On May 4, 2023 Veracyte, Inc. (Nasdaq: VCYT) reported financial results for the first quarter ended March 31, 2023 (Press release, Veracyte, MAY 4, 2023, View Source [SID1234631060]).

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"We had an excellent first quarter, as many areas of our business greatly exceeded our expectations. Not only did we deliver strong Afirma and Decipher growth, we also demonstrated continued financial discipline and outstanding execution, which resulted in a meaningfully higher cash balance than we had projected," said Marc Stapley, Veracyte’s chief executive officer. "In addition, we further enhanced our Afirma test, as well as augmented the clinical evidence in support of adoption and reimbursement of our products, ultimately helping to advance our vision of transforming cancer care for patients all over the world."

Key Business Highlights

Increased first quarter total revenue by 22% to $82.4 million, compared to the first quarter of 2022.
Grew total test volume to 28,788, an increase of 24% compared to the first quarter of 2022.
Enhanced our Afirma offering with the addition of TERT promoter mutation testing to help further guide treatment decisions for patients with suspected or diagnosed thyroid cancer.
Secured four new payer contracts for Afirma GSC, making the test an in-network benefit for over four million additional health plan enrollees.
Presented 17 abstracts for our diagnostic tests and biopharmaceutical offerings at major medical and research conferences.
Published multiple papers that demonstrate the clinical utility of our Decipher Prostate Genomic Classifier and advance the science around prostate cancer.
Published our inaugural Environmental, Social and Governance report.
Ended the first quarter with cash, cash equivalents and short-term investments of $177.9 million, roughly flat to $178.9 million at the end of 2022.
First Quarter 2023 Financial Results

Total revenue for the first quarter of 2023 was $82.4 million, an increase of 22% compared to $67.8 million in the first quarter of 2022. Testing revenue was $72.4 million, an increase of 29% compared to $56.0 million in the first quarter of 2022 driven primarily by the strong performance of our Decipher Prostate and Afirma tests. Product revenue was $3.9 million, an increase of 31% compared to $3.0 million in the first quarter of 2022. Biopharmaceutical and other revenue was $6.1 million, a decrease of 30% compared to $8.8 million in the first quarter of 2022.

Total gross margin for the first quarter of 2023, including the amortization of acquired intangible assets, was 62%, compared to 58% in the first quarter of 2022. Non-GAAP gross margin, excluding the amortization of acquired intangible assets and other acquisition related expenses was 68%, compared to 65% in the first quarter of 2022.

Operating expenses, excluding cost of revenue, were $61.9 million, an increase of 14% compared to the first quarter of 2022. Non-GAAP operating expenses, excluding cost of revenue, amortization of acquired intangible assets, other acquisition related expenses and other restructuring costs, were $58.1 million compared to $49.1 million in the first quarter of 2022.

Net loss for the first quarter of 2023 was $8.1 million, an improvement of 44% compared to the first quarter of 2022. Basic and diluted net loss per common share was $0.11, an improvement of 45% compared to the first quarter of 2022. Net cash used by operating activities in the first three months of 2023 was $2.2 million, an improvement of $6.7 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."

2023 Financial Outlook

The company is raising full-year 2023 total revenue guidance to $330 million to $340 million, representing year-over-year growth of 11% to 15%, and an improvement compared to prior guidance of $325 million to $335 million. Further, the updated outlook reflects testing revenue year-over-year growth of mid-to-high teens.

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/BI87218b059133453fb9c7e07391fd40e3

On May 4, 2023 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultrarare genetic diseases, reported its financial results for the quarter ended March 31, 2023 and reaffirmed its financial guidance for 2023 (Press release, Ultragenyx Pharmaceutical, MAY 4, 2023, View Source [SID1234631059]).

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"In the first quarter we continued the strong growth in product revenue and advanced our clinical programs including completion of the Phase 2 portion of our pivotal study of UX143 in osteogenesis imperfecta. We are analyzing the data and look forward to sharing the results soon. Once completed, we will initiate enrollment of the Phase 3 portion in mid-2023," said Emil D. Kakkis, M.D., Ph.D., chief executive officer and president of Ultragenyx. "We are also making rapid progress in our Phase 1/2 study of GTX-102 in Angelman syndrome, dosing in the expansion cohorts and activating sites in new countries."

