On August 30, 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 its financial results for the quarter ended June 30, 2018 and provided an update on its operations (Press release, RedHill Biopharma, AUG 30, 2018, View Source [SID1234529318]).

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"There is a tremendous amount of enthusiasm among the RedHill team which remains focused on the successful execution of our development plans and commercial operations. With enrollment nearly completed, we are on track to generate top-line results from the confirmatory Phase III study with TALICIA for H. pylori infection in the fourth quarter of this year," said Dror Ben-Asher, Redhill’s CEO. "We are also excited by the robust top-line results from the ground-breaking MAP US Phase III study with RHB-104 for Crohn’s disease which met both its primary and key secondary endpoints. We will provide an update on next steps after we speak with the FDA about the results and the development path to potential approval. Our financial standing is solid, as we remain debt-free with approximately $43 million total cash on hand as of today, with steadily declining cash burn."

Financial highlights for the quarter ended June 30, 20181:

Net Revenue for the second quarter of 2018 was $2.4 million, compared to $0.5 million in the second quarter of 2017. The increase was due to the advancement of promotional activities for Donnatal, and EnteraGam and the initial promotion of Esomeprazole Strontium Delayed-Release Capsules 49.3 mg in late 2017.

Gross Profit for the second quarter of 2018 was $1.6 million, compared to $0.2 million in the second quarter of 2017. Gross margin increased to 69% for the second quarter of 2018 from 44% in the second quarter of 2017.

Research and Development Expenses for the second quarter of 2018 were $6.0 million, a decrease of 28% from $8.4 million for the second quarter of 2017. The decrease was mainly due to the initiation of the Company’s cost reduction plan, the completion of patient treatment for primary endpoint assessment in the Phase III study with RHB-104 and completion of the Phase III and Phase II studies with BEKINDA (RHB-102) for gastroenteritis and IBS-D, respectively.

Selling, Marketing and Business Development Expenses for the second quarter of 2018 were $3.1 million, a decrease of 7% from $3.4 million for the second quarter of 2017. The decrease was due to the continued implementation of the Company’s cost reduction plan and optimization measures.

General and Administrative Expenses for the second quarter of 2018 were relatively flat year-over-year at $2.0 million.

Operating Loss for the second quarter of 2018 was $9.6 million, a decrease of 29% from $13.5 million for the second quarter of 2017. The decrease was due to the increase in net revenue and gross profit, and a decrease in operating expenses by 19%.

Net Cash Used in Operating Activities for the second quarter of 2018 was $8.4 million, a decrease of 14% from $9.7 million for the second quarter of 2017. The decrease was mainly due to the advancement of the Company’s clinical development programs, including completion of patient treatment for primary endpoint assessment in the positive Phase III study of RHB-104 in Crohn’s disease, completion of the Phase III and Phase II studies with BEKINDA in gastroenteritis and IBS-D, respectively, and overall optimization of the Company’s operations.

Cash Balance2 as of June 30, 2018 was $27.9 million. Subsequent to the end of the quarter, on August 14, 2018, RedHill announced the closing of an underwritten offering for gross proceeds of approximately $25 million, before commissions and other offering expenses. RedHill’s cash balance as of August 30, 2018 is approximately $43 million.

NASDAQ Uplisting
On July 20, 2018, RedHill’s American Depositary Shares (ADSs) started trading on the Nasdaq Global Market after being uplisted from the Nasdaq Capital Market. The Company’s ADSs continue to trade under the symbol "RDHL".

Select R&D Highlights:

RHB-104 – Crohn’s disease (positive first Phase III study)

On July 30, 2018, RedHill announced positive top-line safety and efficacy results from the first Phase III study with orally-administered RHB-104 for Crohn’s disease (MAP US study). The study successfully met both its primary endpoint and key secondary endpoints. The randomized, double-blind, placebo-controlled first Phase III study enrolled 331 subjects with moderately to severely active Crohn’s disease (defined as Crohn’s Disease Active Index (CDAI) between 220 and 450) in the U.S., Canada, Europe, Australia, New Zealand and Israel. Subjects were randomized 1:1 to receive RHB-104 or placebo, on-top of baseline background medication, including 5-ASAs, corticosteroids, immunomodulators or anti-TNFα agents.

