On August 7, 2023 Quanterix Corporation (NASDAQ: QTRX), a company fueling scientific discovery through ultrasensitive biomarker detection, reported financial results for the three months ended June 30, 2023 (Press release, Quanterix, AUG 7, 2023, View Source [SID1234633883]).

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!

Second Quarter Financial Highlights

● Total revenue was $31.0 million versus prior year second quarter total revenue of $23.5 million, an increase of 32%. Second quarter revenue also increased 9% sequentially driven by increased Accelerator Lab and consumables revenue. Second quarter revenue includes a one-time benefit of approximately $1.5 million related to product and license agreements.
● GAAP gross margin was 61.7% compared to prior year second quarter GAAP gross margin of 37.1%. Non-GAAP gross margin was 56.4% compared to prior year second quarter non-GAAP gross margin of 29.1%.
● Net loss was $6.1 million compared to prior year second quarter net loss of $24.9 million.
● Net cash flow in the quarter was approximately break-even. Cash and cash equivalents were $329.5 million as of June 30, 2023, as compared to $329.4 million as of March 31, 2023.
"Since launching our corporate transformation, we’re pleased with the accelerating progress as evidenced by both financial and business results," said Masoud Toloue, President and Chief Executive Officer of Quanterix. "In under a year, our talented team has implemented fundamental change to business operations, with cash flow break-even being a key milestone, that said, we intend to continue to invest in scaling and supporting the important work performed on our Simoa platform that we believe will change the way we understand, test, and treat neurological diseases."

Operational and Business Highlights

Accelerating our leadership position in blood-based biomarkers:

● In July, the Company launched LucentAD, a blood-based biomarker test to assist in the evaluation of patients experiencing cognitive symptoms consistent with the early signs of Alzheimer’s disease. This launch includes a new portal for patient and providers.

● Positive readout on Quanterix’s Bio-Hermes clinical study with the Global Alzheimer’s Platform Foundation confirmed high correlation between the LucentAD blood test and amyloid PET scans in early Alzheimer’s patients, a cohort most eligible for therapy. Results will be used to support Quanterix’s FDA filing.

● Quanterix’s Accelerator Lab will now provide Simoa assay testing for tau phosphorylation at residues 217 and 212 (p217+ tau), through a new research use only agreement with Janssen for its proprietary p217+ tau assays. Published studies have shown that the presence of phosphorylated tau in plasma is predictive of central amyloid and tau status and has the potential for diagnosing and staging Alzheimer’s disease.

● Robust reference dataset of serum NfL levels measured using Simoa technology across a wide spectrum of ages, from neonatal to 20-year-old adolescents, was published last week in Lancet Neurology, establishing a new standard with important implications on neuro-testing.
Full Year Business Outlook

With accelerating corporate transformation efforts, management has increased full-year revenue expectations to be in the range of $110 to $116 million, GAAP gross margin percentage to be in the low 50’s, and non-GAAP gross margin percentage to be in the high 40s. The Company also anticipates a lower 2023 cash burn, in the range of $30 to $35 million.

For additional information on the non-GAAP financial measures included in this press release, please see "Use of Non-GAAP Financial Measures" and "Reconciliation of GAAP to Non-GAAP Financial Measures" below.

Conference Call

In conjunction with this announcement, the Company will host a conference call on August 8, 2023 at 8:30 a.m. E.T. Click here to pre-register for the conference call and obtain your dial-in number and passcode.

Interested investors can also access the live webcast from the News & Events page within the Investors section of the Quanterix website at View Source An archived webcast replay will be available on the Company’s website for one year.

On August 7, 2023 QIAGEN (NYSE: QGEN; Frankfurt Prime Standard: QIA) reported the U.S. Food and Drug Administration (FDA) approval of its therascreen PDGFRA RGQ PCR kit (therascreen PDGFRA kit) (Press release, Qiagen, AUG 7, 2023, View Source [SID1234633882]).

