On October 25, 2018 BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) (BioMarin or the Company) reported financial results for the third quarter ended September 30, 2018 (Press release, BioMarin, OCT 25, 2018, View Source [SID1234530636]). The financial results that follow represent a comparison of the third quarter of 2018 to the third quarter of 2017. Total revenues were $391.7 million for the quarter ended September 30, 2018 compared to $334.1 million for the quarter ended September 30, 2017, an increase of 17%. GAAP Net Loss for the quarter ended September 30, 2018 was $12.6 million, or $0.07 loss per basic and diluted share compared to GAAP Net Loss of $12.5 million, or $0.07 loss per basic and diluted share for the quarter ended September 30, 2017.

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Net product revenues for the third quarter of 2018 were $386.3 million, compared to $298.8 million in the third quarter of 2017. The increase in net product revenues was attributed to the following:

Vimizim: increased by $33.0 million, or 37%, due primarily to government ordering patterns in certain countries and an increase in the number of commercial patients;

Brineura: contributed $6.8 million to increased net product revenues, primarily attributed to new patients initiating therapy as the product launched in mid-2017;

Kuvan: increased $7.5 million, or 7%, primarily driven by an increase in the number of commercial patients in North America;

Naglazyme: increased $31.0 million, or 43%, primarily due to government ordering patterns in certain countries and new patients initiating therapy in Europe;

Palynziq: received approval from the Food and Drug Administration (FDA) in May 2018, with commercial sales launching in the third quarter of 2018. Palynziq sales in the third quarter of 2018 were primarily attributed to conversion of clinical patients in the U.S.; and

Aldurazyme: increased $5.2 million, or 23%, primarily as a result of increased volume.

The increase in GAAP Net Loss was primarily due to the following:

Higher research and development (R&D) expense for the expansion of our clinical programs related to vosoritide and valoctocogene roxaparvovec and higher selling, general and administrative (SG&A) expense in support of the U.S. commercial launch of Palynziq and European pre-launch activities, the continued commercial expansion of Kuvan and patient advocacy activities related to our valoctocogene roxaparvovec product candidate and continued commercial expansion of Brineura, partially offset by:

Increased gross profit primarily driven by increased net product revenues across all of our commercial products other than Firdapse.

Non-GAAP Income for the third quarter of 2018 was $60.7 million, compared to Non-GAAP Income of $7.8 million in the third quarter of 2017. The increase in Non-GAAP income in the third quarter of 2018 is primarily attributed to increased gross profit from revenues, offset by higher R&D and SG&A expenses.

As of September 30, 2018, BioMarin had cash, cash equivalents and investments totaling approximately $1.6 billion, as compared to $1.8 billion on December 31, 2017. On October 15, 2018, our 0.75% senior subordinated convertible notes matured and were settled with a combination of $375.0 million in cash for the full principal amount, issuance of common stock for the conversion value in excess of the principal, and cash in lieu of any fractional shares.

Commenting on third quarter results, Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin, said, "BioMarin’s achievements this year give us potential for significant growth in 2019. In the second quarter we received FDA approval of Palynziq, an important new therapy that helps address a significant unmet need in adults with phenylketonuria (PKU) who have been unable to control their blood Phe levels with current treatment options. We also filed for approval of Palynziq in Europe and expect to hear the status of that application in the first half of 2019. The market opportunity with Palynziq is significant and we have been very encouraged by the level of enthusiasm from the PKU community since product launch in July. At the end of the third quarter, 124 U.S. commercial patients were being treated with Palynziq and we are on track to have 250 to 300 U.S. commercial patients by year-end."

"In clinical development, we provided two years of clinical data with the 6e13 vg/kg dose of valoctocogene roxaparvovec gene therapy for severe Hemophilia A from the ongoing Phase 1/2 study in at the World Federation of Hemophilia (WFH) 2018 World Congress in Glasgow, Scotland during the second quarter. The updated data demonstrated the elimination of need for prophylaxis and no spontaneous bleeds for two years. In addition, we amended the protocol for the global GENEr8-1 (Phase 3) pivotal study to evaluate superiority compared to the current standard of care. The number of participants has been increased to 130 and we now anticipate completing enrollment during the second quarter of 2019. Another significant development in the year was the publication of draft guidance from the FDA for hemophilia products. We are encouraged by the considerations included in the guidance document, particularly in relation to the potential for an accelerated approval, and believe it may be pertinent to valoctocogene roxaparvovec if our program results are supportive."

