On September 18, 2020 Autolus Therapeutics plc (Nasdaq: AUTL), a clinical-stage biopharmaceutical company developing next-generation programmed T cell therapies, reported new data highlighting progress on AUTO3, the company’s CAR T cell therapy being investigated in the ALEXANDER study, a Phase 1/2 clinical trial in relapsed/refractory diffuse large B cell lymphoma (DLBCL), during the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Virtual Congress 2020, beginning 18 September (Press release, Autolus, SEP 18, 2020, View Source [SID1234565356]).

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"Today we presented data from our recommended Phase 2 dose cohort from the ALEXANDER trial of AUTO3, a CD19 and CD22 dual targeting CAR T product candidate in DLBCL. The data support a best in class profile with a high level of complete remissions and a well tolerated safety profile," said Dr. Christian Itin, chairman and chief executive officer of Autolus.

"AUTO3 has a tolerable and very favorable safety profile when compared with approved CD19 CAR T therapies," said Dr Craig Moskowitz, Professor of Medicine at Miller School of Medicine, University of Miami Health System. "It has a promising complete response rate of 64%, as demonstrated in the completed cohorts at the recommended Phase 2 dose range and, thus far, these complete remissions also appear durable. Among all dose cohorts with a median follow up of 6 months, 93% of the patients who achieved a complete response did not relapse."

As of the data cut-off date of August 3, 2020, 35 patients in the ALEXANDER Phase 1/2 clinical trial of AUTO3 have been treated and were evaluable for safety. AUTO3 was well tolerated, with no Grade 3 or higher cytokine release syndrome (CRS) with primary infusion and low rates of neurotoxicity (NT). Across all 35 patients, only three cases of NT have been reported, with two having ³Grade 3. Among the 20 patients treated at a dose of ³ 150 x 106 cells with pre-conditioning pembrolizumab on day minus 1 (D-1), which is the declared recommended Phase 2 dose (RP2D), one patient experienced ³Grade 3 NT (patient died due to disease progression and multiorgan failure). None of the patients achieving a complete response (CR) experienced any NT and all cases of NT reported have been atypical in nature and seen in a setting with disease progression and confounding factors.

In terms of efficacy data, of the 35 patients dosed to date, 30 patients were evaluable within their completed cohort. The cohort receiving a dose of ³ 150 x 106 cells and pre-conditioning pembrolizumab D-1 (the RP2D) had an objective response rate (ORR) of 71% and CR rate of 64%. For all patients on study across all dose levels that were evaluable, the ORR was 68% and CR rate of 54%.

Investor call on Friday September 18, 2020

Management will host a conference call and webcast today at 8:00 am EDT/1:00 pm BST to discuss the ESMO (Free ESMO Whitepaper) data. To listen to the webcast and view the accompanying slide presentation, please go to: View Source The call may also be accessed by dialing (866) 652-5200 for U.S. and Canada callers or (412) 317-6060 for International callers. Please ask to be joined into the Autolus Therapeutics call. After the conference call, a replay will be available for one year on Autolus’ website.

On September 18, 2020 Mogrify Limited (Mogrify), a UK company aiming to transform the development of ex vivo cell therapies and pioneer the field of in vivo reprogramming therapies, reported it had won the AstraZeneca Life Science Innovation category of the Business Weekly Awards (Press release, Mogrify, SEP 18, 2020, View Source [SID1234565355]). The award commends Mogrify in applying its proprietary direct cellular conversion technology to further the cause of life science discovery for the benefit of human healthcare. Prof. Julian Gough, Co-founder and CSO of Mogrify, received the Cambridge Enterprise Academic Entrepreneur of the Year award in recognition of his work as a life science innovator and founder of Mogrify.

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Mogrify’s proprietary direct cellular conversion technology determines the conversion factors required to convert (ex vivo or in vivo) any target cell type from any source cell type. The Company is deploying this platform to engineer an evergreen and scalable source of cell types that exhibit efficacy and safety profiles necessary to address diseases with a high unmet clinical need, such as in ophthalmological, immunological, hematological, and other disease areas.

