On May 8, 2018 Sunesis Pharmaceuticals, Inc. (Nasdaq: SNSS) reported financial results for the first quarter ended March 31, 2018. Loss from operations for the three months ended March 31, 2018 was $7.1 million (Press release, Sunesis, MAY 8, 2018, View Source [SID1234526240]). As of March 31, 2018, cash, cash equivalents and marketable securities totaled $25.4 million. This capital is expected to fund the company into early 2019.

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"We remain highly focused on the execution of our Phase 1b/2 trial evaluating our lead program, the non-covalent BTK inhibitor vecabrutinib (SNS-062), to help patients who have developed resistance to covalent BTK inhibitors such as ibrutinib, the current standard of care in treating CLL," said Dayton Misfeldt, Interim Chief Executive Officer of Sunesis. "We believe vecabrutinib represents an important potential new treatment option for B-cell hematologic cancers, and we look forward to providing a data update from the study at a medical meeting in the fall."

Recent Highlights

Phase 1b/2 Study Evaluating Oral Non-Covalent BTK-inhibitor Vecabrutinib (SNS-062) in Adults with Chronic Lymphocytic Leukemia (CLL) and other B-Cell Malignancies. Sunesis’ ongoing Phase 1b/2 study is evaluating the safety, pharmacokinetics, pharmacodynamics, and antitumor activity of its potent non-covalent BTK-inhibitor vecabrutinib in adults with CLL and other B cell malignancies. The Phase 1b portion of the study is an open-label, dose-escalation study with the goal of determining the recommended Phase 2 dose. The Phase 2 portion of the study will explore various cohorts of patients; current cohort concepts include ibrutinib-resistant patients with C481 mutations. The trial is enrolling patients who have relapsed/refractory B cell malignancies after at least 2 lines of standard treatment. For indications such as CLL with approved BTK inhibitors, one of those prior treatments must have been a covalent BTK inhibitor. The study is in the 50 mg cohort. Sunesis expects to reach the recommended Phase 2 dose in the fall of 2018.

Appointed Industry Veteran H. Ward Wolff to the Board of Directors. In February 2018, H. Ward Wolff was appointed to the Board of Directors. Ward brings over 40 years of finance and executive leadership experience to the Board, with 20 years of experience in the life sciences sector, most recently having served as Executive Vice President and Chief Financial Officer of Sangamo Therapeutics, Inc. Mr. Wolff is also designated chairman of the company’s Audit Committee.

Financial Highlights

Cash, cash equivalents, and marketable securities totaled $25.4 million as of March 31, 2018, as compared to $31.8 million as of December 31, 2017. The decrease of $6.4 million was primarily due to $6.6 million of net cash used in operating activities, partially offset by $0.2 million in net

proceeds from the exercise of stock options. This capital is expected to fund the company into early 2019.

Revenue for the three months ended March 31, 2018 was $0.2 million as compared to $0.7 million for the same period in 2017. The decrease between the periods was primarily due to deferred revenue related to the Royalty Agreement with RPI Finance Trust, which was fully amortized to revenue in March 2017.

Research and development expense was $4.0 million for the three months ended March 31, 2018, as compared to $6.2 million for the same period in 2017, primarily relating to the vecabrutinib and the vosaroxin development program in each period. The decrease of $2.2 million was primarily due to $1.7 million decrease in professional services and clinical trials expenses related to higher expenses incurred in the first quarter of 2017 due to the preparation for EMA, and $0.3 million decrease in salary and personnel expenses due to lower headcounts.

General and administrative expense was $3.4 million for the three months ended March 31, 2018, as compared to $3.9 million for the same period in 2017. The decrease of $0.5 million was primarily due to $0.4 million decrease in professional services expenses and $0.1 million decrease in commercial expenses as result of higher expenses incurred in the first quarter of 2017 due to the preparation for EMA.

Interest expense was $0.3 million for the three months ended March 31, 2018, as compared to $0.5 million for the same period in 2017. The decrease was primarily due to the decrease in the outstanding notes payable.

Cash used in operating activities was $6.6 million for the three months ended March 31, 2018, as compared to $9.7 million for the same period in 2017. Net cash used in the 2018 period resulted primarily from the net loss of $7.3 million and changes in operating assets and liabilities of $0.2 million, offset by net adjustments for non-cash items of $0.9 million. Net cash used in the 2017 period resulted primarily from the net loss of $9.8 million and changes in operating assets and liabilities of $0.9 million, partially offset by net adjustments for non-cash items of $1.0 million.

