On November 1, 2022 Atara Biotherapeutics, Inc. (Nasdaq: ATRA), a leader in T-cell immunotherapy, leveraging its novel allogeneic Epstein-Barr virus (EBV) T-cell platform to develop transformative therapies for patients with cancer and autoimmune diseases, reported the Company will release third quarter 2022 financial results after market close on Tuesday, November 8, 2022 (Press release, Atara Biotherapeutics, NOV 1, 2022, View Source [SID1234622691]). Following the release, the Company will host a live conference call and webcast at 4:30 p.m. EST to discuss the Company’s financial results and provide a corporate update.

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Analysts and investors can participate in the conference call by dialing 877-407-8291 for domestic callers and 201-689-8345 for international callers, using the conference ID 13733805. A live audio webcast can be accessed by visiting the Investors & Media – News & Events section of atarabio.com. An archived replay will be available on the Company’s website for 30 days following the live webcast.

On November 1, 2022 Aptose Biosciences Inc. ("Aptose" or the "Company") (NASDAQ: APTO, TSX: APS), a clinical-stage precision oncology company developing highly differentiated oral kinase inhibitors to treat hematologic malignancies, reported financial results for the three and nine-month periods ended September 30, 2022 and provided a corporate update (Press release, Aptose Biosciences, NOV 1, 2022, View Source [SID1234622690]).

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The net loss for the quarter ended September 30, 2022 was $9.8 million ($0.11 per share) compared with $11.3 million ($0.13 per share) for the quarter ended September 30, 2021. The net loss for the nine months ended September 30, 2022 was $31.8 million compared with $41.0 million for the nine months ended September 30, 2021. Total cash and cash equivalents and investments as of September 30, 2022 were $55.4 million. Based on current operations, Aptose expects that cash on hand and available capital provide the Company with sufficient resources to fund planned Company operations including research and development into the first quarter of 2024.

"Aptose assumed responsibility for clinical development of HM43239, Aptose’s well differentiated lead agent now known as tuspetinib, just over 10 months ago, and the execution by our clinical team in that period has been exceptional. We completed the dose escalation and exploration stages of our Phase 1/2 trial having demonstrated not only a superior safety package, but also potent antileukemic activity in multiple mutationally defined populations of extremely challenging relapsed or refractory AML patients that had failed prior treatments," said William G. Rice, Ph.D., Chairman, President and Chief Executive Officer. "We continue to see meaningful responses across dose levels and look forward to providing the full scope of blast reduction and response rate data from the Phase 1/2 trial in the ASH (Free ASH Whitepaper) timeframe next month."

Key Corporate Highlights

"Tuspetinib" adopted as generic name for HM43239 – The United States Adopted Name (USAN) Council recently adopted "tuspetinib" as the generic name for Aptose’s lead drug candidate HM43239, an oral, myeloid kinase inhibitor. Aptose will use "tuspetinib" for all future references of the drug, including in scientific publications and corporate materials. The USAN information on tuspetinib will be posted on the USAN Web site (www.ama-assn.org/go/usan).

Tuspetinib Successfully Completes Dose Escalation and Dose Exploration in Phase 1/2 Trial; To Begin Recruitment of Enriched Patient Populations for Expansion Trial – Aptose has completed Phase 1/2 dose escalation and dose exploration of tuspetinib (formerly HM43239), an oral, myeloid kinase inhibitor in an international Phase 1/2 trial in patients with relapsed or refractory (r/r) acute myeloid leukemia (AML) and will begin recruitment for the expansion trial. The company has identified a safe therapeutic range with a broad therapeutic window, spanning the dose levels of 80, 120 and 160 milligrams, and has selected 120 milligrams as the initiating single agent expansion dose and 80mg as the initiating dose selected for combination with venetoclax. The trial is designed to confirm activity through patient enrichment of specific mutationally defined AML populations, including FLT3-mutant patients who have been failed by a prior FLT3 inhibitor, as supported by fast-track designation and significant response rate to date. In the FLT3-mutated group, tuspetinib demonstrated complete remissions in patients that have co-mutations in important genes including NPM1, NRAS, KRAS, PTPN11, DNMT3A, RUNX1, and MLL-PTD. Some of these mutations are typically associated with resistance to tyrosine kinase inhibitors, yet the patients are still able to respond to tuspetinib. Significant activity, including complete remissions and blast reductions, also have been observed in other diverse AML populations, including in FLT3 wildtype patients that harbor adverse mutations in genes like TP53 and NRAS. Current plans include an expansion trial of tuspetinib as a single agent and in combination with venetoclax in r/r AML patients, with planned segue into registrational trials for accelerated approval in subpopulations of r/r AML patients with high unmet medical needs.