First Quarter 2023 Selected Financial Data Tables and Financial Results

Revenues (dollars in thousands), (unaudited)
Three Months Ended March 31,
2023 2022
Crysvita
Revenue in profit-share territory $ 49,906 $ 45,164
Product revenue 21,234 9,394
Non-cash royalty revenue in European territory 4,882 4,838
Total Crysvita revenue 76,022 59,396
Dojolvi 14,303 12,429
Mepsevii 8,480 4,861
Evkeeza 212 —
Daiichi Sankyo 1,479 3,249
Total revenues $ 100,496 $ 79,935

Net Revenues
Ultragenyx reported $100.5 million in total revenue for the first quarter of 2023, which represents 26% growth compared to the first quarter 2022. The growth is primarily driven by increased demand for Crysvita in Latin America and steady growth from the other regions and products. The first quarter 2023 also included $1.5 million of revenue from the technology transfer which was completed during the quarter as part of the collaboration and license agreement with Daiichi Sankyo.

Selected Financial Data (dollars in thousands, except per share amounts), (unaudited)
Three Months Ended March 31,
2023 2022
Total revenues $ 100,496 $ 79,935
Operating expense:
Cost of sales 12,257 6,100
Research and development 165,698 143,155
Selling, general and administrative 76,646 67,312
Total operating expense 254,601 216,567
Net loss $ (163,972 ) $ (152,320 )
Net loss per share, basic and diluted $ (2.33 ) $ (2.19 )

Operating Expenses
Total operating expenses for the first quarter of 2023 were $254.6 million, including non-cash stock-based compensation of $31.9 million. In 2023, annual operating expenses are expected to decrease as the company manages headcount and increases operational leverage while executing on high-value programs.

Net Loss
For the first quarter of 2023, Ultragenyx reported net loss of $164.0 million, or $2.33 per share basic and diluted, compared with a net loss for the first quarter of 2022 of $152.3 million, or $2.19 per share, basic and diluted.

Cash, Cash Equivalents and Marketable Debt Securities
Cash, cash equivalents, and marketable debt securities were approximately $714.6 million as of March 31, 2023.

2023 Financial Guidance
For the full year 2023, the company reaffirms:

Total revenue in the range of $425 million to $450 million
Crysvita revenue in the range of $325 million to $340 million. This includes all regions where Ultragenyx will recognize revenue, including the royalties in Europe, which have been ongoing, and the royalties in North America, which began in April 2023.
Dojolvi revenue in the range of $65 million to $75 million
Net Cash Used in Operations is expected to be less than $400 million
Recent Updates and Clinical Milestones

UX143 (setrusumab) monoclonal antibody for Osteogenesis Imperfecta (OI): Phase 2 enrollment complete; Phase 2 data and Phase 3 initiation expected in mid-2023
Ultragenyx is continuing to dose patients in the Phase 2/3 Orbit study of UX143 in pediatric and adult patients with OI aged five to <26 years. All patients have completed the Phase 2 portion of the study with data expected in mid-2023. These data are expected to include changes in bone biomarkers response and some data on bone mineral density that will be used to establish the dosing algorithm for the Phase 3 portion of the study. The Phase 3 is expected to initiate in mid-2023.

In addition, in the second quarter of 2023, Ultragenyx intends to initiate Cosmic, a randomized study in children under age five with serious bone disease, which compares bisphosphonates to UX143. Younger pediatric patients with OI often have a much higher fracture rate than other age groups driving clinical urgency for better treatment options. The primary endpoint is expected to be the annualized rate of fractures.

GTX-102 antisense oligonucleotide for Angelman syndrome: Phase 1/2 dosing in the expansion cohorts

Outside of the U.S., the dose escalation phase of this study has been completed and the company recently transitioned to dosing patients in the expansion cohorts, which are designed to verify the GTX-102 dose and treatment regimen that will be used in the Phase 3 program. The study has also expanded geographically with multiple new sites activated in Europe and Australia. Approximately 40 patients will be enrolled across Cohort A (ages four to

Patients from the dose escalation cohorts continue to exhibit encouraging dose and time-dependent clinical activity following longer-term treatment and maintenance dosing. No additional treatment-related serious adverse events or lower extremity weakness adverse events have occurred since our prior update in January 2023. As of May 2023, 14 patients have had at least six months of exposure to GTX-102 including nine patients with more than one year of continuous therapy. The next data update is expected in the second half of 2023.