The top-line results from the MAP US study demonstrated the superiority of RHB-104 over placebo in achieving remission at week 26, defined as CDAI value of less than 150, the primary endpoint of the study. The proportion of patients meeting the primary endpoint was significantly greater in the RHB-104 group compared to placebo (37% vs. 23%, p= 0.013). The study also successfully met key secondary endpoints, demonstrating a consistent benefit to Crohn’s disease patients treated with RHB-104. RHB-104 was found to be generally safe and well tolerated. RedHill will continue to assess additional data as it becomes available. The Company will meet with key opinion leaders and the U.S. Food and Drug Administration (FDA) to present the data package and discuss the development path to potential FDA approval and will continue discussions with potential partners for RHB-104.

On July 2, 2018, RedHill announced that it had received a Notice of Allowance from the United States Patent and Trademark Office (USPTO) and an Intention to Grant from the European Patent Office (EPO) for two new patents covering RHB-104, expected to be valid until at least February 5, 2029, once granted. On August 13, 2018, the Company announced that it has received a Notice of Allowance from the USPTO for a new formulation patent, expected to be valid until at least 2029, that further expands the Company’s intellectual property portfolio covering RHB-104 for Crohn’s disease and RHB-204 for pulmonary nontuberculous mycobacteria (NTM) infections.

TALICIA (RHB-105) – H. pylori infection (confirmatory Phase III) (FDA Fast Track)

To date, over 444 patients have been enrolled in the ongoing confirmatory Phase III study with TALICIA (RHB-105)3 for H. pylori infection (ERADICATE Hp2 study). RedHill expects to announce top-line results in the fourth quarter of 2018.

Subject to a successful outcome of the study and additional regulatory feedback, the ERADICATE Hp2 study is expected to complete the clinical package required for a potential submission of a U.S. New Drug Application (NDA) for TALICIA in early 2019, which is eligible to benefit from priority review of six months.

RHB-204 – nontuberculous mycobacteria (NTM) infections (planned pivotal Phase III) (FDA Fast-Track QIDP status)

A pivotal Phase III study with RHB-204 for the treatment of pulmonary nontuberculous mycobacteria (NTM) infections is expected to be initiated in the first quarter of 2019, subject to completion of a supportive non-clinical program and additional input from the FDA. The study is intended to assess the efficacy and safety of RHB-204 as a first-line treatment for pulmonary NTM infections caused by Mycobacterium avium complex (MAC).

BEKINDA (RHB-102) 24 mg – Gastroenteritis (Phase III)

Following the positive results of the Phase III study with BEKINDA 24 mg for acute gastroenteritis (GUARD study) and guidance provided by the FDA, RedHill is currently in discussions with the FDA on the design of a confirmatory Phase III study to support a potential NDA.

BEKINDA (RHB-102) 12 mg – IBS-D (Phase II)

Following positive results of the Phase II study with BEKINDA 12 mg for diarrhea-predominant irritable bowel syndrome (IBS-D), RedHill is in discussions with the FDA on the design of pivotal Phase III studies and path to potential NDA approval.

An abstract4 (number: 2908495), describing the results of the Phase II study with BEKINDA 12 mg for the treatment of IBS-D, was presented as a Poster of Distinction at Digestive Disease Week (DDW) in June 2018.

YELIVA (ABC294640) – cholangiocarcinoma (Phase IIa) (FDA Orphan Drug designation)

Enrollment in the single-arm Phase IIa study with YELIVA (ABC294640) for the treatment of cholangiocarcinoma (bile duct cancer) is expected to be completed by the end of 2018. The study is being conducted at the Mayo Clinic’s major campuses in Arizona and Minnesota, University of Texas MD Anderson Cancer Center, the Huntsman Cancer Institute, University of Utah Health, and at Emory University. The study is designed to enroll up to 39 patients.

RHB-106 – encapsulated bowel cleanser licensed to Salix Pharmaceuticals

On August 20, 2018, RedHill announced that it had received a Notice of Allowance from the USPTO for a new formulation patent covering RHB-106, which is expected to be valid until at least 2033.

RedHill recently amended its 2014 worldwide license agreement with Salix Pharmaceuticals ("Salix"), a wholly-owned subsidiary of Bausch Health Companies Inc., relating to RHB-106, as well as additional related rights. The amendment clarified Salix’s future development efforts and provides for enhanced involvement by RedHill in certain intellectual property matters and increased the lower end of the range of royalty payments to be paid to RedHill on net sales from low single digits to high single digits. Milestone payments remain unchanged. RedHill continues to assist Salix in the development of RHB-106, as needed.

