GlycoMimetics to Participate in Two Upcoming Investor Conferences

On November 8, 2022 GlycoMimetics, Inc. (Nasdaq: GLYC) reported that management will participate in two upcoming investor conferences (Press release, GlycoMimetics, NOV 8, 2022, View Source [SID1234623496]).

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The Stifel 2022 Healthcare Conference on November 15, 2022 – management will attend and host 1×1 investor meetings.

The Jefferies London Healthcare Conference on November 17, 2022 at 10:50 a.m. EST – management will participate in an in-person fireside chat.

An archived recording of the Jefferies fireside chat will be available for 90 days on the GlycoMimetics website at View Source

MEDIGENE PRESENTS NEW DATA ON REPRODUCIBLE PRODUCTION OF TCR-T CELLS FROM MDG1011 PROGRAM

On November 8, 2022 Medigene AG (Medigene, FSE: MDG1, Prime Standard), an immuno-oncology company focusing on the development of differentiated TCR-T therapies for cancers, reported that new data on reproducible production of TCR-T cells from elderly hematologically-challenged patients with blood cancers at the Cell UK Conference taking place November 7-8, 2022 in London (Press release, MediGene, NOV 8, 2022, View Source [SID1234623454]). The presentation can be found on Medigene´s website: View Source

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MDG1011 is an autologous TCR-T therapy specific for a peptide fragment of PRAME (PReferentially expressed Antigen in MElanoma), a tumor antigen presented on cancer cells by human leukocyte antigen HLA-A2. Generation of MDG1011 in the phase I dose escalation trial (NCT03503968) was complicated by numerous factors impacting the potential quality of T cells, including that patients suffered from blood cancers, were heavily pretreated, and were mostly elderly. Leukemia blasts were detected at high levels in the blood of several patients during the leukapheresis process performed to obtain the starting patient-specific T cells for MDG1011 manufacture, which had the potential to hinder the production and the final purity of MDG1011 drug products. Despite this, a success rate of 92% was achieved in production of TCR-T cells for twelve of thirteen patients using a semi-automated modular manufacturing process. Assessment of immune reactivity demonstrated high antitumor activities for all final MDG1011 products, as potential markers of potency.

Generation of MDG1011 and functional activity

Patients underwent leukapheresis and CD8-positive T cells were enriched and cryopreserved for use in generation of MDG1011 drug products. Following thawing and activation, the PRAME-specific T cell receptor was introduced into CD8-positive T cells via retroviral gene transfer. TCR-T cells were expanded and cryopreserved for later shipment to the clinical centers for thawing and application to the patients as a single infusion. The GMP processes of drug product manufacture and quality control, with subsequent immune assessments of thawed MDG1011 products demonstrated:

* Generation of highly-enriched CD8-positive TCR-T cells was successful, despite high variability in the patient leukapheresis starting materials, with no detection of residual blood cancer cells in final MDG1011 products
* TCR-T cells expressing the PRAME-specific TCR were produced in the required numbers needed for a dose escalation phase I study of MDG1011
* MDG1011 showed high viability through all steps of the process, including before and after cryopreservation
* Poly-functional cytokine secretion and killing capacity for tumor cell lines was seen with all MDG1011 products upon antigen-specific stimulation in vitro directly after thawing
* Diverse subsets of T cells were retained during the manufacturing process of MDG1011 products, including stem cell memory T cells, central memory T cells and other effector memory T cells

Overall, highly successful generation of MDG1011 was feasible for hematologically-challenged patients. The TCR-T cells displayed excellent viability after thawing and antigen-specific poly-functional cytokine secretion was observed for all MDG1011 products manufactured from such patients.

Prof. Dolores Schendel, Chief Scientific Officer at Medigene: "The production of functionally potent MDG1011 product is critical to achieve tangible clinical benefit in patient treatment. We are pleased by this achievement with a very high success rate for MDG1011 products developed for elderly patients with relapsed or refractory blood cancers. This robust process yielded high purity, high quality poly-functional CD8-positive TCR-T cells and the lessons learned here with liquid tumors provide important insights as we develop novel TCR-T therapies for patients with solid cancers."