In the U.S., patients are continuing to receive GTX-102 at the lower loading and maintenance dose. Productive discussions with the FDA are ongoing to harmonize the dosing strategy with the ex-U.S. protocol.

DTX401 AAV gene therapy for Glycogen Storage Disease Type Ia (GSDIa): Dosing in Phase 3 study complete
Ultragenyx has randomized and dosed the last patient in the Phase 3 study. The 48-week study has fully enrolled patients eight years of age and older, randomized 1:1 to DTX401 or placebo. The primary endpoint is the reduction in oral glucose replacement with cornstarch while maintaining glucose control. Phase 3 data are expected in the first half of 2024.

DTX301 AAV gene therapy for Ornithine Transcarbamylase (OTC) Deficiency: Phase 3 study dosing patients
Ultragenyx is randomizing and dosing patients in the ongoing Phase 3 study. The pivotal, 64-week study will include approximately 50 patients, randomized 1:1 to DTX301 or placebo. The primary endpoints are response as measured by removal of ammonia-scavenger medications and protein-restricted diet and change in 24-hour ammonia levels.

UX701 AAV gene therapy for Wilson Disease: Stage 1 of pivotal clinical study dosing patients; expect interim Stage 1 enrollment completion in the second half of 2023
Dosing in the first stage of the pivotal study is ongoing under an amended protocol that removes placebo from the dose finding stage and includes five patients per cohort. During this stage of the study, safety and efficacy of up to three dose levels of UX701 will be evaluated and a dose will be selected for further evaluation in Stage 2. Completion of Stage 1 enrollment is expected in the second half of 2023 with data expected in the first half of 2024 that would include safety and potentially initial signs of clinical activity.

UX053 mRNA for glycogen storage disease type III (GSDIII): Phase 1/2 single ascending dose cohort enrolled; data in 1H23
Dosing in the single ascending dose stage (SAD) of the Phase 1/2 study of UX053 for the treatment of GSDIII has been completed with no safety issues observed. The company has decided to not enroll patients in the multiple ascending dose cohorts at this time to allow greater focus on other late-stage and larger indication clinical programs. The data from the SAD cohort are being analyzed and are expected in the second quarter of 2023.

Conference Call and Webcast Information

Ultragenyx will host a conference call today, Thursday, May 4, 2023, at 2 p.m. PT/ 5 p.m. ET to discuss the first quarter 2023 financial results and provide a corporate update. The live and replayed webcast of the call will be available through the company’s website at View Source To participate in the live call, please register by clicking on the following link (https://register.vevent.com/register/BI1183803cc36b46baa6367e521e12e605), and you will be provided with dial in details. The replay of the call will be available for one year.

On May 4, 2023 Tyra Biosciences, Inc. (Nasdaq: TYRA), a clinical-stage biotechnology company focused on developing next-generation precision medicines that target large opportunities in Fibroblast Growth Factor Receptor (FGFR) biology, reported financial results for the quarter ended March 31, 2023 and highlighted recent corporate progress (Press release, Tyra Biosciences, MAY 4, 2023, View Source [SID1234631058]).

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"2023 is shaping up to be a great year for TYRA. We believe that our lead precision compound, TYRA-300, is well-positioned to become a best-in-class agent in multiple therapeutic areas where FGFR3 plays a major role. Our SURF301 oncology study is progressing well, and we look forward to providing more detail on our clinical plans for TYRA-300 in achondroplasia," said Todd Harris, CEO of TYRA. "In addition, we continued to advance our broader pipeline, with TYRA-200 expected to enter the clinic in 2H 2023, and additional clinical candidates from our in-house SNÅP discovery engine poised for nomination."

