On May 27, 2021 CStone Pharmaceuticals ("CStone", HKEX: 2616), a leading biopharmaceutical company focused on researching, developing, and commercializing innovative immuno-oncology therapies and precision medicines, reported that a registrational clinical trial (GEMSTONE-301 study) of the anti-PD-L1 monoclonal antibody sugemalimab in patients with stage III NSCLC met its primary endpoint at a planned interim analysis reviewed by the independent Data Monitoring Committee (iDMC) (Press release, CStone Pharmaceauticals, MAY 27, 2021, View Source [SID1234583242]). The findings showed that sugemalimab as a consolidation therapy brought statistically significant and clinically meaningful improvement in the Blinded Independent Central Review (BICR) assessed PFS in patients with locally advanced/unresectable NSCLC without disease progression after concurrent or sequential chemoradiotherapy. Investigator assessed PFS showed consistent results as those of the primary endpoint. Sugemalimab was well-tolerated with no new safety signals. Subgroup analyses demonstrated that sugemalimab was associated with clinical benefit regardless of whether patients received concurrent or sequential chemoradiotherapy prior to sugemalimab.

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"Lung cancer is the leading cause of cancer-related mortality globally. There are currently few effective therapies for patients with stage III NSCLC whose disease did not progress after sequential chemoradiotherapy," said Professor Yi-long Wu of Guangdong Provincial People’s Hospital, the Leading Principal Investigator on the GEMSTONE-301 study," The successful results from the study indicate that sugemalimab will meet the urgent treatment needs of these patients."

"We are excited that sugemalimab becomes the first anti-PD-1/PD-L1 monoclonal antibody in the world to cover both stage III and stage IV NSCLC patients," said Dr. Frank Jiang, Chairman and CEO of CStone, "The continued success of sugemalimab in lung cancer demonstrates CStone’s leading research and development capabilities in the field of immuno-oncology. We are working closely with Pfizer and EQRx, our commercial partners for sugemalimab, on the next steps in our joint efforts to deliver this best-in-class drug to patients worldwide."

"Currently, there has not been an approved PD-1 or PD-L1 monoclonal antibody for treating patients in stage III NSCLC who have not developed disease progression after sequential chemoradiotherapy," said Dr. Jason Yang, Chief Medical Officer of CStone, "The GEMSTONE-301 is the first-in-class clinical study design that enrolled patients with either concurrent or sequential chemoradiotherapy to better reflect real-world clinical practice and cover a broader population. CStone is committed to providing treatment options to address the unmet medical needs. The GEMSTONE-301 study will advance the use of multidisciplinary treatment approaches in China to improve the quality of the diagnosis and treatment of stage III NSCLC. We will continue to explore the potential of sugemalimab in registrational clinical trials for patients with hematologic malignancies and advanced gastric and esophageal cancers."

CStone plans to submit an NDA to the NMPA for sugemalimab in stage III NSCLC, and will work with EQRx to hold regulatory discussions on the indications of stage III and stage IV NSCLC with regulators in multiple countries, including the U.S. Food and Drug Administration (FDA). Specific study data will be presented at an upcoming academic conference.

CStone formed a strategic collaboration agreement with Pfizer that includes the development and commercialization of sugemalimab in mainland China, and a framework to bring additional oncology assets to the Greater China market. CStone subsequently formed a strategic collaboration agreement with EQRx, under which EQRx licensed the exclusive rights to two key late-stage immuno-oncology assets, sugemalimab and CS1003 (anti-PD-1 antibody), for global development and commercialization outside of Greater China.

About NSCLC

In recent years, China has had rising lung cancer incidence. According to the latest estimates on the global burden of cancer released by International Agency for Research on Cancer (IARC), in 2020, an estimated 0.82 million new lung cancer cases and 0.71 million new lung cancer deaths occurred in China. Among all Chinese cancer patients, lung cancer is the leading cause of cancer-related deaths. NSCLC is the most common type of lung cancer.