Sunesis reported loss from operations of $7.1 million for the three months ended March 31, 2018, as compared to $9.4 million for the same period in 2017. Net loss was $7.3 million for the three months ended March 31, 2018, as compared to $9.8 million for the same period in 2017.

Conference Call Information

Sunesis will host a conference today at 4:30 p.m. Eastern Time. The call can be accessed by dialing (844) 296-7720 (U.S. and Canada) or (574) 990-1148 (international) and entering passcode 9676198. To access the live audio webcast, or the subsequent archived recording, visit the "Investors and Media – Calendar of Events" section of the Sunesis website at www.sunesis.com. The webcast will be recorded and available for replay on the company’s website for two weeks.

On May 8, 2018 Clovis Oncology, Inc. (NASDAQ:CLVS) reported financial results for the quarter ended March 31, 2018, and provided an update on the Company’s clinical development programs and regulatory and commercial outlook for 2018 (Press release, Clovis Oncology, MAY 8, 2018, View Source [SID1234526239]).

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"We were very pleased to receive the expanded maintenance treatment indication for Rubraca in the U.S. in early April, and while it is early days in the launch, we are receiving very positive feedback to the broader and earlier-line label from clinicians," said Patrick J. Mahaffy, President and CEO of Clovis Oncology. "We are well positioned now with a strong balance sheet and a robust clinical development program for Rubraca, initiating new Clovis-sponsored single-arm studies in ovarian and bladder cancers with Opdivo by year-end, and looking forward to presenting initial data from TRITON2 in prostate cancer at ESMO (Free ESMO Whitepaper) in October. Finally, we are enthusiastic about initiating a global clinical development program for lucitanib, including multiple combination studies."

First Quarter 2018 Financial Results

Clovis reported net product revenue for Rubraca of $18.5 million for the first quarter of 2018. During the first quarter, the supply of free drug distributed to eligible patients through the Rubraca patient assistance program was approximately 22 percent of overall commercial supply. This would have represented an additional $5.1 million in commercial value for the quarter. We expect the supply of free drug to remain at this percentage or slightly higher for the foreseeable future. Net product revenue for the quarter ended March 31, 2017 was $7.0 million, following the initial approval and launch of Rubraca in the treatment setting on December 19, 2016.

Clovis had $463.8 million in cash, cash equivalents and available-for-sale securities as of March 31, 2018. In April 2018, Clovis raised net proceeds of $94.0 million through an offering of 1.8 million shares of common stock and $292 million aggregate principal amount of 1.25% convertible senior notes due 2025. The net proceeds from these offerings were $386.0 million, after deducting underwriting discounts and commissions, and offering expenses.

Clovis reported a net loss for the first quarter of 2018 of $77.7 million, or ($1.54) per share. The net loss for the first quarter of 2017 was $58.5 million, or ($1.33) per share. Net loss for the first quarter of 2018 included share-based compensation expense of $11.9 million, compared to $8.9 million for the first quarter of 2017.

Cash used in operating activities was $100.6 million for the first quarter of 2018, compared with $80.4 million in the first quarter of 2017. This includes product supply costs of $31.5 million in the first quarter of 2018, compared to $18.2 million for the first quarter of 2017. Product supply costs will be approximately $44 million in the second quarter of 2018 and will be approximately $10 million for the remainder of 2018. These costs reflect Clovis’ plan to build additional inventory in advance of the transition to a new manufacturing facility for Rubraca. The Company will also incur final capital costs for the new manufacturing facility of approximately $8 million in late 2018 as well. Additionally, Clovis will make one-time milestone payments to Pfizer of $38 million in the second quarter of 2018 related to product approvals in December 2016 and April 2018 and potentially an additional $20 million milestone payment in the second quarter if the Rubraca Marketing Authorization in Europe is granted prior to June 15, 2018.

Clovis had approximately 50.7 million shares of common stock outstanding as of March 31, 2018.

Research and development expenses totaled $43.5 million for the first quarter of 2018, compared to $32.4 million for the first quarter 2017. Research and development expenses will continue to increase compared to last year as planned Rubraca studies progress.

Selling, general and administrative expenses totaled $39.3 million for the first quarter of 2018, compared to $29.2 million for the first quarter in 2017. Selling, general and administrative expenses will continue to increase compared to last year in support of commercial activities related to Rubraca in the United States and Europe.

