approval of CABOMETYX (cabozantinib) tablets for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib (Press release, Exelixis, NOV 12, 2019, View Source [SID1234550996]).

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"Liver cancer is the fourth leading cause of cancer-related death globally, and there is an urgent need for more treatment options for patients living with this aggressive disease," said Michael M. Morrissey, Ph.D., President and Chief Executive Officer of Exelixis. "The Health Canada approval of CABOMETYX brings a much-needed new therapy to Canadian patients with liver cancer and we look forward to our continued collaboration with Ipsen in our efforts to make our medicines available to patients around the world."

The Health Canada approval was based on results from the CELESTIAL phase 3 pivotal trial of CABOMETYX for patients with advanced HCC who received prior treatment with sorafenib. CABOMETYX demonstrated a statistically significant and clinically meaningful improvement in overall survival (OS) versus placebo.

Under the terms of the Collaboration Agreement with Ipsen, Exelixis will receive a milestone payment of $2 million for the Health Canada approval.

CABOMETYX is also approved in Canada for the treatment of adult patients with advanced renal cell carcinoma (RCC) who have received prior vascular endothelial growth factor targeted therapy and for the first-line treatment of adults with intermediate or poor risk advanced RCC.

Please see Important Safety Information below and full U.S. prescribing information at View Source

About the CELESTIAL Study

CELESTIAL is a randomized, double-blind, placebo-controlled study of cabozantinib in patients with advanced HCC conducted at more than 100 sites globally in 19 countries. The trial was designed to enroll 760 patients with advanced HCC who received prior treatment with sorafenib and may have received up to two prior systemic cancer therapies for HCC and had adequate liver function. Enrollment of the trial was completed in September 2017. Patients were randomized 2:1 to receive 60 mg of cabozantinib once daily or placebo and were stratified based on etiology of the disease (hepatitis C, hepatitis B or other), geographic region (Asia versus other regions) and presence of extrahepatic spread and/or macrovascular invasion (yes or no). No cross-over was allowed between the study arms during the blinded treatment phase of the trial. The primary endpoint for the trial is OS, and secondary endpoints include objective response rate and progression-free survival. Exploratory endpoints include patient-reported outcomes, biomarkers and safety.

In October 2017, Exelixis announced that the independent data monitoring committee for the CELESTIAL study recommended that the trial be stopped for efficacy following review at the second planned interim analysis, with cabozantinib providing a statistically significant and clinically meaningful improvement in OS compared with placebo in patients with previously treated advanced HCC. The data, originally presented at the 2018 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper)’s Gastrointestinal Cancers Symposium (ASCO-GI) in January 2018, was published in The New England Journal of Medicine in July 2018.1

About HCC

Liver cancer is a leading cause of cancer death worldwide, accounting for more than 700,000 deaths and 800,000 new cases each year.2The Canadian Cancer Society estimates that 3,000 Canadians will be diagnosed with liver cancer in 2019.3 In the U.S., the incidence of liver cancer has more than tripled since 1980.4 HCC is the most common form of liver cancer, making up about three-fourths of the estimated nearly 42,000 new cases in the U.S. in 2019.4 HCC is the fastest-rising cause of cancer-related death in the U.S.5 Without treatment, patients with advanced HCC usually survive less than 6 months.6

About the Exelixis and Ipsen Collaboration

In 2016, Exelixis granted Ipsen exclusive rights for the commercialization and further clinical development of cabozantinib outside of the United States and Japan. Under the terms of the Collaboration Agreement with Ipsen, Exelixis is entitled to receive a tiered royalty of 22 percent to 26 percent of annual net sales.

About CABOMETYX (cabozantinib)

In the U.S., CABOMETYX tablets are approved for the treatment of patients with advanced renal cell carcinoma and for the treatment of patients with HCC who have been previously treated with sorafenib. CABOMETYX tablets have also received regulatory approvals in the European Union and additional countries and regions worldwide. In 2017, Exelixis granted exclusive rights to Takeda Pharmaceutical Company Limited for the commercialization and further clinical development of cabozantinib for all future indications in Japan.