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 companion diagnostic is intended for use to aid clinicians in identifying patients with gastrointestinal stromal tumors (GIST) who may be eligible for treatment with AYVAKIT (avapritinib), which is approved in the U.S. for the treatment of adults with unresectable or metastatic GIST harboring a platelet-derived growth factor receptor alpha (PDGFRA) exon 18 mutation, including PDGFRA D842V mutations[1]. The kit is the first platelet-derived growth factor receptor alpha (PDGFRA) assay to receive FDA approval as a companion diagnostic.

QIAGEN and Blueprint Medicines collaboratively developed the PDGFRA companion diagnostic assay. The real-time qualitative PCR in vitro diagnostic assay detects the D842V somatic mutation in the PDGFRA gene to determine which patients may be a candidate for treatment with AYVAKIT. The tyrosine kinase inhibitor (TKI) is designed to target the PDGFRA exon 18 D842V mutation and was approved by the FDA in 2020. GIST patients harboring the D842V mutation in PDGFRA exon 18 show primary resistance to previously approved TKIs.

The therascreen PDGFRA kit uses genomic DNA extracted from a patient’s formalin-fixed paraffin-embedded (FFPE) tumor tissue. FFPE tumor specimens are processed using the QIAamp DSP DNA FFPE Tissue Kit for sample preparation and the Rotor-Gene Q (RGQ) MDx instrument for DNA amplification and mutation detection.

"The therascreen PDGFRA kit is an FDA approved and validated test, delivering results in a fast turnaround time. This ensures that physicians receive results promptly, enabling them to make informed treatment decisions for their GIST patients in a timely and effective manner," said Jonathan Arnold, Vice President, Head of Translational Science and Precision Diagnostics at QIAGEN. "This latest approval confirms QIAGEN’s leadership in companion diagnostics development. It adds to QIAGEN’s extensive list of now 12 FDA-approved companion diagnostics."

GIST is a rare, genomically driven sarcoma of the gastrointestinal tract. Approximately six percent of patients with newly diagnosed GIST have PDGFRA exon 18 mutations, the most common of which is the D842V mutation[2]. Since the introduction of TKIs, including AYVAKIT, the treatment of GIST patients with locally advanced and metastatic disease has dramatically improved[3].

QIAGEN is a pioneer in precision medicine and the leader in collaborating with pharmaceutical and biotechnology companies to develop companion diagnostics. These can detect genetic abnormalities to provide insights that guide clinical decision-making about treatments. From polymerase chain reaction (PCR) and digital PCR (dPCR) to next-generation sequencing (NGS), QIAGEN offers an unmatched breadth of technologies, which means it can tailor products to partners’ needs.

QIAGEN has master collaboration agreements to develop and commercialize companion diagnostics with more than 30 global companies – a deep pipeline that will advance so-called precision medicine, which tailors a patient’s treatment to the genetic profile identified by companion diagnostics testing. Furthermore, QIAGEN has recently announced a series of collaborations with Neuron23 and Helix to develop companion diagnostics in disease areas outside oncology.

AYVAKIT is a registered trademark of Blueprint Medicines. Full prescribing information for AYVAKIT can be found here.

On August 7, 2023 Prime Medicine, Inc. (Nasdaq: PRME), a biotechnology company committed to delivering a new class of differentiated one-time curative genetic therapies, reported financial results and provided business updates for the second quarter ended June 30, 2023 (Press release, Prime Medicine, AUG 7, 2023, View Source [SID1234633880]).

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!

"In recent months, we continued to advance our diversified portfolio of Prime Editing programs while also executing against a strategic partnering strategy aimed at further expanding the broad therapeutic potential of Prime Editing," said Keith Gottesdiener, M.D., President and Chief Executive Officer of Prime Medicine. "PM359, our product candidate for the treatment of CGD, is progressing well, and at the ASGCT (Free ASGCT Whitepaper) Annual Meeting in May, we presented new preclinical data demonstrating its ability to reproducibly correct the CGD disease-causing mutation in CD34+ cells ex vivo with no detectable off-target editing. These findings further support our belief in the potential of this program to change the trajectory of this recurrent debilitating condition, and we look forward to sharing additional in vitro and in vivo data on this program and others later this year."