There are currently limited treatment options for patients with locally advanced/unresectable (stage III) NSCLC. In China, sequential chemoradiotherapy is widely used , while concurrent chemoradiotherapy is with limited use. But both are with unsatisfactory efficacy.

About Sugemalimab (anti-PD-L1 antibody)

Sugemalimab is an investigational anti-PD-L1 monoclonal antibody discovered by CStone. Authorized by the U.S.-based Ligand Corporation, sugemalimab is developed by the OmniRat transgenic animal platform, which can generate fully human antibodies in one stop. As a fully human, full-length anti-PD-L1 monoclonal antibody, sugemalimab mirrors the natural G-type immunoglobulin 4 (IgG4) human antibody, which reduces the risk of immunogenicity and potential toxicities in patients, a unique advantage over similar drugs.

Currently, sugemalimab is being investigated in a number of ongoing clinical trials, including one Phase II registration studies for lymphoma (CS1001-201) and four Phase III registrational studies on stage III NSCLC, stage IV NSCLC, gastric cancer, and esophageal cancer, respectively.

CS1001-201 is a single-arm, multicenter, Phase II pivotal study designed to evaluate the efficacy and safety of sugemalimab as monotherapy for the treatment of adult patients with relapsed or refractory extranodal natural killer/T-cell lymphoma (R/R ENKTL). Based on the encouraging preliminary efficacy results, sugemalimab was granted Orphan Drug Designation for the treatment of T-cell lymphoma and Breakthrough Therapy Designation for the treatment of R/R ENKTL by the U.S. Food and Drug Administration. It has also been granted Breakthrough Therapy Designation by the National Medical Products Administration of China. The proposed indication is R/R ENKTL.

GEMSTONE-301 Study

GEMSTONE-301 study (clinicaltrials.gov registration number: NCT03728556; drug clinical trial registration number: CTR20181429) is a multicenter, randomized, double-blind Phase III clinical trial, designed to evaluate the efficacy and safety of sugemalimab as consolidation therapy in patients with locally advanced/unresectable stage III NSCLC without disease progression after concurrent or sequential chemoradiotherapy. The trial’s primary endpoint was PFS as assessed by BICR according to RECIST v1.1; the secondary endpoints included overall survival, PFS as assessed by investigators and safety profile.

GEMSTONE-302 Study

GEMSTONE-302 (clinicaltrials.gov registration number: NCT03789604; drug clinical trial registration number: CTR20181452) is a randomized, double-blind Phase III study, designed to evaluate the efficacy and safety of anti-PD-L1 monoclonal antibody sugemalimab combined with chemotherapy as the first-line treatment in naïve patients with stage IV non-small cell lung cancer (NSCLC) it vs. placebo combined with chemotherapy. The primary endpoint of the study was investigator-assessed PFS. Secondary endpoints included overall survival, BICR-assessed PFS and safety.

In August 2020, GEMSTONE-302 study met its primary endpoint of significantly prolonging progression-free survival (PFS) and reducing the risk of disease progression or death by 50% with sugemalimab combined with chemotherapy compared to placebo combined with chemotherapy, as assessed by iDMC at the planned interim analysis.

Subgroup analysis showed clinical benefit in patients with squamous versus non-squamous NSCLC, and in patients with PD-L1 expression >=1% versus PD-L1 expression <1%.

Sugemalimab in combination with chemotherapy was well tolerated, no new safety signals were identified. Specific study data were presented in a Proffered Paper Oral Presentation (Late-Breaking Abstract) at ESMO (Free ESMO Whitepaper) Asia 2020. In November 2020, the National Medical Product Administration of China accepted the New Drug Application for sugemalimab combined with chemotherapy for the first-line treatment of advanced squamous and non-squamous non-small cell lung cancer patients.