Prof. Gough is a leading bioinformatician and biotech entrepreneur who led the development and application of the Mogrify platform, which utilizes a systematic big-data approach (Rackham et al., Nature Genetics, 2016) developed over a 10-year period via a multi-national research collaboration. Graduating with a PhD in molecular biology from the University of Cambridge, Julian became Professor of Bioinformatics at the University of Bristol and is now Programme Leader at the MRC Laboratory of Molecular Biology in Cambridge. He has also successfully started a number of biotech companies.

Dr. Darrin M. Disley, OBE, CEO, Mogrify, said: "Our team are committed to transforming healthcare for indications of high unmet clinical need. The award for Life Science Innovation attests to the potential of Mogrify’s platform and pioneering team to overcome safety, efficacy and scalability challenges for cell therapies and we are honored to have this recognized by the judges at this year’s esteemed business awards.

Julian is an outstanding scientist and entrepreneur who has worked tirelessly to advance Mogrify technology and drive its therapeutic application forward, paving the way for future treatments and inspiring those who work alongside him."

Professor Julian Gough, Co-founder and CSO, Mogrify, said: "Following on from our previous success in the ‘Disruptive Technology’ category at last year’s Business Weekly Awards, I am delighted to see Mogrify go from strength to strength and achieve success again in 2020.

I am immensely proud to have been named ‘Academic Entrepreneur of the Year’ and would like to thank my peers who have supported me throughout my career and Mogrify’s talented team for their dedication and hard work in pioneering novel cell therapies."

Since February 2019, Mogrify has raised over $20 million USD in seed and series A rounds and $2.5 million USD in grant funding, and has grown to a headcount over 60 of scientific, operational, and commercial staff.

On September 18, 2020 Case Western Reserve reported that researchers think they have a better idea—and the National Institutes of Health has given them $420,000 to advance it (Press release, Case Western Reserve University, SEP 18, 2020, View Source [SID1234565354]).

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Highly collaborative in nature, the project teams researchers from the university’s schools of dental medicine, medicine and engineering.

Their concept combines imaging and algorithmic technologies to assess whether or not a lesion’s cells are malignant.

Aaron Weinberg, chair and professor in the Department of Biological Sciences in the School of Dental Medicine and professor of otolaryngology and pathology in the School of Medicine,
Aaron Weinberg, chair and professor in the Department of Biological Sciences in the School of Dental Medicine and professor of otolaryngology and pathology in the School of Medicine
"The idea is to quickly, easily, non-invasively and cost-effectively determine if a suspicious lesion is cancerous, or in other cases, to screen individuals on a regular basis to see if their lesion has progressed to cancer," said dental faculty member Aaron Weinberg, the project’s principal investigator.

On average, oral cancer claims the life of someone in America every hour, according to the Oral Cancer Foundation.

Early detection is the key to survival, said Weinberg, chair and professor in the dental school’s Department of Biological Sciences. Their approach not only aims to reduce the need for biopsies, but also provide a much-needed diagnostic alternative in places where pathology services are few and difficult to access.

How it works
Researchers envision a hand-held device that can determine the ratios of two key proteins, where one goes up in cancer while the other one does not. By collecting cells swabbed from suspicious oral lesions, and using fluorescent antibodies that bind specifically to one or the other protein within the cells, the device collects images of fields of cells and an app converts the fluorescence intensity of each protein into numbers and calculates the ratio.

The numerical value above a given threshold indicates cancer.

This procedure has already proven to be accurate and effective in laboratory-based techniques, Weinberg said—and the intention is to convert the lab procedure into a device that will cut the time in obtaining results from two days to about 30 minutes.

"This will address major unmet needs in early oral cancer detection worldwide, especially in resource poor settings where pathology review is lacking and/or unreliable," he said.

Weinberg is joined in the research by Umut Gurkan, the Warren E. Rupp Associate Professor at the Case School of Engineering; Anant Madabhushi, the F. Alex Nason Professor II of Biomedical Engineering and director of the Center for Computational Imaging and Personalized Diagnostics; and Rod Rezaee, associate professor in the Department of Otolaryngology at the School of Medicine and University Hospitals.