Mr. Bienaimé continued, "Our annual Research and Development Day will be held in New York City on November 7, 2018 where we look forward to showcasing our capabilities from pre-clinical work through manufacturing. Specifically, we look forward to providing a 42-month update on vosoritide for the treatment of achondroplasia as well as preclinical data with our next gene therapy product candidate, BMN 307, for the treatment of phenylketonuria. With approximately $1.5 billion in revenues anticipated for the full year 2018, two potentially $1.0 billion-plus late-stage clinical product opportunities, and a growing early-stage pipeline, BioMarin is poised for significant growth."

*Guidance for Palynziq not previously provided

**All Financial Guidance items are calculated based on US GAAP with the exception of Non-GAAP Income. Refer to Non-GAAP Information beginning on page 9 of this press release for a complete discussion of the Company’s Non-GAAP financial information and reconciliations to the comparable GAAP reported information.

Select Agenda Items at Upcoming R&D Day, November 7, 2018 in New York City:

Vosoritide for Achondroplasia:

Drug Development Considerations in Achondroplasia, presented by Ron Rosenfeld, M.D.,

Professor and Chair (emeritus) of Pediatrics, Oregon Health & Science University and Former Senior Vice President and Medical Director, Lucile Packard Foundation for Children’s Health

42-Month Vosoritide Data Update, presented by Jonathan Day, M.B.B.S., Ph.D.,

Executive Medical Director, Clinical Science, BioMarin

PKU Gene Therapy Data and Program update; Research & Development Pipeline Deep Dive:

Genetics, Genomics and the Future of Us presented by Lon Cardon, Ph.D.,

Senior Vice President and Chief Scientific Officer, BioMarin

Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A:

Regulatory Path Considerations, presented by Geoff Nichol, M.B., Ch.B., M.B.A.,

Senior Vice President, Chief Medical Officer and Head of Global Clinical Development, BioMarin

Considerations for Manufacture of AAV-Based Gene Therapy Products, presented by Robert Baffi, Ph.D., M.B.A., Executive Vice President, Technical Operations, BioMarin

Regulatory Policy Advances at the Food and Drug Administration:

The Evolving Regulatory Landscape and Significance of Patient Engagement, presented by Adora Ndu, Pharm.D., J.D., Executive Director and Head of Global Regulatory Policy, Research, and Patient Engagement, BioMarin

Key Program Highlights

Palynziq for phenylketonuria: With the approval in May 2018 of Palynziq in the United States, an injection to reduce blood Phe concentrations in adult patients with phenylketonuria, BioMarin added the seventh commercial product to its portfolio. Palynziq, a PEGylated recombinant phenylalanine ammonia lyase enzyme, is the first approved enzyme substitution therapy to target the underlying cause of PKU by helping the body to break down Phe. The Company expects to learn the status of its Marketing Authorization Application for Palynziq in Europe in the first half of 2019.

Upon approval of Palynziq in May 2018, the Company said it would report two key metrics related to the U.S. commercial launch beginning in the third quarter of 2018. The first metric provides the number of clinics that have prescribed Palynziq. At the end of the third quarter, 50 unique clinics had prescribed Palynziq. The second metric, the number of naïve and clinical trial patients who are receiving reimbursed, commercial treatment with Palynziq totaled 124 at the end of the third quarter. Of the 124 patients, 81 were from the clinical trial study and 43 were formerly naïve to treatment. In addition, the Company anticipates between 250 to 300 adult PKU patients will be on treatment with Palynziq by year-end 2018, including 153 subjects from the clinical studies.

Valoctocogene roxaparvovec gene therapy for hemophilia A:

In May 2018, the Company updated the protocol for the Phase 3 GENEr8-1 study evaluating the 6e13 vg/kg dose and has powered the study to evaluate superiority to the current standard of care, Factor VIII prophylaxis. The Phase 3 GENEr8-1 study will now include 130 participants (an increase of 90 from the original 40) and is expected to be fully enrolled in the second quarter of 2019.

