On February 26, 2019 Clovis Oncology, Inc. (NASDAQ:CLVS) reported financial results for the quarter and year ended December 31, 2018, and provided an update on the Company’s clinical development programs and regulatory and commercial outlook for 2019 (Press release, Clovis Oncology, FEB 26, 2019, View Source [SID1234533683]).

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"We are pleased to have renewed U.S. revenue growth in the fourth quarter, and with our maintenance treatment indication approved in the EU, we look forward to our initial European launch in Germany on March 1," said Patrick J. Mahaffy, President and CEO of Clovis Oncology. "In addition, we are targeting an sNDA filing for BRCA1/2-mutant metastatic castration-resistant prostate cancer (mCRPC) in late 2019, and we expect to present data from both the TRITON2 mCRPC study and the ATLAS study in advanced bladder cancer at a Fall 2019 medical meeting. We are also very pleased to further our clinical collaboration with Bristol-Myers Squibb to not only include a combination study of Opdivo and lucitanib, but also to discuss tumor types for potential additional combination studies with Rubraca."

Fourth Quarter and Year-End 2018 Financial Results

Clovis reported product revenue for Rubraca of $30.4 million for the fourth quarter of 2018 and $95.4 million for the year ended December 31, 2018. For both the fourth quarter and year, the supply of free drug distributed to eligible patients through the Rubraca patient assistance program was approximately 26 percent of overall commercial supply. This represented $10.4 million in commercial value for the fourth quarter and $33.4 million in commercial value for the full year. Net product revenue for the quarter and fiscal year ended December 31, 2017 was $17.0 million and $55.5 million. Rubraca was initially approved and launched in the ovarian cancer treatment setting in the U.S. in December 2016 and in April 2018 the Rubraca label was expanded to include the broader and earlier-line maintenance treatment indication.

Clovis had $520.1 million in cash, cash equivalents and available-for-sale securities as of December 31, 2018. Cash used in operating activities was $82.7 million for the fourth quarter of 2018 and $366.0 million for the year ended December 31, 2018, compared with $65.6 million and $260.9 million for the comparable periods of 2017. This includes product supply costs of $22.7 million in the fourth quarter of 2018 and $98.8 million for the year ended December 31, 2018, compared to $12.0 million and $53.5 million for the comparable periods in 2017. Clovis had approximately 52.8 million shares of common stock outstanding as of December 31, 2018. In April 2018, Clovis raised net proceeds of $93.9 million through an offering of 1.8 million shares of common stock and $290.9 million in proceeds on $300 million aggregate principal amount of 1.25% convertible senior notes due 2025. The net proceeds from these offerings were $384.8 million, after deducting underwriting discounts and commissions, and offering expenses.

Clovis reported a net loss for the fourth quarter of 2018 of $99.3 million, or ($1.88) per share, and $368.0 million or ($7.07) per share for the year ended December 31, 2018. The net loss for the fourth quarter of 2017 was $51.9 million, or ($1.04) per share and $346.4 million, or ($7.36) per share for the year ended December 31, 2017. Net loss for the fourth quarter of 2018 included share-based compensation expense of $11.4 million and $49.1 million for the full year 2018, respectively, compared to $12.5 million and $44.7 million for the comparable periods of 2017.

The net loss for the year ended December 31, 2018 includes a one-time charge of $20 million related to a final settlement reached with the Securities and Exchange Commission which resolves their investigation related to rociletinib, and a charge of $8.0 million related to a legal settlement. The net loss for the year ended December 31, 2017 included a charge of $105.5 million related to the portion of a legal settlement that was paid in Clovis common stock. The adjusted net loss excluding these items was $99.3 million or ($1.88) per share for the fourth quarter and $340.0 million or ($6.53) per share for the fiscal year ended 2018 and $63.4 million or ($1.27) per share for the fourth quarter and $240.9 million or ($5.12) per share for the fiscal year ended 2017.

Research and development expenses totaled $71.2 million for the fourth quarter of 2018, and $231.3 million for the full year 2018, compared to $38.0 million and $142.5 million, respectively, for the comparable periods in 2017. The increase year over year is primarily due to higher research and development costs for rucaparib clinical trials, including increased enrollment in ongoing ovarian and prostate studies, increased costs related to initiating new ovarian and bladder studies during 2018, as well as additional headcount to support these increased rucaparib clinical trial activities.

