On August 9, 2021 Race Oncology Limited ("Race") reported that the first patient has been dosed in the Phase 1b/2 trial in relapsed/refractory Acute Myeloid Leukaemia (ASX Announcement: 22 June 2021) (Press release, Race Oncology, AUG 9, 2021, View Source [SID1234591494]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

This investigator-led trial supervised by Professor Arnon Nagler will use Zantrene (bisantrene dihydrochloride) in a novel three drug combination which has demonstrated compelling efficacy in pre-clinical studies (ASX Announcement: 10 May 2021). Prof Nagler was the Principal Investigator of the Phase 2 investigator-initiated trial where Zantrene was used as a single agent in R/R AML patients and reported an impressive 40% clinical response rate (ASX Announcement: 16 June 2020).

"We are delighted to see the start of this important clinical project which uses a novel combination approach for relapsed or refractory Acute Myeloid Leukaemia. This study is also an important step in our journey towards approval of Zantrene in this area of high unmet medical need."

Race CMO Dr David Fuller
The trial will run in parallel with a separate Australian Phase 2 trial in patients with extramedullary AML (ASX Announcement: 2 June 2021).

Both trials are key components of Race’s Three Pillar strategy.

Even when CR is achieved through intense chemotherapy, approximately half of the younger and 80% of the older patients, relapse. In both clinical situations, refractory and/or relapsed AML, active disease remains a major therapeutic challenge despite recent advances in the clinic.

"This study led by Professor Nagler who has good experience with Zantrene, supports the building of additional data in AML in line with our Three pillar strategy plan. We hope to see improved patient outcomes in what has been historically a difficult to treat disease. We plan on using our trademarked name, Zantrene, in referring to bisantrene dihydrochloride. It’s one of our registered trademarks and its protection is enhanced by its ongoing and appropriate use."

Race CEO & MD Phillip Lynch
Relapsed or Refractory Acute Myeloid Leukemia
Primary refractory or relapsed Acute Myeloid Leukemia is associated with poor prognosis and remains a serious therapeutic challenge. Primary refractory AML is defined by the absence of complete remission (CR), manifested by blast count of ≥5% in bone marrow after one or two cycles of intense induction chemotherapy.

Up to 30% of adults with newly diagnosed AML fail to achieve CR after two courses of intensive chemotherapy.

Clinical Trial Design
The trial is an open-label, Phase 1b/2 study of intravenous FluCloZan (Fludarabine, Clofarabine, Zantrene) in cohorts of up to 12 adult patients with R/R AML with a Phase 1b dose escalation stage to establish the maximum tolerated dose (MTD) or recommended Phase 2 dose of the combined FluCloZan regimen, followed by a Phase 2 expansion stage to determine efficacy and confirm safety of FluCloZan at the recommended Phase 2 dose in up to 17 patients.

Phase 1b, Dose-Escalation (Lead-in Stage)
A two-cohort dose escalation schema using a standard 3 + 3 design will be employed.

Cohort 1 will enroll three patients to receive the FluCloZan regimen for four consecutive days. If no dose limiting toxicities (DLTs) have occurred in the first three patients by day 30 of their first cycle of treatment, then Cohort 2 will receive the treatment for five days (with the extra day representing dose escalation).

Phase 2, Expansion (Efficacy Stage)
Up to 17 patients will be enrolled into a Phase 2 expansion efficacy cohort using a 2-stage Simon design. Initially, 9 patients will be enrolled and treated with the recommended Phase 2 dose of FluCloZan as determined in the Phase 1b part of the study. If there are no patient responses in the first nine subjects according to the response criteria outlined in the European Leukemia Net (ELN) guidelines, the study will be terminated for futility. If at least one patient shows a response, eight more patients will be enrolled and treated. If three or more of the patients treated in Stage 2 respond, the null hypothesis of treatment futility can be rejected.

Efficacy assessments will be based on bone marrow examination at a minimum of two time points on Day 21 and on Day 30. A further bone marrow examination may be performed on Day 42 at the investigator’s discretion, based on patient’s disease and performance status and/or on peripheral blood hematology results during the treatment course and between Day 21 to 42.