Luxeptinib "G3" Formulation Significantly Boosts Oral Availability; Aptose to Proceed with Continuous Dosing – In September, Aptose announced that the G3 formulation of luxeptinib, oral, dual lymphoid and myeloid kinase inhibitor, demonstrated an approximate 18-fold improvement in oral bioavailability relative to the original G1 formulation in testing as a single dose. To date, G3 has been tested as a single dose with dose levels ranging from 10mg to 200mg in 20 patients during ongoing clinical trials. Initial computational modeling of the pharmacokinetic (PK) properties of G3 predicts that plasma steady-state exposure achieved with continuous dosing of 50 mg of G3 (every 12 hours, Q12h) is roughly equivalent to that of 900 mg of G1 Q12h, representing up to an 18-fold improvement in bioavailability with G3. The new G3 formulation could lead to greater absorption and higher steady-state exposure levels. Aptose has amended the protocol of its existing Phase 1 a/b clinical program to incorporate continuous dosing and dose escalation of G3 into the trial and submitted it to the FDA and expects to commence continuous dosing with G3 in patients shortly.

Net loss for the three-month period ended September 30, 2022 decreased by $1.6 million to $9.8 million, as compared to $11.3 million for the comparable period in 2021. The net loss for the nine-month period ended September 30, 2022 decreased by $9.2 million to $31.8 million, as compared to $41.0 million for the comparable period in 2021. Components of net loss are presented below:

Research and Development

Research and development expenses consist primarily of costs incurred related to the research and development of our product candidates and include:

External research and development expenses incurred under agreements with third parties, such as contract research organizations, consultants, members of our scientific advisory boards, external labs and contract manufacturing organizations; and

Employee-related expenses, including salaries, benefits, travel, and stock-based compensation for personnel directly supporting our clinical trials, manufacturing and development activities.
We have ongoing Phase 1 clinical trials for our product candidates tuspetinib and luxeptinib. Tuspetinib was licensed to Aptose in the fourth quarter of 2021, and we assumed sponsorship, and the related costs, of the tuspetinib study effective January 1, 2022. In the fourth quarter of 2021, we discontinued the APTO-253 program and are exploring strategic alternatives for this compound.

We expect our research and development expenses to be higher than current period expenses for the foreseeable future as we advance tuspetinib into larger clinical trials.

The research and development expenses for the three-month and nine-month periods ended September 30, 2022, and 2021 were as follows:

Research and development expenses decreased by $1.1 million to $6.6 million for the three-month period ended September 30, 2022, as compared to $7.7 million for the comparative period in 2021. Changes to the components of our research and development expenses presented in the table above are primarily as a result of the following events:

Program costs for tuspetinib were $3.0 million for the three-month period ended September 30, 2022. The Company in-licensed the development rights of tuspetinib in the fourth quarter of 2021 and assumed sponsorship, and the related costs, of the study effective January 1, 2022.

Program costs for luxeptinib decreased by approximately $3.0 million, primarily due to lower manufacturing costs as a result of the current formulation requiring less API than the prior formulation, partially offset by higher clinical trial costs, mostly related to higher contractor costs required to support the trials.

Program costs for APTO-253 decreased by approximately $701 thousand, due to the Company’s decision on December 20, 2021 to discontinue further clinical development of APTO-253.

Personnel-related expenses decreased by $302 thousand, related to fewer employees in the current three-month period and partially offset by salary increases and certain employees hired during the first half of 2021.