Oncotelic Initiates Clinical Trials Evaluating OT-101 against Pediatric Gliomas

On November 8, 2022 Oncotelic Therapeutics, Inc (OTCQB:OTLC) ("Oncotelic", the "Company" or "We"), a developer of treatments for rare and orphan indications, including Parkinson’s Disease, PDAC, DIPG, and COVID-19, reported that it has submitted a clinical study protocol to the US Food and Drug Administration ("FDA") for the initiation of a Phase 1 Trial (designated "G101") for OT-101, the Company’s transforming growth factor beta 2 ("TGF-β2") inhibitor, as a treatment for patients with recurrent/relapsed DMG (Press release, Oncotelic, NOV 8, 2022, View Source [SID1234623453]).

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G101: An Open-label Dose Escalation Study to Evaluate the Safety and Tolerability of Repeated Cycles of OT-101 in Pediatric Diffuse Midline Glioma ("DMG") Patients, Administered Intraventricularly.

OT-101 is a first-in-class anti-TGF-β2 ribonucleic acid ("RNA") therapeutic that has exhibited single agent activity in relapsed/refractory cancer patients in multiple clinical trials. OT-101 has also demonstrated activity against the COVID-19 virus in our Phase 2 clinical trial- C001.

"Pediatric DMG is a very aggressive brain tumor in children that has a dismal prognosis with a median overall survival of less than one year after standard radiation therapy. Therefore, there is an urgent need for therapeutic innovations for treatment of pediatric DMG", explained Fatih Uckun, MD PhD, the Chief Medical Officer of Oncotelic. "The primary goal of the new study is to carefully evaluate the safety and tolerability of OT-101 when it is administered directly into the cerebral spinal fluid (CSF) of pediatric patients with DMG", he added.

"This is the first of a series of planned clinical trials in pediatric patients with gliomas evaluating clinical benefit while also assessing predictive biomarkers." Dr. Vuong Trieu, CEO and Chairman of Oncotelic. "The groundwork laid down by our successful clinical program in adult gliomas and deep datamining by the team will now help guide the development of OT-101 for pediatric DMG."

About DMG/DIPG

DMG is a highly morbid pediatric central nervous system (CNS) tumor for which there is currently no effective treatment. DMG is responsible for 50% of all childhood high grade glioma ("HGG"). Due to their anatomic location and infiltrative nature, DMGs are not amenable to surgical resection and are most often diagnosed radiographically and treated with radiation therapy, with no effect on survival.

Specifically, 80% of pediatric DMGs harbor somatic mutations in histone H3-encoding genes H3F3A (60%), HIST2H3C or HIST1H3B/C (20%), resulting in lysine-27-to-methionine (H3K27M) conversion that confers a more aggressive clinical course and poorer overall response to therapy. As such, DMG, H3 K27-mutant was first introduced in the 2016 World Health Organization ("WHO") classification of CNS tumors as a newly defined entity and these tumors are WHO grade 4 gliomas associated with local infiltration and a poor prognosis with 2-year survival rate of < 10%, regardless of histological grading. This entity represents the majority of DIPGs as well as tumors found along the midline (e.g., brainstem, midbrain, thalamus, and spine).

About OT-101

OT-101, is a first-in-class anti-TGF-β2 RNA therapeutic that exhibited single agent activity in some relapsed/refractory cancer patients in clinical trial settings. HGGs are characterized by a T-cell exhaustion signature and pronounced T-cell hypo responsiveness of their tumor microenvironment ("TME"). TGF-β2 has been implicated as a key contributor to the immunosuppressive landscape of the TME in HGG. OT-101 is designed to abrogate the immunosuppressive actions of TGF- β2. In a completed Phase 2 clinical study, OT-101 exhibited clinically meaningful single-agent activity and induced durable complete and partial responses in recurrent and refractory adult HGG patients, including young adults with Glioblastoma Multiforme or Amyloidosis.

OT-101 has been granted orphan designation by the FDA under the Orphan Drug Act ("ODA"). ODA provides for granting special status to a drug to treat a rare disease or condition upon request of a drug company. Orphan designation qualifies the sponsor of the drug for various development incentives of the ODA, including tax credits for qualified clinical testing. OT-101 also been granted Rare Pediatric Designation for DIPG. The FDA grants rare pediatric disease designation for diseases with serious or life-threatening manifestations that primarily affect people aged from birth to 18 years, and that affect fewer than 200,000 people in the U.S. Under the FDA’s Rare Pediatric Disease Priority Review Voucher program, a sponsor who receives an approval of a new drug application or biologics license application for a product for the prevention or treatment of a rare pediatric disease may be eligible for a voucher, which can be redeemed to obtain priority review for any subsequent marketing application and may be sold or transferred.