Treatment will be terminated upon any sign of progressive/recurrent disease and/or referral to pre-transplant conditioning therapy for (allogeneic) stem cell transplantation.

Patients who do not progress or experience any DLTs may receive a second course of treatment for the same duration as in their first cycle.

All patients will be actively followed-up every three months for a further 12 months following completion treatment for disease free survival (DFS) and overall survival (OS).

Indicative Costs and Timelines
The trial is expected to take 36 to 40 months to complete with full patient recruitment over approximately 18 months. Given the trial is open-label, Race expects that data will be reported at interim points throughout the trial.

Race will pay Chaim Sheba a total fee of USD $668,739 over the study’s life. Payments will be made to Chaim Sheba upon reaching key milestones and the total trial cost will depend on the number of patients recruited and other operational variables.

Clinical Trial Summary
Study Title An Open-label, Phase Ib/II, Two-stage, Study of Zantrene (Bisantrene) in combination with Fludarabine and Clofarabine as Salvage Therapy for Adult Patients with Relapsed or Refractory Acute Myeloid Leukaemia (AML)
Registration NCT04989335
Phase of Development Phase 1b/2
Active Ingredient Bisantrene dihydrochloride, Fludarabine, Clofarabine (FluCloZan)
Study Description Phase 1b/2 study of FluCloZan, IV, in cohorts of adult patients with R/R AML using a 2-stage design: a Phase 1b lead-in dose escalation stage to establish the MTD or RP2D of FluCloZan and a Phase 2 expansion stage to determine efficacy and confirm safety of the FluCloZan regimen at the RP2D.
Principal Investigator Professor Arnon Nagler
Sponsor Race Oncology
Indication/population Adult men and women 18 to 65 years of age with relapsed and/ or refractory Acute Myeloid Leukemia (R/R AML) presenting with non-CNS extramedullary disease.
Number of Subjects Phase 1b: up to 12 patients Phase 2: up to 17 patients in the expansion phase
Study Period 36 – 40 months
Study Design A two-cohort dose escalation schema using a standard 3 + 3 design will be employed followed by an expansion phase at the RP2D. As the patient population is considered relapsed and/or refractory to existing treatments, a comparator arm will not be used.
Statistical methods Simon 2 stage design
End Points Primary
Phase 1b Dose Escalation: number of subjects experiencing a DLT in each cohort Phase 2 Expansion: Overall Response Rate (ORR) defined as the proportion of subjects with CR and CRi between Day 30 to Day 42 Secondary: Transplant/allo-HSCT rates (for transplant/allo-HSCT-eligible subjects); Combined CR and CRi and PR response rate; Morphologic leukemia-free state (MLFS); Partial remission (PR); Stable disease (SD); Progressive disease (PD); Relapse; Disease free survival (DFS); Overall survival (OS); Time to next treatment (for transplant/allo-HSCT-ineligible subjects)
Participating Centres 1 Chaim Sheba Medical Center, Tel Hashomer, Israel
Dates First patient August 6, 2021; Last patient (anticipated): Q3 CY2023

On August 9, 2021 Sareum Holdings plc (AIM: SAR), the specialist drug development company, reported that it has raised £1,000,000, before expenses, through a subscription by a high net worth individual (the "Subscriber") for 12,121,212 new ordinary shares of 0.025p each in the capital of the Company ("Ordinary Shares") (the "Subscription Shares") at a price of 8.25p per share (the "Subscription Price") (the "Subscription") (Press release, Sareum, AUG 9, 2021, View Source [SID1234586711]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Under the terms of the Subscription, the new Subscriber will also be issued one and a quarter five-year warrants, exercisable at the Subscription Price, for every Subscription Share issued (the "Subscription Warrants"), which can only be exercised following the Company’s closing middle market share price being above 10.25p per Ordinary Share for five consecutive days. The Subscription Price represents a discount of approximately 6.8 per cent. to the closing middle market price for Sareum shares on 6 August 2021.