On May 27, 2021 Immunocore (Nasdaq: IMCR), a late-stage biotechnology company pioneering the development of a novel class of T cell receptor (TCR) bispecific immunotherapies designed to treat a broad range of diseases, including cancer, infection and autoimmune disease, reported that management will present at the following investor conferences in June (Press release, Immunocore, MAY 27, 2021, View Source [SID1234583272]).

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Jefferies Virtual Healthcare Conference

Fireside Chat: Thursday, June 3, 2021 at 8:00 a.m. ET

Goldman Sachs 42nd Annual Global Healthcare Conference

Fireside Chat: Wednesday, June 9, 2021 at 10:30 am ET

The investor conferences will each be held in a virtual format and a live webcast of each fireside chat can be accessed in the ‘Investors’ section of Immunocore’s website at www.immunocore.com. For conferences that offer replays, presentations will be made available for a limited time.

On May 27, 2021 OPKO Health, Inc. (NASDAQ: OPK) reported that management will be participating in the Jefferies Virtual Healthcare Conference being held June 1-4, 2021 (Press release, Opko Health, MAY 27, 2021, View Source [SID1234580673]). Management will be interviewed in a fireside chat on Wednesday, June 2 at 11:30 a.m. Eastern time and will also be holding one-on-one virtual meetings with investors throughout the day.

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The fireside chat will be webcast live and archived in the Investors section of OPKO Health’s website and will be available here.

On May 27, 2021 RedHill Biopharma Ltd. (Nasdaq: RDHL) ("RedHill" or the "Company"), a specialty biopharmaceutical company, reported its financial results and operational highlights for the first quarter ended March 31, 2021 (Press release, RedHill Biopharma, MAY 27, 2021, View Source [SID1234580691]).

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Dror Ben-Asher, RedHill’s Chief Executive Officer, said: "The progress of our two novel, oral COVID-19 programs has put RedHill at the forefront of oral COVID-19 therapeutics development. Opaganib, one of the most advanced and promising novel, dual-mode of action, oral drug candidates in development for COVID-19, now has almost 100% enrollment in its global 464-patient Phase 2/3 study in severe COVID-19." Mr. Ben-Asher continued: "Commercially, a strong end to the first quarter has set up 2021 for growth, reversing a slow start to the year across the industry. Movantik’s new prescriptions in the first quarter outperformed the same quarter last year, while Talicia’s growth in prescription volume, repeat prescribing and new prescribers will be key growth drivers going forward. With the U.S. now emerging from the shadows of COVID-19, patients returning to clinics and travel resuming, positively affecting Aemcolo’s prospects, we are excited for the promise 2021 holds."

Micha Ben Chorin, Chief Financial Officer at RedHill, added: "A strong March helped rebalance quarterly revenues, as we maintained cash burn rate at previous quarter levels. With a healthy balance sheet, we are well-positioned to drive our late-stage R&D programs forward, as we work diligently to build on the upward trends across our core business."

Financial highlights for the quarter ended March 31, 2021[iii]

Net Revenues were approximately $20.6 million for the first quarter of 2021, a decrease of $0.9 million compared to the fourth quarter of 2020. The decrease was mainly attributable to typical cyclical trends in Movantik sales.

Gross Profit was approximately $10.3 million for the first quarter of 2021, a decrease of $0.5 million compared to the fourth quarter of 2020, maintaining a consistent gross margin of approximately 50%. The decrease was mainly attributable to the decrease in net revenues.

Research and Development Expenses were approximately $7.5 million for the first quarter of 2021, an increase of $1.3 million compared to the fourth quarter of 2020, mainly attributable to the progression of our COVID-19 development programs.

Selling, Marketing and General and Administrative Expenses were approximately $21.0 million for the first quarter of 2021, a decrease of $3.3 million compared to the fourth quarter of 2020. The decrease was mainly attributable to large non-recurring marketing investments made in the fourth quarter of 2020.