As previously reported, on March 31, 2022, we entered into a joint venture, or JV, with Dragon Overseas Capital Ltd. (Dragon Overseas) and GMP Biotechnology Ltd. (GMP Bio). The JV and Oncotelic will develop and ultimately market OT-101, individually and/or in combination with other products. Oncotelic would receive up to $50 million on sale of the RPD voucher, following marketing approval of OT-101 for diffuse intrinsic pontine glioma, or DIPG, by the US Food and Drug Administration.

Adaptimmune Reports Increased Response Rate and Durability of Response in Its Phase 1 SURPASS trial; ORR Now 52% Across Ovarian, Urothelial, and Head & Neck Cancers

On November 8, 2022 Adaptimmune Therapeutics plc (NASDAQ: ADAP), a leader in cell therapy to treat cancer, reported that updated clinical data from its MAGE-A4 franchise (Press release, Adaptimmune, NOV 8, 2022, View Source [SID1234623452]). Clinical data continue to support the potential of Adaptimmune’s engineered T-cell therapies for people with cancer across multiple solid tumor indications . The company will provide further details on a call to be held today, November 8, 2022, at 8:00 a.m. EST (1:00 p.m. GMT) during which management will be available for Q&A.

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"The data in the SURPASS trial continue to demonstrate the potential of our next-generation cell therapy targeting MAGE-A4 in a broad range of difficult-to-treat, late-stage solid tumors," said Elliot Norry, Adaptimmune’s Chief Medical Officer. "We are initiating a Phase 2 trial, SURPASS-3, for people with ovarian cancer in collaboration with The GOG Foundation, Inc. I am pleased to announce that we recently received FDA RMAT Designation for ADP-A2M4CD8 for the treatment of patients with platinum resistant ovarian cancer. We also plan to gather data in urothelial and head & neck cancers with two additional cohorts that will focus on earlier lines of treatment. Finally, we will evaluate further development opportunities in the ongoing signal finding Phase 1 SURPASS trial in both monotherapy as well as in combination with the checkpoint inhibitor nivolumab."

New positive data reported in the Phase 1 SURPASS trial

Since data were reported at ESMO (Free ESMO Whitepaper) in September, there have been additional clinical responses including a new complete response in urothelial cancer and one new response in ovarian cancer
The objective response rate (ORR) has increased to 52% in heavily pre-treated patients with late -stage ovarian, urothelial, and head & neck cancers after a single dose of ADP-A2M4CD8 (ORR of 44% reported at ESMO (Free ESMO Whitepaper))
The overall ORR has increased to 37% (ORR of 33% reported at ESMO (Free ESMO Whitepaper))
Increased ORR in ovarian cancer to 43% (ORR of 36% reported at ESMO (Free ESMO Whitepaper))
Increased ORR in urothelial cancer to 57% (ORR of 43% reported at ESMO (Free ESMO Whitepaper))
There have been no new responses reported in head & neck cancer since the last update of 3 out of 4 patients responding as presented at ESMO (Free ESMO Whitepaper)
Durability continues to increase in this ongoing trial and is now at a median of ~20 weeks; previously reported as ~12 weeks at ESMO (Free ESMO Whitepaper)
Development plans for the SURPASS family of trials

The Phase 1 signal finding SURPASS trial is ongoing in both a monotherapy and a combination cohort evaluating ADP-A2M4CD8 with nivolumab (a checkpoint inhibitor)
Adaptimmune is working with The GOG Foundation, Inc and has initiated a Phase 2 trial (SURPASS-3) evaluating ADP-A2M4CD8 in both monotherapy and in combination with nivolumab in platinum-resistant ovarian cancer
The Company recently received FDA RMAT Designation for ADP-A2M4CD8 for the treatment of patients with platinum resistant ovarian cancer
The Company plans to pursue two new cohorts in the SURPASS Phase 1 trial with ADP–A2M4CD8
In combination with a checkpoint inhibitor in a second-line setting for advanced urothelial cancer
In combination with a checkpoint inhibitor in the first-line setting for advanced head & neck cancer
Conference Call Information

The Company will host a live teleconference and webcast to provide additional details at 8:00 a.m. EST (1:00 p.m. GMT) today, November 8, 2022. A live webcast of the conference call and replay can be accessed at View Source Call in information is as follows: (800)- 319-4610 (US or Canada) or +1 (416)- 915-3239 (International and additional options available HERE).