U.S. Important Safety Information

Hemorrhage: Severe and fatal hemorrhages occurred with CABOMETYX. The incidence of Grade 3 to 5 hemorrhagic events was 5% in CABOMETYX patients. Discontinue CABOMETYX for Grade 3 or 4 hemorrhage. Do not administer CABOMETYX to patients who have a recent history of hemorrhage, including hemoptysis, hematemesis, or melena.
Perforations and Fistulas: GastrointestinaI (GI) perforations, including fatal cases, occurred in 1% of CABOMETYX patients. Fistulas, including fatal cases, occurred in 1% of CABOMETYX patients. Monitor patients for signs and symptoms of perforations and fistulas, including abscess and sepsis. Discontinue CABOMETYX in patients who experience a fistula that cannot be appropriately managed or a GI perforation.
Thrombotic Events: CABOMETYX increased the risk of thrombotic events. Venous thromboembolism occurred in 7% (including 4% pulmonary embolism) and arterial thromboembolism in 2% of CABOMETYX patients. Fatal thrombotic events occurred in CABOMETYX patients. Discontinue CABOMETYX in patients who develop an acute myocardial infarction or serious arterial or venous thromboembolic event requiring medical intervention.
Hypertension and Hypertensive Crisis: CABOMETYX can cause hypertension, including hypertensive crisis. Hypertension occurred in 36% (17% Grade 3 and <1% Grade 4) of CABOMETYX patients. Do not initiate CABOMETYX in patients with uncontrolled hypertension. Monitor blood pressure regularly during CABOMETYX treatment. Withhold CABOMETYX for hypertension that is not adequately controlled with medical management; when controlled, resume at a reduced dose. Discontinue CABOMETYX for severe hypertension that cannot be controlled with anti-hypertensive therapy or for hypertensive crisis.
Diarrhea: Diarrhea occurred in 63% of CABOMETYX patients. Grade 3 diarrhea occurred in 11% of CABOMETYX patients. Withhold CABOMETYX until improvement to Grade 1 and resume at a reduced dose for intolerable Grade 2 diarrhea, Grade 3 diarrhea that cannot be managed with standard antidiarrheal treatments, or Grade 4 diarrhea.
Palmar-Plantar Erythrodysesthesia (PPE): PPE occurred in 44% of CABOMETYX patients. Grade 3 PPE occurred in 13% of CABOMETYX patients. Withhold CABOMETYX until improvement to Grade 1 and resume at a reduced dose for intolerable Grade 2 PPE or Grade 3 PPE.
Proteinuria: Proteinuria occurred in 7% of CABOMETYX patients. Monitor urine protein regularly during CABOMETYX treatment. Discontinue CABOMETYX in patients who develop nephrotic syndrome.
Osteonecrosis of the Jaw (ONJ): ONJ occurred in <1% of CABOMETYX patients. ONJ can manifest as jaw pain, osteomyelitis, osteitis, bone erosion, tooth or periodontal infection, toothache, gingival ulceration or erosion, persistent jaw pain, or slow healing of the mouth or jaw after dental surgery. Perform an oral examination prior to CABOMETYX initiation and periodically during treatment. Advise patients regarding good oral hygiene practices. Withhold CABOMETYX for at least 28 days prior to scheduled dental surgery or invasive dental procedures. Withhold CABOMETYX for development of ONJ until complete resolution.
Wound Complications: Wound complications were reported with CABOMETYX. Stop CABOMETYX at least 28 days prior to scheduled surgery. Resume CABOMETYX after surgery based on clinical judgment of adequate wound healing. Withhold CABOMETYX in patients with dehiscence or wound healing complications requiring medical intervention.
Reversible Posterior Leukoencephalopathy Syndrome (RPLS): RPLS, a syndrome of subcortical vasogenic edema diagnosed by characteristic finding on MRI, can occur with CABOMETYX. Evaluate for RPLS in patients presenting with seizures, headache, visual disturbances, confusion, or altered mental function. Discontinue CABOMETYX in patients who develop RPLS.
Embryo-Fetal Toxicity: CABOMETYX can cause fetal harm. Advise pregnant women and females of reproductive potential of the potential risk to a fetus. Verify the pregnancy status of females of reproductive potential prior to initiating CABOMETYX and advise them to use effective contraception during treatment and for 4 months after the last dose.
Adverse Reactions: The most commonly reported (≥25%) adverse reactions are: diarrhea, fatigue, decreased appetite, PPE, nausea, hypertension, and vomiting.
Strong CYP3A4 Inhibitors: If coadministration with strong CYP3A4 inhibitors cannot be avoided, reduce the CABOMETYX dosage. Avoid grapefruit or grapefruit juice.
Strong CYP3A4 Inducers: If coadministration with strong CYP3A4 inducers cannot be avoided, increase the CABOMETYX dosage. Avoid St. John’s wort.
Lactation: Advise women not to breastfeed during CABOMETYX treatment and for 4 months after the final dose.
Hepatic Impairment: In patients with moderate hepatic impairment, reduce the CABOMETYX dosage. CABOMETYX is not recommended for use in patients with severe hepatic impairment.