Stock-based compensation decreased by approximately $169 thousand in the three months ended September 30, 2022, compared to the three months ended September 30, 2021, primarily due to stock options granted with lower grant date fair values, in the current period.
Research and development expenses decreased by $4.5 million to $21.3 million for the nine-month period ended September 30, 2022, as compared to $25.8 million for the comparative period in 2021. Changes to the components of our research and development expenses presented in the table above are primarily as a result of the following events:

Program costs for tuspetinib were approximately $6.6 million for the nine-month period ended September 30, 2022. The Company in-licensed the development rights of HM43239 in the fourth quarter of 2021 and assumed sponsorship, and the related costs, of the study effective January 1, 2022.

Program costs for luxeptinib decreased by approximately $7.5 million, primarily due to lower manufacturing costs as a result of the current formulation requiring less API than the prior formulation, and partially offset by lower clinical trial costs, mostly related to higher contractor costs required to support the trials.

Program costs for APTO-253 decreased by approximately $2.6 million, due to the Company’s decision on December 20, 2021 to discontinue further clinical development of APTO-253.

Personnel-related expenses increased by $119 thousand, mostly related to certain employees hired in 2021 to support our clinical trials and manufacturing activities, salary plan, and offset by lower personnel in the nine months ended September 2022.

Stock-based compensation decreased by approximately $1.1 million in the nine months ended September 30, 2022, as compared to the nine months ended September 30, 2021, primarily due to stock options granted with lower grant date fair values, in the current period.
General and Administrative

General and administrative expenses consist primarily of salaries, benefits and travel, including stock-based compensation for our executive, finance, business development, human resources, and support functions. Other general and administrative expenses are professional fees for auditing and legal services, investor relations and other consultants, insurance and facility-related expenses.

We expect that our general and administrative expenses will increase for the foreseeable future as we incur additional costs to support the expansion of our pipeline of activities. We also expect our intellectual property related legal expenses to increase as our intellectual property portfolio expands.

General and administrative expenses for the three-month period ended September 30, 2022 were $3.5 million, as compared to $3.6 million for the comparative period in 2021, a decrease of approximately $193 thousand. The decrease was primarily due the following:

General and administrative expenses, other than stock-based compensation and depreciation of equipment, increased by approximately $424 thousand in the three months ended September 30, 2022, primarily as a result of higher salaries expenses, higher travel expenses, and higher professional fees.

Stock-based compensation decreased by approximately $606 thousand in the three months ended September 30, 2022, as compared to the three months ended September 30, 2021, mostly as a result of a lower number of options granted in the current period and that those options granted in the current period had a lower grant date fair value.
General and administrative expenses for the nine-month period ended September 30, 2022 were $10.9 million as compared to $15.3 million for the comparative period, a decrease of approximately $4.4 million. The decrease was primarily due to the following:

General and administrative expenses, other than share-based compensation and depreciation of equipment, increased by approximately $833 thousand in the nine months ended September 30, 2022, primarily as a result of higher salaries expenses, higher travel expenses and higher professional fees.

Stock-based compensation decreased by approximately $5.2 million in the nine months ended September 30, 2022, compared to the nine months ended September 30, 2021, primarily due to lower grant date fair value of options granted in the current period, and additional compensation recognized in the comparative period for modifications made to then vested and unvested stock options for one officer, as part of a separation and release agreement.
*Please note the change in platform. Analysts interested in participating in the question-and-answer session will pre-register for the event from the participant registration link above to receive the dial-in numbers and a personal PIN, which are required to access the conference call. They also will have the option to take advantage of a new Call Me button and the system will automatically dial out to connect to the Q&A session.

The audio webcast can also be accessed through a link on the Investor Relations section of Aptose’s website here. A replay of the webcast will be available on the company’s website for 30 days.

The press release, the financial statements and the management’s discussion and analysis for the quarter and nine months ended September 30, 2022 will be available on SEDAR at www.sedar.com and EDGAR at www.sec.gov/edgar.shtml.

On November 1, 2022 Apollo Endosurgery, Inc. ("Apollo") (NASDAQ:APEN), a global leader in less invasive medical devices for gastrointestinal and bariatric procedures, reported financial results for the third quarter ended September 30, 2022 and recent corporate highlights (Press release, Apollo Endosurgery, NOV 1, 2022, View Source [SID1234622689]).