Reata Pharmaceuticals, Inc. Announces Third Quarter 2022 Financial Results and Provides an Update on Clinical Development Programs

On November 8, 2022 Reata Pharmaceuticals, Inc. (Nasdaq: RETA) ("Reata," the "Company," "our," "us," or "we"), a clinical-stage biopharmaceutical company, reported financial results for the third quarter of 2022 and provided an update on the Company’s business operations and clinical development programs (Press release, Reata Pharmaceuticals, NOV 8, 2022, View Source [SID1234623451]).

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Recent Company Highlights

Omaveloxolone in Patients with Friedreich’s Ataxia

In May 2022, the U.S. Food and Drug Administration ("FDA") accepted our New Drug Application ("NDA") for filing and granted Priority Review for omaveloxolone for patients with Friedreich’s ataxia. We completed a Mid-Cycle Communication Meeting with the FDA and submitted additional data and analyses to the FDA after the meeting. The FDA determined that these submissions were a major amendment to our NDA and extended the Prescription Drug User Fee Act ("PDUFA") date by three months to provide time for a full review of the new data and analyses. The PDUFA date is now February 28, 2023.

We recently completed a Late-Cycle Meeting with the FDA. The purpose of the Late-Cycle Meeting is for the FDA to discuss any substantive issues identified, and the Division’s objectives for the remainder of the review. The meeting does not address the final regulatory decision for the NDA. While we have not received formal minutes from the FDA, in the preliminary agenda for, and during, the Late-Cycle Meeting, the FDA stated that they continue to review the analyses and data included in the recent NDA submissions. The FDA made no request for additional data or analyses but stated that additional data may be requested as reviews are ongoing. The FDA confirmed that no information requests were outstanding. The FDA reiterated that they do not currently plan to hold an advisory committee meeting.

The FDA stated that no issues related to risk management have been identified to date. During the meeting, the FDA indicated that post-marketing requirements and label review are ongoing. With respect to post-marketing requirements and commitments, FDA stated that if omaveloxolone is approved, they anticipate requiring a drug-drug interaction trial with CYP3A4 modulators, a thorough QT trial, and an evaluation of pregnancy outcomes. FDA stated that other post-marketing requirements and commitments may be considered depending on the findings of the review. With respect to label review, during the meeting we noted that the original proposed label language did not reflect the data and analyses included in the amendments to the NDA and that we have updated it in connection with the planned filing of our Marketing Authorization Application ("MAA") in Europe later this year. We committed to submit the updated proposed label language to the NDA. The FDA indicated that post-marketing requirements and label comments will be communicated in early in 2023.

We have advanced our commercial launch preparations in the United States and are building the infrastructure necessary to support the commercialization of omaveloxolone for the treatment of Friedreich’s ataxia, if and when we receive regulatory approval. We have designed our patient access programs and our product distribution network. The payer field team has been hired and deployed. Hiring of the sales leadership team is underway and we intend to onboard our sales organization and reimbursement specialists in the first quarter of 2023, pending regulatory advancement.

We plan to submit an MAA to the European Medicines Agency ("EMA") for omaveloxolone this year.

Third Quarter Financial Highlights

Cash and Cash Equivalents

On September 30, 2022, we had cash and cash equivalents and marketable securities of $435.9 million, as compared to $590.3 million of cash and cash equivalents on December 31, 2021.

GAAP and Non-GAAP Research and Development ("R&D") Expenses

R&D expenses according to generally accepted accounting principles in the U.S. ("GAAP") were $43.5 million for the third quarter of 2022, as compared to $39.4 million for the same period of the year prior.

Non-GAAP R&D expenses were $36.8 million for the third quarter of 2022, as compared to $34.0 million, for the same period of the year prior.1

GAAP and Non-GAAP General and Administrative ("G&A") Expenses

GAAP G&A expenses were $27.3 million for the third quarter of 2022, as compared to $25.7 million, for the same period of the year prior.