Operating Loss and Net Loss were approximately $18.2 million and $22.9 million, respectively, for the first quarter of 2021, compared to $19.7 million and $24.3 million, respectively, in the fourth quarter of 2020. The decrease was mainly attributable to the decrease in marketing expenses, as detailed above.

Net Cash Used in Operating Activities was approximately $12.3 million for the first quarter of 2021, a decrease of $0.4 million compared to the fourth quarter of 2020.

Net Cash Provided by Financing Activities was approximately $58.7 million for the first quarter of 2021, comprised primarily of proceeds from equity offerings.

Cash Balance1 as of March 31, 2021, was approximately $92.1 million.

Commercial Highlights

Movantik (naloxegol)[iv]

Movantik ended the quarter strongly with a 4% increase in new prescriptions compared to the first quarter of 2020. Movantik market leadership position is holding strong at 75% U.S. market share, with focus on growth in 2021 and beyond. Movantik also continues to enjoy excellent coverage without restrictions in the PAMORA class for both commercial & government segments, with 88% of American commercial lives covered.

In March 2021, the Company announced that RedHill Biopharma Inc., AstraZeneca AB, AstraZeneca Pharmaceuticals LP and Nektar Therapeutics had entered into a settlement and license agreement with MSN Pharmaceuticals, Inc. and MSN Laboratories PVT. LTD. (MSN) resolving their patent litigation in the U.S. in response to MSN’s Abbreviated New Drug Application (ANDA) seeking approval by the U.S. Food and Drug Administration (FDA) to market a generic version of Movantik. Under the terms of the settlement agreement, MSN may not sell a generic version of Movantik in the U.S. until October 1, 2030 (subject to FDA approval) or earlier under certain circumstances.

In February 2021, the Company also announced an agreement with Cosmo Pharmaceuticals N.V. to manufacture Movantik, securing high-quality manufacturing capacity for our current largest commercial product.

Talicia (omeprazole magnesium, amoxicillin and rifabutin)[v]

Talicia continued to achieve new launch year milestones. Talicia achieved 11% growth in prescription volume and a 39% increase in the number of repeat prescribers compared to the previous quarter, positioning the brand and RedHill for continued growth in 2021 as clinician visits resume and diagnostic labs reopen fully. The quarter ended strongly, resulting in the highest levels of monthly and weekly prescription volume and number of prescribers since launch. Overall, Talicia continues to show growth in total prescribers and repeat prescribing, and March’s performance indicates ongoing momentum for accelerated growth for the remainder of 2021.

Talicia’s growth is supported by an increased commercial coverage of 77%, compared to 69% in the fourth quarter of 2020. Further formulary additions are expected, adding to the previously announced listings of Talicia on the national formularies of Prime Therapeutics, EnvisionRx and Express Scripts.

Aemcolo (rifamycin)[vi]

RedHill has implemented plans, including re-launching active field promotion, to support, and build on, the initial momentum that Aemcolo was generating pre-COVID-19 travel restrictions. The Company expects that these plans will drive a resurgence of interest in Aemcolo once travel restrictions are lifted and international travel from the U.S. returns to significant levels.

In January 2021, the Company reported that its partner, Cosmo Pharmaceuticals, announced it had successfully completed its Phase 2 Proof-of-Concept (POC) clinical trial of rifamycin SV-MMX 600 mg in patients with diarrhea-predominant irritable bowel syndrome (IBS-D). As part of an exclusive license agreement between RedHill and Cosmo Pharmaceuticals from October 2019 for the U.S. rights to Aemcolo (rifamycin), RedHill maintains certain rights, including a right of first refusal, in relation to rifamycin SV-MMX 600 mg in the U.S.

R&D Highlights

COVID-19 Program: Opaganib (ABC294640, Yeliva)[vii]

The global Phase 2/3 study of orally-administered, opaganib in patients with severe COVID-19 pneumonia requiring hospitalization and treatment with supplemental oxygen (NCT04467840) is now almost 100% enrolled. Last patient out will occur approximately six weeks after the final patient is randomized. This puts opaganib amongst the first novel investigational COVID-19 oral pills to deliver late-stage data.

