On September 19, 2022 Epigenomics Inc, a wholly-owned subsidiary of Epigenomics AG (Frankfurt Prime Standard: ECX, OTCQX: EPGNY; the "Company"), reported it has enrolled its first subject in a new pivotal clinical trial supporting the Company’s "Next-Generation" blood-based diagnostic screening test for the detection of colorectal cancer (CRC) (Press release, Epigenomics, SEP 19, 2022, View Source [SID1234619641]).

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The prospective, multi-center study titled CRC-DRAW (CRC–Detection Reliable Assessment With Blood) will enroll screening eligible participants 45 years of age and older who are at average risk for colorectal cancer. Over the course of the trial, it is expected to enroll over 15,000 subjects to achieve the proposed statistical endpoints needed for the study. Clinical outcomes generated from CRC-DRAW will be used to support the planned Pre-Market Approval (PMA) submission of the Company’s "Next-Generation" CRC screening test with the U.S. Food and Drug Administration (FDA).

"CRC is still the number two cause of cancer deaths in the U.S. because numerous patients remain unscreened due to barriers with access" said Greg Hamilton, CEO of Epigenomics. "More than ever physicians need options that patients will embrace and we believe that a reliable blood-based test that is effective and affordable provides a meaningfully differentiated solution to this problem. We have initiated our CRC-DRAW trial because we are confident the improved performance of our "Next-Gen" test will give patients, physicians and providers an easy-to-use non-invasive option that will dramatically increase screening rates and ultimately save lives."

"Epigenomics has been a long-standing pioneer in the liquid biopsy space with the first ever FDA approved cancer test from blood," said Dr. Andrew A. Lukowiak, President and Chief Scientific Officer. "We have utilized this know-how and experience to augment our previous test with additional biomarkers to significantly improve the performance. We look forward to presenting preliminary test performance data within the next couple of months."

On September 19, 2022 GENFIT (Nasdaq and Euronext: GNFT), a late-stage biopharmaceutical company dedicated to improving the lives of patients with severe chronic liver diseases, reported it has entered into an exclusivity agreement with a view to acquire all the share capital and voting rights of Versantis, a private Swiss-based clinical stage biotechnology company focused on addressing the growing unmet medical needs in liver diseases (Press release, Genfit, SEP 19, 2022, https://ir.genfit.com/news-releases/news-release-details/genfit-acquire-clinical-stage-biopharmaceutical-company [SID1234619640]).

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The acquisition of Versantis fits perfectly within GENFIT’s strategic vision of becoming a global leader in ACLF (acute-on-chronic liver failure) and is another critical milestone in the execution of GENFIT’s strategic plan. With this acquisition, GENFIT consolidates its position in ACLF via the integration of a clinically advanced asset presenting a solid scientific rationale supported by encouraging Phase 1b and preclinical data. GENFIT will also further expand its pipeline in other liver diseases characterized by high unmet medical needs with additional product candidates developed by Versantis. In addition, GENFIT’s know-how in ACLF will broaden, with the integration of Versantis’ team of experts, joining forces to accelerate both research and development.

ACLF is an underserved medical condition associated with short-term mortality (23% to 74% mortality at 28 days, depending on severity grade) and a significant cost of care. No drugs have been approved in this indication so far and incidence is growing at epidemic rates due to an aging population and a higher prevalence of diabetes, obesity, NASH, as well as alcohol and drug-induced liver injury. From a patient perspective, the ACLF syndrome is characterized by an abrupt life-threatening worsening of a pre-existing advanced chronic liver disease resulting in liver and extrahepatic organ failure (brain, kidneys, cardiovascular and respiratory). The cascade of multiple organ failures, including the development of a neuropsychiatric condition called hepatic encephalopathy (HE), together, lead to major complications in patients with ACLF, who can rapidly progress into coma and death. Every year, an estimated 137’000 patients are hospitalized in the US with ACLF, with very few therapeutic options. This represents an important unmet medical need.

As a pioneer in ACLF, Versantis has acquired a unique expertise, developing clinical-stage technology and assets aiming to improve ACLF patients’ outcomes.

