On November 10, 2021 Exscientia (Nasdaq: EXAI), an AI-driven pharmatech company committed to discovering, designing and developing the best possible drugs in the fastest and most effective manner, reported that it will report financial results for the third quarter ended September 30, 2021, on Wednesday, November 17, 2021 after U.S. market close (Press release, Exscientia, NOV 10, 2021, View Source [SID1234595141]). Following the announcement, the Company will host a conference call and webcast at 1:30 p.m. GMT / 8:30 a.m. ET on Thursday, November 18, 2021, to provide additional detail on the different business models underlying the company’s pipeline of more than 25 programmes.

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A webcast of the live call can be accessed by visiting the "Investors and Media" section of the Company’s website at investors.exscientia.ai. Alternatively, the live conference call can be accessed by dialing +1 (760) 294 1674 (U.S.), +44 203 059 5869 (U.K.), +44 203 059 8128 (International) and mentioning Exscientia. A replay will be available for 90 days under "Events and Presentations" in the "Investors and Media" section of the Exscientia website.

On November 10, 2021 Taiga Biotechnologies, a cell-based immunotherapy company, reported that the Company has completed the site initiation session for the first site to participate in the TBX-2400-01 clinical trial, a Phase 1/2 study to evaluate the safety and early efficacy of TBX-2400 in acute myeloid leukemia (AML) and myelofibrosis patients undergoing hematopoietic stem cell transplant (HSCT), in Croatia (Press release, Taiga Biotechnologies, NOV 10, 2021, View Source [SID1234595157]).

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Taiga’s platform technology, TBX-4000 uses the MYC protein to regulate cellular function. When exposed to immune cells, TBX-4000 delivers MYC across the cell membrane directly to the nucleus where MYC drives the activation and proliferation of the cell. Hematopoietic stem cells (HSCs) derived from either bone marrow or GCSF-mobilized apheresis material treated with TBX-4000 form a new product designated as TBX-2400. TBX-2400 is an allogeneic stem cell therapy that improves the rate of engraftment and reconstitution of the immune system for patients receiving a bone marrow transplant.

Taiga has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA), Rare Pediatric Disease Designation from the FDA and Advanced Therapy Medicinal Product Designation from the EMA.

"The initiation of this study, under the aegis of the Croatian Ministry of Health and the EMA, represents a significant milestone for our TBX-2400 clinical development program," said Dr. Yosef Refaeli, Chief Executive Officer of Taiga Biotechnologies. "We believe TBX-2400 has the potential to dramatically change the HSCT market by reducing the risk and increasing the success rate of HSCT, reducing time and cost by as much as 50%, thereby, turning it into a commonplace procedure. In addition, TBX-2400 requires 1000x fewer cells than current procedures, which is expected to eliminate the supply-demand issues that currently limit the number of procedures performed and should increase the number of eligible procedures for acute cases, such as leukemia and lymphoma. Importantly, it has the potential to treat other immune-based diseases, such as diabetes and multiple sclerosis." This clinical study will be managed by Optimapharm, a full service European CRO headquartered in Zagreb, Croatia.

"We hope to positively impact difficult to perform HSCT transplants with the TBX-2400 program. We are encouraged by earlier pre-clinical data that support TBX-2400’s ability to enhance homing to the specialized HSC niches in the bone marrow as well as to improve engraftment," said Brian C. Turner, P.D., Co-founder, President and Chief Scientific Officer of Taiga Biotechnologies.

"We are keen to test the ability of HSCTs treated with TBX-2400 in the context of adult bone marrow failure syndrome as well as in hematologic oncology indications in this study as it holds the promise to significantly enhance and improve outcomes for these very sick patients who are faced with limited treatment options," said Nadira Durakovic, M.D., Associate Professor at the University of Zagreb School of Medicine and University Hospital in Croatia and a principal investigator of the study.

About TBX-2400-01
TBX-2400-01 is a Phase I study to assess the safety and early efficacy of TBX-2400 in enhancing engraftment in patients undergoing allogeneic hematopoietic stem cell transplant (HSCT) for the treatment of acute myelogenous leukemia or myelofibrosis. This is an interventional clinical trial that seeks to recruit 10-20 patients in 3 clinical sites (The University Hospital in Zagreb, The Hadassah Medical Center in Jerusalem, Israel, and the Rambam Medical Center in Haifa, Israel). The primary endpoint relates to the incidence of HSCT related severe adverse events as well as the frequency of successful transplant engraftment. In addition, the secondary endpoints include specific assessments of immune cell function following transplantation and engraftment.