The net proceeds from the Subscription will be used to progress the Company’s SDC-1801 and SDC-1802 TYK2/JAK1 inhibitor drug development programmes as well as for working capital purposes. As noted in the Company’s Trading Update of 25 May 2021, the Company is targeting the filing of a Clinical Trials Approval for SDC-1801 in Q4 2021, subject to successful progress and no further delays with the final preclinical studies. Clinical trial plans, including priority autoimmune indications and potential Covid-19 application, will also be developed in parallel, subject to additional funding being raised.

Application will be made for the 12,121,212 Subscription Shares, which will rank pari passu with the Company’s existing Ordinary Shares, to be admitted to trading on the AIM market of the London Stock Exchange ("AIM") ("Admission"). It is anticipated that Admission will become effective at 8.00 am on 17 August 2021. The Subscription is subject to normal conditions including, inter alia, Admission.

Total Voting Rights

For the purpose of the Disclosure Guidance and Transparency Rules, following the above issue of equity, the issued share capital of the Company will comprise 3,365,701,148 Ordinary Shares. The above figure may be used by shareholders as the denominator for the calculations by which they will determine if they are required to notify their interest in, or a change to their interest in, the Company, under the Disclosure Guidance and Transparency Rules.

Dr Tim Mitchell, CEO of Sareum Holdings plc, said: "This new subscription is Sareum’s fourth since 1 June this year, reflecting the strong and continuing interest in our proprietary TYK2/JAK1 development programmes in autoimmune diseases, including the immune overreaction to Covid-19, and cancer. With the funds raised during this period now totalling over £4.5 million, our financial position has been significantly strengthened and we are looking forward to completing the preclinical studies with SDC-1801 and advancing it into clinical development, and to progressing the preclinical development of our second TYK2/JAK1 inhibitor SDC-1802."

The information contained within this announcement is deemed by the Company to constitute inside information as stipulated under the Market Abuse Regulation (EU) No. 596/2014 as it forms part of United Kingdom domestic law by virtue of the European Union (Withdrawal) Act 2018.

On August 9, 2021 QIAGEN (NYSE:QGEN; Frankfurt Prime Standard: QIA) and OncXerna Therapeutics, Inc. ("OncXerna"), a precision medicine company using an innovative RNA-expression based biomarker platform to predict patient responses to its targeted oncology therapeutic candidates, reported signing a master companion diagnostics (CDx) agreement to develop a NGS CDx for OncXerna’s product candidate, Navicixizumab, and a non-exclusive license to the Xerna TME panel (Press release, Qiagen, AUG 9, 2021, View Source [SID1234586597]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

QIAGEN and OncXerna have agreed to collaborate to advance the Xerna TME panel towards IVD (in-vitro diagnostic) regulatory approval as a NGS companion diagnostic for Navicixizumab, which is being developed by OncXerna as a treatment for patients with ovarian cancer. The diagnostic will be used to determine if patients with ovarian cancer whose dominant tumor biology is driven by angiogenesis are more likely to benefit from treatment with Navicixizumab.

The Xerna TME panel uses proprietary RNA-based gene expression data and a machine learning-based algorithm to classify patients based on the dominant biology of their cancer so that patients can be matched with therapies that directly address these biologies. Pursuant to the non-exclusive license, which is for research use only ("RUO") and clinical development programs, OncXerna has granted QIAGEN the rights to integrate the Xerna TME panel into their NGS workflow solutions, which broadens QIAGEN’s NGS CDx custom panel development capabilities.

"We are very pleased to complete these agreements with QIAGEN, which we see as providing important external validation for the Xerna TME panel and our broader RNA-based biomarker platform," stated Dr. Laura Benjamin, OncXerna Founder and CEO. "As an NGS industry-leader in precision medicine diagnostics with impressive global development, manufacturing and commercial capabilities, we believe QIAGEN is uniquely positioned to help advance the Xerna TME panel towards regulatory approval as an NGS companion diagnostic and, if approved, drive the panel’s adoption. Moreover, integration of the Xerna TME Panel into their workflow solutions could enable QIAGEN to provide a new RNA-based offering to strengthen their overall immune oncology solutions for biopharma customers."