U.S. Commercial Highlights:

On June 28, 2018, RedHill announced that it had entered into a co-promotion agreement with Napo Pharmaceuticals, a human health company developing and commercializing novel gastrointestinal prescription products, granting RedHill the exclusive right to co-promote Mytesi (crofelemer 125 mg delayed-release tablets)5 in the U.S. to certain gastroenterologists and primary care physicians for the approved indication in people living with HIV/AIDS. Mytesi is an FDA-approved anti-diarrheal prescription drug indicated for the symptomatic relief of non-infectious diarrhea in adults with HIV/AIDS on anti-retroviral therapy (ART). On July 25, 2018, RedHill announced that it had initiated the promotion of Mytesi. Mytesi is the fourth product being promoted by RedHill’s gastrointestinal-focused U.S. salesforce, in preparation for potential U.S. launch of RedHill’s late clinical-stage products.

Conference Call and Webcast Information:

The Company will host a conference call today, August 30, 2018 at 8:30 a.m. EDT to review the financial results and business highlights.

To participate in the conference call, please dial one of the following numbers 15 minutes prior to the start of the call: United States: +1-800-263-0877; International: +1-646-828-8143; and Israel: +972-3-721-9463. The access code for the call is: 9460445.

The conference call will be broadcasted live and will be available for replay on the Company’s website, View Source, for 30 days. Please access the Company’s website at least 15 minutes ahead of the conference call to register, download and install any necessary audio software.

On August 30, 2018 Leap Therapeutics, Inc. (NASDAQ: LPTX), a biotechnology company developing targeted and immuno-oncology therapeutics, reported that Christopher K. Mirabelli, Ph.D., Chairman, President and Chief Executive Officer, will present a corporate overview at the H.C. Wainwright 20TH Annual Global Investment Conference, being held in New York City on September 4-6, 2018 (Press release, Leap Therapeutics, AUG 30, 2018, View Source;p=RssLanding&cat=news&id=2365574 [SID1234529242]).

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Leap Presentation Details:

H.C. Wainwright 20th Annual Global Investment Conference
Date: Thursday, September 6, 2018
Time: 9:35 A.M.

The presentation will be webcast live and may be accessed on the Investors page of the company’s website at www.investors.leaptx.com, where a replay of the event will also be available for a limited time.

On August 30, 2018 Portola Pharmaceuticals, Inc. (Nasdaq:PTLA) reported that the company will participate in the following conferences in September (Press release, Portola Pharmaceuticals, AUG 30, 2018, View Source;p=RssLanding&cat=news&id=2365495 [SID1234529231]):

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Citi’s 13th Annual Biotech Conference on Thursday, September 6, 2018 in Boston, MA. The Company will conduct one-on-one meetings with institutional investors at this conference.

Morgan Stanley 16th Annual Global Healthcare Conference on Wednesday, September 12, 2018, at 2:15 p.m. Eastern Time in New York, NY. The Company will participate in a fireside chat presentation at the conference, which will be webcast live and available for replay from Portola’s website at www.portola.com in the Investor Relations section.

On August 30, 2018 MEI Pharma, Inc. (Nasdaq: MEIP), a late-stage pharmaceutical company focused on advancing new therapies for cancer, reported results for its fiscal year ended June 30, 2018 (Press release, MEI Pharma, AUG 30, 2018, View Source [SID1234529228]).

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"We begin the 2019 fiscal year in our strongest position ever, with progress across all four clinical-stage oncology candidates, and with particular focus on the planned initiation of the ME-401 Phase 2 accelerated approval study by year-end. With ME-401 joining pracinostat as the second candidate in a global study to support marketing authorization, along with our robust cash position, we are well situated to pursue our development strategy," said Daniel P. Gold, Ph.D., president and chief executive officer of MEI Pharma.

Dr. Gold continued, "We are also very pleased to report that in recent discussions with FDA on a ME-401 accelerated approval registration strategy, FDA expressed support of our proposed randomized Phase 2 trial in which we will evaluate continuous and intermittent dosing schedules in patients with relapsed or refractory follicular lymphoma. As we execute on our plans to start this study around year-end, we also look forward to reporting additional progress across our pipeline over the coming quarters, including clinical data from our CDK9 inhibitor, voruciclib, and clinical updates on pracinostat and ME-344."