More information on this clinical trial can be found at View Source;draw=2&rank=1.

About TBX-2400
Taiga has developed a proprietary recombinant protein that can passively enter cells without the need for binding and uptake by a cell surface receptor, which can transiently alter intracellular levels of a critical protein involved in survival and proliferation called "MYC." Hematopoietic stem cells (HSCs) derived from either bone marrow or GCSF-mobilized apheresis material treated with TBX-4000 form a new product designated as TBX-2400. The treatment of HSCs with TBX-4000 leads to improved homing to the specialized HSC stem cell niches in the bone marrow as well as improved engraftment. The TBX-2400-01 clinical trial aims to test the safety and early efficacy of TBX-2400 cells in adults who suffer from myelofibrosis or relapsed/refractory acute myeloid leukemia. TBX-2400 has been awarded Orphan Disease Designation by the U.S. FDA and EMA. In addition, this program has also been awarded Rare Pediatric Disease designation by the U.S. FDA.

On November 10, 2021 KemPharm, Inc. (NASDAQ: KMPH), a specialty pharmaceutical company focused on the discovery and development of proprietary prodrugs, reported its financial results for the third quarter ended September 30, 2021 (Press release, KemPharm, NOV 10, 2021, View Source [SID1234595279]). In addition, the Company announced that Richard W. Pascoe, lead independent director on the Board of Directors, has been named Executive Chairman to support execution of the Company’s strategic growth objectives to expand its pipeline and commercialization capabilities. Travis C. Mickle, Ph.D., remains the Company’s President and Chief Executive Officer, and as a member of the Board of Directors.

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"I am delighted to partner with Rich in his new role as Executive Chairman as we build upon our successful track record of clinical development and U.S. regulatory approvals to create an innovative biopharmaceutical company with an expanded pipeline and commercial capabilities," stated Dr. Mickle. "Rich and I have built a strong working relationship since he joined our Board in 2014, and I am proud of our many accomplishments at KemPharm to date. Working together to expand the Company’s growth opportunities will be exciting as together we pursue our shared goal to increase shareholder value."

"I am excited for the opportunity to continue my service to KemPharm as its Executive Chairman and to partner with Travis and the executive team as we seek to grow into a profitable, fully integrated, biopharmaceutical company," said Mr. Pascoe. "Travis, the Board of Directors, and I are committed to this objective which we will seek to accomplish by successfully building, developing, and commercializing a pipeline of innovative product candidates focused on the treatment of neurodegenerative/central nervous system indications."

Mr. Pascoe has a strong track record building and leading life sciences organizations. He has served in key leadership roles in companies that successfully raised over $300 million in equity capital, taken private companies public, closed more than $2 billion of value in business development transactions, obtained regulatory approvals for two products in both the U.S. and Europe, and led commercial launches of prescription drugs in the U.S. across multiple therapeutic categories.

Most recently, Mr. Pascoe served as Chief Executive Officer of Histogen Inc., and his prior experiences include serving as Chief Executive Officer at Apricus Biosciences Inc. and Somaxon Pharmaceuticals, Inc. Mr. Pascoe’s career is also highlighted by several senior management roles, including Chief Operating Officer at ARIAD Pharmaceuticals, Inc., and Senior Vice President of the Neuroscience Division at King Pharmaceuticals, Inc. In addition to serving as Executive Chairman of KemPharm, Inc., Mr. Pascoe is currently a member of the board of directors of Seelos Therapeutics, Inc., and the board of directors of the Johnny Mac Soldiers Fund, a charity for military veterans. Mr. Pascoe is a graduate of the Unites States Military Academy at West Point.

Q3 2021 Corporate and Financial Results:

"The third quarter of 2021 was highlighted by the U.S. commercial launch of AZSTARYS, a seminal event in KemPharm’s history, which occurred during a year with numerous transformative milestones for the company," said Dr. Mickle. "Corium’s commercialization of AZSTARYS continues to proceed as planned, and their team has recently reported to us their estimate that more than 50 million commercial and Medicaid lives now have access to AZSTARYS with more progress expected in the coming months. Additionally, research involving AZSTARYS and serdexmethylphenidate (SDX) was presented at three recent ADHD medical conferences, including data from the pivotal study of AZSTARYS demonstrating the drug’s 30-minute onset-of-action and 13-hour duration-of-effect. At only 100 days post-launch, we are encouraged by the early progress, and we believe that Corium’s commercialization efforts will continue to gain traction with payors, providers, prescribers and patients."