The study has passed four Data Safety Monitoring Board reviews, including a futility review. The fourth DSMB review, conducted in April, was based on an analysis of unblinded safety data from the first 255 patients treated for at least 14 days, extending the total opaganib safety database to approximately 380 patients.

Opaganib has shown dual anti-inflammatory and antiviral activity and is host-targeted, and therefore expected to be effective against emerging viral variants with various mutations in the spike protein.

The Company recently announced receipt of a Notice of Allowance for a U.S. patent application covering the use of opaganib for the treatment of COVID-19 with a term extending until at least 2041. The Company also previously announced that it had signed collaborations with several U.S., European and Canadian suppliers, including with Cosmo Pharmaceuticals for large-scale ramp-up of opaganib manufacturing, further strengthening manufacturing capabilities and capacity of opaganib.

In view of the upcoming completion of enrollment, RedHill is evaluating the regulatory path for opaganib with a focus on those countries currently most affected by COVID-19. The regulatory path, including potential submissions of emergency use applications in those countries, is subject to whether the data generated by the ongoing Phase 2/3 study is sufficiently positive and supportive, as well as the specific requirements in each country. The strength of the safety and efficacy data generated from the opaganib studies will be key to regulatory applications. Additional studies to support the potential of such applications and the use or marketing of opaganib are likely to be required. For example, the FDA has indicated we will need to complete additional studies to support applications in the U.S. Evaluations and discussions continue with the FDA, EMA and regulators in other countries.

The Company continues its discussions with U.S. and other government agencies and non-governmental organizations around potential funding to support the development and manufacturing scale-up of opaganib.

COVID-19 Program: RHB-107 (upamostat)[viii]

In February 2021, RedHill announced dosing of the first patient in the U.S. Phase 2/3 COVID-19 study with novel, orally-administered, RHB-107 (upamostat). The study with once-daily RHB-107 is evaluating treatment of non-hospitalized patients with symptomatic COVID-19 who do not require supplemental oxygen – the vast majority of COVID-19 patients.

RHB-107 is a novel, orally-administered, serine protease inhibitor. It is also host-targeting and therefore also expected to be effective against emerging viral variants with mutations in the spike protein. In previously announced in vitro results, RHB-107 strongly inhibited SARS-CoV-2 viral replication.

The Company recently announced receipt of a Notice of Allowance for a U.S. patent application covering the use of RHB-107 for the treatment of COVID-19 with a term extending until at least 2041.

RHB-204 – Pulmonary Nontuberculous Mycobacteria (NTM) Disease

A U.S. Phase 3 study is ongoing to evaluate the efficacy and safety of RHB-204 in adults with pulmonary NTM disease caused by Mycobacterium avium Complex (MAC) infection.

The FDA also granted Fast Track designation for RHB-204 in January 2021, providing early and frequent communications and a rolling review of any New Drug Application (NDA). RHB-204 is also eligible for NDA Priority Review and Accelerated Approval.

RHB-204 was granted FDA Orphan Drug designation and Qualified Infectious Disease Product designation, extending its U.S. market exclusivity to a potential total of 12 years upon potential FDA approval.

Opaganib – Cholangiocarcinoma and Prostate Cancer

The Phase 2a study evaluating the activity of opaganib in advanced cholangiocarcinoma (bile duct cancer) is ongoing. Enrollment has been completed for the first cohort of 39 patients, evaluating the activity of orally-administered opaganib as a stand-alone treatment. Preliminary data from this cohort indicated a signal of activity in a number of subjects with advanced cholangiocarcinoma. Enrollment is ongoing for a second cohort, evaluating opaganib in combination with hydroxychloroquine, an anti-autophagy agent.