"We believe that the Xerna TME panel that we can now offer our customers will further enhance our strong portfolio in companion diagnostics. Through this agreement, we aim to foster additional NGS-based collaborations with pharmaceutical companies for the development of drug treatments for immune oncology and promote early clinical adoption of precision medicine diagnostics such as our therascreen portfolio and the Xerna TME panel," said Jonathan Arnold, Vice President, Head of Oncology and Precision Diagnostics at QIAGEN. "We are also thrilled to have the master CDx agreement in place and look forward to working with OncXerna to develop an NGS companion diagnostic for Navicixizumab based on our extensive track record with the development of companion diagnostics for a variety of cancers."

QIAGEN is a pioneer in Precision Medicine and the global leader in collaborations with pharmaceutical and biotechnology companies to co-develop companion diagnostics, which detect clinically relevant genetic abnormalities to provide insights that guide clinical decision-making in diseases such as cancer. QIAGEN has an unmatched depth and breadth of technologies from next-generation sequencing (NGS) to polymerase chain reaction (PCR) for companion diagnostic development. QIAGEN has ten PCR based companion diagnostic indications that are FDA approved, including therascreen EGFR for non-small cell lung cancer, therascreen KRAS for colorectal cancer, therascreen FGFR for urothelial cancer, therascreen PIK3CA for breast cancer based on tissue or plasma samples and the therascreen BRAF kit for colorectal cancer.

Currently, QIAGEN is working under master collaboration agreements with more than 25 companies to develop and commercialize companion diagnostic tests for their drug candidates – a deep pipeline of potential future products to advance Precision Medicine for the benefit of patients.

About OncXerna Therapeutics, its Xerna RNA-based Biomarker Platform, and Xerna TME Panel

OncXerna is a precision medicine company using an innovative RNA-expression based biomarker platform to predict patient responses to its targeted oncology therapeutic candidates. OncXerna is working to expand next-generation precision medicine to a larger group of cancer patients by leveraging the company’s Xerna platform to prospectively identify patients based on the dominant biology of their cancer. OncXerna’s approach pairs those patients with OncXerna’s clinical-stage therapeutic candidates and known mechanism of action that directly address these biologies, with the goal to substantially improve patient outcomes. The Xerna TME Panel uses proprietary RNA-based gene expression data and a machine learning-based algorithm to classify patients based on the interplay between angiogenic and immunogenic dominant biologies of the tumor microenvironment (TME), and has been developed as a clinical assay. The Xerna TME Panel is an investigational assay that has not been approved, and has not been demonstrated to be safe or effective for any use.

About Navicixizumab

Navicixizumab is an anti-DLL4/VEGF bispecific antibody product candidate that demonstrated antitumor activity in patients who were previously treated with Avastin (bevacizumab) in a Phase 1b clinical trial. The U.S. Food and Drug Administration granted Fast Track designation to navicixizumab for the treatment of high-grade ovarian, primary peritoneal, or fallopian tube cancer in patients who have received at least three prior therapies and/or prior treatment with Avastin. Navicixizumab is an investigational agent that has not been approved, and it has not been demonstrated to be safe or effective for any use, including for the treatment of advanced ovarian cancer.

On August 9, 2021 Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, reported that management will participate in a fireside chat at the 2021 Wedbush PacGrow Healthcare Conference on Wednesday, August 11th at 2:55pm Eastern Time (Press release, Sangamo Therapeutics, AUG 9, 2021, View Source [SID1234586252]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The virtual session will be webcast live and may be accessed on the Sangamo Therapeutics website in the Investors and Media section under Events and Presentations. The presentation will be available on the Sangamo Therapeutics website after the event.