Dr. Mickle continued, "In addition to Rich’s appointment as Executive Chairman, we also announced several other corporate advancements, including the appointment of Tamara Seymour to our Board of Directors and the uplisting of our common stock to The Nasdaq Global Select Market. These developments continue a period of transformation for KemPharm as we position ourselves to capitalize on pipeline development opportunities that, we believe, will ultimately translate to enhanced shareholder value. The advancement of our SDX program is also moving ahead, with data from the ongoing SDX clinical trial expected to be announced prior to year-end. This information will provide valuable insights into the potential path forward for developing SDX-based product candidates designed for therapeutic indications with underserved patient populations."

Financial results for Q3 2021 included revenue of $2.0 million, as compared to Q3 2020 revenue of $1.9 million, which was derived primarily from service fee revenue. The service fee revenue is being earned under consulting arrangements which contractually continue through March 2022.

KemPharm’s net loss for Q3 2021 was $1.8 million, or $0.05 per basic share, compared to a net loss of $3.0 million, or a loss of $0.68 per basic and diluted share for the same period in 2020. Net loss for Q3 2021 was driven primarily by operating loss of $2.2 million, partially offset by non-cash fair value adjustment income of $0.3 million related to derivative and warrant liability and net interest income and other items of $0.1 million. The net operating loss of $2.2 million for Q3 2021 was a change of $1.0 million compared to net operating loss of $1.2 million in the same period in 2020, which was primarily due to increases in operating expenses period over period. The net increase in operating expenses was primarily due to increases in research and development expense of $0.5 million and general and administrative expenses of $0.5 million.

As of September 30, 2021, total cash and cash equivalents was $131.5 million, which was a decrease of $0.8 million compared to $132.3 million as of June 30, 2021.

As of September 30, 2021, total shares of common stock outstanding was 35,317,313 shares, and fully diluted common shares outstanding was 46,553,727 shares, which included 4,252,600 shares issuable upon exercise of warrants. In addition, no preferred stock is outstanding as of September 30, 2021.

Conference Call Information:

KemPharm will host a conference call and live audio webcast with slide presentation on Wednesday, November 10, 2021, at 4:30 p.m. ET, to discuss its corporate and financial results for the third quarter 2021.

Telephone Access: To access the conference call telephonically, interested participants and investors will be required to register via the following online form: View Source

Once registered, all individuals will be provided with participant dial-in numbers, a passcode and a registrant ID, which can then be used to access the conference call.

Participants may register at any time. It is recommended that the registration process be completed at least 15 minutes prior to the start of the call.
Webcast Access: The live audio webcast with slide presentation will be accessible via the Investor Relations section of KemPharm’s website, View Source An archive of the webcast and presentation will be available for 90 days beginning at approximately 5:30 p.m. ET, on November 10, 2021.

About AZSTARYS:

AZSTARYS is an FDA-approved, once-daily product for the treatment of attention deficit hyperactivity disorder (ADHD) in patients age six years or older. AZSTARYS consists of SDX, KemPharm’s prodrug of d-methylphenidate (d-MPH), co-formulated with immediate release d-MPH.

The complete approved prescribing information for AZSTARYS may be downloaded in PDF format here:
View Source

On November 10, 2021 MEI Pharma, Inc. (NASDAQ: MEIP), a late-stage pharmaceutical company focused on advancing new therapies for cancer, reported results for the quarter ended September 30, 2021 and highlighted recent corporate progress (Press release, MEI Pharma, NOV 10, 2021, View Source [SID1234595295]).

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"Fiscal year 2022 is off to an exciting start as we look towards reporting data from the follicular lymphoma cohort in the pivotal TIDAL study evaluating zandelisib in patients with follicular and marginal zone lymphomas which, subject to discussions with FDA, we intend to use to support the submission of our first New Drug Application," said Daniel P. Gold, Ph.D., president and chief executive officer of MEI Pharma. "Beyond TIDAL, our development efforts include studies intended to expand the commercial opportunity for zandelisib in additional indications, like the Phase 3 COASTAL study evaluating follicular and marginal zone lymphoma patients after one prior line of therapy, and the Phase 2 CORAL study that will evaluate zandelisib plus venetoclax and rituximab in patients with chronic lymphocytic leukemia. Our goal is to have zandelisib incorporated as a component of standard therapy across multiple B-cell indications."