On September 18, 2020 Checkmate Pharmaceuticals, Inc. (NASDAQ: CMPI) ("Checkmate"), a clinical stage biotechnology company focused on developing proprietary technology to harness the power of the immune system to combat cancer, reported second quarter 2020 financial results and provided an update on recent progress (Press release, Checkmate Pharmaceuticals, SEP 18, 2020, View Source [SID1234565352]).

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"This is an exciting time for our company as we rapidly develop CMP-001 across multiple tumor types in combination with checkpoint inhibitors," said Barry Labinger, Chief Executive Officer. "Our recent public offering positions us well to execute on our clinical and strategic plans, including advancement of CMP-001 towards registration in melanoma and towards demonstration of proof of concept in additional tumor types, including head and neck cancer. We are on track to initiate key new clinical trials by late 2020/early 2021 as planned."

Recent Progress

In August 2020, Checkmate closed its initial public offering of 5,000,000 shares of common stock at $15 per share. Including the subsequent partial exercise of the underwriters’ overallotment option, total gross proceeds were approximately $76.6 million, before deducting underwriting commissions and offering expenses.
In July 2020, the U.S. Food and Drug Administration granted Fast Track designation to Checkmate’s product candidate, CMP-001, a differentiated Toll-like receptor 9 (TLR9) agonist, in combination with a programmed death receptor 1 (PD-1) blocking antibody (nivolumab or pembrolizumab) in both anti-PD-1 refractory melanoma and first-line metastatic melanoma.
Checkmate is actively engaging with potential clinical sites and remains on track to initiate three Phase 2 trials combining CMP-001 with PD-1 blockade by late 2020/early 2021 for the treatment of:
First-line head and neck cancer
Anti-PD-1 refractory melanoma
First-line metastatic or unresectable melanoma
Second Quarter 2020 Financial Results

Cash and cash equivalents: Cash and cash equivalents were $80.3 million as of June 30, 2020 (excluding proceeds from the IPO which was completed in August 2020).
Research and development expenses (R&D): R&D expenses were $6.5 million for the quarter ended June 30, 2020, compared to $5.8 million for the quarter ended June 30, 2019. The increase was primarily attributable to start-up costs for upcoming clinical trials as well as manufacturing and process development costs.
General and administration expenses (G&A): G&A expenses were $1.8 million for the quarter ended June 30, 2020, compared to $1.1 million for the quarter ended June 30, 2019. The increase was primarily attributable to expenses incurred in connection with the Checkmate’s preparation for operating as a publicly-traded company.
Net loss and comprehensive loss: Net loss and comprehensive loss was $8.3 million for the quarter ended June 30, 2020, compared to $6.9 million for the quarter ended June 30, 2019.

On September 18, 2020 IntegraGen (FR0010908723 – ALINT – Eligible PEA PME), a company specializing in the decryption of the human genome, which performs interpretable genomic analyzes for academic and private laboratories, reported its financial results for the first half of 2020 (Press release, Integragen, SEP 18, 2020, View Source [SID1234565351]). The accounts were approved by the Board of Directors at a meeting held on 17 September 2020.

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Bernard Courtieu, Chairman and CEO of IntegraGen, said: "The results IntegraGen the first half 20 20 confirms the performance we achieved for the second half of 2019, with an EBITDA positive operating performance. We are also announcing business growth of 15% on a comparable basis to 2019 in an environment particularly complicated by the COVID-19 health crisis during the second quarter of this year. This performance confirms the soundness of our model of focusing on genomics and has been enabled by the trust and loyalty of our customers in our ability to meet their needs and by the efforts and dedication of all IntegraGen employees.

Finally, we are very happy to be able to lead the industrial and commercial merger project with the Belgian company OncoDNA. In fact, our complementary assets will enable us to create a European market leader in Genomic Services and to accelerate our international efforts while sustaining and developing jobs within the two companies."