Its main asset, VS-01, is a first-in-class innovative liposomal-based therapeutic product candidate currently in clinical development as a potential first-line therapy for the timely recovery of ACLF and UCD. If approved, it would be the first drug to use the intraperitoneal route to simultaneously support the liver, kidney and brain, the organs that most often fail in cirrhotic patients. VS-01 operates to clear toxic metabolites from the body following paracentesis, by extracting them from the blood into the peritoneal (abdominal) cavity, where they are captured by proprietary scavenging liposomes which are then drained from the body. A planned 60-patient, randomized and controlled Phase 2 Proof-of-concept trial of VS-01 in ACLF is expected to launch in the fourth quarter 2022. Efficacy and safety interim data are expected as early as the first half of 2024. The US Food and Drug Administration (FDA) granted VS-01 with the Orphan Drug Designation (ODD) in ACLF and in UCD and with the Rare Pediatric Diseases Designation (RPDD) for the acute treatment of UCD. The European Medicines Agency (EMA) also granted VS-01 with ODD in acute liver failure. Given the unmet medical need and the current standard of care, GENFIT intends to seek approval of these candidates via expedited regulatory pathways.

VS-02 is a pre-clinical oral, small molecule drug candidate being developed for the chronic management of HE, considered an endemic disease worldwide. HE is a nervous system disorder brought on by advanced chronic liver disease. VS-02 will be developed as a unique colon-active formulation designed to minimize systemic absorption of ammonia and act where ammonia is primarily produced, while reducing glutamine levels in the brain.

GENFIT will also be able to develop TS-01, a unique point-of-care diagnostic device in prototype development for the at-home measurement of ammonia in the blood, the primary cause of HE.

The deal should be completed during the fourth quarter 2022, following completion of the consultation of GENFIT’s employees representative bodies.

In parallel, GENFIT continues the development of its other program evaluating NTZ in ACLF, with a pre-IND meeting scheduled with the FDA in the coming weeks, following encouraging Phase 1 data.

Pascal Prigent, Chief Executive Officer of GENFIT, commented: "Versantis has an exciting portfolio that is complementary to GENFIT’s. We are also thrilled to welcome a talented team that has developed a strong scientific expertise in ACLF. We believe that significant synergies exist and that this acquisition will accelerate the development of several promising drug candidates in areas of high unmet needs." Jean-François Mouney, Co-founder and Chairman of the Board of GENFIT, added: "This agreement is a new chapter in the implementation of GENFIT’s strategy, which expands and diversifies our portfolio with assets presenting a significant market potential." Vincent Forster, PhD, Chief Scientific Officer and Meriam Kabbaj, PhD, Chief Operations Officer, both Board Members and Co-founders of Versantis, concluded: "We are enthusiastic to be part of GENFIT, considering their experience in exploring severe and underserved conditions. We think that GENFIT’s knowledge and experience in the development of programs targeting complex liver diseases will be invaluable to accelerate and maximize the probability of success of our programs. We also are excited by the new opportunities offered by GENFIT’s research capabilities."

Financials
The deal includes an initial consideration of CHF40 million due at closing, with contingent consideration of up to CHF65 million upon positive Phase 2 results for VS-01 and VS-02 and regulatory approval of VS-01. In addition, Versantis is eligible to receive 1/3 of the net proceeds resulting from the potential sale of the Pediatric Review Voucher of VS-01’s pediatric application by GENFIT to a third party, or 1/3 of the fair market value of this Voucher if GENFIT opts to apply it to one of its own programs. GENFIT will finance the base acquisition consideration from its current cash and cash equivalents. Based on our development plan for our current programs and for Versantis’ programs, the revenue expected from our partnership agreements, and accounting for transaction costs, we anticipate that funding of the Group’s corporate development is secured for approximately 2 years.

GENFIT will host a conference call on September 19, 2022 at 08:00am ET / 1.00pm GMT / 2.00pm CET in English and in French

Both the English and French conference calls will be accessible on the investor page of our website, under the events section at https://ir.genfit.com/ or by calling 888-394-8218 (toll-free US and Canada), 0800 358 6377 (toll-free UK) or 0805 101 219 (France toll-free) five minutes prior to the start time (confirmation code: 3338254). A replay will be available shortly after the call.

Upcoming calls and events

Live conference call today, September 19, at 8:00am ET / 1:00pm GMT / 2:00pm CET in English and in French
Half-year financial results (press release) on September 28, 2022
Pipeline days on October 5 (French session in Paris, France) and October 19 (English session in NYC, United States)

On September 19, 2022 BeiGene (NASDAQ: BGNE; HKEX: 06160; SSE: 688235), a global biotechnology company focused on developing innovative and affordable oncology medicines to improve treatment outcomes and access for patients worldwide, reported that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a positive opinion recommending approval of BRUKINSA (zanubrutinib) for the treatment of adult patients with marginal zone lymphoma (MZL) who have received at least one prior anti-CD20-based therapy (Press release, BeiGene, SEP 19, 2022, View Source [SID1234619638]).