Fiscal Year 2018 and Recent Company Highlights

Financial

In May 2018, the Company completed a private placement of common stock, along with warrants to purchase common stock, resulting in net proceeds to MEI of approximately $70 million.
As of June 30, 2018, MEI had $102.7 million in cash, cash equivalents and short-term investments, with no outstanding debt. The cash balance includes the proceeds of our private placement completed in May 2018.
Research and development expenses were $17.0 million for the year ended June 30, 2018, compared to $7.2 million for 2017. The increase was primarily related to increased activities in all clinical programs including the acquisition and development costs associated with voruciclib.
General and administrative expenses were $9.8 million for the year ended June 30, 2018, compared to $8.6 million for 2017. The increase primarily relates to professional services expenses, share-based compensation, and general corporate expenses incurred during the year ended June 30, 2018.
The Company recognized revenues of $1.6 million for the year ended June 30, 2018. Revenues resulted from the recognition of fees allocated to research and development activities related to the Helsinn License Agreement. Revenue decreased due to lower levels of research and development activities during the year ended June 30, 2018.
Cash expenditures for operations were $21.3 million for the year ended June 30, 2018, compared to $16.5 million for 2017. Cash expenditures were $3.8 million for the fourth quarter ended June 30, 2018, compared to $3.1 million for the same period in 2017.
Net loss was $40.1 million, or $0.97 per share, for the fiscal year ended June 30, 2018, compared to net income of $2.7 million, or $0.07 per share for 2017. The Company’s net loss includes a $9.7 million change in the fair value of the warrants issued in connection with the May 2018 financing and $2.4 million in transaction costs related to the May 2018 financing recorded as financing expense on the statement of operations. The Company is required to calculate the change in fair value of the warrants and record the non-cash charge to the statement of operations at each reporting date.
ME-401 – a next-generation selective oral inhibitor of PI3K delta

In June 2018, the Company presented results from a Phase 1b study evaluating ME-401 in patients with relapsed/refractory follicular lymphoma, chronic lymphocytic leukemia and small lymphocytic lymphoma at the 2018 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting as well as the European Hematology Association (EHA) (Free EHA Whitepaper) Congress. The data demonstrated a 90% overall objective response rate in patients. Based on these data, the Company is advancing ME-401 into a Phase 2, single-agent study for the treatment of adults with relapsed or refractory follicular lymphoma. The Phase 2 study is intended to support accelerated approval and is planned to begin around the end of 2018.
In July 2018 the Company discussed with FDA a ME-401 monotherapy accelerated approval strategy in patients with relapsed or refractory follicular lymphoma (FL). The FDA communicated support for the Company’s proposed randomized Phase 2 trial evaluating continuous and intermittent dosing schedules. Accelerated approval of ME-401 will be subject to FDA review of the improvement provided by ME-401 over other therapies available at the time of the regulatory action.
MEI expects to report data updates from the Phase 1b study, including at a medical meeting late in Q4 2018. By year-end 2018, MEI also plans to initiate the Phase 2 study to support accelerated approval of ME-401 in relapsed or refractory follicular lymphoma.
Pracinostat – an oral HDAC inhibitor (partnered with Helsinn Healthcare, SA)

In January 2018, the European Medicines Agency granted Orphan Drug Designation to pracinostat, currently in a Phase 3 study in combination with azacitidine for the treatment of acute myeloid leukemia (AML) in adult patients unfit for induction chemotherapy.
In May 2018, the Company and Helsinn announced a successful interim analysis from the Phase 2 study evaluating the combination of pracinostat and azacitidine in high and very high-risk myelodysplastic syndrome (MDS) patients previously untreated with hypomethylating agents. Based on the successful planned interim analysis, the study expanded open-label enrollment to 60 patients. MEI is responsible for the conduct of the Phase 2 study, the cost of which will be shared by Helsinn. Helsinn is responsible for funding any further studies.
MEI expects to provide updates to the pracinostat program at a medical meeting late in Q4 2018.
Voruciclib – an oral, selective CDK inhibitor with robust CDK9 inhibition

In January 2018, the U.S. Food and Drug Administration cleared the Company’s Investigational New Drug Application (IND) for voruciclib.
MEI expects to report updates regarding the ongoing Phase 1 study, and the initiation of dosing of voruciclib in combination with VENCLEXTA (venetoclax) in relapsed or refractory B-cell malignancies, at medical meetings in 2019.
ME-344 – a novel mitochondrial inhibitor

