On August 8, 2022 CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, reported financial results for the second quarter ended June 30, 2022 (Press release, CRISPR Therapeutics, AUG 8, 2022, View Source [SID1234617737]).

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"Strong progress continues across our broad portfolio of gene edited therapies and we remain on track to achieve important 2022 milestones," said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. "We and our partner Vertex presented new clinical data at EHA (Free EHA Whitepaper) highlighting the potentially transformative profile of exa-cel in patients with TDT or SCD. We also presented encouraging new clinical data for CTX130 for the treatment of both solid tumors and certain hematologic malignancies, and we expect to report data from our ongoing trial of CTX110 targeting CD19+ B-cell malignancies later this year. In addition, we and our partner ViaCyte continue to enroll and dose patients in the Phase 1 clinical trial of VCTX210 for T1D. We remain well positioned and well capitalized to bring transformative medicines for patients suffering from serious diseases."

Dr. Kulkarni further added, "During our Innovation Day in June, we presented the depth and breadth of our pipeline and highlighted our platform innovations to create the next generation of genomic medicines. We are poised to significantly expand our pipeline programs of potentially curative therapies with the addition of new development candidates as we continue to innovate our genome editing, delivery, and cell engineering capabilities."

Recent Highlights and Outlook

Hemoglobinopathies

In June, CRISPR Therapeutics and Vertex Pharmaceuticals presented new clinical data at the European Hematology Association (EHA) (Free EHA Whitepaper) Congress on exagamglogene autotemcel (exa-cel), formerly known as CTX001, highlighting the potentially transformative profile of this investigational therapy for people with transfusion-dependent beta thalassemia (TDT) or severe sickle cell disease (SCD). Data from 75 patients (44 with TDT, 31 with SCD) from the CLIMB-111, CLIMB-121 and CLIMB-131 studies with follow-up ranging from 1.2 to 37.2 months after exa-cel infusion continued to demonstrate that exa-cel has the potential to be a durable, one-time functional cure. All 31 patients with severe SCD, characterized by recurrent vaso-occlusive crises (VOCs), were free of VOCs after exa-cel infusion through the duration of follow-up, with follow-up ranging from 2.0 to 32.3 months. Of the 44 patients with TDT, 42 were transfusion-free with follow-up ranging from 1.2 to 37.2 months. Two patients who were not yet transfusion-free had 75% and 89% reductions in transfusion volume, respectively. The safety profile was generally consistent with myeloablative conditioning with busulfan and autologous stem cell transplant. The CLIMB-111 and CLIMB-121 trials are in Phase 3 and fully enrolled.

CRISPR Therapeutics and Vertex have initiated two additional Phase 3 clinical trials, CLIMB-131 and CLIMB-141, for exa-cel in pediatric patients with TDT or SCD.

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. Discussions with the FDA are ongoing.

In June, CRISPR Therapeutics hosted an Innovation Day focused on research and development, during which it presented preclinical data on its anti-CD117 (c-Kit) antibody-drug conjugate (ADC). The Company plans to advance this 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 June, CRISPR Therapeutics presented new T-cell lymphoma clinical data at the EHA (Free EHA Whitepaper) Congress from the Company’s ongoing Phase 1 COBALT-LYM trial evaluating the safety and efficacy of CTX130, its wholly-owned allogeneic CAR-T investigational therapy targeting CD70, for both solid tumors and certain hematologic malignancies. The preliminary data demonstrate that CTX130 has the potential to provide meaningful clinical benefit with a well-tolerated safety profile. The Company continues to enroll and dose patients in the dose expansion trial.

In June, at CRISPR Therapeutics’ Innovation Day, the Company provided the following updates regarding its wholly-owned oncology programs:
Presented new clinical data from the Company’s ongoing Phase 1 COBALT-RCC trial evaluating the safety and efficacy of CTX130 for the treatment of relapsed or refractory renal cell carcinoma (RCC). The preliminary data demonstrates the first signs of meaningful activity in solid tumors with an allogeneic cell therapy and a well-tolerated safety profile.
Announced plans to advance next-generation investigational therapies with additional gene edits that have the potential to improve upon first-generation programs. The Company expects to advance two next-generation constructs to IND by year end: CTX131 and CTX112 targeting CD70 and CD19, respectively.
Announced plans for a next-generation allogeneic CAR-T therapy targeting B-cell maturation antigen (BCMA) that incorporates proprietary edits to enhance the potency of the CAR-T cells.
Announced collaborations with top cancer centers on new targets. One with Moffitt Cancer Center will seek to advance a first-in-human trial for an autologous CAR-T investigational therapy targeting CD83 for the potential treatment of acute myeloid leukemia (AML) and other oncology and autoimmune indications. A second with Roswell Park Comprehensive Cancer Center will seek to advance an initial trial for an autologous, gene-edited CAR-T therapy targeting GPC3 for the potential treatment of solid tumors.