Based on recent draft guidelines published by the FDA on the development of gene therapy products for the treatment of hemophilia, BioMarin believes that an accelerated approval path forward may be an option with valoctocogene roxaparvovec. In addition, if the Company pursues an accelerated approval, it will inform investors of that decision in the second half of 2019.

Vosoritide for achondroplasia: On November 7, 2018, the Company intends to provide a 42-month update at R&D Day on vosoritide, an analog of C-type natriuretic peptide (CNP). It is being studied in children with achondroplasia. The program includes four distinct areas of focus to support global approval. Currently enrolling,

the global Phase 3 study is a randomized, placebo-controlled study of vosoritide in approximately 110 children with achondroplasia ages 5-14 for 52 weeks. The study will be followed by a subsequent open-label extension. Children in this study will have completed a minimum six-month baseline study to determine their respective baseline growth velocity prior to entering the Phase 3 study. In addition, the Company expects to have over 5 years of clinical data from the long-term, open-label Phase 2 program to corroborate maintenance of effect.

Tralesinidase alfa (formerly referred to as BMN 250) for MPS IIIB (Sanfilippo Syndrome, Type B): In September 2018, the Company provided an update at the Society for the Study of Inborn Errors of Metabolism (SSIEM) meeting from the Phase 1/2 trial with tralesinidase alfa. Of the seven subjects who have been treated with the 300 mg/kg weekly dose, heparan sulfate levels were normalized in the brain fluid. All subjects also experienced normalization of the enlargement of their liver and spleen. Development Quotient (DQ), a measure of cognitive function normalized to age, was also monitored. Five of the seven subjects have experienced encouraging trends in brain function based on DQ measures.

Other Ongoing Clinical Development Programs:

BMN 290 for Friedreich’s Ataxia: In 2017, BioMarin announced that it had selected as its next clinical drug development candidate, BMN 290, a selective chromatin modulation therapy intended for treatment of Friedreich’s ataxia. Currently, there are no approved disease modifying therapies for Friedreich’s ataxia. The Company is currently conducting additional pre-clinical work on BMN 290 and could potentially file an investigational new drug (IND) application in the first half of 2019 based on the outcome of those data.

Gene therapy product candidate for phenylketonuria (PKU): As previously announced, the Company expects to submit an investigational new drug (IND) application for a gene therapy product for the treatment of PKU in the second half of 2019. In preclinical models, BioMarin’s PKU gene therapy product candidate demonstrated sustained, normalized Phe levels in an ongoing study and out to 60 weeks at the last observation. The product candidate is an AAV vector containing the DNA sequence that codes for the phenylalanine hydroxylase enzyme that is deficient in people with PKU.

BioMarin will host a conference call and webcast to discuss third quarter 2018 financial results today, Thursday, October 25, 2018 at 4:30 p.m. ET. This event can be accessed on the investor section of the BioMarin website at www.biomarin.com.

On October 25, 2018 Gilead Sciences, Inc. (Nasdaq: GILD) reported its results of operations for the third quarter ended September 30, 2018 (Press release, Gilead Sciences, OCT 25, 2018, View Source [SID1234530106]).

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The financial results that follow represent a year-over-year comparison of the third quarter 2018 to the third quarter 2017. Total revenues were $5.6 billion in 2018 compared to $6.5 billion in 2017. Net income was $2.1 billion or $1.60 per diluted share in 2018 compared to $2.7 billion or $2.06 per diluted share in 2017. Non-GAAP net income was $2.4 billion or $1.84 per diluted share in 2018 compared to $3.0 billion or $2.27 per diluted share in 2017.

Three Months Ended Nine Months Ended
September 30, September 30,
(In millions, except per share amounts) 2018 2017 2018 2017
Product sales $ 5,455 $ 6,402 $ 15,996 $ 19,825
Royalty, contract and other revenues 141 110 336 333
Total revenues $ 5,596 $ 6,512 $ 16,332 $ 20,158

Net income attributable to Gilead $ 2,097 $ 2,718 $ 5,452 $ 8,493
Non-GAAP net income $ 2,403 $ 2,990 $ 6,855 $ 9,311

Diluted earnings per share $ 1.60 $ 2.06 $ 4.15 $ 6.44
Non-GAAP diluted earnings per share $ 1.84 $ 2.27 $ 5.22 $ 7.06

Product Sales

Total product sales for the third quarter of 2018 were $5.5 billion compared to $6.4 billion for the same period in 2017. Product sales for the third quarter of 2018 were $4.1 billion in the United States, $873 million in Europe and $451 million in other locations. Product sales for the third quarter of 2017 were $4.5 billion in the United States, $1.2 billion in Europe and $663 million in other locations.