Key Milestones and Objectives for Rubraca

U.S. Approval for Ovarian Cancer Maintenance Treatment Indication

On April 6, the U.S. Food and Drug Administration (FDA) approved Rubraca (rucaparib) tablets for the maintenance treatment of adult patients with recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in a complete or partial response to platinum-based chemotherapy. FDA granted regular approval for Rubraca in this second, broader and earlier-line indication on a priority review timeline based on positive data from the phase 3 ARIEL3 clinical trial. Biomarker testing is not required for patients to be prescribed Rubraca in this maintenance treatment indication. In addition to granting Rubraca approval in this second indication, the FDA converted the approval of the initial treatment indication from an accelerated to a regular approval.

European Union (EU) Treatment Approval Anticipated in Q2 2018

In late March, the Company announced that the European Union’s (EU) European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending the granting of a conditional marketing authorization for Rubraca as monotherapy treatment of adult patients with platinum-sensitive, relapsed or progressive, BRCA mutated (germline and/or somatic), high-grade epithelial ovarian, fallopian tube, or primary peritoneal cancer, who have been treated with two or more prior lines of platinum-based chemotherapy, and who are unable to tolerate further platinum-based chemotherapy. Clovis continues to anticipate the grant of the Marketing Authorization by the European Commission to follow in Q2 2018, and plans to submit a variation to the Marketing Authorization for the maintenance treatment indication, with the CHMP opinion for maintenance anticipated by the end of 2018. Clovis continues to establish its EU organization to support the planned launch of Rubraca in Europe.

Also during the quarter, the Company initiated an early access program in Europe for rucaparib for treatment and as maintenance therapy in recurrent ovarian cancer. The program, to be known as the Rucaparib Access Program (RAP), will enable participation from certain countries in Europe, where permitted by applicable rules, procedures and regulatory authorities. The RAP protocol allows for rucaparib treatment of an individual patient with third-line or greater BRCA mutant epithelial, fallopian tube, or primary peritoneal ovarian cancer who has platinum-sensitive disease and is unable to tolerate further platinum-based chemotherapy or has platinum-resistant disease and needs treatment with single agent rucaparib. The RAP protocol will also provide access to rucaparib for maintenance therapy of an individual patient with recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer who has received at least two prior platinum-based treatment regimens, has platinum-sensitive disease, and is in a complete or partial response to the most recent platinum-based regimen. In all cases, the patient must have a special clinical need that cannot be met by current licensed available medicines. Patients must be ineligible for Clovis’ ARIEL4 clinical trial or unable to access a participating ARIEL4 site to qualify for Clovis’ early access program. Questions or inquiries regarding the RAP should be directed to [email protected].

Rubraca Clinical Development

Clovis has a robust clinical development program underway in multiple tumor types, including Clovis-sponsored, partner-sponsored and investigator-initiated trials. The following clinical studies are open for enrollment or are anticipated to open during the next several months:

The Clovis-sponsored ARIEL4 confirmatory study in the treatment setting is a Phase 3 multicenter, randomized study of Rubraca versus chemotherapy in relapsed ovarian cancer patients with BRCA mutations who have failed two prior lines of therapy. This study is currently enrolling patients.
The Clovis-sponsored Phase 3 ATHENA study in advanced ovarian cancer in the first-line maintenance treatment setting evaluating Rubraca plus Opdivo (PD-1 inhibitor), Rubraca, Opdivo and placebo in newly-diagnosed patients who have completed platinum-based chemotherapy. This study, as part of a broad clinical collaboration with Bristol-Myers Squibb, is expected to begin in the first half of 2018.
The Clovis-sponsored TRITON3 study, a Phase 3 comparative study in mCRPC enrolling BRCA mutant and ATM mutant (both inclusive of germline and somatic) patients who have progressed on AR-targeted therapy and who have not yet received chemotherapy in the castrate-resistant setting is also open for enrollment. TRITON3 compares Rubraca to physician’s choice of AR-targeted therapy or chemotherapy in these patients. This study is currently enrolling patients.
The Clovis-sponsored TRITON2 study in mCRPC, a Phase 2 single-arm study enrolling patients with BRCA mutations and ATM mutations (both inclusive of germline and somatic) or other deleterious mutations in other homologous recombination (HR) repair genes. All patients will have progressed after receiving one line of taxane-based chemotherapy and one or two lines of androgen-receptor (AR) targeted therapy. This study is currently enrolling patients. The Company plans to present initial data from the ongoing TRITON2 study at ESMO (Free ESMO Whitepaper) in October 2018, pending abstract acceptance.
A Clovis-sponsored single-arm Phase 2 open-label monotherapy study of Rubraca in recurrent, metastatic bladder cancer titled ATLAS: A Study of Rucaparib in Patients with Locally Advanced or Metastatic Urothelial Carcinoma. This study is currently enrolling patients.
The Phase 1 RUCA-J study, sponsored by Clovis, initiated during the quarter with the first patient dosed with rucaparib in Japan. The Phase 1 study seeks to identify the recommended dose of rucaparib in Japanese patients, which will enable development of a bridging strategy and potential inclusion of Japanese sites in planned or ongoing global studies.
A Phase 2, open-label, multi-cohort study evaluating the combination of Rubraca and Opdivo in patients with relapsed, BRCA wild-type ovarian cancer and in patients with locally advanced or metastatic bladder carcinoma who are ineligible for treatment with cisplatin. This study is sponsored by Clovis and is expected to begin in the second half of 2018.
The Phase 3 pivotal study in advanced triple-negative breast cancer (TNBC) to evaluate Opdivo and Rubraca in combination. This study is sponsored by Bristol-Myers Squibb.
The Phase 2 combination study of Opdivo with Rubraca for the treatment of mCRPC. This study, sponsored by Bristol-Myers Squibb, is being conducted as an arm of a larger sponsored prostate cancer study. This study is currently enrolling patients.
The Phase 1b combination study of the cancer immunotherapy Tecentriq (atezolizumab; anti-PDL1) and Rubraca for the treatment of ovarian and triple-negative breast cancers. This study is sponsored by Roche and is currently enrolling patients.
Exploratory studies in other tumor types are also underway.

Lucitanib Clinical Development

Lucitanib is an oral, potent inhibitor of the tyrosine kinase activity of vascular endothelial growth factor receptors 1 through 3 (VEGFR1-3), platelet-derived growth factor receptors alpha and beta (PDGFR α/β) and fibroblast growth factor receptors 1 through 3 (FGFR1-3), which was previously evaluated in breast and lung cancers in partnership with Servier. Clovis has received notice from Servier that they will return their ex-US rights (excluding China) for lucitanib later in 2018. Clovis therefore will own global rights (excluding China) to lucitanib. There are no payments from Clovis to Servier related to the return of these ex-US rights.

Lucitanib was originally developed by Clovis and Servier with the hypothesis of activity in FGFR driven tumors; data in breast and lung cancer were insufficient to move the program forward. Recent data for a similar drug that inhibits these same three pathways – when combined with a PD-1 inhibitor – are extremely encouraging and represent a validated and alternative hypothesis for the development of lucitanib in combination with a PD(L)-1 inhibitor, and a Clovis-sponsored combination study is now being planned. Clovis also intends to initiate a study of lucitanib in combination with rucaparib, based on encouraging data of VEGF and PARP inhibitors in combination. Each of these studies is expected to initiate before the end of Q1 2019.

Conference Call Details

Clovis will hold a conference call to discuss Q1 2018 results this afternoon, May 8, at 4:30pm ET. The conference call will be simultaneously webcast on the Company’s web site at www.clovisoncology.com, and archived for future review. Dial-in numbers for the conference call are as follows: US participants 866.489.9022, International participants 678.509.7575, conference ID: 4198438.

About Rubraca (rucaparib)

Rubraca is an oral, small molecule inhibitor of PARP1, PARP2 and PARP3 being developed in ovarian cancer as well as several additional solid tumor indications. Studies open for enrollment or under consideration include ovarian, prostate, breast, gastroesophageal, pancreatic, lung and bladder cancers. Clovis holds worldwide rights for Rubraca.

In the United States, Rubraca is approved for the maintenance treatment of adult patients with recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in a complete or partial response to platinum-based chemotherapy. Rubraca is also approved in the United States for the treatment of adult patients with deleterious BRCA mutation (germline and/or somatic) associated epithelial ovarian, fallopian tube, or primary peritoneal cancer who have been treated with two or more chemotherapies, and selected for therapy based on an FDA-approved companion diagnostic for Rubraca.

Rubraca is an unlicensed medical product outside of the U.S.