Sales revenue for the first half 20 20 was up by 15 % to 4,706 thousand euros on a comparable basis against 4,102 thousand euros during the same period in 2019, excluding income of diagnostics related activities, the assets of which were sold at the start of 2020. After considering transfer charges and reversal of depreciation and amortization, revenues were 4,745 thousand euros compared to 4,183 thousand euros during the first half of 2019, which included 62 thousand euros in diagnostic revenue.

The operating expenses amounted to 4,613 thousand euros, down 1% compared to the first half of 2020. Optimization of resources enabled a second consecutive 6-month period to achieve balance. Reagent costs remain stabled. As of June 2020 the company had 47 employees. The operating profit before tax, depreciation and amortization (EBITDA) was a positive 248 thousand euros during the first half 2020 compared to a loss of 325 thousand euros in the first half 2019.

The operating profit before tax showed a gain of 132 thousand euros, also a very clear improvement compared to the same period of 2019 (-484 thousand euros). Of note was the exceptional result of-166 thousand and Credit of Tax Innovation (CTI) related to the research conducted during the period. The net result was a profit of 17 thousand euros, compared to a loss of 538 thousand euros in the first half of 2020.

The net cash of IntegraGen as of June 30, 2020 totaled 5,730 thousand euros compared to 2,749 thousand euros at the end of December 2019. This variation is mainly explained by the obtaining of a State Guaranteed Loan (PGE) of 1,800 thousand euros through the implementation of various adaptation measures aimed at preserving cash flow during the COVID-19 pandemic and by cash flow generated by day-to-day operations.

ACTIVITY

▪ Genomic Services Sales growth resulted from an increase in all sequencing activities, in particular the SeqOIA platform (+33%), activity relating to outsourced platforms (+18%), and, finally, services dedicated to laboratories, research and the teams responsible for clinical research (+9%).

▪ Software As a reminder, the company sells interpretive software and consulting services to its customers. The sales of software are growing but showed limited development during the first half of 2020 due to the impact of the COVID-19 pandemic which affect results and planned of projects. In March the company announced that the Dana-Farber Cancer Institute, a leading cancer referral center in the United States, would begin using MERCURYTM, IntegraGen’s proprietary software, to support the efforts to analyze and report tumor sequencing data for patients with cancer.

▪ Impact of COVID-19 The company has fulfilled its commitments to its customers and employees by rigorously adjusting its resources. The team based at the laboratory of Evry and one based at the Institut Pasteur have continued their activities. The operations of SeqOIA laboratory were suspended for a few weeks but now have resumed. The company has set up a continuity plan, including teleworking devices for all IT and sales teams, but also, since March 20th, the company implemented short term working measures for several employees and the deferral of payment for some charges. The company also obtained a $1,800 thousand euros loan guaranteed by the State to Société Générale in order to save cash and reduce risk.

▪ Perspectives 2020: Confirmed growth IntegraGen has succeeded in maintaining high activities for both research services and with the company’s industrial sequencing platform for clinical use over the past several months. The company expects an acceleration of software sales in the coming months and the continuation its sales efforts to implement new and strong partnerships and sustain business with leading research institutes, as developed with the Dana-Farber Cancer Institute, whose collaboration has been in place since March 2020. F

RIENDLY PUBLIC TENDER OFFER OF ONCO DNA On July 9, 2020, OncoDNA SA and IntegraGen SA announced that they had signed an agreement on July 8, 2020 under which the Belgian company OncoDNA filed a draft friendly cash tender offer relating to IntegraGen shares, including securities trading on the Euronext Growth market of Euronext Paris, at a unit price of € 2.20, valuing 100% of IntegraGen securities for €14.5m. The Autorité des Marchés Financiers (AMF) and the Ministry of the Economy in charge of enforcing the regulations applicable to foreign investments in France have approved this offer which was launched as of September 17, 2020.

OncoDNA is a Belgian company who has an international business related to precision medicine for oncology. The company has developed products for clinicians that helps guide the choice of the best treatment for patients with advanced cancers. OncoDNA also provides liquid biopsy monitoring solutions to better track the progression of the disease. The merger between the two companies will, among other things, create a European leader in genomic services, present in France, Belgium and Spain, employing more than 100 individuals.