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Note: Non-GAAP financial information excludes acquisition-related, up-front collaboration, stock-based compensation and other expenses, fair value adjustments of marketable equity securities and measurement period adjustments relating to the enactment of the 2017 Tax Cuts and Jobs Act (Tax Reform). A reconciliation between GAAP and non-GAAP financial information is provided in the tables on page 7, 8 and 9.

HIV product sales(1) were $3.7 billion for the third quarter of 2018 compared to $3.3 billion for the same period in 2017. The increase was primarily due to the continued uptake of products containing emtricitabine (FTC) and tenofovir alafenamide (TAF), which include Biktarvy (bictegravir 50 mg/emtricitabine 200 mg/tenofovir alafenamide 25 mg), Descovy (emtricitabine 200 mg/tenofovir alafenamide 25 mg), Genvoya (elvitegravir 150 mg/cobicistat 150 mg/emtricitabine 200 mg/tenofovir alafenamide 10 mg) and Odefsey (emtricitabine 200 mg/rilpivirine 25 mg/tenofovir alafenamide 25 mg).
Chronic hepatitis C (HCV) product sales, which consist of Epclusa (sofosbuvir 400 mg/velpatasvir 100 mg), Harvoni (ledipasvir 90 mg/sofosbuvir 400 mg), Vosevi (sofosbuvir 400 mg/velpatasvir 100 mg/voxilaprevir 100 mg) and Sovaldi (sofosbuvir 400 mg), were $902 million for the third quarter of 2018 compared to $2.2 billion for the same period in 2017. The decline was primarily due to lower sales of Harvoni and Epclusa across all major markets as a result of increased competition.
Yescarta (axicabtagene ciloleucel), which was launched in the United States in October 2017, generated $75 million in sales during the third quarter of 2018.
Other product sales, which include products from Gilead’s chronic hepatitis B (HBV), cardiovascular, oncology and other categories inclusive of Vemlidy (tenofovir alafenamide 25 mg), Viread (tenofovir disoproxil fumarate 300 mg), Letairis (ambrisentan 5 mg and 10 mg), Ranexa (ranolazine 500 mg and 1000 mg), Zydelig (idelalisib 150 mg) and AmBisome (amphotericin B liposome for injection 50 mg/vial), were $751 million for the third quarter of 2018 compared to $874 million for the same period in 2017.
Operating Expenses

Three Months Ended Nine Months Ended
September 30, September 30,
(In millions) 2018 2017 2018 2017
Research and development expenses (R&D) $ 939 $ 789 $ 3,068 $ 2,584
Non-GAAP R&D expenses $ 844 $ 745 $ 2,579 $ 2,446

Selling, general and administrative expenses (SG&A) $ 948 $ 879 $ 2,925 $ 2,626
Non-GAAP SG&A expenses $ 852 $ 806 $ 2,576 $ 2,440

During the third quarter of 2018, compared to the same period in 2017:

R&D and SG&A expenses increased primarily due to higher costs to support the growth of Gilead’s business following the acquisition of Kite Pharma, Inc. (Kite) and stock-based compensation expenses associated with Gilead’s acquisition of Kite.
Non-GAAP R&D and non-GAAP SG&A expenses increased primarily due to higher costs to support the growth of Gilead’s business following the acquisition of Kite.
Cash, Cash Equivalents and Marketable Securities

As of September 30, 2018, Gilead had $30.8 billion of cash, cash equivalents and marketable securities compared to $31.7 billion as of June 30, 2018. During the third quarter of 2018, Gilead generated $2.2 billion in operating cash flow. Gilead repaid $1.8 billion principal amount of senior unsecured notes due in September 2018, paid cash dividends of $742 million and utilized $449 million on stock repurchases.

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(1) Excludes sales of Viread as Viread is primarily used for treatment of chronic HBV.