Dr. Gottesdiener continued, "Also in the second quarter, we entered into a research collaboration with Cimeio Therapeutics, gaining access to Cimeio’s powerful CD117 immunotherapy technology for genetic diseases. We are pleased to be working together to evaluate combining Prime Editing enabled protective shielding with multiplexed therapeutic edits to potentially reduce the toxicity and increase the efficiency of existing HSC transplant regimens. These combined technologies may enable us to more gently and effectively treat a wider range of genetic diseases than currently possible, and future applications may include selection of in vivo edited HSCs, which could allow for the treatment of genetic diseases without transplantation. This partnership reflects the tremendous breadth of our Prime Editing technology, as well as our commitment to leveraging its versatility, precision and efficiency to improve the care and treatment of patients worldwide."

Recent Business Updates

Pipeline

•Prime Medicine continued to advance its strategic pipeline of eighteen programs and remained on track to initiate investigational new drug (IND)-enabling studies of PM359, its development candidate for chronic granulomatous disease (CGD), in 2023.
•In May 2023, Prime Medicine presented new preclinical data at the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 26th Annual Meeting. These data further demonstrated the potential for Prime Editing to correct the causative mutation of CGD and showcased the potential application of the Prime Assisted Site-Specific Integrase Gene Editing (PASSIGE) platform to generate multiplex-edited CAR-T cells for the treatment of certain cancers and immune diseases. Read the full data here.

Corporate

•In June 2023, Prime Medicine announced a research collaboration with Cimeio Therapeutics to develop Prime Edited Shielded Cell and Immunotherapy Pairs (SCIP) for genetic diseases, acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS). The goal of the research is to reduce the toxicity of

conditioning regimens and introduce new therapeutic options to meaningfully expand the utility of hematopoietic stem cell (HSC) transplant and enable the in vivo selection of edited HSCs to potentially remove the need for transplantation entirely. Under the terms of the agreement, Prime Medicine will develop a Prime Editor for Cimeio’s CD117 shielding variant that will then be evaluated by both companies; if the research collaboration is successful, the companies will grant exclusive license options to each other for their technologies. If the companies exercise their exclusive license options, they will each be eligible to receive economics on net sales of licensed products.

Anticipated Upcoming Milestones

Prime Medicine expects the following activities and next steps to drive the Prime Editing platform forward:

Pipeline

•Initiate investigational new drug (IND)-enabling studies for PM359 in CGD in 2023.
•Expand preclinical proof-of-concept in vivo data, with plans to share data from in vivo rodent studies and large animal studies from several programs in the second half of 2023.
•Share in vitro preclinical data in additional liver, eye and neuromuscular programs.
•Complete first IND filing as early as 2024 and additional IND filings anticipated in 2025.

Platform

•Continue to develop and optimize non-viral and viral delivery systems and share additional proof-of-concept data from in vivo rodent and large animal studies in the second half of 2023.
•Further demonstrate superior off-target profiles for Prime Editing programs.
•Expand Prime Editing using proprietary recombinase technologies for new and existing programs.
•Maximize Prime Editing’s broad therapeutic potential and create value through strategic business development that extends the reach and impact of Prime Editing to areas beyond Prime Medicine’s current areas of focus.

Second Quarter 2023 Financial Results:

•R&D Expenses: Research and development (R&D) expenses were $34.6 million for the three months ended June 30, 2023, as compared to $18.9 million for the three months ended June 30, 2022. This increase was primarily due to increases in lab supplies, personnel, and facilities costs as the company continues to expand and build out its R&D activities and function.
•G&A Expenses: General and administrative (G&A) expenses were $10.7 million for the three months ended June 30, 2023, as compared to $7.4 million for the three months ended June 30, 2022. This increase was primarily due to an increase in professional and consultant costs and personnel costs primarily attributable to the build-out of the company’s G&A team to support our R&D function.
•Net Loss: Net loss was $42.4 million for the three months ended June 30, 2023, as compared to $29.3 million for the three months ended June 30, 2022.
•Cash Position: As of June 30, 2023, cash, cash equivalents, investments and restricted cash were $221.1 million, as compared to $263.0 million as of March 31, 2023.