On May 8, 2018 OPKO Health, Inc. (NASDAQ: OPK) reported financial results and business highlights for the three months ended March 31, 2018 (Press release, Opko Health, MAY 8, 2018, View Source [SID1234526238]).

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Financial Highlights

Consolidated revenues for the three months ended March 31, 2018 were $254.9 million compared with $266.4 million for the comparable period of 2017.

Revenue from services were $211.3 million for the three months ended March 31, 2018 compared with $228.6 million for the comparable 2017 period. While not directly comparable, this represents a marked improvement from the most recently completed quarter ended December 31, 2017.

Revenue from products included $3.7 million of revenue from RAYALDEE during the first quarter of 2018. Revenues from RAYALDEE were deferred during the initial launch period so there is no prior-year comparison.
During the three months ended March 31, 2018, operating expenses included investment in commercial activities supporting the launch of RAYALDEE of $7.3 million and continued investment in our pharmaceutical pipeline, with R&D expense increasing $6.3 million to $32.9 million compared with $26.6 million for the 2017 period.

Net loss of $43.1 million or $0.08 per share during the three months ended March 31, 2018 compares with a net loss of $34.5 million or $0.06 per share for the comparable period of 2017

Cash, cash equivalents and marketable securities were $99.9 million as of March 31, 2018.

"We are pleased to report steady sequential-quarter growth for RAYALDEE and 4Kscore, as well as improvement in our lab business from Q4 of last year," said Phillip Frost, Chairman and Chief Executive Officer of OPKO Health. "The improved first quarter results were in line with our expectations, starting 2018 positively coming off challenging fourth quarter 2017 results. These factors, taken together, reflect progress across the breadth of our commercial and laboratory services."

Business Highlights

RAYALDEE total prescriptions reported by IMS for Q1 2018 increased 730% compared with Q1 2017 and 38% compared with Q4 2017: As of May 1, 2018, more than 79% of patients had access to RAYALDEE under their insurance plans.

4Kscore utilization in Q1 2018 increased 13% compared with Q1 2017: Five abstracts of studies further demonstrating the utility of the 4Kscore test in the management of prostate cancer are to be presented at the American Urological Association meeting May 18-21.

Appointed Geoff Monk as General Manager, BioReference Laboratories: Mr. Monk is well prepared to lead BRL with more than 20 years of management experience in the diagnostic laboratory business. Mr. Monk was previously Managing Director of the New York and New Jersey unit of Quest Diagnostics.

Advanced the Phase 2b trial for our SARM (selective androgen receptor modulator) to treat benign prostatic hyperplasia (BPH): Enrollment is ongoing in this dose-ranging study for our orally administered SARM. This medicine is expected to improve the symptoms of BPH by reducing prostate size and, on the basis of data from a previous trial in 350 men, increase muscle mass and bone strength and decrease body fat. BPH affects approximately 50 million men in the U.S.

Initiated a Phase 2b clinical trial for OPK88003, our once-weekly oxyntomodulin dual GLP1-Glucagon agonist to treat type 2 diabetes and obesity: In a previous Phase 2 trial in 420 overweight patients with type 2 diabetes, the drug was shown to be safe and effective. The current trial is to study a new dosing schedule to achieve even greater weight loss.

Premarket Approval (PMA) application for Claros point-of-care PSA test under review by FDA: OPKO has submitted a PMA for a PSA test utilizing the Claros 1 immunoassay analyzer, a novel diagnostic instrument that can provide rapid, quantitative blood test results in 10 minutes in the physician’s office with only a finger stick drop of whole blood. A second product utilizing the Claros platform to measure testosterone is advancing toward a 510(k) submission to the FDA later this year.

Global and Japanese Phase 3 registration trials for hGH-CTP in pediatric growth hormone deficient children are continuing to enroll patients: The global pediatric study compares a single weekly administration with daily injections of a currently marketed growth hormone product. The global and Japanese pediatric studies utilize the pen device and formulation that will be launched commercially upon approval. The pediatric segment represents more than 80% of the market for treatment of hGH deficiency.

Initiation of three additional Phase 2 clinical trials are anticipated in 2018:

RAYALDEE line extension in dialysis patients with secondary hyperparathyroidism (SHPT): Together with our partners, Vifor Fresenius and Japan Tobacco, OPKO is developing RAYALDEE for Stage 5 chronic kidney disease (CKD) patients with SHPT undergoing dialysis and anticipates initiating a global Phase 2 trial during Q3 of this year.