Revised Full Year 2018 Guidance

Gilead revised its full year 2018 guidance, initially provided on February 6, 2018 and revised on July 25, 2018:

(In millions, except percentages and per share amounts) Initially Provided
February 6, 2018
Reiterated
May 1, 2018 Updated
July 25, 2018

Updated

October 25, 2018

Net Product Sales $20,000 – $21,000 $20,000 – $21,000 $20,800 – $21,300
Non-GAAP
Product Gross Margin 85% – 87% 85% – 87% 85% – 87%
R&D Expenses $3,400 – $3,600 $3,400 – $3,600 $3,400 – $3,600
SG&A Expenses $3,400 – $3,600 $3,400 – $3,600 $3,400 – $3,600
Effective Tax Rate 21.0% – 23.0% 19.0% – 21.0% 18.0% – 20.0%
Diluted EPS Impact of Acquisition-related, Up-front Collaboration, Stock-based Compensation and Other Expenses $1.41 – $1.51 $1.50 – $1.60 $1.50 – $1.60

Corporate Highlights

Announced that John F. Milligan, Ph.D., will step down as President and Chief Executive Officer (CEO).
John C. Martin, Ph.D., announced his intent to step down from the Board at the time a new CEO joins the company.
Announced plans to launch authorized generic versions of Epclusa and Harvoni in the United States through a newly created subsidiary, Asegua Therapeutics LLC.
Announced that Laura Hamill has joined the company as Executive Vice President, Worldwide Commercial Operations.
Announced that Gregg Alton has been appointed Chief Patient Officer and that Diana Brainard, M.D., has been promoted to Senior Vice President, HIV and Emerging Viral Infections. Also announced that Andrew Cheng, M.D., Ph.D., Chief Medical Officer, decided to leave Gilead to pursue another opportunity.
Announced that Michael Amoroso has joined the company as Senior Vice President and Head of Worldwide Commercial, Cell Therapy.
Product and Pipeline Updates announced by Gilead during the Third Quarter of 2018 include:

HIV and Liver Diseases Programs

Announced a strategic collaboration with Precision BioSciences (Precision) to develop therapies targeting the in vivo elimination of HBV virus with Precision’s proprietary genome editing platform, ARCUS.
Announced that the China National Drug Administration has approved Genvoya for the treatment of HIV-1 infection.
Presented data at the 22nd International AIDS Conference, which included the announcement of a retrospective nationwide analysis of the impact of Truvada (emtricitabine 200mg and tenofovir disoproxil fumarate 300mg) for pre-exposure prophylaxis (PrEP) use across all 50 U.S. states and the District of Columbia. Conducted in collaboration with researchers at Emory University Rollins School of Public Health and the Centers for Disease Control and Prevention, these data demonstrated that use of once-daily oral Truvada for PrEP has had an independent and significant impact on the number of new HIV infections diagnosed in the United States from 2012 to 2016.
Oncology and Cell Therapy Programs

Announced a license agreement with Trianni, Inc. (Trianni) that grants Gilead the use of the Trianni transgenic human monoclonal antibody discovery platform to support drug discovery efforts.
Announced that the European Commission has granted Marketing Authorization for Yescarta as a treatment for adult patients with relapsed or refractory diffuse large B-cell lymphoma and primary mediastinal large B-cell lymphoma, after two or more lines of systemic therapy.
Announced a strategic collaboration with Gadeta B.V. (Gadeta) to develop novel gamma delta T cell receptor therapies in various cancers.
Inflammation Programs

Announced that FINCH 2, a global, randomized, placebo-controlled, Phase 3 study of filgotinib, an investigational, selective JAK1 inhibitor, in adults with moderately-to-severely active rheumatoid arthritis and prior inadequate response/intolerance to biologic agents, achieved its primary endpoint in the proportion of patients achieving an American College of Rheumatology 20 percent response at week 12.
Announced that the randomized, placebo-controlled Phase 2 TORTUGA study of filgotinib achieved its primary efficacy endpoint in adults with moderately to severely active ankylosing spondylitis (AS). In the study, patients treated with filgotinib achieved significantly greater improvements in AS Disease Activity Score, the primary endpoint, at week 12, with a mean change from baseline of -1.5 versus -0.6 for those treated with placebo (p<0.0001).