On November 12, 2019 NantHealth, Inc. (NASDAQ: NH), a next-generation, evidence-based, personalized healthcare company, reported FDA authorization of Omics Core℠, the first whole exome tumor-normal in vitro diagnostic (IVD) that measures overall tumor mutational burden (TMB) in cancer tissue (Press release, NantHealth, NOV 12, 2019, View Source [SID1234550995]).

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The Omics Core assay is the nation’s first FDA authorized custom-targeted whole exome sequencing platform to report both overall tumor mutation burden in tumor specimens from 19,396 protein-coding genes (whole exome) and somatic alterations (point mutations, small insertions and deletions) in 468 cancer-relevant genes.

TMB is reported via two metrics:

Total number of somatic non-synonymous exonic variants within the 19,396 genes (whole exome) surveyed.
An estimate of mutation rate by counting all somatic, synonymous and non-synonymous variants detected in gene coding regions and dividing by the approximate size of the whole exome.
"Tumor mutation burden (TMB) is now recognized as a key biomarker across multiple tumor types. Studies have shown that immunotherapy treated patients with high TMB had better outcomes compared to those with low TMB. Since the potential for TMB as a precision medicine tool is so high, it is imperative that the most accurate and comprehensive method of analysis be applied to enable physicians to determine which tumors could benefit from checkpoint inhibitors and immunotherapy," said Patrick Soon-Shiong, MD, Chairman & CEO NantHealth. "I am so proud of the scientific, regulatory and bioinformatics teams who have spent almost a decade to perfect this important test that measures the absolute number of mutations occurring in 19,396 protein-coding genes (whole exome) targeting 39 million base pairs (39 Mb) of the human genome from both a tumor and patient-matched normal control sample (tumor-normal). We believe that this comprehensive diagnostic will provide greater accuracy than the widely-used formulaic extrapolation of TMB from a limited gene panel sequence ," said Soon-Shiong.

"Omics Core is the first whole exome test for TMB authorized by the FDA, and as such, marks a watershed moment in oncology. Clinicians can now directly measure the mutations in a patient’s tumor specimen accurately via tumor-normal sequencing and have confidence that the results they receive are fully validated to help support better therapeutic decisions. Also, the breadth of a whole exome means that many more neoepitopes and novel targets may be identified to support vaccine development, novel drug development, and therapies for previously undruggable targets," said Sandeep Bobby Reddy, MD – Chief Medical Officer, NantHealth

"Multiple groups, including our own presentation at ASCO (Free ASCO Whitepaper) in 2018, have shown the importance of performing whole exome sequencing to measure the comprehensive TMB. We calculated a three-fold overestimation of TMB when extrapolating from panel-based methods, potentially leading physicians to over-prescribe checkpoint inhibitors for patients that are unlikely to respond. Given the high cost and the possibility of adverse events with these therapies, it is critical we identify the most appropriate population as accurately as possible," said Steve Benz, PhD – President, Genomics, ImmunityBio.