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Third Quarter and Recent Corporate Highlights

Grew third quarter 2022 U.S. revenue by 29% and OUS revenue by 9% (18% in constant currency) over the same period in 2021;
Grew third quarter 2022 Endoscopic Suturing System (ESS) revenue 41% (45% in constant currency) over the same period in 2021;
Increased third quarter 2022 revenue from the Company’s top 10 direct accounts by approximately 80% over the same period in 2021;
Secured FDA marketing authorizations for Apollo ESGTM and Apollo REVISETM, new endoscopic systems for treatment of patients with obesity (BMI 30-50 kg/m2); and
Announced publication of the landmark MERIT study for the Endoscopic Sleeve Gastroplasty procedure in The Lancet, demonstrating significant, durable weight loss results and an outstanding patient safety profile.
Said Chas McKhann, president and CEO, "The third quarter delivered record revenue and our seventh consecutive quarter of double-digit revenue growth. Our strong growth demonstrated continued acceleration in endobariatrics, where medical professionals are achieving clinically meaningful weight loss for patients with greatly reduced recovery time and side effect risk compared to traditional surgeries. We also expanded adoption in our G.I. defect closure business, particularly with our innovative X-Tack line, which has nearly doubled in revenue over last year and surpassed Orbera in third quarter revenue in the U.S."

"We also secured FDA marketing authorizations for our Apollo ESGTM and Apollo REVISETM endoscopic systems and announced publication of the landmark MERIT study in The Lancet," added McKhann. "These two milestones position Apollo to play a significant role in addressing the global obesity epidemic with clinically validated and relevant solutions backed by thousands of cases in the published clinical literature demonstrating excellent weight loss and safety profiles. Based on our strong operating results and important strategic milestones, we feel that Apollo is well positioned to revolutionize patient care in both G.I. health and obesity with advanced endoscopic solutions."

Financial Results for Third Quarter 2022

Total worldwide revenues increased 20% to $19.6 million for the third quarter of 2022, compared to $16.4 million in revenue during the third quarter of 2021. Revenue results in the third quarter include $0.7 million of foreign currency impact. On a constant currency basis, total revenue in the third quarter of 2022 increased 24% compared with the prior year third quarter. Compared to the third quarter of 2021, U.S. product sales increased 29% and OUS increased 9% (or 18% on a constant currency basis).

Compared to the third quarter of 2021, total ESS product sales increased $4.2 million, or 41%, due to the continued strong global demand for OverStitch and X-Tack products. Total IGB product sales decreased $0.9 million compared to the prior year quarter primarily due to summer seasonality and macroeconomic conditions impacting patient demand.

Gross margin was 55% for the third quarter of 2022, compared to 56% in the third quarter of 2021, reflecting foreign exchange headwinds, a lower revenue mix from IGB products, which have a higher gross margin profile, and a higher revenue mix from OUS distributors, which have a lower gross margin profile.

Total operating expenses increased $3.3 million compared to the third quarter of 2021. The increase primarily was due to the expansion of the U.S. salesforce and increased marketing to drive revenue growth as the Company scales the business and targets sales growth and product utilization.

Net loss for the third quarter of 2022 was $11.4 million compared to $6.7 million for the third quarter of 2021, including changes in unrealized foreign exchange of $3.3 million between periods.

Non-GAAP adjusted EBITDA, which excludes interest, taxes, depreciation, amortization, unrealized foreign exchange and stock-based compensation, in the third quarter 2022 was a loss of $3.6 million, compared to a loss of $2.5 million in the third quarter 2021.

The Company had $132.9 million in cash and committed cash at September 30, 2022, including cash, cash equivalents and restricted cash of $67.9 million and $65.0 million of future draws available under the Company’s credit facility with Innovatus Capital Partners should the Company meet certain milestones.

2022 Outlook

The Company is raising its fiscal year 2022 revenue guidance to be in the range of $75 million to $76 million, inclusive of up to $3 million in foreign currency headwinds. The Company continues to monitor the potential and uncertain impact of adverse economic conditions in the U.S. and OUS. Continued or sustained COVID-19 pandemic, inflationary or recessionary pressures could impact the Company’s ability to achieve these financial projections.

