On February 22, 2024 Veracyte, Inc. (Nasdaq: VCYT) reported financial results for the fourth quarter and full year ended December 31, 2023 (Filing, 3 mnth, DEC 31, Veracyte, 2024, FEB 22, 2024, View Source [SID1234640375]).

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 closed 2023 with another quarter of excellent results, driven by our Afirma and Decipher businesses," said Marc Stapley, Veracyte’s chief executive officer. "Looking to 2024 and beyond, we will leverage our Veracyte Diagnostics Platform to continue to drive near- and long-term revenue. With multiple growth catalysts over the coming years and with our strong financial discipline, we expect to achieve positive cash flow for the third consecutive year and going forward."

Key Business Highlights
•Grew total test volume to 33,836 in the fourth quarter and 126,977 for the full year 2023, an increase of 21% and 24%, respectively, compared to the prior year periods.
•Enhanced our Endocrinology offering in 2023 with the launch of TERT promoter mutation testing for Afirma, the introduction of our Afirma GRID research tool, and enhancements to our online physician ordering portal.
•Continued to strengthen the body of evidence for Decipher Prostate in 2023, adding 8 publications and 16 abstracts to the clinical-evidence library, reinforcing Decipher’s status as the only molecular test to receive Level 1 evidence designation in the National Comprehensive Cancer Network’s (NCCN) prostate cancer guidelines.
•Acquired C2i Genomics Inc., adding whole-genome minimal residual disease (MRD) capabilities to our novel Veracyte Diagnostics Platform and expanding our ability to serve patients across the cancer care continuum.
•Signed an agreement with Illumina to develop some of our tests as in vitro diagnostics (IVDs) for use on their NextSeq 550Dx next-generation sequencing (NGS) instrument, as part of our multi-platform strategy to accelerate global expansion of our tests as IVDs.
•Published the clinical validation study for our Percepta Nasal Swab test in CHEST.
•For the full year, generated $44 million of cash from operations and ended the year with $216 million of cash and cash equivalents.

Fourth Quarter 2023 Financial Results
Total revenue for the fourth quarter of 2023 was $98.2 million, an increase of 22% compared to $80.3 million reported in the fourth quarter of 2022. Testing revenue was $90.4 million, an increase of 29% compared to $70.3 million in the fourth quarter of 2022, driven primarily by the strong performance of our Decipher Prostate and Afirma tests. Product revenue was $3.7 million, an increase of 13% compared to $3.2 million in the fourth quarter of 2022. Biopharmaceutical and other revenue was $4.1 million, a decrease of 39% compared to $6.8 million in the fourth quarter of 2022.

Total gross margin for the fourth quarter of 2023, including the amortization of acquired intangible assets, was 66%, compared to 61% in the fourth quarter of 2022. Non-GAAP gross margin, excluding the amortization of acquired intangible assets and other acquisition related expenses was 70%, compared to 67% in the fourth quarter of 2022.

Operating expenses, excluding cost of revenue, were $100.3 million, which included an impairment charge of $32.0 million associated with the impairment of HalioDx developed biopharmaceutical services technology,

customer relationships and customer backlog finite-lived intangible assets, compared to $54.1 million in the fourth quarter of 2022. Non-GAAP operating expenses, which excludes cost of revenue, amortization of acquired intangible assets, impairment charges, other acquisition related expenses and other restructuring costs, grew 28% to $65.6 million, including a technology access fee of $3.5 million to develop our IVD kitted tests on an NGS platform, compared to $51.1 million in the fourth quarter of 2022.

Net loss for the fourth quarter of 2023 was $28.3 million, and basic and diluted net loss per common share was $0.39, which includes the aforementioned $32.0 million impairment charge. Net cash provided by operating activities in the fourth quarter of 2023 was $15.6 million, an improvement of $5.8 million compared to the same period in 2022.

A reconciliation of GAAP to non-GAAP financial measures has been provided in the tables included in this press release. An explanation of these measures is also included below under the heading "Note Regarding Use of Non-GAAP Financial Measures."

Full Year 2023 Financial Results
Total revenue for 2023 was $361.1 million, an increase of 22% compared to $296.5 million in 2022. Testing revenue was $326.5 million, an increase of 30% compared to $250.5 million in 2022 driven primarily by the strong performance of our Decipher and Afirma tests. Product revenue was $15.6 million, an increase of 23% compared to $12.6 million in 2022. Biopharmaceutical and other revenue was $18.9 million, a decrease of 43% compared to $33.4 million in 2022, driven by the reduction of customer projects given overall spending constraints across the industry.

Total gross margin for the full year 2023, including the amortization of acquired intangible assets, was 64%, compared to 59% in 2022. Non-GAAP gross margin, excluding the amortization of acquired intangible assets and other acquisition related expenses was 69%, compared to 66% in 2022.

Operating expenses, excluding cost of revenue, were $315.5 million, an increase of 46% compared to $216.8 million in 2022. Non-GAAP operating expenses, which excludes cost of revenue, amortization of acquired intangible assets, impairment charges, other acquisition related expenses and other restructuring costs, grew 20% to $240.7 million compared to $200.3 million in 2022.