Selling, general and administrative expenses totaled $49.1 million for the fourth quarter of 2018, and $175.8 million for the full year 2018, compared to $38.5 million and $138.9 million for the comparable periods in 2017. The increase year over year is primarily due to higher selling, general and administrative expenses related to the commercialization of Rubraca in the U.S. and the EU, including facility expenses and personnel expenses associated with establishing the EU infrastructure.

Key Milestones and Objectives for Rubraca

Recurrent Ovarian Cancer Maintenance Treatment Indication Approved in both U.S. and the EU

In April 2018, Rubraca was granted its second indication by the U.S. FDA as 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. This approval, in a broader and earlier-line indication, was 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 January 2019, the European Commission (EC) also approved the use of Rubraca for its second indication, as monotherapy for the maintenance treatment of adults with platinum-sensitive relapsed high-grade epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in response (complete or partial) to platinum-based chemotherapy. This approval expands rucaparib’s indication beyond its initial marketing authorization in the EU granted in May 2018, and in this indication Rubraca is available to eligible patients regardless of their BRCA mutation status. Rucaparib was the first PARP inhibitor licensed for an ovarian cancer treatment indication in Europe and is the first to be available for both treatment and maintenance treatment among eligible patients. Clovis has established its EU organization to support its initial launch in Germany this Friday, March 1 and anticipates launching in other EU countries later in 2019 and 2020.

Regulatory Path for Supplemental NDA in BRCA-mutant Advanced Prostate Cancer Based on TRITON2 Dataset and Breakthrough Therapy Designation

Initial data from the Company’s ongoing TRITON studies of Rubraca in advanced prostate cancer were presented at the ESMO (Free ESMO Whitepaper) 2018 Congress (European Society for Medical Oncology) in October 2018. The initial TRITON2 data showed a 44% confirmed objective response rate (ORR) by investigator assessment in 25 RECIST1/PCWG3** response-evaluable patients with a BRCA1/2 alteration, and results by independent assessment were consistent. The median duration of response in these patients had not yet been reached. In addition, a 51% confirmed prostate specific antigen (PSA) response rate was observed in 45 PSA response-evaluable patients with a BRCA1/2 alteration. Preliminary safety data for Rubraca in men with mCRPC were consistent with those observed in patients with ovarian cancer and other solid tumors.

The TRITON2 results were the basis for Breakthrough Therapy designation for Rubraca as a monotherapy treatment of adult patients with BRCA1/2 mutated mCRPC who have received at least one prior androgen receptor (AR)-directed therapy and taxane-based chemotherapy, which was granted on October 1, 2018 by the U.S. Food and Drug Administration (FDA). Both studies in the TRITON program, TRITON2 and TRITON3, continue to enroll patients.

Pending data maturity, Clovis is targeting the filing of a supplemental NDA (sNDA) for Rubraca in BRCA-mutant advanced prostate cancer in late 2019.

____________________

1
Response Evaluation Criteria in Solid Tumors (RECIST) is a standardized methodology for determining therapeutic response to anticancer therapy using changes in lesion appearance on imaging studies.

**
Prostate Cancer Working Group (PCWG3) is an international expert committee of prostate cancer clinical investigators who have recommended modifications to RECIST for use in the conduct of trials in metastatic castration-resistant prostate cancer (mCRPC) which were adopted in the TRITON2 protocol.

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 currently enrolling patients.
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 androgen-receptor (AR)-targeted therapy and who have not yet received chemotherapy in the castration-resistant setting. 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 in patients with BRCA mutations (inclusive of germline and somatic) and is also enrolling patients with deleterious mutations of 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 AR-targeted therapy. This study is currently enrolling patients. Updated data from the ongoing TRITON2 study are anticipated at a Fall 2019 medical meeting.
The Clovis-sponsored ATLAS study is a single-arm Phase 2 open-label monotherapy study of Rubraca in recurrent, metastatic bladder cancer. This study is currently enrolling patients, and initial data from the ongoing ATLAS study are anticipated at a Fall 2019 medical meeting.
The Phase 1 RUCA-J study, sponsored by Clovis, has identified the 600mg BID dose of rucaparib as the recommended dose in Japanese patients; this will enable development of a bridging strategy and potential inclusion of Japanese sites in planned or ongoing global studies. This study is currently enrolling patients.
The Phase 2, open-label, multi-cohort ARIES study evaluating the combination of Rubraca and Opdivo in patients with relapsed ovarian cancer and patients with locally advanced or metastatic bladder carcinoma. This study is sponsored by Clovis and is expected to begin enrolling patients in the first half of 2019.
The Phase 1/2 combination study of sacituzumab govitecan and Rubraca for the treatment of advanced metastatic triple-negative breast cancer, relapsed platinum-resistant ovarian cancer and metastatic urothelial cancers is sponsored by Clovis and is expected to begin enrolling patients in 2019.
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 in the CHECKMATE 9KD prostate cancer study, and is currently enrolling patients.
Exploratory studies in other tumor types are also underway, as well as active discussions with Bristol-Myers Squibb regarding additional potential combination studies.

