BioXcel Therapeutics Reports Second Quarter 2024 Financial Results

On August 6, 2024 BioXcel Therapeutics, Inc. (Nasdaq: BTAI), a biopharmaceutical company utilizing artificial intelligence to develop transformative medicines in neuroscience and immuno-oncology, reported its financial results for the second quarter of 2024 (Press release, BioXcel Therapeutics, AUG 6, 2024, View Source [SID1234645410]).

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"We are on track with our business priorities as we focus on bringing BXCL501 to the greatest number of patients in need," said Vimal Mehta, Ph.D., CEO of BioXcel Therapeutics. "We are pleased with the progress with our SERENITY and TRANQUILITY programs and our focused market-access strategy for IGALMI. Our confidence in our lead neuroscience asset is underpinned by its broad therapeutic potential across multiple neuropsychiatric conditions and its growing intellectual property portfolio."

Late-Stage Clinical Programs

· SERENITY At-Home* Pivotal Phase 3 Trial: designed to evaluate the safety of a 120 mcg dose of BXCL501 in the at-home setting for agitation associated with bipolar disorders or schizophrenia.

o Recently received feedback on protocol from U.S. Food and Drug Administration (FDA).

· TRANQUILITY In-Care Pivotal Phase 3 Trial: designed to evaluate the efficacy and safety of a 60 mcg dose of BXCL501 for agitation associated with Alzheimer’s dementia (AAD).

o Protocol being finalized for planned submission to FDA.

IGALMI (dexmedetomidine) Sublingual film

Post-marketing Requirement (PMR) Study

· Reported positive topline results from PMR study evaluating PRN (as-needed) treatment of IGALMI for agitation associated with bipolar disorders or schizophrenia.

o Study achieved its objective and demonstrated no evidence of tachyphylaxis, tolerance, or withdrawal with 180 mcg dose (highest approved dose).

o Although this PMR study was not statistically powered to evaluate repeat dose efficacy, a reduction in agitation was observed for each episode occurring during the seven-day study period, and no serious adverse events were reported following treatment.

Commercialization

· IGALMI net revenue grew 90% in Q2 2024 over Q1 2024 driven by focused market-access strategy and increased contracting with psychiatric care clinics and behavioral health facilities using a small commercial team.

Patent Portfolio

The Company continues to strengthen its intellectual property portfolio for IGALMI.

· Recently received a U.S. Patent and Trademark Office (USPTO) Notice of Allowance for U.S. Patent Application No. 18/526,686 for IGALMI. Once issued by the USPTO, the patent is expected to have an expiration date of January 12, 2043, and will be submitted for listing in the FDA’s Approved Drug Products with Therapeutic Equivalence Evaluations (commonly known as the "Orange Book").

· This is expected to be the 11th listed U.S. patent for IGALMI in the Orange Book.

TG Therapeutics Reports Second Quarter 2024 Financial Results and Raises BRIUMVI® (ublituximab-xiiy) Full Year Revenue Guidance

On August 6, 2024 TG Therapeutics, Inc. (NASDAQ: TGTX) (the Company or TG Therapeutics) reported its financial results for the second quarter of 2024, along with recent company developments and provided an update on 2024 revenue guidance (Press release, TG Therapeutics, AUG 6, 2024, View Source [SID1234645426]).

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Michael S. Weiss, the Company’s Chairman and Chief Executive Officer stated, "We are pleased to report another quarter of outperformance across all aspects of our business. From a financial standpoint, our second quarter U.S. BRIUMVI net revenues exceeded expectations, leading us to raise our full year guidance. On the R&D side, we also had an exciting quarter with the first patients now treated with subcutaneous ublituximab in a newly launched Phase 1 study and clearance of our IND for azer-cel, our allogeneic "off-the-shelf" CD19 CAR-T, for patients with progressive MS." Mr. Weiss continued, "We are also excited to announce our new $250 million credit facility with HealthCare Royalty and Blue Owl Capital that enables us to accelerate the initiation of a share repurchase program and pay down our current debt, while preserving our current cash to continue building our commercial infrastructure, ramping up our marketing efforts, and investing in our R&D programs. We look forward to continuing the positive momentum into the second half of 2024."