Conference Call

Apollo will host a live webcast audio call with slides today at 3:30 p.m. CT / 4:30 p.m. ET. Investors are invited to join the live call via webcast from the Investors section of the Company’s corporate website at www.apolloendo.com. An audio-only option is available is available by dialing +1-973-528-0011 and referencing access code 399827 or the "Apollo Endosurgery Third Quarter 2022 Earnings Call." Investors who opt for audio-only will need to download the related slides at www.apolloendo.com.

A replay of the webcast will be made available on Apollo’s website, www.apolloendo.com, shortly after completion of the call.

Non-GAAP Financial Measures

To supplement the Company’s financial statements presented in accordance with U.S. generally accepted accounting principles (GAAP), the Company reports certain non-GAAP financial measures, including non-GAAP adjusted operating expenses, non-GAAP Adjusted EBITDA and non-GAAP product sales percentage change in constant currency. Adjusted operating expense is calculated as operating expense less stock-based compensation. Adjusted EBITDA is calculated as GAAP net loss, plus depreciation and amortization, interest expense, net, income tax expense, stock-based compensation, unrealized foreign exchange, and gain on forgiveness of PPP loan. Product sales percentage change in constant currency is calculated by translating current foreign currency sales at last year’s exchange rate. These supplemental measures of the Company’s performance are not required by, and are not determined in accordance with GAAP. The Company believes that these non-GAAP financial measures provide investors with an additional tool for evaluating the Company’s core performance, which management uses in its own evaluation of continuing operating performance, and a baseline for assessing the future earnings potential of the Company. The Company’s non-GAAP financial measures may not provide information that is directly comparable to that provided by other companies in the Company’s industry, as other companies in the industry may calculate non-GAAP financial results differently. Non-GAAP financial results should be considered in addition to, and not as a substitute for, or superior to, financial measures calculated in accordance with GAAP. Included in the supplemental tables to this press release is a reconciliation of non-GAAP adjusted EBITDA to GAAP net loss.

On November 1, 2022 CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, reported financial results for the third quarter ended September 30, 2022 (Press release, CRISPR Therapeutics, NOV 1, 2022, View Source [SID1234622687]).

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"We are poised to end the year having made significant strides advancing our broad clinical pipeline of potentially curative gene edited therapies," said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. "CRISPR Therapeutics and Vertex remain on course to submit exa-cel for rolling FDA review this month and for EMA and MHRA review by year end, working with urgency to advance a gene edited therapy with potential to transform the lives of patients with sickle cell disease and beta thalassemia. Concurrently, we continue to enroll and dose patients in our immuno-oncology trials, CTX110 and CTX130, and remain on track to bring our next generation CAR-T programs to the clinic early next year. Finally, we’ve progressed our regenerative medicine portfolio for the treatment of type 1 diabetes with the clearance of our CTA for VCTX211 by Health Canada."

Recent Highlights and Outlook

Hemoglobinopathies

In September, CRISPR Therapeutics and Vertex Pharmaceuticals concluded discussions with the U.S. Food and Drug Administration (FDA), and the FDA granted exagamglogene autotemcel (exa-cel) a rolling review for the potential treatment of sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). The companies expect to submit the biologics licensing applications (BLA) for exa-cel for rolling review, beginning in November 2022 and expect to complete the submission by the end of Q1 2023. In the U.S., exa-cel has been granted Fast Track, Regenerative Medicine Advanced Therapy (RMAT), Rare Pediatric Disease and Orphan Drug Designations.

CRISPR Therapeutics and Vertex have completed discussions with the European Medicines Agency (EMA) and the Medicines and Healthcare products Regulatory Agency (MHRA) on the submission package for exa-cel and are on track to submit for regulatory approvals of exa-cel for SCD and TDT in Europe and the U.K. by the end of 2022. Exa-cel has been granted Orphan Drug Designation from the European Commission and Priority Medicines (PRIME) designation from the EMA, for both SCD and TDT.

Two additional Phase 3 clinical trials, CLIMB-141 and CLIMB-151, for exa-cel in pediatric patients with TDT and SCD respectively, are ongoing.

CRISPR Therapeutics continues to advance its anti-CD117 (c-Kit) antibody-drug conjugate (ADC), its internal targeted conditioning program, towards clinical studies.