Financial Guidance

Based on its current operating plans, Prime Medicine expects that its cash, cash equivalents and investments as of June 30, 2023, will be sufficient to fund its anticipated operating expenses and capital expenditure requirements into 2025.

On August 7, 2023 Astellas Pharma Inc. (TSE: 4503, President and CEO: Naoki Okamura, "Astellas") and Poseida Therapeutics, Inc. (NASDAQ: PSTX, CEO: Mark Gergen, "Poseida") reported a strategic investment to support the advancement of Poseida’s commitment to redefining cancer cell therapy (Press release, Poseida Therapeutics, AUG 7, 2023, View Source [SID1234633879]).

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 transaction agreements, Astellas will invest a total of $50 million, including $25 million to acquire 8,333,333 shares of common stock of Poseida (approximately 8.8% of the outstanding common stock of Poseida) at $3.00 per share in a private placement and a one-time $25 million payment for a right of exclusive negotiation and first refusal to license one of Poseida’s clinical stage programs: P-MUC1C-ALLO1, an allogeneic CAR-T cell therapy in development for multiple solid tumor indications. In addition, Poseida has granted Astellas a board observer seat, which includes the ability to attend Poseida’s scientific advisory board meetings, and certain notice rights related to any potential change of control of Poseida.

Astellas has established the Focus Area Approach for its research and development strategy. One of Astellas’ Primary Focuses within the strategy is immuno-oncology. Astellas is committed to developing next-generation immuno-oncology drugs using multi-functional platforms. Its portfolio includes oncolytic viruses, bispecific immune cell engagers, small molecules, and cell therapy platforms.

Poseida is engaged in the research and development of cell and gene therapies for cancer and rare genetic diseases by leveraging its proprietary genetic editing platforms. In oncology, Poseida has a broad pipeline of allogeneic CAR-T cell therapy product candidates for both solid and liquid tumors, including P-MUC1C-ALLO1, which is in Phase 1 development for the treatment of multiple solid tumor indications.

"By leveraging our extensive expertise, experience in cancer biology and unique technologies, we are focused on reinvigorating the immune system’s ability to discover, disarm and destroy cancers in more patients. We are pursuing this ambitious goal through innovative and multifunctional modality platforms, using the capabilities at our global R&D sites as well as through partnership with external expert partners," said Adam Pearson, Chief Strategy Officer, Astellas.

"We believe that this investment fits strategically with our long-term vision of expanding our capability in immuno-oncology and will ultimately lead to the development of new therapeutics for patients in need of cancer immunotherapy."

"We are excited to enter this strategic relationship with Astellas, a premier biopharmaceutical company that shares our long-term vision that cell and gene therapies represent an exciting growth area for the development of innovative medicines for improving patient care," said Mark Gergen, Poseida’s Chief Executive Officer. "This investment further validates the potential of our proprietary genetic engineering technology platform and cell therapy approach, and we look forward to working with Astellas to advance our shared vision and explore future opportunities for collaboration to further unlock that potential."

About P-MUC1C-ALLO1

P-MUC1C-ALLO1 is an allogeneic CAR-T product candidate in Phase 1 development for multiple solid tumor indications. Poseida believes P-MUC1C-ALLO1 has the potential to treat a wide range of solid tumors derived from epithelial cells, such as breast, ovarian, colorectal, lung, pancreatic and renal carcinomas, as well as other cancers expressing a cancer-specific form of the Mucin 1 protein, or MUC1-C. P-MUC1C-ALLO1 is designed to be fully allogeneic, with genetic edits to eliminate or reduce both host-vs-graft and graft-vs-host alloreactivity. Poseida has demonstrated the elimination of tumor cells to undetectable levels in preclinical models of both breast and ovarian cancer. Additional information about the Phase 1 study is available at www.clinicaltrials.gov using identifier: NCT05239143.