OPKO’s NK-1 antagonist to treat pruritus (itching) in Stage 5 CKD patients undergoing dialysis: An Investigational New Drug application was submitted to the FDA and plans are being finalized to begin a single-dose Phase 2a trial in dialysis patients to treat severe itching. Approximately 50% of renal dialysis patients experience pruritus.

OPKO’s AntagoNAT oligonucleotide OPK88001 to treat Dravet’s syndrome: Three clinical research centers in the United States are expected to participate in this first-in-human trial of OPKO’s AntagoNAT to treat Dravet’s syndrome.
Conference Call & Webcast Information

OPKO’s senior management will provide a business update and discuss results in greater detail in a conference call and live audio webcast at 4:30 p.m. Eastern time today. The conference call dial-in and webcast information is as follows:

WHEN: Tuesday, May 8, 2018 at 4:30 p.m. Eastern time.
DOMESTIC DIAL-IN: 866-634-2258
INTERNATIONAL DIAL-IN : 330-863-3454
PASSCODE: 5976529
WEBCAST: View Source

For those unable to participate in the live conference call or webcast, a replay will be available beginning May 8, 2018 two hours after the close of the conference call. To access the replay, dial (855) 859-2056 or (404) 537-3406. The replay passcode is: 5976529. The replay can be accessed for a period of time on OPKO’s website at View Source.

On May 8, 2018 Kura Oncology, Inc., (Nasdaq: KURA) a clinical-stage biopharmaceutical company focused on the development of precision medicines for oncology, reported first quarter 2018 financial results and provided a corporate update (Press release, Kura Oncology, MAY 8, 2018, View Source [SID1234526237]).

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"I am very pleased with the progress we made over the past quarter, highlighted by a successful end of Phase 2 meeting with the FDA," said Troy Wilson, Ph.D., J.D., President and Chief Executive Officer of Kura Oncology. "We are working diligently to initiate AIM-HN, our registration-directed trial of tipifarnib in HRAS mutant HNSCC, as well as SEQ-HN, our concurrent screening and outcomes study. We believe that tipifarnib has the potential to become an important treatment option for patients with HRAS mutant HNSCC, and we are committed to executing the clinical and regulatory strategy that best positions it for success."

"We are also excited about the potential to expand the clinical utility of tipifarnib to other solid tumor and hematologic indications," continued Dr. Wilson. "We are evaluating tipifarnib in multiple biomarker-guided Phase 2 clinical trials, and our goal is to generate proof-of-concept data in one or more of these additional indications by year end. We believe we have the cash runway to advance tipifarnib and our emerging pipeline of drug candidates through a series of upcoming potential data catalysts, and we look forward to providing further updates on our progress in the months ahead."

Corporate Update

Registration-directed trial of tipifarnib in HRAS mutant HNSCC – Following a positive Phase 2 trial in HRAS mutant HNSCC and a successful end of Phase 2 meeting with the FDA, Kura is planning to conduct a global, registration-directed trial of its lead drug candidate tipifarnib in at least 59 recurrent or metastatic patients with HRAS mutant HNSCC. The primary endpoint of the trial will be objective response rate. Based on feedback from the FDA, Kura believes that the single-arm trial, if positive, could support an application for accelerated approval. Kura anticipates that the trial, called AIM-HN, will require fewer than 100 clinical sites worldwide and take approximately two years to enroll. Kura expects to initiate the AIM-HN trial in the second half of 2018.

Screening and outcomes study in HRAS mutant HNSCC – Concurrent with the AIM-HN trial, Kura is also planning to conduct a non-interventional, case-control study in HNSCC, called SEQ-HN. The study is expected to facilitate the identification of patients with HRAS mutations for potential enrollment into the AIM-HN trial. In addition, SEQ-HN is designed to characterize the natural history of patients with HRAS mutant HNSCC, which may support any future discussions with regulatory agencies concerning the appropriateness and nature of a potential approval.

Tipifarnib in HRAS mutant lung squamous cell carcinoma (LSCC) – Kura recently presented new preclinical data showing that tipifarnib is highly active in HRAS mutant LSCC tumor models. The data, presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in April 2018, illustrate the potential for tipifarnib in the treatment of HRAS mutant LSCC. Kura is collaborating with the Spanish Lung Cancer Group, a cooperative group consisting of more than 150 public and private oncology centers in Spain, on a proof-of-concept trial of tipifarnib in HRAS mutant LSCC. Kura anticipates this investigator-sponsored trial to initiate later this year.