Immuno-Oncology

CRISPR Therapeutics continues to enroll and dose patients in the pivotal trial of CTX110, its wholly-owned allogeneic chimeric antigen receptor T cell (CAR-T) investigational therapy targeting CD19+ B-cell malignancies. The Company expects to report additional data in 2022.

In September, CRISPR Therapeutics announced that the FDA granted RMAT designation to CTX130, the Company’s wholly-owned allogeneic CAR-T cell therapy targeting CD70, for the treatment of Mycosis Fungoides and Sézary Syndrome (MF/SS), types of cutaneous T-cell lymphoma (CTCL).

In October, CRISPR Therapeutics announced the acceptance of an oral presentation for CTX130 for the treatment of relapsed or refractory renal cell carcinoma (RCC) and a poster presentation of pre-clinical anti-CD83 CAR-T data at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 37th Annual Meeting, taking place in Boston, MA or virtually from November 8 to 12, 2022.

CRISPR Therapeutics announced the clearance of its Investigational New Drug (IND) application by the FDA for CTX112, its next generation chimeric antigen receptor T cell (CAR-T) candidate targeting CD19+ B-cell malignancies. The Company expects to initiate clinical trials for CTX112 in the first half of 2023.

Regenerative Medicine

Enrollment and dosing are ongoing in the Phase 1 clinical trial evaluating the safety and tolerability of VCTX210 for the treatment of type 1 diabetes (T1D). VCTX210 is an investigational, allogeneic, gene-edited, stem cell-derived product candidate, developed in collaboration with ViaCyte that applies CRISPR Therapeutics’ gene-editing technology to ViaCyte’s proprietary stem cell capabilities for the generation of pancreatic cells designed to evade recognition by the immune system.

In addition, CRISPR Therapeutics and ViaCyte, Inc. announced the clearance of its Clinical Trial Application (CTA) by Health Canada for VCTX211, its next generation, allogeneic, gene-edited, stem cell-derived product candidate for T1D, which includes novel edits to promote cell survival. This immune-evasive cell replacement therapy is designed to enable patients to produce their own insulin. The companies continue to progress their third program, VCTX212, in pre-clinical development for the treatment of Type 1 and Type 2 diabetes. In the third quarter of 2022, Vertex announced it had acquired ViaCyte.

In Vivo

Based upon ongoing progress with its in vivo approaches for liver gene editing utilizing both non-viral and viral delivery vehicles, CRISPR Therapeutics expects to move multiple programs utilizing in vivo approaches into the clinic in the next 12 to 15 months, including programs in cardiovascular disease. The Company’s lead program, CTX310, targeting angiopoietin-related protein 3 (ANGPTL3) is currently in IND-enabling studies.
Third Quarter 2022 Financial Results

Cash Position: Cash, cash equivalents and marketable securities were $1,973.1 million as of September 30, 2022, compared to $2,379.1 million as of December 31, 2021. The decrease in cash of $406.0 million was primarily driven by cash used in operating activities to support ongoing research and development of the Company’s clinical and pre-clinical programs.
Revenue: Total collaboration revenue was $0.1 million for the third quarter of 2022, compared to $0.3 million for the third quarter of 2021.
R&D Expenses: R&D expenses were $116.6 million for the third quarter of 2022, compared to $83.5 million for the third quarter of 2021. The increase in expense was driven by development activities supporting the advancement of our pipeline programs, as well as increased headcount costs to support these programs.
G&A Expenses: General and administrative expenses of $27.0 million for the third quarter of 2022 were consistent with general and administration expenses of $23.7 million for the third quarter of 2021.
Collaboration Expense: Collaboration expense, net, was $38.9 million for the third quarter of 2022, compared to $22.5 million for the third quarter of 2021. The increase in collaboration expense, net, was primarily driven by increased pre-commercial and manufacturing scale-up costs associated with our hemoglobinopathies programs under our collaboration with Vertex.
Net Loss: Net loss was $174.5 million for the third quarter of 2022, compared to net loss of $127.2 million for the third quarter of 2021.
About exagamglogene autotemcel (exa-cel)
Exa-cel, formerly known as CTX001, is an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy that is being evaluated for patients with TDT or SCD characterized by recurrent VOCs, in which a patient’s own hematopoietic stem cells are edited to produce high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is the form of the oxygen-carrying hemoglobin that is naturally present during fetal development, which then switches to the adult form of hemoglobin after birth. The elevation of HbF by exa-cel has the potential to alleviate transfusion requirements for patients with TDT and reduce painful and debilitating sickle crises for patients with SCD. Earlier results from these ongoing trials were published in The New England Journal of Medicine in January of 2021.