First Quarter 2023 and Recent Corporate Highlights

TYRA-300

•
Advanced SURF301 Phase 1/2 Study for Oncology. TYRA continued to advance its SURF301 Phase 1/2 clinical study of TYRA-300, an investigational agent and a once-daily oral FGFR3-selective inhibitor, with an initial focus on patients with metastatic urothelial carcinoma of the bladder and urinary tract. SURF301 (Study in Untreated and Resistant FGFR3+ Advanced Solid Tumors) (NCT05544552) is a multi-center, open label study designed to determine the optimal and maximum tolerated doses and the recommended Phase 2 dose of TYRA-300, as well as to evaluate the preliminary antitumor activity of TYRA-300. During the first quarter of 2023, TYRA continued to enroll and dose patients in SURF301 at multiple clinical sites.
•
Expanded Development into Achondroplasia. In March 2023, TYRA announced the expansion of development of TYRA-300 into achondroplasia based on positive preclinical results in a study performed in collaboration with the Imagine Institute in Paris, France. TYRA is on track to submit an Investigational New Drug application (IND) to the U.S. Food and Drug Administration (FDA) to enable a Phase 2 study of TYRA-300 in pediatric achondroplasia in 2024.

TYRA-200

•
IND Cleared by FDA; Phase 1 Study on Track for 2H 2023. In March 2023, TYRA announced that the FDA cleared its IND to proceed with a Phase 1 clinical study of TYRA-200, an FGFR1/2/3 inhibitor with potency against activating FGFR2 gene alterations and resistance mutations. The trial will be focused on intrahepatic cholangiocarcinoma resistant to prior FGFR inhibitors. TYRA expects the first patient will be dosed in this trial in the second half of 2023.

SNÅP Platform and Pipeline

•
TYRA continued to advance its in-house precision medicine discovery engine, SNÅP, to develop therapies in targeted oncology and genetically defined conditions including FGF19+/FGR4-driven cancers, and RET (REarranged during Transfection kinase) driven cancers.

First Quarter 2023 Financial Results

•
First quarter 2023 net loss was $11.9 million compared to $14.8 million for the same period in 2022.
•
First quarter 2023 research and development expenses were $10.4 million compared to $9.6 million for the same period in 2022.
•
First quarter 2023 general and administrative expenses were $3.9 million compared to $5.2 million for the same period in 2022.
•
As of March 31, 2023, TYRA had cash and cash equivalents of $241.7 million that will support TYRA through important clinical and operational milestones over at least the next two years.

About TYRA-300

TYRA-300 is the Company’s lead precision medicine program stemming from its in-house SNÅP platform. TYRA-300 is an investigational, oral, FGFR3-selective inhibitor currently in development for the treatment of cancer and skeletal dysplasias including achondroplasia. TYRA-300 is being evaluated in a multi-center, open label Phase 1/2 clinical study, SURF301 (Study in Untreated and Resistant FGFR3+ Advanced Solid Tumors). SURF301 (NCT05544552) was designed to determine the optimal and maximum tolerated doses (MTD) and the recommended Phase 2 dose (RP2D) of TYRA-300, as well as to evaluate the preliminary antitumor activity of TYRA-300. SURF301 is currently enrolling adults with advanced urothelial carcinoma and other solid tumors with FGFR3 gene alterations. In skeletal dysplasias, TYRA-300 has demonstrated positive preclinical results and the Company expects to submit an IND for the initiation of a Phase 2 clinical study in pediatric achondroplasia in 2024.

On May 4, 2023 TG Therapeutics, Inc. (NASDAQ: TGTX) reported that Michael S. Weiss, the Company’s Chairman and Chief Executive Officer, will participate in the Bank of America Securities 2023 Health Care Conference, being held at the Encore Hotel, in Las Vegas, NV on Tuesday May 9th through Thursday May 11th, 2023 (Press release, TG Therapeutics, MAY 4, 2023, View Source [SID1234631057]). The fireside chat is scheduled to take place on Tuesday, May 9, 2023, at 3:00 PM PT.

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A live webcast of the fireside chat will be available on the Events page, located within the Investors & Media section, of the Company’s website at View Source

On May 4, 2023 Surface Oncology (Nasdaq: SURF), a clinical-stage immuno-oncology company developing next-generation immunotherapies that target the tumor microenvironment, reported financial results and corporate highlights for the first quarter 2023, as well as anticipated near-term milestones (Press release, Surface Oncology, MAY 4, 2023, View Source [SID1234631056]).

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"We remain highly encouraged by the progress of our two clinical programs, SRF388 a potential first-in-class IL-27 inhibitor, and SRF114, a potentially best-in-class anti-CCR8 antibody, both of which have been enrolling well," said Rob Ross, M.D., chief executive officer of Surface. "At the recent AACR (Free AACR Whitepaper) Annual Meeting, we presented compelling new SRF114 preclinical data which indicate that therapeutic depletion of CCR8 positive tumor infiltrating Tregs results in robust anti-tumorigenic activity across multiple tumor models. With respect to SRF388 and our ongoing Phase 2 trials in liver and lung cancer, we look forward to providing a clinical data update in both indications later in the second quarter."