Net loss for the full year 2023 was $74.4 million,and basic and diluted net loss per common share was $1.02,which includes impairment charges of $68.3 million in the year. Net cash provided by operating activities in 2023 was $44.2 million, an increase of $36.7 million compared to 2022.

A reconciliation of GAAP to non-GAAP financial measures has been provided in the tables included in this press release. An explanation of these measures is also included below under the heading "Note Regarding Use of Non-GAAP Financial Measures."

2024 Financial Outlook
The company is maintaining full-year 2024 total revenue guidance of $394 million to $402 million and consistent with prior guidance, expects cash, cash equivalents and short-term investments at the end of the year to be $230 million to $234 million.

Conference Call and Webcast Details
Veracyte will host a conference call and webcast today at 4:30 p.m. Eastern Time to discuss the company’s financial results and provide a general business update. The conference call will be webcast live from the company’s website and will be available via the following link: View Source The webcast should be accessed 10 minutes prior to the conference call start time. A replay of the webcast will be available for one year following the conclusion of the live broadcast and will be accessible on the company’s website at View Source

The conference call dial-in can be accessed by registering at the following link: https://register.vevent.com/register/BI38de228ea0a04e0ab786cfe56be5ca1b

On February 21, 2024 Ono Pharmaceutical Co., Ltd. (Osaka, Japan; President and CEO: Gyo Sagara; "Ono") reported that it has entered into a drug discovery collaboration agreement with Epsilon Molecular Engineering, INC (Saitama, Japan; President and CEO: Naoto Nemoto; "EME"), to generate novel VHH antibodies, aiming at the creation of innovative VHH antibody drugs (Press release, Ono, FEB 21, 2024, View Source [SID1234646260]).

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 this agreement, EME will obtain novel humanized VHH antibodies against multiple targets selected by Ono, by leveraging EME’s proprietary humanized VHH screening platform, "The Month". Ono will conduct various tests to evaluate the activities (in vitro and in vivo assay) of humanized VHH antibodies obtained by EME to discover and develop antibody drug candidates.
 Ono will hold option rights to exclusively develop and commercialize the antibody drug candidates generated through the collaboration worldwide. Ono will pay to EME an upfront payment, and milestone payments based on the progress of research and clinical development.

About EME’s VHH screening platform "The Month"
 "The Month" is a high throughput antibody screening platform based on cDNA display technology. By combining that with EME’s proprietary humanized VHH antibody artificial library, PharmaLogical Library, EME can acquire a large number of VHH antibodies in about one month against tough targets that are difficult to obtain with conventional methods. This library has a vast size ranging from 10 trillion to 100 trillion and is specifically designed to reflect the structural features of VHH antibodies, demonstrating antigen recognition distinct from conventional antibodies.

ON February 21, 2024 Blacksmith Medicines, Inc. (Blacksmith), a leading biopharma dedicated to discovering and developing medicines targeting metalloenzymes, reported the release of $3.3M from its contract with the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health (NIH), Department of Health and Human Services (HHS), to conduct IND-enabling and Phase 1 studies of FG-2101, its novel non-hydroxamate LpxC inhibitor, using both intravenous (IV) and oral administration for the treatment of urinary tract infections caused by multidrug-resistant Gram-negative bacteria (Press release, Blacksmith Medicines, FEB 21, 2024, View Source [SID1234643532]).

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

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

                  Schedule Your 30 min Free Demo!

The company’s recent successful completion of certain Base Period activities including preclinical toxicology studiestriggered the release of this award under its contract with NIAID (75N93022C00060). The total funding could be up to $17.2M over five years, depending on contract options exercised. The new funding will be used for GMP clinical material manufacturing and IND preparation/submission.

"With support from NIAID, we have successfully completed preclinical toxicology studies including 7-day and 14-day repeat dose assessments (non-GLP and GLP) and safety pharmacology evaluations including cardiovascular, respiratory and CNS assessments of our novel non-hydroxamate LpxC inhibitor FG-2101," said Andrew Tomaras, Ph.D., Senior Vice President of Blacksmith. "Next, with the achievement of these safety studies, we will initiate the preparation of an investigational new drug (IND) application as well as the manufacturing campaign of GMP material for upcoming clinical studies. We are extremely grateful to the NIAID team for their continued support for the candidacy of our potential first-in-class antibacterial agent."

About LpxC

LpxC, a zinc metalloenzyme, is an attractive and highly sought-after antibiotic target – it is conserved across Gram-negative bacteria and not found in Gram-positive bacteria or human cells. Inhibiting LpxC results in potent killing of Gram-negative bacteria with the presumed benefit of sparing Gram-positive bacteria such as those residing in the protective microbiome of the gut which help to deter opportunistic C. difficile infections.

Other LpxC inhibitors have been evaluated by biopharma in the past but chemistry limitations (e.g. hydroxamic acid) have yielded ineffective compounds that suffer from poor drug-like properties. Thus, there are no approved therapeutics targeting LpxC. Blacksmith, using its proprietary chemistry platform, has developed novel non-hydroxamate inhibitors of LpxC that are safe and effective in animal models of Gram-negative infection and are able to kill Gram-negative ‘superbugs’ where other antibiotics are ineffective.