Lucitanib Clinical Development

Lucitanib is an investigational, 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 (PDGFRα/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 global rights (excluding China) for lucitanib.

Recent data for a drug that inhibits these same three pathways – when combined with a PD-1 inhibitor – are extremely encouraging and represent a scientific rationale for the development of lucitanib in combination with a PD-1 inhibitor, and a Clovis-sponsored study of lucitanib in combination with Opdivo is planned in gynecologic cancers. Clovis also intends to initiate a study of lucitanib in combination with rucaparib in ovarian cancer, based on encouraging data of VEGF and PARP inhibitors in combination. Each of these Phase 1b/2 studies is expected to initiate during the first half of 2019.

Conference Call Details

Clovis will hold a conference call to discuss Q4/FY 2018 results this morning, February 26, at 8:30am 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 866.393.4306, International participants 734.385.2616, conference ID: 6675335.

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.

In the EU, Rubraca is approved for the maintenance treatment of adults with platinum-sensitive relapsed high-grade epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in response (complete or partial) to platinum-based chemotherapy. This expands rucaparib’s indication beyond its initial marketing authorization in the EU granted in May 2018 and with this label expansion, rucaparib is now available to patients regardless of their BRCA mutation status. Rubraca is also approved in the EU for the 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.

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

On February 26, 2019 Cerus Corporation (Nasdaq: CERS) reported complete financial results for the fourth quarter and year ended December 31, 2018 (Press release, Cerus, FEB 26, 2019, View Source [SID1234533682]).

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Recent developments and highlights include:

Record fourth quarter product revenue of $16.5 million.
Provided 2019 annual product revenue guidance of $70 million to $73 million, representing a 15% to 20% increase over 2018 reported product revenue.
Filed CE Mark registration for the Company’s INTERCEPT Blood System for red blood cells (RBCs).
Initiated enrollment in ReCePI, Cerus’ U.S. Phase 3 study evaluating the safety and efficacy of the INTERCEPT Blood System for RBCs in patients undergoing complex cardiac surgery.
Expanded the executive management team with the appointment of William Moore as senior vice president of manufacturing operations and supply chain.
The recently issued annual planned FDA guidance agenda from CBER (Center for Biologics Evaluation and Research) indicates that a final platelet bacterial safety guidance document is planned for 2019.
"We believe we are entering a transformational period in the U.S., with a final platelet bacterial guidance document now anticipated by the end of this year," said William ‘Obi’ Greenman, Cerus’ president and chief executive officer. "Our recent momentum is expected to continue into 2019 as we push forward on our mission to establish INTERCEPT as the standard of care for transfused blood components globally by executing on our commercial strategy and advancing our pipeline opportunities."

Revenue

Product revenue during the fourth quarter of 2018 was $16.5 million, compared to $16.2 million during the same period in 2017. Strong gains in fourth quarter platelet kit sales were partially offset by a year-over-year decline in illuminator sales. During the fourth quarter of 2017, total product revenue benefited from illuminator shipments pursuant to the Company’s expanded supply agreement with EFS, the French National Blood Service, and large plasma kit orders to distributors. Full-year 2018 product revenue totaled $60.9 million, an increase of 40% compared to 2017 product revenue.

Government contract revenue from the Company’s Biomedical Advanced Research and Development Authority (BARDA) agreement was $3.7 million during the fourth quarter of 2018, compared to $2.4 million during the same period in 2017, as a result of increasing INTERCEPT red blood cell clinical and development activities. Government contract revenue from the Company’s BARDA agreement for the year ended December 31, 2018, was $15.1 million compared to $7.8 million for the year ended December 31, 2017. The total potential value of the current BARDA agreement is $201 million with $25 million recognized as revenue to date.