"Determining the tumor mutational burden from whole exome sequencing is the first step in defining neoepitopes. These unique tumor mutations are recognized by T cells and elicit an immunological anti-tumor response. Patients with high TMB typically have more neoepitopes that attract cancer killing T cells to the tumor microenvironment. Identifying neoepitopes from whole exome TMB enables the development of neoepitope-targeted vaccines for the >95% of protein-encoding genes not covered by limited gene panel tests and the >99% of genes not directly targeted by drugs today. The clearance of Omics Core based upon its analytical performance and validity in reporting TMB establishes a new chapter in the era of precision cancer immunotherapy," said Shahrooz Rabizadeh, PhD – Chief Scientific Officer, ImmunityBio.

About Omics Core

The Omics Core assay is a qualitative in vitro diagnostic test that uses targeted next generation sequencing of formalin-fixed paraffin-embedded tumor tissue matched with normal specimens from patients with solid malignant neoplasms to detect tumor gene alterations in a broad multi gene panel. The test is intended to provide information on somatic mutations (point mutations and small insertions and deletions) and tumor mutational burden (TMB) for use by qualified health care professionals in accordance with professional guidelines, and is not conclusive or prescriptive for labeled use of any specific therapeutic product. Omics Core is a single-site assay performed at NantHealth, Inc. The Omics Core assay is protected by US Patents 9,652,587; 9,646,134; 9,824,181; 10,249,384; 9,721,062; 10,242,155; 10,268,800.

On November 12, 2019 Akebia Therapeutics, Inc. (Nasdaq: AKBA), a biopharmaceutical company focused on the development and commercialization of therapeutics for people living with kidney disease, reported financial results for the third quarter ended September 30, 2019 (Press release, Akebia, NOV 12, 2019, View Source [SID1234550994]). The Company will host a conference call today, Tuesday, November 12, 2019, at 9:00 a.m. Eastern Time to discuss its third quarter 2019 financial results and recent business highlights.

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Akebia also announced that it has entered into a $100 million non-dilutive, definitive term loan agreement with funds managed by Pharmakon Advisors LP, the investment manager of the BioPharma Credit funds. The loans provide Akebia with up to $100 million of borrowing capacity available in two tranches. Subject to the satisfaction of customary conditions, Akebia expects to draw $80 million at an initial closing later this month, and an additional tranche of $20 million is available for draw at Akebia’s option until December 31, 2020. Additional information on the loan agreement will be included in the Company’s Quarterly Report on Form 10-Q for the quarterly period ended September 30, 2019 that is expected to be filed with the U.S. Securities and Exchange Commission today, November 12, 2019.

"Akebia continues to make great progress advancing our strategy. We achieved a primary objective of the Company by strengthening our balance sheet with $80 to $100 million non-dilutive, tranched term loans, on very competitive terms, to further support our clinical development program for vadadustat, our investigational oral hypoxia-inducible factor prolyl hydroxylase inhibitor (HIF-PHI) for the treatment of anemia due to chronic kidney disease (CKD), and other strategic goals. Importantly, we believe these loans, the first tranche of which is expected to close later this month, in combination with our other cash resources, are expected to extend our cash runway into 2021, well past our expected top-line data readouts of our global Phase 3 clinical studies of vadadustat. Auryxia product revenue allows us to service the debt," stated John P. Butler, Chief Executive Officer of Akebia. "As we believe we are now within two quarters of our first readout of our global Phase 3 studies of vadadustat with top-line data of INNO2VATE on track for Q2’FY20 and PRO2TECT for mid-2020, subject to the accrual of major adverse cardiovascular events (MACE), we’re working to sharpen the timelines for completion and keying in on NDA and MAA related activities and commercial plans for vadadustat, upon approval."

Butler continued, "We have a tremendous amount of confidence in the program that we’ve designed for vadadustat and believe we are positioned well for clinical, regulatory and commercial success. We expect vadadustat to be the first drug of the HIF class to deliver clear data that directly compares its outcomes to the current standard of care in both dialysis and non-dialysis patients for the treatment of anemia due to CKD. We believe these data will be highly informative for physicians, patients and payers as they make important decisions about patient care, and a key consideration when differentiating between HIFs in the class."