About metalloenzymes and the Blacksmith platform

Metalloenzymes utilize a metal ion cofactor in the enzyme active site to perform essential biological functions. This diverse class of targets has historically been difficult to drug due to small molecule chemistry limitations that have plagued the industry. The Blacksmith metalloenzyme platform has solved this problem by leveraging the following:

A large proprietary fragment library of metal-binding pharmacophores (MBPs);
A comprehensive database containing a full characterization of the metalloenzyme genome including functions, metal cofactors, and associations to disease;
A first-of-its-kind metallo-CRISPR library of custom single guide RNAs;
An industry-leading metalloenzyme computational toolkit for docking, modeling and structure-based drug design; and
A robust and blocking intellectual property estate covering bioinorganic, medicinal, and computational chemistry approaches for metalloenzyme-targeted medicines.

On February 21, 2024 Aleta Biotherapeutics (Aleta), a clinical stage, immuno-oncology company with a CAR T-Cell Engager (CTE) platform that enables cell cancer therapies to work more effectively, and Cancer Research UK’s Centre for Drug Development, reported the first patient was dosed in a Phase 1/2 clinical trial (Press release, Aleta Biotherapeutics, FEB 21, 2024, View Source [SID1234640353]). This trial is evaluating the Company’s first-in-class biologic CAR T-Cell Engager, ALETA-001, for the treatment of patients with B-cell malignancies who are relapsed/refractory to CD19-targeting CAR T-cell therapy.

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!

Cancer Research UK’s Centre for Drug Development is sponsoring and conducting the Phase 1/2 clinical trial of ALETA-001, the lead agent in Aleta’s portfolio.

"It is very exciting and meaningful to have ALETA-001 now in the clinic. Clinical evaluation of ALETA-001 is a key milestone toward a much-needed treatment option for the many cancer patients whose CD19-targeted CAR T-cell therapies ultimately stop working. ALETA-001 restores and increases the effectiveness with which CAR T-cells can kill cancer cells, and we believe that it will enable more patients to successfully benefit from cell therapies," stated Dr. Paul Rennert, President and Chief Scientific Officer, Aleta Biotherapeutics.

The ALETA-001 Phase 1/2 clinical trial is an open-label, dose-expansion trial which will evaluate safety and tolerability, pharmacokinetic and pharmacodynamic effects, and early signals of clinical efficacy of ALETA-001 as a single agent. Chief Investigator Dr. Sridhar Chaganti is leading the trial from University Hospital Birmingham NHS Foundation Trust in Birmingham, UK, and will enroll patients who are relapsed/refractory following treatment with available CD19-targeting CAR T-cell therapies. For more information on this trial, please visit clinicaltrials.gov (NCT06045910)

Dr. Lars Erwig, Cancer Research UK’s Director of Drug Development, said, "A significant number of patients with blood cancers unfortunately relapse following CD19-directed CAR T-cell therapy. ALETA-001 is being developed to provide patients with these cancers a better chance for a successful treatment outcome. At Cancer Research UK, we are very excited to study the clinical potential of ALETA-001 to transform a patient’s treatment journey – which can possibly be lifesaving in many cases."

"Aleta-001 is a uniquely designed new molecule with a novel mechanism of action, coating tumor cells densely with the target antigen, thereby stimulating the tumor killing ability of activated CAR T-cells," commented Dr. Sridhar Chaganti, Chief Investigator, University Hospital Birmingham NHS Foundation Trust, Birmingham, UK. "This is an important new strategy being investigated to improve outcomes for patients with relapsed or refractory lymphomas who experience disease progression after CAR T-cell therapy and have a poor prognosis."

About CAR T-Cell Therapy Engager (CTE) ALETA-001

Aleta’s lead clinical stage program, ALETA-001, was designed specifically for the treatment of B-cell malignancies in patients who have received a CD19-directed CAR T-cell therapy and are at risk of treatment failure. Developed to improve the effectiveness of CD19-directed CAR T-cell therapies by increasing CD19 antigen density and restoring lost CD19 expression on the cancer cell, ALETA-001 contains the CD19 target protein which is further linked to an CD20 antibody domain. This allows CD19+/CD20+ cancer cells to be easily recognized and killed by CD19-directed CAR T-cells that were previously administered and are already circulating within a patient.

Aleta previously secured landmark clinical support and funding from Cancer Research UK for the ALETA-001 Phase 1/2 clinical trials, and ALETA-001 has received a UK Innovation Passport under the Innovative Licensing and Access Pathway (ILAP) from the U.K. Medicines and Healthcare products Regulatory Agency (MHRA). ILAP designation is granted to medicines that address life-threatening or seriously debilitating conditions, and where there exists a significant patient or public health need.

On February 21, 2024 Oncoinvent AS, a clinical stage company advancing alpha emitter therapy across a variety of solid cancers, reported that members of management will present at the Evercore ISI 2024 Emerging Biotech Conference on Wednesday, February 28, 2024 at 11:20 a.m. ET (Press release, Oncoinvent, FEB 21, 2024, View Source [SID1234640352]).

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!