BARDA is part of the Office of the Assistant Secretary for Preparedness and Response within the U.S. Department of Health and Human Services. The development of the INTERCEPT red blood cell program has been funded in whole or in part with Federal funds from the Department of Health and Human Services; Office of the Assistant Secretary for Preparedness and Response; Biomedical Advanced Research and Development Authority, under Contract No. HHSO100201600009C.

Gross Margins

Gross margins on product revenue during the fourth quarter of 2018 were 49%, compared to 44% for the fourth quarter of 2017. Gross margins in the quarter benefited from a favorable product mix and higher average selling prices for platelet kits. Gross margins on product revenue for the full-year 2018 and 2017 totaled 48%.

Operating Expenses

Total operating expenses for the fourth quarter 2018 were $27.3 million compared to $20.3 million for the same period the prior year. Full-year 2018 operating expenses totaled $99.4 million compared to $86.3 million for the full-year 2017.

Selling, general, and administrative (SG&A) expenses for the fourth quarter of 2018 totaled $14.8 million, compared to $12.6 million for the fourth quarter of 2017. The year-over-year increase was primarily tied to higher commercial activity in the U.S. Full-year 2018 SG&A expenses totaled $56.8 million, compared to $52.6 million for the full-year 2017 with the increase primarily tied to higher headcount and compensation related costs.

Research and development (R&D) expenses for the fourth quarter of 2018 were $12.4 million, compared to $7.8 million for the fourth quarter of 2017. The increase in year-over-year R&D expenses was primarily due to additional activities and costs tied to the development of INTERCEPT red blood cell system, including preparation for the CE Mark submission, trials and activities in pursuit of a potential FDA approval of INTERCEPT red blood cells and activities aimed at obtaining expanded label claims for INTERCEPT platelets and plasma. Full-year 2018 R&D expenses totaled $42.6 million, compared to $33.7 million for the full-year 2017. The increase in full-year 2018 R&D expenses compared to full-year 2017 R&D expenses was primarily due to costs associated with clinical development of INTERCEPT red blood cell system, the pursuit of supplemental approvals for the platelet and plasma systems, and activities related to the BARDA agreement.

Net Loss

Net loss for the fourth quarter of 2018 was $16.2 million, or $0.12 per diluted share, compared to a net loss of $11.5 million, or $0.10 per diluted share, for the fourth quarter of 2017. Net loss for the year ended December 31, 2018, was $57.6 million, or $0.44 per diluted share, compared to a net loss of $60.6 million, or $0.56 per diluted share, for the same period in 2017.

Cash, Cash Equivalents and Investments

At December 31, 2018, the Company had cash, cash equivalents and short-term investments of $117.6 million, compared to $60.7 million at December 31, 2017.

At December 31, 2018, the Company had approximately $29.9 million in outstanding debt under its loan agreement compared to $29.8 million at December 31, 2017.

2019 Product Revenue Guidance

The Company expects 2019 product revenue to be in the range of $70 million to $73 million, representing 15% to 20% growth compared to 2018 reported product revenue.

QUARTERLY CONFERENCE CALL

The Company will host a conference call and webcast at 4:30 P.M. ET this afternoon, during which management will discuss the Company’s financial results and provide a general business overview and outlook. To access the live webcast, please visit the Investor Relations page of the Cerus website at View Source Alternatively, you may access the live conference call by dialing (866) 235-9006 (U.S.) or (631) 291-4549 (international).

A replay will be available on the Company’s website, or by dialing (855) 859-2056 (U.S.) or (404) 537-3406 (international) and entering conference ID number 2392137. The replay will be available approximately three hours after the call through March 12, 2019.

On February 26, 2019 Karyopharm Therapeutics Inc. (Nasdaq:KPTI), a clinical-stage pharmaceutical company, reported that NASDAQ has halting trading of the Company’s common stock. The U.S. Food and Drug Administration (FDA) Oncologic Drugs Advisory Committee (ODAC) is holding a meeting today from 12:30 p.m. to 5:00 p.m. ET to discuss Karyopharm’s New Drug Application (NDA) requesting accelerated approval for selinexor, a first-in-class, oral Selective Inhibitor of Nuclear Export (SINE) compound (Press release, Karyopharm, FEB 26, 2019, View Source [SID1234533681]). The proposed indication to be discussed at this ODAC meeting is for selinexor in combination with dexamethasone for the treatment of patients with relapsed refractory multiple myeloma who have received at least three prior therapies and whose disease is refractory to at least one proteasome inhibitor, one immunomodulatory agent, and one anti-CD38 monoclonal antibody.