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"There are currently no BTK inhibitors approved for MZL in Europe and with this positive opinion, we are one step closer to bringing forward a chemotherapy-free treatment option for this rare blood cancer. We look forward to a decision from the European Commission in the coming months," said Mehrdad Mobasher, M.D., M.P.H., Chief Medical Officer, Hematology at BeiGene. "We have undertaken a broad development program to evaluate BRUKINSA as a potential treatment for various B-cell malignancies in over 4,500 patients, which continues to generate evidence to support BRUKINSA as an effective and well-tolerated treatment option for blood cancer patients around the world."

The CHMP recommendation is based on positive results from the Phase 2, open-label, multicenter, single-arm MAGNOLIA trial (NCT03846427) in 66 patients with relapsed or refractory (R/R) MZL who received at least one anti-CD20-based regimen. In the study, the overall response rate (ORR) was 68% (95% CI: 55.6, 79.1) with 26% of patients achieving complete response (CR) and 42% achieving partial response (PR). The median time to response was 2.8 months (range: 1.7 to 11.1 months) and ORRs for MZL subtypes extranodal, nodal, splenic, and unknown were 64%, 76%, 67%, and 50%, respectively.

BRUKINSA demonstrated favorable and well-defined tolerability consistent with its known safety profile. The most common (≥30%) adverse reactions, including laboratory abnormalities, in the pooled safety population of 847 patients were decreased neutrophil count, upper respiratory tract infection, decreased platelet count, and hemorrhage. The observed cardiac safety profile was consistent with previous BRUKINSA studies, with low rates of atrial fibrillation (3%) and atrial flutter (0.4%). BRUKINSA was well-tolerated, as demonstrated by low rates of discontinuation due to adverse events (6%).

Pier Luigi Zinzani, MD., PhD., Full Professor of Haematology at the Institute of Haematology "Seràgnoli", University of Bologna, Italy commented, "Marginal zone lymphoma encompasses a number of subtypes. As a highly selective BTK inhibitor, the clinical trial data for BRUKINSA showed deep and sustained overall responses regardless of subtype, along with a well-established safety profile. If approved, BRUKINSA has the potential to deliver meaningful outcomes for MZL patients in Europe who otherwise have no approved treatment options."

"This opinion is an important milestone as we work to bring a BTK inhibitor for the first time to European MZL patients," notes Gerwin Winter, Senior Vice President, Head of Europe at BeiGene. "We look forward to combining our global scale and local expertise to deliver innovative medicines to patients across Europe."

Following the CHMP positive opinion, the European Commission will consider BeiGene’s Marketing Application, with a final decision expected within 67 days of the CHMP opinion. The decision will be applicable to all 27 member states of the European Union (EU) plus Iceland and Norway. BRUKINSA is currently approved in the EU for the treatment of adult patients with Waldenström’s macroglobulinemia (WM) who have received at least one prior therapy or for the first-line treatment of patients unsuitable for chemo-immunotherapy.

BeiGene has obtained reimbursement for BRUKINSA for the treatment of Waldenström’s macroglobulinemia in Austria, Belgium, Denmark, England and Wales, Germany, Ireland, Spain, and Switzerland while additional EU countries are currently going through the reimbursement process.

About Marginal Zone Lymphoma
MZL is a group of ultra-rare, slow growing B-cell malignancies that begin in the marginal zones of lymph tissue.i Epidemiological data from Europe is limited, but the incidence rate of MZL is estimated to range between 20 and 30 per million per year.ii,iii,iv There are three different subtypes of MZL: extranodal marginal zone B-cell lymphoma, or mucosa-associated lymphoid tissue (MALT), which is most common; nodal marginal zone B-cell lymphoma which develops in the lymph nodes and is rare; and splenic marginal zone B-cell lymphoma which develops in the spleen, bone marrow, or both, and is the rarest form of the disease.v

About BRUKINSA
BRUKINSA is a small molecule inhibitor of Bruton’s tyrosine kinase (BTK) discovered by BeiGene scientists that is currently being evaluated globally in a broad clinical program as a monotherapy and in combination with other therapies to treat various B-cell malignancies. Because new BTK is continuously synthesized, BRUKINSA was specifically designed to deliver targeted and sustained inhibition of the BTK protein by optimizing bioavailability, half-life, and selectivity. With differentiated pharmacokinetics compared to other approved BTK inhibitors, BRUKINSA has been demonstrated to inhibit the proliferation of malignant B cells within a number of disease relevant tissues.