On August 9, 2021 Reata Pharmaceuticals, Inc. (Nasdaq: RETA) ("Reata," the "Company," "our," "us," or "we"), a clinical-stage biopharmaceutical company, reported financial results for the quarter ended June 30, 2021, and provided an update on the Company’s business operations and clinical development programs (Press release, Reata Pharmaceuticals, AUG 9, 2021, View Source [SID1234586250]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Recent Company Highlights

Omaveloxolone in Patients with Friedreich’s Ataxia ("FA")

Based on a communication received from the U.S. Food and Drug Administration ("FDA") regarding omaveloxolone for the treatment of FA, we withdrew our request for a Type C meeting and requested a pre-NDA meeting with the FDA. The pre-NDA meeting request has been granted, a pre-NDA meeting has been scheduled during the third quarter of this year, and we have submitted briefing materials for the meeting. We recently received a communication from the FDA requesting the estimated date of our New Drug Application ("NDA") for its planning purposes. We plan to submit the NDA during the first quarter of 2022.

Bardoxolone Methyl ("Bardoxolone") in Patients with Alport Syndrome

The NDA for bardoxolone for the treatment of patients with chronic kidney disease ("CKD") caused by Alport syndrome is currently under review by the FDA. The FDA completed a bio-research monitoring inspection of Reata. We did not receive any observations. We also recently completed a mid-cycle communication meeting with the FDA. While we have not yet received formal minutes from the FDA, in the preliminary agenda for, and during, the meeting, the FDA identified four significant clinical and statistical review issues for us to address. The FDA invited us to respond to its identified issues in follow-up submissions to the NDA, and we believe each of the identified issues is addressable with additional data and analyses. The FDA did not designate any safety issues as significant issues, and it stated that, based on its current review, it does not believe a Risk Evaluation and Mitigation Strategies ("REMS") program is needed. The FDA also advised us that an Advisory Committee meeting is tentatively scheduled for December 8, 2021. The Prescription Drug User Fee Act ("PDUFA") date, the FDA action date for the application, is scheduled for February 25, 2022.

We reaffirm our plan to submit a Marketing Authorization Application ("MAA") with the European Medicines Agency ("EMA") in the fourth quarter of 2021 for marketing approval of bardoxolone for the treatment of CKD caused by Alport syndrome in the European Union.

On July 27, 2021, Kyowa Kirin Co., Ltd. ("KKC"), our strategic collaborator in CKD in Japan, submitted an NDA in Japan to the Ministry of Health, Labour and Welfare ("MHLW") for bardoxolone for the treatment of patients with CKD caused by Alport syndrome. Based on this submission, we have earned a milestone payment under the KKC license agreement.

Bardoxolone in Patients with Autosomal Dominant Polycystic Kidney Disease ("ADPKD")

FALCON is an international, multi-center, randomized, double-blind, placebo-controlled, registrational Phase 3 trial studying the safety and efficacy of bardoxolone in patients with ADPKD randomized one-to-one to bardoxolone or placebo. We recently had a Type B meeting with the FDA regarding the ADPKD development program, and we have not yet received minutes from the meeting. Based on the discussion during the meeting, we plan to modify the protocol so that the primary endpoint will be the Year 2 off-treatment analysis. Additionally, we will not unblind the trial until after its completion, we will add an eight-week off-treatment visit to the study, and we will incorporate the FDA’s feedback on data related to patients who discontinued treatment early in the study. We may need to increase the patient enrollment sample size from the current target of 550 patients. More than 370 patients are currently enrolled in the study. We continue to expect to enroll 550 patients in the FALCON study by the end of 2021. However, if we decide to increase the target enrollment, we will provide updated guidance on our enrollment timeline.

Bardoxolone in Patients with CKD at Risk of Rapid Progression

MERLIN is a multi-center, double-blind, placebo-controlled, Phase 2 trial to evaluate the safety and efficacy of bardoxolone in patients with CKD due to multiple etiologies at meaningful risk of progression to end-stage kidney disease. We have completed enrollment in the MERLIN trial and expect to have top-line data in the fourth quarter of 2021. If the results of this study are positive, we may proceed to a larger Phase 3 with similar eligibility criteria.