Non-GAAP G&A expenses were $19.5 million for the third quarter of 2022, as compared to $17.5 million for the same period of the year prior.1

GAAP and Non-GAAP Net Loss

The GAAP net loss for the third quarter of 2022, was $79.0 million, or $2.16 per share, on both a basic and diluted basis, as compared to a GAAP net loss of $71.8 million, or $1.97 per share, on both a basic and diluted basis, for the same period of the year prior.

The non-GAAP net loss for the third quarter of 2022, was $53.9 million, or $1.47 per share on both a basic and diluted basis, as compared to a non-GAAP net loss of $46.2 million, or $1.27 per share, on both a basic and diluted basis, for the same period of the year prior.1

[1]See "Non-GAAP Financial Measures" below for a description of non-GAAP financial measures and a reconciliation between GAAP and non-GAAP R&D expenses, GAAP and non-GAAP G&A expenses, and GAAP and non-GAAP net loss, respectively, appearing later in the press release.

Cash Guidance

The Company reaffirms that its existing cash and cash equivalents and marketable debt securities will be sufficient to enable it to fund operations through the end of 2024.

Non-GAAP Financial Measures

This press release contains non-GAAP financial measures, including non-GAAP R&D expenses, non-GAAP G&A expenses, non-GAAP operating expenses, non-GAAP net loss and non-GAAP net loss per common share – basic and diluted. These measures are not in accordance with, or an alternative to, GAAP, and may be different from non-GAAP financial measures used by other companies.

The Company defines non-GAAP R&D expenses as GAAP R&D expenses, excluding stock-based compensation expense; non-GAAP G&A expenses as GAAP G&A expenses, excluding stock-based compensation expense; non-GAAP operating expenses as GAAP operating expenses, excluding stock-based compensation expense; non-GAAP net loss as GAAP net loss, excluding stock-based compensation expense and non-cash interest expense from liability related to sale of future royalties; and non-GAAP net loss per common share – basic and diluted as GAAP net loss per common share – basic and diluted, excluding stock-based compensation expense and non-cash interest expense from liability related to sale of future royalties. The Company has excluded the impact of stock-based compensation expense, which may fluctuate from period to period based on factors including the variability associated with performance-based grants of stock options and restricted stock units and changes in the Company’s stock price, which impact the fair value of these awards. The Company has excluded the impact of accreted non-cash interest expense from liability related to sale of future royalties as it may be calculated differently from, and therefore may not be comparable to, peer companies who also provide non-GAAP disclosures. The Company has excluded the impact of stock-based compensation expense and non-cash interest expense from liability related to sale of future royalties because the Company believes its impact makes it difficult to compare its results to prior periods and anticipated future periods.

Because management believes certain items, such as stock-based compensation expense and non-cash interest expense from liability related to sales of future royalties, can distort the trends associated with the Company’s ongoing performance, the following measures are often provided, excluding special items, and utilized by the Company’s management, analysts, and investors to enhance consistency and comparability of year-over-year results, as well as to industry trends, and to provide a basis for evaluating operating results in future periods: non-GAAP net loss; non-GAAP net loss per common share – basic and diluted; non-GAAP R&D expenses; non-GAAP G&A expenses; and non-GAAP operating expenses.

The Company believes the presentation of these non-GAAP financial measures provides useful information to management and investors regarding the Company’s financial condition and results of operations. When GAAP financial measures are viewed in conjunction with these non-GAAP financial measures, investors are provided with a more meaningful understanding of the Company’s ongoing operating performance and are better able to compare the Company’s performance between periods. In addition, these non-GAAP financial measures are among those indicators the Company uses as a basis for evaluating performance, allocating resources, and planning and forecasting future periods. These non-GAAP financial measures are not intended to be considered in isolation or as a substitute for GAAP financial measures. A reconciliation between these non-GAAP measures and the most directly comparable GAAP measures is provided later in this press release.

Conference Call Information

Reata’s management will host a conference call on November 8, 2022, at 8:30 am ET. The conference call will be accessible by dialing (844) 200-6205 (toll-free domestic) or (929) 526-1599 (international) using access code 756839. The webcast link is View Source

Third quarter 2022 financial results to be discussed during the call will be available on the Company’s website shortly before the call at View Source and will be available for 12 months after the call. The audio recording and webcast of the conference call will be accessible for at least 90 days after the event at View Source.