In light of preclinical findings demonstrating tumor regression following combination treatment with opaganib and RHB-107 (upamostat), RedHill plans to add an additional cohort to the ongoing Phase 2a study, evaluating opaganib in combination with RHB-107, subject to discussions with the FDA. Opaganib was granted FDA Orphan Drug designation for the treatment of cholangiocarcinoma.

An additional Phase 2 study with opaganib in prostate cancer is ongoing at the Medical University of South Carolina (MUSC). The study is supported by a National Cancer Institute grant awarded to MUSC with additional support from RedHill.

Conference Call and Webcast Information:

The Company will host a webcast today, Thursday, May 27, 2021, at 8:30 a.m. EDT, during which it will present key highlights for the first quarter of 2021.

The webcast including slides will be broadcast live on the Company’s website, View Source, and will be available for replay for 30 days.

To participate in the conference call, please dial one of the following numbers 15 minutes prior to the start of the call: United States: +1-877-870-9135; International: +1-646-741-3167 and Israel:
+972-3-530-8845; the access code for the call is: 8506238. (Press release, RedHill Biopharma, MAY 27, 2021, View Source [SID1234580691])

On May 27, 2021 Leaps by Bayer, the impact investment arm of Bayer AG reported that it has led a USD 90 million Series B investment in Berlin-based digital health company Ada Health GmbH ("Ada") (Press release, Bayer, MAY 27, 2021, View Source [SID1234580707]). Additional investors joined the round, including Samsung Catalyst Fund, Vitruvian Partners, Inteligo Bank, F4 and Mutschler Ventures.

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The investment will help advance Ada’s popular health assessment technology, enabling the digital health pioneer to accelerate its path towards becoming the world’s leading personalized operating system for health, as well as to further grow the company’s leading position in the United States. In addition to today’s announcement, Bayer and Ada Health are in discussions about entering into a longer-term strategic partnership to support the company’s healthcare businesses.

Ada Health has developed a powerful AI-based health assessment and care navigation platform that helps users to understand their symptoms, to identify and differentiate conditions with a high degree of medical accuracy, and to navigate safely to the right care, at the right time. Ada’s consumer app has become the world’s most popular and highest-rated symptom assessment app, with over 23 million assessments completed since its global launch.

Ada’s core technology is also available in a suite of AI-driven enterprise solutions. The company is collaborating with a range of leading health systems, insurers, life sciences companies, and global non-profit organizations to integrate its symptom assessment and care navigation solutions into a range of digital care journeys to improve outcomes for patients, consumers and healthcare providers.

"Investing in breakthrough technologies that drive digital change in healthcare is one of the strategic imperatives for Leaps by Bayer and for the entire field of healthcare," said Dr. Jürgen Eckhardt, Head of Leaps by Bayer. "Ada’s truly transformative technology, combining powerful artificial intelligence with an emphasis on medical rigor and high levels of clinical accuracy will lead the way in helping more patients and consumers in achieving better health outcomes sooner by intervening earlier in their healthcare journey."

The timely detection of diseases is one of the biggest challenges in guiding consumers and patients to appropriate care, with under-diagnosis, misdiagnosis, and long patient journeys to diagnosis and care unfortunately being commonplace. In addition, global healthcare systems are overburdened, with organizations required to meet pressures such as growing and ageing populations, emerging comorbidity requiring treatment, and the ongoing pandemic. Ada can help shorten time to diagnosis by providing medical guidance that is relevant, actionable and effective, and will increasingly leverage personalized data insights to support the prediction and prevention of diseases.

"Today’s investment will help bring us closer to our goal of improving healthcare outcomes for more than 1 billion people around the world. We’re proud to have investors on board who truly believe in Ada’s technology and vision of transforming the way people access healthcare," Daniel Nathrath, CEO and co-founder of Ada Health comments on today’s announcement. "We are thrilled to have Leaps by Bayer as the lead investor particularly as our values are very much aligned with putting science and technology as well as a commitment to medical quality and user-centricity at the heart of everything we do."