On October 25, 2018 Aurinia Pharmaceuticals Inc., (NASDAQ:AUPH) (TSX:AUP) reported that it will release its third quarter 2018 financial results on Thursday, November 8, 2018, after the market closes (Press release, Aurinia Pharmaceuticals, OCT 25, 2018, View Source [SID1234530151]). Aurinia’s management will host a conference call to discuss the company’s third quarter 2018 financial results and provide a general business update.

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The conference call and webcast is scheduled for November 8, 2018 at 4:30pm EDT. In order to participate in the conference call, please dial +1-877-407-9170 (Toll-free U.S. & Canada). An audio webcast can be accessed under "News/Events" through the "Investors" section of the Aurinia corporate website at www.auriniapharma.com. A replay of the webcast will be available on Aurinia’s website.

On October 25, 2018 RedHill Biopharma Ltd. (Nasdaq: RDHL) (Tel-Aviv Stock Exchange: RDHL) ("RedHill" or the "Company"), a specialty biopharmaceutical company primarily focused on proprietary drugs for gastrointestinal diseases, reported that Mr. Adi Frish, Senior VP Business Development and Licensing, will present a corporate overview at the BIO-Europe 2018 Conference, on Tuesday, November 6, 2018, at 4:15 p.m. CET, at the Bella Center in Copenhagen, Denmark (Press release, RedHill Biopharma, OCT 25, 2018, View Source [SID1234530282]).

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A copy of the presentation to be delivered by Mr. Frish will be available on the Company’s website and may be viewed at: View Source

On October 25, 2018 Can-Fite BioPharma Ltd. (NYSE American: CANF) (TASE:CFBI), a biotechnology company advancing a pipeline of proprietary small molecule drugs that address liver and inflammatory diseases, reported that its CEO, Dr. Pnina Fishman, will present at The NYC Oncology Investor Conference 2018 View Source to take place on October 30-31 (Press release, Can-Fite BioPharma, OCT 25, 2018, View Source [SID1234530150]). Her lecture will be delivered on the 31st at 11:40 am.

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Dr. Fishman will present the molecular mechanism mediating the anti-cancer effects of Namodenoson and will describe clinical data from the Company’s earlier Phase I/II liver cancer study. In addition, she will present the status of the current Phase II study in patients with advanced hepatocellular carcinoma, Child Pugh B.

The global Phase II study is being conducted in the U.S., Europe and Israel. Patients with advanced HCC, Child Pugh B, who failed Nexavar (sorafenib) as a first line treatment are treated twice daily with 25 mg of oral Namodenoson or placebo using a 2:1 randomization. The primary endpoint of the Phase II study is Overall Survival (OS). Secondary endpoints include Progression Free Survival (PFS), safety, and the relationship between outcomes and A3AR expression.

Advanced liver cancer is categorized into 3 subclasses including Child Pugh A, mostly treated with Nexavar, Child Pugh B and Child Pugh C. Although a few drugs for the treatment of advanced liver cancer have recently launched, none are specifically aimed at treating patients who have reached the Child Pugh B stage. This represents a major unmet need and potentially positions Namodenoson as an important drug candidate to treat this patient population.

Accumulated safety data to date continues to indicate a favorable safety profile, with no clinically significant novel or emerging events attributed to chronic treatment with Namodenoson.

"We are pleased to present at the NYC Oncology Conference and share with investors our unique approach to treat patients with advanced HCC. This tumor is resistant to chemotherapy whereas Namodenoson has shown in an earlier pre-clinical study to induce specific apoptosis towards the liver cancer cells while sparing the normal liver cells," commented Dr. Pnina Fishman, company CEO.

About Namodenoson

Namodenoson is a small orally bioavailable drug that binds with high affinity and selectivity to the A3 adenosine receptor (A3AR). Namodenoson is being evaluated in Phase II trials for two indications, as a second line treatment for hepatocellular carcinoma, and as a treatment for non-alcoholic fatty liver disease (NAFLD) and non-alcoholic steatohepatitis (NASH). A3AR is highly expressed in diseased cells whereas low expression is found in normal cells. This differential effect accounts for the excellent safety profile of the drug.