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The briefing materials can be found on the FDA website at: View Source

The ODAC is an independent panel of experts that evaluates data concerning the efficacy and safety of marketed and investigational products for use in the treatment of cancer and makes appropriate recommendations to the FDA. Although the FDA will consider the recommendation of the panel, the final decision regarding the approval of the product is made by the FDA solely, and the recommendations by the panel are non-binding.

Karyopharm’s NDA seeking accelerated approval for oral selinexor in combination with dexamethasone as a treatment for patients with triple class refractory multiple myeloma who have received at least three prior therapies is under Priority Review by FDA with an action date of April 6, 2019, under the Prescription Drug User-Fee Act (PDUFA).

The Company has also submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) requesting conditional approval for selinexor in combination with dexamethasone for the treatment of patients with relapsed refractory multiple myeloma who have received at least three prior lines of therapy and whose disease is refractory to at least one PI, one IMiD, and one anti-CD38 monoclonal antibody. The selinexor MAA has been granted accelerated assessment by the EMA’s Committee for Medicinal Products for Human Use.

About Selinexor

Selinexor is a first-in-class, oral Selective Inhibitor of Nuclear Export (SINE) compound. Selinexor functions by binding with and inhibiting the nuclear export protein XPO1 (also called CRM1), leading to the accumulation of tumor suppressor proteins in the cell nucleus. This reinitiates and amplifies their tumor suppressor function and is believed to lead to the selective induction of apoptosis in cancer cells, while largely sparing normal cells. In 2018, Karyopharm reported positive data from the Phase 2b STORM study evaluating selinexor in combination with low-dose dexamethasone in patients with triple class refractory myeloma who have been previously exposed to all five of the most commonly prescribed anti-myeloma therapies currently available. Selinexor has been granted Orphan Drug Designation in multiple myeloma and Fast Track designation for the patient population evaluated in the STORM study. Karyopharm’s New Drug Application (NDA) has been accepted for filing and granted Priority Review by the FDA, and oral selinexor is currently under review by the FDA as a possible new treatment for patients with triple class refractory multiple myeloma who have received at least three prior therapies. The Company has also submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) with a request for conditional approval and was granted accelerated assessment. Selinexor is also being studied in patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL). In 2018, Karyopharm reported positive top-line results from the Phase 2b SADAL study evaluating selinexor in patients with relapsed or refractory DLBCL after at least two prior multi-agent therapies and who are ineligible for transplantation, including high dose chemotherapy with stem cell rescue. Selinexor has received Fast Track designation from the FDA for the patient population evaluated in the SADAL study. Selinexor is also being evaluated in several other mid-and later-phase clinical trials across multiple cancer indications, including in multiple myeloma in a pivotal, randomized Phase 3 study in combination with Velcade (bortezomib) and low-dose dexamethasone (BOSTON), as a potential backbone therapy in combination with approved therapies (STOMP), in liposarcoma (SEAL), and an investigator-sponsored study in endometrial cancer (SIENDO), among others. Additional Phase 1, Phase 2 and Phase 3 studies are ongoing or currently planned, including multiple studies in combination with approved therapies in a variety of tumor types to further inform Karyopharm’s clinical development priorities for selinexor. Additional clinical trial information for selinexor is available at www.clinicaltrials.gov.

On February 26, 2019 Kura Oncology, Inc. (Nasdaq: KURA), a clinical-stage biopharmaceutical company focused on the development of precision medicines for oncology, reported that it will report fourth quarter and full year 2018 financial results after the close of U.S. financial markets on Tuesday, March 5, 2019 (Press release, Kura Oncology, FEB 26, 2019, View Source [SID1234533680]). Kura’s management will host a webcast and conference call at 4:30 p.m. ET / 1:30 p.m. PT that day to discuss the financial results and provide a corporate update.

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The live call may be accessed by dialing 877-516-3514 for domestic callers and 281-973-6129 for international callers and entering the conference code: 8996475. A live webcast and archive of the call will be available online from the investor relations section of the company website at www.kuraoncology.com.