Based on progress in this program to date, exa-cel has been granted Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the FDA for both TDT and SCD. Exa-cel has also been granted Orphan Drug Designation from the European Commission, as well as Priority Medicines (PRIME) designation from the European Medicines Agency (EMA), for both TDT and SCD.

Among gene-editing approaches being evaluated for TDT and SCD, exa-cel is the furthest advanced in clinical development.

About CLIMB-111 and CLIMB-121
The ongoing Phase 1/2/3 open-label trials, CLIMB-111 and CLIMB-121, are designed to assess the safety and efficacy of a single dose of exa-cel in patients ages 12 to 35 years with TDT or with SCD, characterized by recurrent VOCs, respectively. The trials are now closed for enrollment. Patients will be followed for approximately two years after exa-cel infusion. Each patient will be asked to participate in CLIMB-131, a long-term follow-up trial.

About CLIMB-131
This is a long-term, open-label trial to evaluate the safety and efficacy of exa-cel in patients who received exa-cel in CLIMB-111, CLIMB-121, CLIMB-141 or CLIMB-151. The trial is designed to follow participants for up to 15 years after exa-cel infusion.

About CLIMB-141 and CLIMB-151
The ongoing Phase 3 open-label trials, CLIMB-141 and CLIMB-151, are designed to assess the safety and efficacy of a single dose of exa-cel in patients ages 2 to 11 years with TDT or with SCD, characterized by recurrent VOCs, respectively. The trials are now open for enrollment and currently enrolling patients ages 5 to 11 years of age and will plan to extend to patients 2 to less than 5 years of age at a later date. Each trial will enroll approximately 12 patients. Patients will be followed for approximately two years after infusion. Each patient will be asked to participate in CLIMB-131, a long-term follow-up- trial.

About the CRISPR-Vertex Collaboration
CRISPR Therapeutics and Vertex Pharmaceuticals entered into a strategic research collaboration in 2015 focused on the use of CRISPR/Cas9 to discover and develop potential new treatments aimed at the underlying genetic causes of human disease. Exa-cel represents the first potential treatment to emerge from the joint research program. Under a recently amended collaboration agreement, Vertex will lead global development, manufacturing and commercialization of exa-cel and split program costs and profits worldwide 60/40 with CRISPR Therapeutics.

About CTX110 and CARBON Trial
CTX110, a wholly owned program of CRISPR Therapeutics, is a healthy donor-derived gene-edited allogeneic CAR-T investigational therapy targeting cluster of differentiation 19, or CD19. CTX110 is being investigated in the ongoing CARBON trial, a Phase 1 single-arm, multi-center, open label clinical trial, CARBON, is designed to assess the safety and efficacy of several dose levels of CTX110 for the treatment of relapsed or refractory B-cell malignancies. CTX110 has been granted RMAT designation by the FDA.

About CTX130 and COBALT Trials
CTX130, a wholly-owned program of CRISPR Therapeutics, is a healthy donor-derived gene-edited allogeneic CAR-T investigational therapy targeting cluster of differentiation 70, or CD70, an antigen expressed on various solid tumors and hematologic malignancies. CTX130 is being developed for the treatment of both solid tumors, such as renal cell carcinoma (COBALT-RCC), and T-cell and B-cell hematologic malignancies (COBALT-LYM). CTX130 is being investigated in two ongoing independent Phase 1, single-arm, multi-center, open-label clinical trials that are designed to assess the safety and efficacy of several dose levels of CTX130 for the treatment of relapsed or refractory renal cell carcinoma and various subtypes of lymphoma, respectively. CTX130 has been granted Orphan Drug designation for the treatment of T-cell lymphoma by the FDA and RMAT designation for the treatment of relapsed or refractory Mycosis Fungoides and Sézary Syndrome (MF/SS), types of cutaneous T-cell lymphoma (CTCL).