Regenerative Medicine

Enrollment and dosing are ongoing in the Phase 1 clinical trial of VCTX210 for the treatment of T1D. VCTX210 is an investigational, allogeneic, gene-edited, stem cell-derived product 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. This immune-evasive cell replacement therapy is designed to enable patients to produce their own insulin.

In June, at CRISPR Therapeutics’ Innovation Day, the Company provided the following updates regarding its regenerative medicine programs:
Announced plans to expand its regenerative medicine pipeline with two next-generation approaches. The first, VCTX211, features novel edits to promote cell survival. The Company plans to file a CTA for VCTX211 in the second half of 2022. The second program, VCTX212, is in early-stage development for the treatment of Type 1 and Type 2 diabetes.
In Vivo
In June, at CRISPR Therapeutics’ Innovation Day, the Company provided the following updates regarding its in vivo programs:
Based upon ongoing progress with its in vivo approaches for liver gene editing utilizing both viral and non-viral delivery vehicles, CRISPR Therapeutics continues to expect to move multiple programs utilizing in vivo approaches into the clinic in the next 12 to 18 months, including programs in cardiovascular disease. The Company’s lead program, CTX310, targeting angiopoietin-related protein 3 (ANGPTL3) is currently in IND-enabling studies.
Announced the establishment of CRISPR-X, a dedicated group within CRISPR Therapeutics focused on emerging technologies, including those to allow HDR-independent and/or AAV-free whole gene correction and insertion.
Other Corporate Matters

In May, CRISPR Therapeutics announced the appointment of Phuong Khanh (P.K.) Morrow, M.D., FACP, as Chief Medical Officer. Dr. Morrow brings more than a decade of leadership experience in global drug development and joined CRISPR Therapeutics to lead the Company’s global clinical development and regulatory operations.
Second Quarter 2022 Financial Results

Cash Position: Cash, cash equivalents and marketable securities were $2,073.7 million as of June 30, 2022, compared to $2,379.1 million as of December 31, 2021. The decrease in cash of $305.4 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 $0.2 million for the second quarter of 2022, compared to $900.2 million for the second quarter of 2021. Revenue for the second quarter of 2021 was primarily associated with the $900.0 million upfront payment from Vertex in connection with the Amended and Restated Joint Development and Commercialization Agreement.

R&D Expenses: R&D expenses were $123.2 million for the second quarter of 2022, compared to $82.3 million for the second quarter of 2021. The increase in expense was driven by development activities supporting the advancement of our wholly-owned immuno-oncology programs, as well as expenses related to our new U.S. research and development headquarters.

G&A Expenses: General and administrative expenses of $26.3 million for the second quarter of 2022 were consistent with general and administration expenses of $28.8 million for the second quarter of 2021.

Collaboration Expense: Collaboration expense, net, was $33.9 million for the second quarter of 2022, compared to $26.9 million for the second 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 $185.8 million for the second quarter of 2022, compared to net income of $759.2 million for the second 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 Regenerative Medicine Advanced Therapy designation from 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 from the FDA.

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 August 8, 2022 ALX Oncology Holdings Inc., ("ALX Oncology") (Nasdaq: ALXO) a clinical-stage immuno-oncology company developing therapies that block the CD47 checkpoint pathway, reported financial results for the second quarter ended June 30, 2022 and provided clinical development and operational highlights (Press release, ALX Oncology, AUG 8, 2022, View Source [SID1234617773]).

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"The second quarter of 2022 was marked by substantial progress advancing our lead program, evorpacept, through multiple clinical trials," said Jaume Pons, Ph.D., Founder, President and Chief Executive Officer of ALX Oncology. "Notable accomplishments included the U.S. Food and Drug Administration ("FDA") granting Fast Track designation to evorpacept for the first-line treatment of adult patients with PD-L1 positive advanced head and neck squamous cell carcinoma ("HNSCC") in combination with KEYTRUDA (pembrolizumab), Merck’s anti-PD-1 therapy, and Orphan Drug Designation ("ODD") to evorpacept for the treatment of patients with acute myeloid leukemia ("AML")."