Dr. Gold continued: "We are also excited about development plans for our other pipeline candidates, such as evaluating voruciclib’s potential to synergize with venetoclax in patients with AML, and the potential of ME-344 plus bevacizumab in patients with colorectal cancer. And, with $145.5 million in cash at the end of the quarter, plus an additional $10 million milestone subsequently paid to MEI by Kyowa Kirin, as well as promising clinical data across our pipeline, we believe we are in a strong position to achieve our goals in the year ahead, continue to advance our pipeline, and develop best-in-class cancer therapies for patients in need."

First Quarter Fiscal Year 2022 Financial and Drug Candidate Pipeline Highlights

Initiated COASTAL, a Phase 3 study evaluating zandelisib in combination with rituximab in follicular and marginal zone lymphoma patients who received one or more prior lines of treatment. This study is intended to support FDA approval for additional indications and act as the required confirmatory study for the potential accelerated approval of zandelisib in patients with relapsed or refractory follicular lymphoma or marginal zone lymphoma.
Received a $10,000,000 milestone payment from Kyowa Kirin Co. pursuant to the 2020 global license, development and commercialization agreement between the companies triggered in August 2021 by the dosing of the first patient in the Phase 3 COASTAL study.
Triggered an additional $10,000,000 milestone payment from Kyowa Kirin Co. pursuant to the 2020 global license, development and commercialization agreement between the companies for the dosing in September 2021 of the first patient in Japan in the Phase 3 COASTAL study. The milestone payment was received in October 2021, and was recorded as a receivable in the company’s financial statements as of September 30, 2021.
Expected Drug Candidate Pipeline Developments

Zandelisib – Oral PI3K delta inhibitor for the treatment of various B-cell malignancies

Report data from the Phase 2 TIDAL study by the end of calendar year 2021 from the follicular lymphoma cohort of the study. Data from the follicular lymphoma cohort of the Phase 2 TIDAL study data are intended, subject to discussions with the U.S. Food and Drug Administration, to be submitted in support of an initial accelerated approval marketing application.
Initiate a Phase 2 study evaluating zandelisib plus venetoclax and rituximab in patients with chronic lymphocytic leukemia in the first half of calendar year 2022.
Provide an update from the arm of a Phase 1b study evaluating zandelisib plus zanubrutinib, including in expansion cohorts enrolling patients with relapsed or refractory mantle cell and follicular lymphomas in mid calendar year 2022.
Voruciclib – Oral CDK9 inhibitor for the treatment of B-cell malignancies and acute myeloid leukemia

Program update at the 63rd Annual American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, reporting safety and pharmacokinetic data from the monotherapy portion of the Phase 1 program evaluating voruciclib in patients with acute myeloid leukemia and B-cell malignancies.
ME-344 – Tumor selective mitochondrial inhibitor

Initiate a Phase 2 study of ME-344 in relapsed colorectal cancer in the mid calendar year 2022.
Pracinostat – Oral HDAC Inhibitor

MEI and Helsinn have mutually agreed to terminate the Helsinn License Agreement. MEI does not intend to develop pracinostat further for any use and does not anticipate any future material financial obligations regarding the compound.
First Quarter Fiscal Year 2022 Financial Results

As of September 30, 2021, MEI had $145.5 million in cash, cash equivalents, and short-term investments with no outstanding debt.
For the quarter ended September 30, 2021, cash used in operations was $7.7 million, compared to $9.1 million for 2020. The decrease in cash used reflects the receipt of a $10.0 million milestone payment from Kyowa Kirin Co., partially offset by increased costs associated with our clinical development programs.
Research and development expenses were $20.0 million for the quarter ended September 30, 2021, compared to $13.0 million for 2020. The increase was primarily related to increased development costs associated with zandelisib, including increased activity in the TIDAL study and start-up costs related to the Phase 3 COASTAL study, as well as increased personnel costs to support clinical trial activities.
General and administrative expenses were $7.9 million for the quarter ended September 30, 2021, compared to $5.9 million for 2020. The increase primarily relates to personnel costs and general corporate expenses to support our activities, including preparation for commercial launch of zandelisib.
MEI recognized revenues of $13.4 million for the quarter ended September 30, 2021, compared to $3.8 million for 2020. The increase in revenue primarily related to the license agreement with Kyowa Kirin and reflects the recognition of fees allocated to research and development obligations.
Net loss was $11.9 million, or $0.11 per share, for the quarter ended September 30, 2021, compared to net loss of $2.1 million, or $0.02 per share for 2020. The Company had 112,678,498 shares of common stock outstanding as of September 30, 2021, compared with 112,522,001 shares as of September 30, 2020.
The adjusted net loss for the quarter ended September 30, 2021, excluding non-cash expenses related to changes in the fair value of the warrants (a non-GAAP measure), was $14.5 million, compared to an adjusted net loss of $15.3 million for 2020.