About VCTX210
VCTX210 is an investigational, allogeneic, gene-edited, immune-evasive, stem cell-derived therapy for the treatment of T1D. VCTX210 is being developed under a co-development and co-commercialization agreement between CRISPR Therapeutics and ViaCyte, Inc.

On November 1, 2022 Ligand Pharmaceuticals Incorporated (NASDAQ: LGND) reported the company has completed the expected tax-free spin-off of its OmniAb antibody discovery business which has become OmniAb, Inc., an independent publicly traded company (Press release, Ligand, NOV 1, 2022, View Source [SID1234622686]). OmniAb will begin regular-way trading November 2, 2022 on NASDAQ under the stock ticker symbol "OABI." Ligand continues to trade under the stock ticker symbol "LGND."

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"For the last decade, Ligand has been focused on providing the tools and technologies needed by the pharmaceutical industry and sharing in the promise of drug innovation with our shareholders. Today, Ligand has a diversified portfolio of growing royalty revenue and a large portfolio of late-stage programs," said John Higgins, CEO. "With the spin-off completed, we are focused on creating financial growth for years to come. We wish Matt Foehr and the OmniAb team well as they continue to enable development of innovative therapeutics as an independent publicly traded company."

Ligand Leadership Updates

In conjunction with the spin-off, Ligand is also announcing Executive management appointments. Ligand is led by a team of experienced executives with a record of building businesses with strong financial growth and disciplined business investing.

"I’m delighted to announce changes in leadership at Ligand. Matt Korenberg and Tavo Espinoza are expanding their responsibilities within Ligand, and we are pleased to have Andrew Reardon join our executive leadership team. Matt has been promoted to President and Chief Operating Officer. He has led the financial and strategic efforts at Ligand over the previous seven years as Chief Financial Officer. I am excited for Matt to continue his leadership in this elevated and expanded role," said John Higgins, Chief Executive Officer.

"Tavo joins our executive leadership team as Chief Financial Officer after six years leading the Accounting and Finance functions at Ligand. Andrew joins the team as Chief Legal Officer and brings a wealth of knowledge and experience in royalty transactions. I am confident this experienced team will continue to drive the pursuit of growth through Ligand’s exciting new chapter."

Octavio (Tavo) Espinoza is joining Ligand’s executive leadership team as Chief Financial Officer. Mr. Espinoza has been with Ligand since 2016 serving in a variety of finance leadership roles at Ligand, most recently as Senior Vice President, Finance. Prior to joining Ligand, Mr. Espinoza served as Senior Director, Finance for Receptos, a publicly traded drug-discovery company, that was acquired by Celgene (now Bristol-Myers Squibb). Before Receptos, Mr. Espinoza was Senior Director, Accounting for Illumina, a publicly traded life sciences analytics and genetics sequencing tools company, and prior to Illumina, served as Senior Manager, Accounting at Intuit. Mr. Espinoza began his professional career in public accounting at PricewaterhouseCoopers and holds a B.S. in business administration from San Diego State University and a license as a Certified Public Accountant in the State of California.

Andrew Reardon joins Ligand’s executive leadership team as Chief Legal Officer. Prior to joining Ligand, Mr. Reardon was the Chief Legal Officer at HealthCare Royalty Management, a manager of investment funds investing in biopharmaceutical products primarily through royalty and structured credit transactions. Before joining HealthCare Royalty, Mr. Reardon was a senior associate with the law firm Willkie Farr & Gallagher, where he focused on corporate transactions, including finance and mergers and acquisitions. Mr. Reardon is a former officer with the United States Army National Guard. He earned his J.D. from the University of Iowa College of Law and received his B.A. from the State University of New York College at Old Westbury.

As part of the planned business separation, in connection with the closing of the business combination, Jennifer Cochran, Ph.D., Sarah Boyce and Sunil Patel stepped down from the Ligand board of directors and joined the OmniAb board. These changes reduce Ligand’s number of directors to eight.

Other Updates

In conjunction with the spin-out OmniAb will retain the current Ligand address for its corporate headquarters and Ligand will transfer its headquarters to Las Vegas, Nevada in approximately two months. Ligand has leased office space in Nevada since 2019 and Nevada has been the primary distribution point for Captisol shipments originating in the United States since that time.