In June 2018, the Company presented interim results from an investigator-initiated Phase 1 study in HER2 negative breast cancer in combination with bevacizumab (marketed as Avastin) at ASCO (Free ASCO Whitepaper). ME-344 in combination with Avastin demonstrated inhibition of tumor proliferation as measured by Ki-67 reductions in HER2 negative breast cancer patients. These results support continuation of the ongoing Phase 1 study.
MEI expects to report additional data from the investigator-sponsored Phase 1 study at medical meetings in 2019.
Operational Highlights

In February 2018, the Company announced the appointment of industry veteran Frederick W. Driscoll to the board of directors. Mr. Driscoll serves on the audit committee.
In July 2018, the Company announced that David M. Urso, J.D., senior vice president of corporate development and general counsel, was promoted to chief operating officer. Mr. Urso is also continuing as the Company’s general counsel and head of corporate development.
Conference Call and Webcast

MEI Pharma will host a conference call with simultaneous webcast today, August 30, 2018, at 5:00 p.m. Eastern time to provide a corporate update. To access the live call, please dial (866) 939-3921 (United States) or (678) 302-3550 (International), conference ID 47469059. The conference call will also be webcast live and can be accessed at www.meipharma.com. A replay of the webcast will be available approximately one hour after the conclusion of the call.

On August 30, 2018 SOTIO, a biotechnology company owned by the PPF Group, reported the completion of the acquisition of Cytune Pharma by PPF (Press release, SOTIO, AUG 30, 2018, View Source [SID1234529225]). SOTIO will continue to develop the lead program SO-C101 (RLI-15) within its pipeline and intends to initiate first-in- human clinical trials in early 2019. SOTIO is spearheading all of PPF’s activities in the biotech sector and closely cooperated during Cytune’s acquisition process. All Cytune projects will be developed as part of SOTIO’s pipeline.

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Cytune Pharma develops IL-15 based therapies for the treatment of cancer. The lead molecule of Cytune’s pipeline, SO-C101 (RLI-15), is a human fusion protein of IL-15 and the high-affinity binding domain of IL-15Rα and acts as a specific IL-2/IL-15Rβγ agonist.

The deal announced in 2015 between PPF Group and Cytune involved investments into Cytune and a staged-acquisition of the company, which has now been completed with the acquisition of all outstanding shares of Cytune. Cytune becomes an important part of SOTIO’s activities in the biotech sector and will also closely collaborate with its affiliate Accord Research on the future development of the lead program SO-C101, and other earlier-stage targeted RLI-15 programs. SOTIO is planning to initiate a first-in-human study with SO-C101 in selected European countries and the US in early 2019. A key part of this early clinical program is the combination with immune checkpoint inhibitors.

"We are excited about the completion of the acquisition and welcome Cytune to the PPF Group. Cytune and SOTIO have been closely and very successfully collaborating over the course of the last few years and we very much look forward to initiating the first clinical trials with SO-C101 in a few months," commented Radek Spisek, CEO of SOTIO. "SO-C101 will become a core program of our pipeline, together with our clinical programs in ovarian, lung and prostate cancer based on our autologous dendritic cell therapy platform DCVAC."

"I’m proud that our products, originating from INSERM and the University of Nantes and supported by Bpifrance and Atlanpole Biotherapies, hold the potential to lead to new therapeutic options for the treatment of cancer patients. Such IL-2/IL-15Rβγ agonists represent a new and very promising class of drugs in the immune-oncology segment as they have shown to re-induce responses in patients after failure to checkpoint inhibitors treatment," adds David Bechard, President and COO of Cytune. "The continued financial support by PPF combined with SOTIO’s clinical development expertise will be instrumental in accelerating the development of SO-C101 in multiple clinical trials, ultimately for the benefit of cancer patients."

The financial terms of the transaction are not disclosed.

About SO-C101
Molecule SO-C101 (RLI-15) is a human fusion protein of IL-15 and the high-affinity binding domain of IL-15Rα which acts as a specific agonist of the intermediate-affinity IL-2/IL-15Rβγ. It is a novel immunotherapeutic approach with potential applications in a variety of oncology indications. In preclinical experiments, SO-C101 has been shown to stimulate and induce proliferation of immune effector cells, such as cytotoxic T cells and NK cells, without expanding the CD4+ T regulatory cells. Based on the preclinical experiments, SO-C101 is more potent and better tolerated compared to the unmodified IL-15 or IL-2. SO-C101 and other products based on this platform allow for combinations with other immunotherapeutic strategies, including checkpoint inhibitors.