Patent protection for tipifarnib in hematologic malignancies – Kura recently announced the issuance of U.S. patent 9,956,215, "Methods of Treating Cancer Patients with Farnesyltransferase Inhibitors." The newly issued patent includes multiple claims directed to the use of tipifarnib as a method of treating patients with CXCL12-expressing peripheral T-cell lymphoma (PTCL) and acute myeloid leukemia (AML) and has an expiration date of November 2037, excluding any possible patent term extension. This patent comes less than one year after the U.S. Patent and Trademark Office issued a similar patent for tipifarnib in HRAS mutant HNSCC, reinforcing the potential of Kura’s broader strategy to generate intellectual property related to its drug candidates and their use in treating genetically defined patient populations.

Potential biomarker for KO-947 in squamous cell carcinomas – At the AACR (Free AACR Whitepaper) Annual Meeting in April 2018, Kura presented new preclinical data for its ERK inhibitor, KO-947, including the identification of 11q13 amplification as a potential biomarker of activity in squamous cell carcinomas. Amplification of chromosomal region 11q13 is a common genetic alteration in squamous cell carcinomas, comprising approximately 20% of HNSCC and 50% of esophageal squamous cell carcinoma.

Upcoming Milestones

Initiation of AIM-HN, a registration-directed trial of tipifarnib in HRAS mutant HNSCC, and SEQ-HN, a non-interventional, case-control study in HRAS mutant HNSCC, in the second half of 2018

Additional data from RUN-HN, an ongoing Phase 2 trial of tipifarnib in HRAS mutant HNSCC, in the second half of 2018

Biomarker-enriched data from ongoing Phase 2 trials of tipifarnib in hematologic malignancies in the second half of 2018

Initiation of a proof-of-concept study of tipifarnib in HRAS mutant LSCC sponsored by the Spanish Lung Cancer Group in 2018

Data from a Phase 1 dose-escalation trial of KO-947 in solid tumors in the second half of 2018

Submission of an investigational new drug application for KO-539, a potent and selective inhibitor of the menin-MLL interaction, in late 2018 or early 2019

Financial Results

Research and development expenses for the first quarter of 2018 were $11.6 million, compared to $5.5 million for the first quarter of 2017.

General and administrative expenses for the first quarter of 2018 were $3.4 million, compared to $2.1 million for the first quarter of 2017.

Net loss for the first quarter of 2018 was $14.6 million, compared to $7.5 million for the first quarter of 2017.

Cash, cash equivalents and short-term investments totaled $138.2 million as of March 31, 2018, which included $57.7 million in net proceeds under an ATM facility in January 2018, compared with $93.1 million as of December 31, 2017.

Management expects that current cash, cash equivalents and short-term investments will be sufficient to fund its current operations into the first half of 2020.

Conference Call and Webcast

Kura’s management will host a webcast and conference call today at 4:30 p.m. ET / 1:30 p.m. PT today, May 8, 2018, to discuss the financial results for the first quarter of 2018 and provide a corporate update. The live call may be accessed by dialing (877)

516-3514 for domestic callers and (281) 973-6129 for international callers and using conference ID #7199738. A live webcast of the call will be available from the Investors and Media section of the company website at www.kuraoncology.com, and will be archived there for 30 days.

On May 8, 2018 Jazz Pharmaceuticals plc (Nasdaq: JAZZ) reported financial results for the first quarter of 2018 and updated financial guidance for 2018 (Press release, Jazz Pharmaceuticals, MAY 8, 2018, View Source;p=RssLanding&cat=news&id=2347828 [SID1234526236]).

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"The first quarter was highlighted by strong revenue growth, cash flow generation and execution across the organization that led to significant progress toward our 2018 goals," said Bruce Cozadd, chairman and chief executive officer of Jazz Pharmaceuticals. "The recent submission of our supplemental NDA for Xyrem for pediatric narcolepsy patients and FDA acceptance of our NDA for solriamfetol for excessive sleepiness in patients with narcolepsy or OSA result from our focused investment in advancing our promising R&D pipeline. Over the next 18 months, we look forward to fueling our portfolio with innovative product candidates and delivering on multiple regulatory milestones and product launches."

GAAP net income for the first quarter of 2018 was $46.0 million, or $0.75 per diluted share, compared to $86.5 million, or $1.41 per diluted share, for the first quarter of 2017.