"We continue to expand our clinical development efforts with the introduction of a new clinical study, ASPEN-07, a Phase 1 trial of evorpacept for the treatment of patients with urothelial carcinoma ("UC") and anticipate initiation in the fourth quarter of 2022. We are continuing enrollment in ASPEN-03 and ASPEN-04, two distinct Phase 2 studies for the treatment of patients with advanced HNSCC. In addition, we continue to advance ASPEN-06, our Phase 2/3 study testing evorpacept and CYRAMZA (ramucirumab), Eli Lilly and Company’s anti-VEGFR2 antibody, added to trastuzumab and paclitaxel for the treatment of patients with HER2-positive gastric cancer or gastroesophageal junction ("GEJ") cancer," Dr. Pons continued.

Recent Clinical Developments for Evorpacept

FDA Granted Fast Track Designation as First-Line Treatment for HNSCC
In August 2022, ALX Oncology announced that the FDA granted Fast Track designation to evorpacept, a next-generation CD47 blocker, in combination with KEYTRUDA (pembrolizumab) for the first-line treatment of patients with PD-L1 expressing metastatic or unresectable, recurrent HNSCC.
ALX Oncology continues to advance ASPEN-03 and ASPEN-04, which are two distinct randomized Phase 2 studies for the treatment of patients with advanced HNSCC in combination with KEYTRUDA (pembrolizumab) with or without chemotherapy. Patient enrollment for ASPEN-03 and ASPEN-04 continues as planned with results expected to be presented by mid-2024.
FDA Granted ODD for Evorpacept for the Treatment of Patients with AML
In June 2022, ALX Oncology announced that the FDA granted ODD to evorpacept for the treatment of patients with AML.
The Phase 1 dose escalation portion of ASPEN-05, a Phase 1/2 clinical trial of evorpacept in combination with venetoclax and azacitidine for the treatment of patients with AML, has successfully completed enrollment with no safety concerns to date up to the highest protocol defined dose level of 60 mg/kg evorpacept once every four weeks.
Patient enrollment was paused before proceeding into the Phase 1 dose optimization portion of ASPEN-05 pending completion of the Phase 1 portion of ASPEN-02, a Phase 1/2 study of evorpacept in combination with azacitidine in patients with myelodysplastic syndrome ("MDS"). Data from ASPEN-02 will be used to inform the optimal dose(s) of evorpacept to be studied in the ASPEN-05 study in combination with venetoclax and azacitidine. Ongoing patients in ASPEN-05 will continue to be treated and followed per protocol.
Additional Evorpacept Clinical Program Updates
In June 2022, ALX Oncology announced expected initiation of ASPEN-07, a Phase 1 trial of evorpacept for the treatment of patients with UC. ASPEN-07 will investigate evorpacept in combination with an antibody-drug conjugate ("ADC"), PADCEV (enfortumab vedotin-ejfv), for the treatment of patients with UC in the fourth quarter of 2022.
ALX Oncology continues to advance ASPEN-06, a randomized Phase 2 (open-label) / Phase 3 (double-blind), international, multi-center study to evaluate the efficacy of evorpacept and CYRAMZA (ramucirumab) added to trastuzumab and paclitaxel for the treatment of patients with HER-positive gastric/GEJ cancer whose tumors have progressed following treatment with HER2-targeted therapy and chemotherapy. ASPEN-06 is being conducted in collaboration with Eli Lilly and Company. Patient enrollment continues to progress and results from the Phase 2 portion of ASPEN-06 are expected to be presented in 2023.
Second Quarter 2022 Financial Results:

Cash, Cash Equivalents and Investments: Cash, cash equivalents and investments as of June 30, 2022 were $324.2 million. ALX Oncology recently updated its cash forecast and believes its cash, cash equivalents and investments are sufficient to fund planned operations through the fourth quarter of 2024.
Research and Development ("R&D") Expenses: R&D expenses consist primarily of pre-clinical, clinical and manufacturing expenses related to the development of ALX Oncology’s current lead product candidate, evorpacept, and R&D employee-related expenses. These expenses for the three months ended June 30, 2022, were $26.7 million, compared to $11.2 million for the prior-year period. The increase in expenses during the three months ended June 30, 2022 compared to the three months ended June 30, 2021 were primarily attributable to an increase of $10.3 million in clinical and development costs primarily due to manufacturing of clinical trial materials to support a higher number of active clinical trials and future expected patient enrollment related to the advancement of our lead product candidate, as well as an increase of $1.6 million related to the Tallac Collaboration for work related to the IND filing planned for 2023; an increase of $0.4 million in preclinical costs primarily related to development of new targets; an increase of $1.8 million in personnel and related costs due primarily to an increase driven by headcount growth and a portion of a retention bonus payable to ScalmiBio stockholders; an increase of $2.0 million in stock-based compensation expense due to additional awards granted since June 30, 2021; and an increase of $1.1 million in other research costs.
General and Administrative ("G&A") Expenses: G&A expenses consist primarily of administrative employee-related expenses, legal and other professional fees, patent filing and maintenance fees, and insurance. These expenses for the three months ended June 30, 2022, were $7.0 million, compared to $5.1 million for the prior-year period. The expense increases during the three months ended June 30, 2022 compared to the three months ended June 30, 2021 were primarily attributable to an increase of $1.6 million in stock-based compensation expense due to additional stock option awards granted since June 30, 2021 and an increase of $0.3 million in personnel and related costs primarily driven by headcount growth.
Net loss: GAAP net loss was $32.9 million for the second quarter ended June 30, 2022, or $0.81 per basic and diluted share, as compared to a net loss of $16.3 million for the second quarter ended June 30, 2021, or $0.40 per basic and diluted share. Non-GAAP net loss was $27.1 million for the second quarter ended June 30, 2022, as compared to a net loss of $14.0 million for the second quarter ended June 30, 2021. A reconciliation of GAAP to non-GAAP financial results can be found at the end of this news release.

On August 8, 2022 Quanterix Corporation (NASDAQ: QTRX), a company expanding the limits of exploration with ultrasensitive biomarker detection, reported operating results for the three months ended June 30, 2022 (Press release, Quanterix, AUG 8, 2022, View Source [SID1234617789]).

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Second Quarter 2022 Financial Highlights

Q2 total revenue was $23.5M versus prior year Q2 total revenue of $25.4M, a decrease of 7.4%;
Q2 product revenue was $14.8M versus prior year Q2 product revenue of $18.7M, a decrease of 20.8%;
Q2 service and other revenue was $8.5M versus prior year Q2 service and other revenue of $5.6M, an increase of 51.3%; and
Q2 gross margin was 37.1% versus prior year Q2 gross margin of 54.7%, a reduction of 1,760 bps; Q2 pro-forma gross margin was 28.3% versus prior year Q2 pro-forma gross margin of 47.5%, a reduction of 1,920 bps.
For additional information on the pro-forma financial measures included in this press release, please see "Use of Pro-Forma Financial Measures" and "Reconciliation of GAAP to Pro-Forma" below.

Second quarter revenues were impacted by a reduction in consumable revenue as the Company addressed assay quality challenges and process improvement initiatives. While these challenges are a near-term headwind to revenue growth, the Company believes underlying market demand for the SIMOA technology remains robust.

The reduction in GAAP gross margin reflects the reallocation of resources, principally headcount, to ongoing quality-related activities, resulting in an increase in cost of goods sold and a corresponding decrease in operating expense. Pro-forma gross margin is adjusted to include the portion of product-related freight and shipping costs that are not billed to the customer. These amounts are included in operating expenses on a GAAP basis. The Company expects to report pro-forma gross margin on a go forward basis. The increases to cost of goods sold described above have a corresponding reduction in operating expense, with no overall change to the Company’s total expenses. The Company believes that including these expenses in cost of goods sold reflects the nature of these expenses and will provide better visibility into the Company’s progress toward its quality process improvement initiatives. Gross margins were also impacted by a decrease in sales of higher-margin consumable products as well as a reduction in overall volume.

Strategic Business Re-Alignment Plan

Following a strategic review and top-down assessment of its operations, the Company announced a comprehensive plan that includes restructuring and business re-alignment in order to fully realize the potential of its SIMOA platforms and continue its leadership role in ultrasensitive translational biomarker detection. In connection with these changes, the Company has set in motion an assay redevelopment program with the ultimate objective of improving its ability to manufacture and deliver high-quality assays at scale. The company expects to make initial progress toward this initiative in 2022 and that the program will be completed in 2023. The plan also aligns the Company’s investments to best serve the needs of customers, focus innovation efforts on key platforms and provide the foundation for the Company’s entry into translational pharma and clinical markets, which it believes will be required to access new growth categories. The changes will improve the Company’s cost structure by aligning our expenses with anticipated future revenues. These actions will result in a reduction in force affecting approximately 130 employees across the Company’s world-wide operations. The Company also announced that it is reviewing alternatives with respect to additional facility space that it currently leases in Bedford, Massachusetts.