On February 26, 2019 Ophthotech Corporation (Nasdaq: OPHT) reported financial and operating results for the fourth quarter and full year ended December 31, 2018 and provided a business update (Press release, Ophthotech, FEB 26, 2019, View Source [SID1234533679]).

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"2018 was a significant year for transforming the Company by executing multiple business development transactions that resulted in the addition of four gene therapy research and development programs targeting orphan inherited retinal diseases and an age-related therapeutic development program to our retina portfolio," stated Glenn P. Sblendorio, Chief Executive Officer and President of Ophthotech. "We welcomed Versant Ventures as a major shareholder of Ophthotech through the acquisition of Inception 4 and expanded our Board of Directors by adding three industry leaders. We look forward to topline data from our Phase 2b clinical trial of Zimura for the treatment of geographic atrophy secondary to dry age-related macular degeneration in the fourth quarter of 2019. We are committed to advancing and potentially expanding our pipeline of therapies for retinal diseases and creating value for our shareholders."

Gene Therapy Program Highlights of 2018
In 2018, the Company initiated multiple innovative gene therapy collaborations to discover and develop next-generation therapies for the treatment of inherited retinal diseases.

In June 2018, the Company entered into an exclusive global license agreement with the University of Florida Research Foundation (UFRF), Incorporated and the Trustees of the University of Pennsylvania (Penn) for rights to develop and commercialize a novel adeno-associated virus (AAV) gene therapy product candidate for the treatment of rhodopsin-mediated autosomal dominant retinitis pigmentosa (RHO-adRP), an orphan monogenic retinal disease. The novel AAV gene therapy construct is designed to knock down toxic rhodopsin and deliver a transgene for healthy rhodopsin via a single AAV vector in a mutation independent manner. The Company expects to initiate a Phase 1/2 clinical trial in RHO-adRP in 2020.

In August, proof-of-concept study results of this product candidate in a naturally occurring canine disease model were published in the prestigious journal Proceedings of the National Academy of Sciences of the USA. This publication entitled: "Mutation-independent Rhodopsin Gene Therapy by Knockdown and Replacement with a Single AAV vector" was published by scientists at Penn and University of Florida.

In October 2018, the Company entered into its second series of gene therapy agreements with Penn and UFRF, including an exclusive option agreement for rights to negotiate to acquire an exclusive global license to develop and commercialize novel AAV gene therapy product candidates

for the treatment of diseases impacted by the bestrophin, or BEST1, gene. The Company expects to initiate a Phase 1/2 clinical trial in Best disease in 2021.

In February 2018, the Company entered into a series of sponsored research agreements with the University of Massachusetts Medical School (UMMS) and its Horae Gene Therapy Center. The research seeks to utilize a minigene strategy to create novel AAV gene therapy product candidates for Leber Congenital Amaurosis type 10 due to CEP290 mutations (the most common type of LCA), and autosomal recessive Stargardt disease due to ABCA4 mutations, both of which are orphan inherited retinal diseases, and to evaluate different AAV gene delivery methods for application in the eye.

Therapeutic Program Highlights of 2018

Complement Factor C5 Inhibitor Program: Zimura

In October 2018, the Company completed patient enrollment for its ongoing randomized, double-masked, sham controlled, multi-center Phase 2b clinical trial of Zimura for the treatment of geographic atrophy secondary to dry AMD. The Company expects initial top-line data for this trial to be available in the fourth quarter of 2019.

Patient enrollment in the Phase 2b randomized, double-masked, sham-controlled, multi-center clinical trial assessing the efficacy and safety of Zimura in patients with autosomal recessive Stargardt disease (STGD1) is completed and initial top-line data is expected to be available in in the second half of 2020.

Corporate Highlights of 2018

In October 2018, Ophthotech acquired Inception 4, Inc., a privately held company backed by Versant Ventures. Ophthotech gained worldwide development and commercialization rights to Inception 4’s small molecule inhibitors of HtrA1 (high temperature requirement A serine peptidase 1 protein). In addition, Versant Ventures agreed to help Ophthotech identify opportunities to expand the pipeline. Ophthotech obtained approximately $6.1 million in cash through its acquisition of Inception 4 and issued approximately 5.2 million shares to the shareholders of Inception 4. After giving effect to the transaction, funds affiliated with Versant Ventures own approximately 12.5% of the outstanding shares of Ophthotech’s common stock.