On November 10, 2021 Epigenomics AG (FSE: ECX, OTCQX: EPGNY, the "Company") reported financial results (IFRS, unaudited) for the first nine months of 2021 (Press release, Epigenomics, NOV 10, 2021, View Source [SID1234594995]).

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9M 2021 FINANCIAL RESULTS

Total revenue in the first nine months of 2021 increased to EUR 6,022 thousand (9M 2020: EUR 541 thousand), due to the sale of samples from the Company’s "biobank" in the amount of EUR 5,675 thousand. Product revenue decreased from EUR 453 thousand to EUR 330 thousand in the reporting period compared to the same period of the previous year due to the Covid-19 pandemic.
Research and development costs decreased from EUR 3,412 thousand to EUR 2,226 thousand in the nine-month period due to a reduction in clinical study costs related to Covid-19; however, patient enrollment for the Epi pro Colon post-approval study has recently begun to increase closer to pre Covid 19 levels.
Selling and administrative expenses increased slightly from EUR 5,442 thousand to EUR 5,809 thousand primarily due to the costs associated with the sale of the Biobank samples.
EBITDA (before share-based payment costs) improved significantly to EUR -209 thousand in the reporting period compared to EUR -8,090 thousand in the same period of the previous year. This is also primarily due to the biobank samples sale.
The net loss for the period also decreased significantly to EUR -691 thousand (9M 2020: EUR -9,109 thousand); accordingly, the loss per share decreased from EUR 1.59 to EUR 0.07 compared to the same period of the previous year.
Cash consumption decreased to EUR 3,598 thousand in the first nine months of 2021 (9M 2020: EUR 7,492 thousand).
As of 30 September 2021, the Company had cash and cash equivalents (including marketable securities) of EUR 23,555 thousand (31 December 2020: EUR 3,566 thousand).
OPERATIONAL DEVELOPMENTS

Epigenomics initiated clinical trial plans for Epi proColon "Next-Gen", an updated version of the assay that meets CMS reimbursement criteria based upon performance from prospectively collected banked clinical samples. The Company plans to begin enrollment in the summer of 2022.
In addition, Epigenomics executed a EUR 5.7 million sale of samples from the Company’s extensive biobank. The Company has retained the necessary samples to complete development activities for Epi proColon "Next-Gen".
In September the company successfully completed the placement of a subordinated non-interest-bearing mandatory convertible bond that raised EUR 16.5 million in gross proceeds.
OUTLOOK 2021

Revenue

As a result of the sale of the biobank to New Horizon Health Limited, the revenue and earnings guidance for the full year 2021 was adjusted on August 17, 2021: The Company expects revenues of EUR 6.0 million for the financial year 2021 (previously: EUR 0.4 million to EUR 1.0 million).
EBITDA / cash consumption

For EBITDA before share-based payment costs, Epigenomics forecasts a range of EUR -3.0 million to EUR -4.0 million (previously: EUR -7.0 million to EUR -9.0 million) for the full year 2021. Based on the Company’s 2021 business plan, cash consumption is expected to range between EUR 3.5 million to EUR 4.5 million (previously: EUR 7.0 million to EUR 9.0 million).
Further information

The financial report for the first nine months of 2021 is available on the Epigenomics’ website at: View Source." target="_blank" title="View Source." rel="nofollow">View Source

Conference call for analysts and investors

Epigenomics AG will host a conference call for analysts and investors today at 4.00 pm (CET) / 10.00 am (EDT). The webcast can be accessed at the following link: View Source

The dial-in numbers for the conference call are (for registered participants):

Dial-in number Germany: +49 30 232531508
Dial-in number UK: +44 1635 598058
Dial-in number U.S.A.: +1 516-269-8975

Participants are asked to dial in 10 minutes prior to the start of the conference call and to register using the link above.

An audio replay of the conference call will be provided on the Company’s website following the call.