Recent Highlights

Auryxia (ferric citrate) net product revenue increased 13 percent year-over-year to $30.0 million for the third quarter of 2019. Total Auryxia prescriptions increased 15.5 percent year-over-year to 51,700 in the third quarter of 2019.
In November, Mitsubishi Tanabe Pharma Corporation (MTPC), Akebia’s development and commercialization collaboration partner in Japan for vadadustat, presented positive 24-week and 52-week data from two Phase 3 active-controlled pivotal studies evaluating the efficacy and safety of vadadustat in Japanese patients with anemia due to CKD, at the American Society of Nephrology (ASN) Kidney Week 2019. Each study met its primary endpoint based on mean hemoglobin level at week 20 and 24, and showed vadadustat’s effect on hemoglobin was sustained through to 52 weeks in each study. (See recent related press release here.) In July, MTPC submitted a Japanese New Drug Application (JNDA) to the Ministry of Health, Labor and Welfare in Japan for marketing approval of vadadustat as a treatment for anemia due to CKD. The JNDA is the first regulatory submission for marketing approval of vadadustat and, if approved, is expected to lead to the first launch of vadadustat worldwide, next year.
Nine abstracts, including several associated with vadadustat and Auryxia, Akebia’s FDA-approved drug, were presented at ASN in November.
In October, the Independent Data Monitoring Committee reviewed unblinded safety and efficacy data from Akebia’s global Phase 3 studies of vadadustat, as planned, and recommended continuation of the studies without modifications.
In October, Akebia filed a complaint in federal district court against the Centers for Medicare & Medicaid Services (CMS) and the U.S. Department of Health and Human Services (HHS). The lawsuit challenges a September 2018 decision by CMS that rescinded Medicare Part D coverage of Auryxia, when used for the treatment of iron deficiency anemia (IDA) in adult patients with CKD not on dialysis. The legal action also seeks to reverse a related decision by CMS that imposed a prior authorization requirement for Auryxia when used for the control of serum phosphorus levels in adult patients with CKD on dialysis.
In April and August, Akebia completed enrollment in its global Phase 3 program, INNO2VATE and PRO2TECT, respectively, evaluating the safety and efficacy of vadadustat in dialysis-dependent and non-dialysis dependent CKD subjects with anemia due to CKD. The Company continues to expect to report top-line data from the INNO2VATE and PRO2TECT studies in Q2’FY20 and mid-2020, respectively, subject to the accrual of MACE.
Financial Results

Total revenue for the third quarter of 2019 was $92.0 million, compared to $53.2 million in the pre-merger third quarter of 2018.

Auryxia net product revenue for the third quarter of 2019 was $30.0 million, compared to $26.6 million, as reported by Keryx Biopharmaceuticals, Inc. (Keryx) prior to its merger with the Company, during the same period in 2018. This represents a 13 percent increase in net product revenue from the third quarter of 2018.

Collaboration and license revenue for the third quarter of 2019 was $62.0 million, compared with $53.2 million in the third quarter of 2018. The increase was primarily due to increased collaboration revenue of $6.8 million from Otsuka Pharmaceutical Co. Ltd (Otsuka). In accordance with the Company’s collaboration agreements, Otsuka began funding 80 percent of the development costs for vadadustat in the second quarter of 2019.

Cost of goods sold was $38.3 million for the third quarter of 2019, consisting of $11.2 million of costs associated with the manufacture of Auryxia and non-cash charges of $27.1 million related to the application of purchase accounting as a result of the merger with Keryx. These non-cash, merger-related charges include a $18.0 million inventory step-up charge and $9.1 million of amortization of intangibles.

Research and development expenses were $74.5 million for the third quarter of 2019 compared to $70.6 million for the third quarter of 2018. The increase was primarily attributable to an increase in headcount and other costs to support its research and development programs and clinical and preclinical activities. These increases were partially offset by a decrease in external costs related to PRO2TECT and INNO2VATE Phase 3 studies as they advance toward readout.

Selling, general and administrative expenses were $34.2 million for the third quarter of 2019 compared to $10.4 million for the third quarter of 2018. The increase was primarily attributable to commercialization costs associated with Auryxia, as there were no comparable commercialization costs in the third quarter of 2018.

The Company reported a net loss for the third quarter of 2019 of $54.6 million, or ($0.46) per share, as compared to a net loss of $26.0 million, or ($0.46) per share, for the third quarter of 2018. The Company’s net loss for the third quarter of 2019 includes the impact of non-cash charges of $27.1 million related to the application of purchase accounting as a result of the merger with Keryx, offset by an income tax benefit of $1.3 million.