First Quarter and Subsequent Corporate Highlights

In April 2023, Surface presented new preclinical data on SRF114, a fully human anti-CCR8 antibody, at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2023 in Orlando. A poster presentation highlighted new in vivo data showing SRF114 promotes a pro-inflammatory tumor microenvironment resulting in robust antitumor activity in checkpoint inhibition-resistant and checkpoint inhibition-susceptible tumor models. The poster also demonstrated that anti-CCR8 therapy resulted in the depletion of Treg cells selectively in tumor tissue and anti-CCR8 and anti-PD-1 combination therapy increased tumor immune cell infiltration, cytokine production and improved overall survival in a checkpoint inhibitor-resistant melanoma model.

In January 2023, Surface announced the first patient had been dosed in a Phase 1/2 study evaluating SRF114 as a monotherapy in patients with advanced solid tumors. The dose escalation portion of the study is ongoing and will enroll up to 30 patients evaluating the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy of SRF114.
Selected Anticipated Near-term Corporate Milestones

In the second quarter, Surface expects to share updated clinical data from the ongoing Phase 2 studies investigating SRF388 as a monotherapy in lung cancer and in combination therapy in liver cancer.
Initial safety and efficacy data from the ongoing SRF114 Phase 1/2 clinical study are anticipated in 2024.
Financial Results

As of March 31, 2023, cash, cash equivalents and marketable securities were $102.1 million, compared to $124.8 million on December 31, 2022.

General and administrative (G&A) expenses were $5.9 million for the first quarter ended March 31, 2023, compared to $6.5 million for the same period in 2022. The decrease primarily relates to personnel-related costs from reduced headcount and a reduction in professional fees. G&A expenses included $1.0 million in stock-based compensation expense for the first quarter ended March 31, 2023.

Research and development (R&D) expenses were $13.8 million for the first quarter ended March 31, 2023, compared to $16.6 million for the same period in 2022. This decrease was primarily driven by a reduction in manufacturing costs for our SRF388 program and the strategic decision to pause the SRF617 program as part of our corporate restructuring in November 2022. R&D expenses included $0.6 million in stock-based compensation expense for the first quarter ended March 31, 2023.

For the first quarter ended March 31, 2023, net loss was $19.7 million, or basic and diluted net loss per share of $0.33. Net income was $6.2 million for the same period in 2022, or basic net income per share of $0.13 and diluted net income per share of $0.13.

Surface Oncology continues to project that current cash and cash equivalents are sufficient to fund the company into the third quarter of 2024.

About SRF388

SRF388 is a fully human anti-IL-27 antibody designed to inhibit the activity of this immunosuppressive cytokine. Surface Oncology has identified particular tumor types, including liver and lung cancer, where IL-27 appears to play an important role in the immunosuppressive tumor microenvironment and may contribute to resistance to treatment with checkpoint inhibitors. SRF388 targets the rate-limiting p28 subunit of IL-27, and preclinical studies have shown that treatment with SRF388 blocks the immunosuppressive biologic effects of IL-27, resulting in immune cell activation in combination with other cancer therapies including anti-PD-1 therapy, as well as potent anti-tumor effects as a monotherapy. Furthermore, Surface Oncology has identified a potential biomarker associated with IL-27 that may be useful in helping to identify patients most likely to respond to SRF388. In November 2020, Surface announced that SRF388 was granted Orphan Drug designation and Fast Track designation for the treatment of refractory hepatocellular carcinoma from the FDA.

About SRF114

SRF114 is a fully human, afucosylated anti-CCR8 antibody designed to preferentially deplete CCR8+ Treg cells within the tumor microenvironment. In preclinical studies, Surface Oncology has shown that SRF114 induces antibody-dependent cellular cytotoxicity (ADCC) and/or antibody-dependent cellular phagocytosis (ADCP) pathways to deplete intratumoral Treg cells. In addition, SRF114 reduced tumor growth in murine models. These findings support the advancement of SRF114 as a therapeutic candidate that holds the potential to drive anti-tumor immunity in patients.