On August 7, 2023 Keros Therapeutics, Inc. ("Keros" or the "Company") (Nasdaq: KROS), a clinical-stage biopharmaceutical company focused on developing and commercializing novel therapeutics to treat a wide range of patients with disorders that are linked to dysfunctional signaling of the transforming growth factor-beta ("TGF-ß") family of proteins, reported a business update and announced financial results for the quarter ended June 30, 2023 (Press release, Keros Therapeutics, AUG 7, 2023, View Source [SID1234633877]).

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!

"We are excited to share our initial development plans for KER-065—an activin receptor ligand trap that is designed to increase skeletal muscle and bone mass, increase fat metabolism and reduce fibrosis—in Duchenne muscular dystrophy ("DMD"), a debilitating disease with a serious unmet need, and look forward to moving this important program into the clinic in the first quarter of 2024," said Jasbir S. Seehra, Ph.D., President and Chief Executive Officer of Keros. "As highlighted by the progression of KER-065 and the initiation of our Phase 2 clinical trial of KER-012 in patients with pulmonary arterial hypertension ("PAH"), we continue to execute on our strategy of developing our pipeline of differentiated assets designed to harness the powerful biology of the TGF-ß family of proteins."

Recent Program Highlights

•KER-050 for the treatment of ineffective hematopoiesis to address cytopenias
◦Following recommendation by the Safety Review Committee, dosing for Cohort 4 of Keros’ ongoing Phase 2 clinical trial of KER-050 in patients with myelofibrosis-associated cytopenias was initiated at 4.5 mg/kg in both combination and monotherapy arms. Keros expects to announce dose escalation data from and initiate Part 2 of this trial in the second half of 2023.
•KER-047 for the treatment of functional iron deficiency
◦Keros initiated an open-label Phase 2 clinical trial in myelodysplastic syndromes ("MDS") and myelofibrosis patients with functional iron deficiency. The Company expects to report initial data from this trial in the first half of 2024.
•KER-012 for the treatment of PAH and for the treatment of cardiovascular disorders
◦In July 2023, Keros announced that the U.S. Food and Drug Administration cleared the Company’s investigational new drug application to conduct a randomized, double-blind, placebo-controlled, global Phase 2 clinical trial, which Keros refers to as the TROPOS trial, of KER-012 in combination with background therapy in patients with PAH.
◦The Company is hosting a conference call and webcast tomorrow, August 8, 2023, at 8:00 a.m. Eastern time to provide an overview of the TROPOS trial design.
•KER-065 for the treatment of neuromuscular disease
◦KER-065, which was nominated out of Keros’ proprietary library of activin receptor type II ligand traps, is being developed for the treatment of neuromuscular diseases, with an initial focus on DMD.
◦The Company plans to commence a randomized, double-blind, placebo-controlled, two-part Phase 1 clinical trial to evaluate the safety and tolerability of single and multiple ascending doses of KER-065 in healthy volunteers in the first quarter of 2024.

Second Quarter 2023 Financial Results

Keros reported a net loss of $37.5 million in the second quarter of 2023 as compared to a net loss of $27.3 million in the second quarter of 2022. The increase of $10.2 million for the second quarter was largely due to increased research and development efforts as well as additional investments to support the achievement of Keros’ clinical and corporate goals.

Research and development expenses were $32.5 million for the second quarter of 2023 as compared to $23.3 million for the same period in 2022. The increase of $9.3 million was primarily due to additional research and development efforts, manufacturing activities and personnel expenses to support the advancement of Keros’ pipeline.

General and administrative expenses were $8.8 million for the second quarter of 2023 as compared to $7.4 million for the same period in 2022. The increase of $1.4 million was primarily due to increase in personnel expenses and other external expenses to support Keros’ organizational growth.

Keros’ cash and cash equivalents as of June 30, 2023 was $322.0 million compared to $279.0 million as of December 31, 2022. Keros expects that the cash and cash equivalents it had on hand at June 30, 2023 will enable Keros to fund its operating expenses and capital expenditure requirements into the fourth quarter of 2025.