The Company ended the quarter with cash, cash equivalents and available-for-sale securities of $145.6 million. "We are pleased to have further strengthened our balance sheet and extended our operating cash runway with our very recent non-dilutive, tranched term loans for up to $100 million, with the first $80 million tranche expected to close later this month. We expect these loans, coupled with the committed research and development funding from our collaborators and the receipt of a regulatory milestone from MTPC, assuming approval of vadadustat in Japan, to provide us with the cash resources to fund our current operating plan into Q1 of 2021," stated Jason A. Amello, Chief Financial Officer of Akebia.

Conference Call

Akebia will host a conference call today, Tuesday, November 12, 2019, at 9:00 a.m. Eastern Time to discuss its third quarter 2019 financial results and recent business updates. To listen to the conference call, please dial (877) 458-0977 (domestic) or (484) 653-6724 (international) using conference ID number 9996464. The call will also be webcast LIVE and can be accessed via the Investors section of the Company’s website at View Source

A replay of the conference call will be available two hours after the completion of the call through November 18, 2019. To access the replay, dial (855) 859-2056 (domestic) or (404) 537-3406 (international) and reference conference ID number 9996464. An online archive of the conference call can be accessed via the Investors section of the Company’s website at View Source

On November 12, 2019 Phio Pharmaceuticals Corp. (NASDAQ: PHIO), a biotechnology company developing the next generation of immuno-oncology therapeutics based on its proprietary self-delivering RNAi (INSTASYL) therapeutic platform, reported its financial results for the third quarter ended September 30, 2019 and provided a business update (Press release, Phio Pharmaceuticals, NOV 12, 2019, View Source [SID1234550993]).

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"We remain focused on advancing our immuno-oncology pipeline toward meaningful value inflection points," said Dr. Gerrit Dispersyn, Phio’s President and CEO. "During the quarter, we continued to execute on our R&D objectives, in particular for our lead programs in targeted PD-1 receptor silencing, as well as leverage our unique self-delivering RNAi platform to establish additional research collaborations with leading companies and academic institutions as part of our ongoing effort to maximize the potential value of our technology. We’ve also presented several posters demonstrating the value of our platform and highlighting our pipeline progress to an audience of potential partners and future prescribers. We look forward to continuing to update the market as our programs advance."

Quarter in Review and Recent Corporate Updates

Entered into a research collaboration with the Helmholtz Zentrum München for the design and development of new targets based on Phio’s INSTASYL platform for use in cancer immunotherapies
Announced a research collaboration with Carisma Therapeutics to evaluate potential of Phio’s technology to enhance the immune function of Carisma’s chimeric antigen receptor macrophages (CAR-M) as a novel adoptive cell therapy for use in cancer treatment
Presented three posters at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 2019 Annual Meeting highlighting Phio’s proprietary INTASYL technology for "weaponizing" T cells against cancer, reflecting internal work and from collaborations with Iovance Biotherapeutics and the Karolinska Institutet
Select Quarterly Financial Results

Cash Position

At September 30, 2019, the Company had cash of $8.7 million as compared with $14.9 million at December 31, 2018. The Company expects its cash and ability to raise cash through utilization of the Lincoln Park Capital equity line to provide funding for at least the next twelve months.

Research and Development Expenses

Research and development expenses for the quarter ended September 30, 2019, were $1.0 million as compared with $0.8 million for the quarter ended September 30, 2018. The increase was primarily due to an increase in consulting and outside professional service fees in support of preclinical research for the Company’s immuno-oncology programs.

General and Administrative Expenses

General and administrative expenses for the quarter ended September 30, 2019, were $1.1 million as compared with $0.7 million for the quarter ended September 30, 2018. The increase was primarily driven by an increase in payroll-related expenses and legal fees.

Net Loss

Net loss for the quarter ended September 30, 2019, was $2.1 million or $0.08 per share, compared with $1.5 million or $0.34 per share for the quarter ended September 30, 2018. The increase was primarily due to changes in operating expenses, as discussed above.