The Company expects to incur restructuring and related charges of approximately $7 million to $10 million in Q3 2022. The Company also expects to realize run-rate savings of approximately $25 million on an annualized basis. The Company is currently unable to estimate facility-related charges.

FY2022 Guidance and Long-Term Outlook

The Company announced that it now expects its total 2022 revenue to be flat compared to 2021. The Company expects to return to double digit revenue growth by 2024 as the benefits of its restructuring and re-alignment plan are fully realized, and to accelerate at a faster pace once new growth categories are unlocked.

"We are taking decisive action to focus and improve the quality and scalability of our products, support our customers and open the door to new growth opportunities," said Masoud Toloue, President and Chief Executive Officer of Quanterix. "By embarking on this next chapter, we will emerge a stronger company, innovating at a faster pace and well-timed to enable new disease modifying therapies expected to come to market in the coming years."

Conference Call

In conjunction with this announcement, Quanterix Corporation will host a conference call on August 8, 2022 at 4:30 p.m. EST. Individuals interested in listening to the conference call may do so by pre-registering here and obtaining a dial-in number and passcode.

A live webcast will also be available at: View Source You may also access the live webcast by visiting the News & Events page within the Investors section of the Quanterix website at www.quanterix.com. The webcast will be available on the Company’s website for one year following completion of the call.

Financial Highlights

Use of Pro-Forma Financial Measures

To supplement the Company’s financial statements presented on a GAAP basis, the Company has provided certain pro-forma financial measures, including pro-forma gross profit, pro-forma gross margin percentage, pro-forma operating expenses and pro-forma loss from operations. Management uses these pro-forma measures to evaluate the Company’s operating performance in a manner that allows for meaningful period-to-period comparison and analysis of trends in its business. Management believes that such measures are important in comparing current results with prior period results and are useful to investors and financial analysts in assessing the Company’s operating performance. The pro-forma financial information presented here should be considered in conjunction with, and not as a substitute for, the financial information presented in accordance with GAAP. Investors are encouraged to review the reconciliation of these pro-forma measures to their most directly comparable GAAP financial measures set forth below.

On August 8th 2022, Orion’s collaboration partner Bayer reported the U.S. Food and Drug Administration (FDA) has approved a supplemental New Drug Application (sNDA) for the oral androgen receptor inhibitor (ARi) darolutamide in combination with docetaxel for the treatment of patients with metastatic hormone-sensitive prostate cancer (mHSPC) (Press release, Orion , AUG 8, 2022, View Source [SID1234617804]).

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The approval is based on positive results of the Phase III ARASENS trial that demonstrated darolutamide plus androgen deprivation therapy (ADT) and docetaxel significantly reduced the risk of death by 32.5% in patients with metastatic hormone-sensitive prostate cancer (mHSPC) compared to ADT plus docetaxel. These results were recently published in The New England Journal of Medicine.1 Darolutamide is approved in more than 70 markets around the world, including the U.S., the European Union (EU), Japan and China, under the brand name Nubeqa, for the treatment of patients with non-metastatic castration-resistant prostate cancer (nmCRPC), who are at high risk of developing metastatic disease. The compound is also being investigated in further studies across various stages of prostate cancer.

Prostate cancer remains the second leading cancer-related cause of death among men in the U.S., with up to one-third of patients developing metastatic disease.2, 3 The incidence of mHSPC has increased by 72% in the U.S. over the past 10 years.4 Approximately one in three patients who are diagnosed with mHSPC survive the disease five years or longer, with most eventually experiencing progression to castration-resistant prostate cancer (CRPC).3,4

The application received Priority Review designation granted by the FDA and was submitted under the FDA’s Real-Time Oncology Review (RTOR) pilot program, which aims to provide a more efficient review process of applications to ensure that safe and effective cancer treatments are available to patients as early as possible. Ongoing reviews are also being conducted under the FDA Oncology Center of Excellence’s (OCE) Project Orbis initiative, which provides a framework for concurrent submission and review of cancer treatments among participating international health authorities.

Darolutamide is developed jointly by Orion and Bayer.