During 2018, the Company expanded its Board of Directors by adding leading industry experts: Adrienne L. Graves, Ph.D., former Chief Executive Officer of Santen, Inc., and Jane Pritchett Henderson, Chief Financial Officer of Turnstone Biologics and former Chief Financial Officer and Senior Vice President of Corporate Development at Voyager Therapeutics. Effective January 1, 2019, Calvin W. Roberts, M.D., Senior Vice President and Chief Medical Officer, Eye Care at Bausch Health Companies and Clinical Professor of Ophthalmology at Weill Medical College of Cornell University was elected to Ophthotech’s Board of Directors.

2019 Operational Update

As of December 31, 2018, the Company had $131.2 million in cash and cash equivalents.
The Company estimates its year end 2019 cash and cash equivalents will range between $80 million and $85 million based on its current 2019 business plan, including expansion of the Company’s gene therapy research and development programs, expansion of its HtrA1 development program, and the continuation of its clinical development programs for

Zimura (avacincaptad pegol sodium). This estimate does not reflect any additional expenditures resulting from the potential in-licensing or acquisition of additional product candidates or technologies or associated development that the Company may pursue.

2018 Financial Highlights

Gain on Extinguishment of Royalty Purchase Liability: The Company recognized a gain on extinguishment of its royalty purchase liability of $125 million for the quarter and year ended December 31, 2018, due to the December 2018 termination of the Company’s royalty purchase and sale agreement with Novo Holdings A/S. The termination of the agreement relieved the Company of any obligation to pay Novo Holdings A/S future product royalties on sales of certain platelet derived growth factor (PDGF) antagonists and did not impact the Company’s cash balance.

Revenues: The Company did not have any collaboration revenue for the quarters ended December 31, 2018 and 2017 or during the year ended December 31, 2018, compared to $210 million for the year ended December 31, 2017. Collaboration revenue decreased for 2018 as compared to 2017 as the Company had recognized all deferred revenue associated with the completion of the Company’s licensing and commercialization agreement with Novartis Pharma AG during the third quarter of 2017 and had no collaboration revenue in 2018.

R&D Expenses: Research and development expenses were $16.1 million for the quarter ended December 31, 2018, compared to $7.9 million for the same period in 2017. Research and development expenses increased primarily due to $6.9 million in charges associated with the Company’s October 2018 acquisition of Inception 4 and its HtrA1 inhibitor program and increases in costs associated with the Company’s ongoing Zimura and gene therapy development programs. For the year ended December 31, 2018, research and development expenses were $41.7 million compared to $66.3 million for 2017. Research and development expenses decreased primarily due to decreases in expenses related to the discontinuation of the Company’s Fovista Phase 3 clinical program and decreases in costs associated with the Company’s 2017 reduction in personnel program partially offset by charges associated with the Company’s October 2018 acquisition of Inception 4 and its HtrA1 inhibitor program and increases in costs associated with the Company’s ongoing Zimura and gene therapy development programs.

G&A Expenses: General and administrative expenses were $5.7 million for the quarter ended December 31, 2018, compared to $6.9 million for the same period in 2017. For the year ended December 31, 2018, general and administrative expenses were $23.6 million compared to $35.7 million for 2017. General and administrative expenses decreased primarily due to decreases in costs to support the Company’s operations and infrastructure and decreases in costs associated with its 2017 reduction in personnel program, which included facilities lease termination expenses incurred during the first quarter of 2017.

Net Income: The Company reported net income for the quarter ended December 31, 2018 of $104.1 million, or $2.62 per diluted share, compared to net loss of $9.5 million, or ($0.26) per diluted share, for the same period in 2017. For the year ended December 31, 2018, the Company reported net income of $63.1 million, or $1.70 per

diluted share, compared to net income of $114.2 million, or $3.17 per diluted share, for the same period in 2017.

Conference Call/Web Cast Information
Ophthotech will host a conference call/webcast to discuss the Company’s financial and operating results and provide a business update. The call is scheduled for February 26, 2019 at 8:00 a.m. Eastern Time. To participate in this conference call, dial 888-224-1005 (USA) or 323-994-2093 (International), passcode 6840989. A live, listen-only audio webcast of the conference call can be accessed on the Investor Relations section of the Ophthotech website at: www.ophthotech.com. A replay will be available approximately two hours following the live call for two weeks. The replay number is 888-203-1112 (USA Toll Free), passcode 6840989.