Recent Presentations

Abstracts highlighting results from our various programs in CKD and FA have been selected for presentation at recent international medical conferences. Posters that have been presented can be found on our website at View Source." target="_blank" title="View Source." rel="nofollow">View Source

Dr. David Lynch, MD, PhD, Director, Friedreich’s Ataxia Program, Division of Neurology, Children’s Hospital of Philadelphia, Philadelphia, PA, presented the talk Efficacy of Omaveloxolone in Patients with Friedreich’s Ataxia: Baseline-Controlled Study at the National Ataxia Foundation’s Ataxia Investigators Meeting 2021, which was held virtually from May 24 – 27, 2021.
Dr. Bradley A. Warady, MD, Director, Division of Pediatric Nephrology, Children’s Mercy Kansas City, Kansas City, MO presented the talk Safety of Bardoxolone Methyl in Pediatric Patients with Alport Syndrome in CARDINAL Phase 3 Trial at the 58th ERA-EDTA Congress, which was held virtually from June 5 – 8, 2021.
Dr. Arlene Chapman, MD, Professor of Medicine, University of Chicago, Chicago, IL, presented the poster Trial Design for Phase 3 FALCON: Evaluation of the Safety, Tolerability, and Efficacy of Bardoxolone Methyl in Patients with Autosomal Dominant Polycystic Kidney Disease at the PKD CON:NECT Conference, which was held virtually from June 25 – 26, 2021.
Second Quarter Financial Highlights

Cash and Cash Equivalents

At June 30, 2021, we had cash and cash equivalents of $755.7 million. The decrease in cash and cash equivalents during the second quarter of 2021 was $21.9 million, as compared to $40.5 million in the first quarter of 2021. This decrease in cash used is primarily due to a $22.9 million tax refund related to the Coronavirus Aid, Relief and Economic Security Act that was received during the second quarter of 2021. We reaffirm our current cash runway to last through mid-2024.

Collaboration Revenue

Collaboration revenue was $2.2 million in the second quarter of 2021, as compared to $3.1 million for the same period of the year prior.

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 $40.1 million for the second quarter of 2021, as compared to $36.8 million, for the same period of the year prior.

Non-GAAP R&D expenses were $34.8 million for the second quarter of 2021, as compared to $29.3 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 $22.0 million for the second quarter of 2021, as compared to $16.6 million, for the same period of the year prior.

Non-GAAP G&A expenses were $14.0 million for the second quarter of 2021, as compared to $9.3 million for the same period of the year prior.1

GAAP and Non-GAAP Net Loss

The GAAP net loss for the second quarter of 2021 was $72.7 million, or $2.00 per share, on both a basic and diluted basis, as compared to a GAAP net loss of $67.6 million, or $2.03 per share, on both a basic and diluted basis, for the same period of the year prior.

The non-GAAP net loss for second quarter of 2021 was $48.0 million, or $1.32 per share on both a basic and diluted basis, as compared to a non-GAAP net loss of $40.9 million, or $1.23 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.

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 increases in GAAP and non-GAAP net loss are driven primarily by increased clinical study and manufacturing activities, commercial launch readiness activities, and increased personnel and personnel-related costs to support the growth of our development activities compared to the same period of the year prior.

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, non-cash interest expense from liability related to sale of future royalties, loss on extinguishment of debt, and gain on lease termination; 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, non-cash interest expense from liability related to sale of future royalties, and loss on extinguishment of debt. 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 for stock options and restricted stock units and changes in the Company’s stock price, which impacts 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 loss on extinguishment of debt and gain on lease termination as they are non-recurring transactions that make it difficult to compare its results to peer companies who also provide non-GAAP disclosures. The Company has excluded the impact of stock-based compensation expense, non-cash interest expense from liability related to sale of future royalties, loss on extinguishment of debt, and gain on lease termination 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, non-cash interest expense from liability related to sales of future royalties, loss on extinguishment of debt, and gain on lease termination, 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 August 9, 2021, at 4:30 pm ET. The conference call will be accessible by dialing (866) 270-1533 (toll-free domestic) or (412) 317-0797 (international) using the access code: 10157197. The webcast link is View Source

Second quarter financial results to be discussed during the call will be included in an earnings press release that 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 will be accessible for at least 90 days after the event at View Source." target="_blank" title="View Source." rel="nofollow">View Source