Adjusted net income for the first quarter of 2018 was $182.4 million, or $2.98 per diluted share, compared to $141.2 million, or $2.31 per diluted share, for the first quarter of 2017. Reconciliations of applicable GAAP reported to non-GAAP adjusted information are included at the end of this press release.

Total revenues increased 18% in the first quarter of 2018 compared to the same period in 2017 due to an increase in net product sales of Xyrem and the launch of Vyxeos.

Xyrem net product sales increased 16% in the first quarter of 2018 compared to the same period in 2017.

Erwinaze/Erwinase net product sales in the first quarter were consistent with the same period in 2017. The company is currently experiencing supply disruptions and expects that there may be further supply challenges during 2018.

Defitelio/defibrotide net product sales in the first quarter of 2018 were consistent with the same period in 2017. The company continues to expect inter-quarter variability in Defitelio net sales given that veno-occlusive disease is an ultra-rare disease.

Vyxeos net product sales were $26.2 million in the first quarter of 2018. Vyxeos launched in the U.S. in August 2017.

Operating expenses changed over the prior year period primarily due to the following:

Selling, general and administrative (SG&A) expenses increased in the first quarter of 2018 compared to the same period in 2017 on a GAAP and on a non-GAAP adjusted basis due to higher expenses resulting from expansion of the company’s business, including expenses supporting the potential EU launch of Vyxeos and U.S. launch of solriamfetol. SG&A expenses in the first quarter of 2018 on a GAAP basis also included an estimated loss contingency of $57.0 million related to an ongoing U.S. Department of Justice (DOJ) investigation of our support of 501(c)(3) organizations that provide financial assistance to Medicare patients. In April 2018, the company reached an agreement in principle with the DOJ on a proposal for a civil settlement of potential claims relating to the investigation, subject to negotiation of a definitive settlement agreement and other contingencies.
Research and development (R&D) expenses increased in the first quarter of 2018 compared to the same period in 2017 on a GAAP and on a non-GAAP adjusted basis due to an increase in expenses related to the company’s ongoing pre-clinical and clinical development programs and regulatory activities. R&D expenses in the first quarter of 2018 on a GAAP basis also included milestone payments of $11.0 million related to the U.S. Food and Drug Administration’s (FDA) acceptance for filing of the company’s New Drug Application (NDA) for solriamfetol.
Cash Flow and Balance Sheet

As of March 31, 2018, cash, cash equivalents and investments were $708.2 million, and the outstanding principal balance of the company’s long-term debt was $1.8 billion. During the first quarter of 2018, we generated $162.4 million of cash from operations, used $34.5 million to repurchase approximately 238,000 ordinary shares under the company’s share repurchase program at an average cost of $145.34 per ordinary share and made milestone payments totaling $11.0 million.

Recent Developments

In March 2018, the FDA accepted for filing with standard review the company’s NDA seeking marketing approval for solriamfetol, an investigational medicine for the treatment of excessive sleepiness in adult patients with narcolepsy or obstructive sleep apnea (OSA). The Prescription Drug User Fee Act (PDUFA) date for an FDA decision is December 20, 2018.

In April 2018, the company entered into an agreement with Spark Therapeutics, Inc. to purchase a rare pediatric disease priority review voucher (PRV) for $110 million that will allow the company to accelerate the review process by the FDA for one of its future regulatory submissions.

In April 2018, the company submitted a supplemental NDA to the FDA seeking marketing approval for Xyrem in the treatment of pediatric narcolepsy patients with cataplexy and excessive daytime sleepiness.

Conference Call Details

Jazz Pharmaceuticals will host an investor conference call and live audio webcast today at 4:30 p.m. EDT (9:30 p.m. IST) to provide a business and financial update and discuss its 2018 first quarter results. The live webcast may be accessed from the Investors section of the company’s website at www.jazzpharmaceuticals.com. Please connect to the website prior to the start of the conference call to ensure adequate time for any software downloads that may be necessary. Investors may participate in the conference call by dialing +1 855 353 7924 in the U.S., or +1 503 343 6056 outside the U.S., and entering passcode 9868758.

A replay of the conference call will be available through May 15, 2018 by dialing +1 855 859 2056 in the U.S., or +1 404 537 3406 outside the U.S., and entering passcode 9868758. An archived version of the webcast will be available for at least one week in the Investors section of the company’s website at www.jazzpharmaceuticals.com.