BRUKINSA is supported by a broad clinical program which includes more than 4,500 subjects in 35 trials across 28 markets. To date, BRUKINSA has received more than 20 approvals covering more than 50 countries and regions, including the United States, China, the EU, Great Britain, Canada, Australia, and additional international markets. Currently, more than 40 additional regulatory submissions are in review around the world.

BeiGene Oncology
BeiGene is committed to advancing best- and first-in-class clinical candidates internally or with like-minded partners to develop impactful and affordable medicines for patients across the globe. We have a growing R & D and medical affairs team of approximately 3,300 colleagues dedicated to advancing more than 100 clinical trials that have involved more than 16,000 subjects. Our expansive portfolio is directed predominantly by our internal colleagues supporting clinical trials in more than 45 countries and regions. Hematology-oncology and solid tumor targeted therapies and immuno-oncology are key focus areas for the Company, with both mono- and combination therapies prioritized in our research and development. BeiGene currently has three approved medicines discovered and developed in our own labs: BTK inhibitor BRUKINSA in the U.S., China, the European Union, Great Britain, Canada, Australia, and additional international markets; and the non-FC-gamma receptor binding anti-PD-1 antibody, tislelizumab, as well as the PARP inhibitor, pamiparib, in China.

BeiGene also partners with innovative companies who share our goal of developing therapies to address global health needs. We commercialize a range of oncology medicines in China licensed from Amgen, Bristol Myers Squibb, EUSA Pharma and Bio-Thera. We also plan to address greater areas of unmet need globally through our other collaborations including with Mirati Therapeutics, Seagen, and Zymeworks.

In January 2021, BeiGene and Novartis announced a collaboration granting Novartis rights to co-develop, manufacture, and commercialize BeiGene’s anti-PD1 antibody, tislelizumab, in North America, Europe, and Japan. Building upon this productive collaboration, BeiGene and Novartis announced an option, collaboration, and license agreement in December 2021 for BeiGene’s TIGIT inhibitor, ociperlimab, that is in Phase 3 development. Novartis and BeiGene also entered into a strategic commercial agreement through which BeiGene will promote five approved Novartis Oncology products across designated regions of China.

On September 19, 2022 Allakos Inc. (Nasdaq: ALLK), a biotechnology company developing lirentelimab (AK002) and AK006 for the treatment of allergic and inflammatory diseases, reported the pricing of an underwritten offering of 29,882,000 shares of its common stock at an offering price of $5.02 per share (Press release, Allakos, SEP 19, 2022, View Source [SID1234619637]). Investors who have agreed to purchase shares in the offering include Logos Capital, Alta Partners, Braidwell LP, BVF Partners L.P., Commodore Capital, Deep Track Capital, Frazier Life Sciences, New Enterprise Associates (NEA), RTW Investments, L.P., Surveyor Capital (a Citadel Company), TCGX, and Vivo Capital. Before deducting the underwriting discounts and commissions and estimated offering expenses, the company expects to receive total gross proceeds of approximately $150 million. All of the shares are to be sold by the company. The offering is expected to close on or about September 21, 2022, subject to satisfaction of customary closing conditions.

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Jefferies is acting as the lead book-running manager and Cowen and LifeSci Capital are acting as joint book-running managers for the offering.

The shares are being offered by the company pursuant to a Registration Statement on Form S-3 previously filed and declared effective by the SEC. A prospectus supplement and accompanying prospectus relating to the offering will also be filed with the SEC. These documents can be accessed for free through the SEC’s website at www.sec.gov.

When available, a copy of the prospectus supplement and the accompanying prospectus relating to the offering may also be obtained from:

Jefferies LLC, Attention: Equity Syndicate Prospectus Department, 520 Madison Avenue, New York, NY 10022, by email at [email protected], or by telephone at (877) 821-7388; or

Cowen and Company, LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, Attn: Prospectus Department, by email at [email protected] or by telephone at (833) 297-2926.

LifeSci Capital LLC, Attention: Syndicate Prospectus Department, 250 West 55th Street, 34th Floor, New York, NY 10019, by email at [email protected], or by telephone at (646) 876-5059.

This press release does not constitute an offer to sell or a solicitation of an offer to buy, nor will there be any sale of these securities in any state or other jurisdiction in which such offer, solicitation, or sale would be unlawful before registration or qualification under the securities laws of that state or jurisdiction.

On September 19, 2022 Roche reported that it will publish its Sales Results for the 3rd Quarter of 2022 prior to the opening of the Swiss Stock Exchange on Tuesday, 18 October 2022 (Press release, Hoffmann-La Roche, SEP 19, 2022, View Source [SID1234619636]).

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