About the ARASENS Trial

The ARASENS trial is a randomized, Phase III, multi-center, double-blind, placebo-controlled trial which was prospectively designed to investigate the efficacy and safety of oral darolutamide, an androgen receptor inhibitor (ARi), plus androgen deprivation therapy (ADT) and the chemotherapy docetaxel in patients with metastatic hormone-sensitive prostate cancer (mHSPC). A total of 1,306 newly diagnosed patients were randomized in a 1:1 ratio to receive 600 mg of darolutamide twice a day or matching placebo, plus ADT and docetaxel.

The primary endpoint of this trial was overall survival (OS). Secondary endpoints included time to castration-resistant prostate cancer (CRPC), time to pain progression, time to first symptomatic skeletal event (SSE), time to initiation of subsequent anticancer therapy, all measured at 12‐week intervals, as well as adverse events (AEs) as a measure of safety and tolerability. Results from this trial were published in the New England Journal of Medicine1. The ARASENS trial demonstrated that darolutamide plus androgen deprivation therapy (ADT) and docetaxel significantly reduced the risk of death by 32.5% compared to ADT plus docetaxel1. Improvements in the secondary endpoints supported the benefit observed in the primary endpoint, overall survival1.

About Metastatic Hormone-Sensitive Prostate Cancer

Prostate cancer is the second most commonly diagnosed malignancy in men worldwide. In 2020, an estimated 1.4 million men were diagnosed with prostate cancer, and about 375,000 died from the disease worldwide.5

At the time of diagnosis, most men have localized prostate cancer, meaning their cancer is confined to the prostate gland and can be treated with curative surgery or radiotherapy. Upon relapse, when the disease will metastasize or spread, androgen deprivation therapy (ADT) is the cornerstone of treatment for this hormone-sensitive disease. Current treatment options for men with metastatic hormone-sensitive prostate cancer (mHSPC) include hormone therapy, such as ADT, androgen receptor pathway inhibitors plus ADT or a combination of docetaxel chemotherapy and ADT. Despite these treatments, a large proportion of men with mHSPC will eventually experience progression to metastatic castration-resistant prostate cancer (mCRPC), a condition with limited survival.

About darolutamide

Darolutamide is an oral androgen receptor inhibitor (ARi) with a distinct chemical structure that binds to the receptor with high affinity and exhibits strong antagonistic activity, thereby inhibiting the receptor function and the growth of prostate cancer cells. The low potential for blood-brain barrier penetration for darolutamide is supported by preclinical models and neuroimaging data in healthy humans.6, 7 This is supported by the overall low incidence of central nervous system (CNS)-related adverse events (AEs) compared to placebo as seen in the ARAMIS Phase III trial and the maintained verbal learning and memory observed in the darolutamide arm of the Phase II ODENZA trial.8

The product is approved under the brand name Nubeqa in more than 70 markets around the world, including the U.S., EU, Japan, China, for the treatment of patients with non-metastatic castration-resistant prostate cancer (nmCRPC), who are at high risk of developing metastatic disease. The compound is also being investigated in further studies across various stages of prostate cancer, including in the ARANOTE Phase III trial evaluating darolutamide plus androgen deprivation therapy (ADT) versus ADT alone for metastatic hormone-sensitive prostate cancer as well as the Australian and New Zealand Urogenital and Prostate Cancer Trials Group (ANZUP) led international co-operative group Phase III trial, evaluating darolutamide as an adjuvant treatment for localized prostate cancer with very high risk of recurrence (DASL-HiCaP, ANZUP1801). Information about these trials can be found at www.clinicaltrials.gov.

On August 8, 2022 VolitionRx Limited (NYSE AMERICAN: VNRX) ("Volition") reported it will host a conference call on Thursday August 11, at 08.30 a.m. Eastern Time to discuss its financial and operating results for the second quarter 2022, in addition to providing a business update (Press release, VolitionRX, AUG 8, 2022, View Source [SID1234617820]).

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Cameron Reynolds, President and Chief Executive Officer of Volition, will host the call along with Terig Hughes, Chief Financial Officer, Dr. Tom Butera, Chief Executive Officer of Volition Veterinary Diagnostics Development LLC, and Scott Powell, Executive Vice President, Investor Relations. The call will provide an update on important events which have taken place in the second quarter of 2022 and upcoming milestones.

A live audio webcast of the conference call will also be available on the investor relations page of Volition’s corporate website at View Source In addition, a telephone replay of the call will be available until August 25, 2022. The replay dial-in numbers are 1-844-512-2921 (toll-free) in the U.S. and Canada and 1-412-317-6671 (toll) internationally. Please use replay pin number 13732149.