Recent Highlights & Developments

United States (U.S.) Commercialization of BRIUMVI (ublituximab-xiiy)

BRIUMVI U.S. net product revenue of $72.6 million for the second quarter of 2024, representing >350% growth over the second quarter of 2023

Approximately 5,850 BRIUMVI new patient prescriptions received by the TG Therapeutics hub since launch, from approximately 950 healthcare providers at approximately 525 centers, including more than 1,400 prescriptions received in the second quarter of 2024

Awarded a national contract with the Department of Veterans Affairs (VA) for BRIUMVI to be the preferred anti-CD20 agent listed on the VA National Formulary for patients with relapsing forms of multiple sclerosis (RMS)

Development Updates & General Business

Initiated a phase 1 clinical trial evaluating subcutaneous ublituximab in RMS, with the first patients now dosed

Received clearance by the U.S. Food and Drug Administration (FDA) of an Investigational New Drug (IND) application for azer-cel in progressive forms of multiple sclerosis (MS)

Obtained three additional patents from the United States Patent and Trademark Office (USPTO) for BRIUMVI, extending patent protection through 2042

Corporate Finance Updates

Established a new 5-year, $250 million credit facility with HealthCare Royalty and Blue Owl Capital, set to mature in 2029, primarily to repay $107 million in outstanding debt and accrued interest, which was set to mature in multiple tranches from mid-2025 to January 2026, and to fund the buyback of up to $100 million of currently outstanding shares of the Company’s common stock. The remainder will be available for working capital purposes, providing the Company with additional operational flexibility.

2024 Updated Target U.S. BRIUMVI Guidance

Updating BRIUMVI U.S. net product revenue target to approximately $290 to $300 million for the full year 2024 (prior guidance of $270 to $290 million for full year 2024)

Remaining 2024 Development Pipeline Anticipated Milestones

Study BRIUMVI in an additional autoimmune disease outside of MS

Commence a clinical trial evaluating azer-cel in autoimmune diseases, starting with progressive MS

Present additional data from the ENHANCE Phase 3b CD20 switch trial

Financial Results for Second Quarter 2024

Product Revenue, net: Product revenue, net was approximately $72.6 million and $123.1 million for the three and six months ended June 30, 2024, respectively, compared to $16.0 million and $23.8 million for the three and six months ended June 30, 2023, respectively. Product revenue, net for both the three and six months ended June 30, 2024 and 2023, consisted of net product sales of BRIUMVI in the United States.

License, milestone, royalty and other revenue: License, milestone, royalty and other revenue was approximately $0.9 million and $13.9 million for the three and six months ended June 30, 2024, respectively, compared to less than $0.1 million for both the three and six months ended June 30, 2023, respectively. License, milestone, royalty and other revenue for the six months ended June 30, 2024 is predominantly comprised of a $12.5 million milestone payment under the Neuraxpharm Commercialization Agreement for the first key market commercial launch of BRIUMVI in the European Union (EU) which occurred in the first quarter of 2024.

R&D Expenses: Total research and development (R&D) expense was approximately $17.6 million and $50.3 million for the three and six months ended June 30, 2024, respectively, compared to $28.1 million and $44.0 million for the three and six months ended June 30, 2023, respectively. The decrease in R&D expense during the three months ended June 30, 2024 was primarily attributable to reduced clinical trial related expense and license milestones incurred during the period ended June 30, 2024. The increase in R&D expense during the six months ended June 30, 2024 was primarily attributable to license and milestone expense related to the license agreement with Precision BioSciences, Inc., as well as additional manufacturing and development costs incurred in connection with our ublituximab subcutaneous development work during the period.

SG&A Expenses: Total selling, general and administrative (SG&A) expense was approximately $38.8 million and $73.4 million for the three and six months ended June 30, 2024, respectively, compared to $30.7 million and $58.8 million for the three and six months ended June 30, 2023, respectively. The increase in both periods was primarily due to the scale-up of the BRIUMVI commercial launch, including personnel.

Net Income (Loss): Net income (loss) was $6.9 million and $(3.8) million for the three and six months ended June 30, 2024, respectively, compared to a net loss of $(47.6) million and $(86.8) million for the three and six months ended June 30, 2023, respectively.

Cash Position and Financial Guidance: Cash, cash equivalents and investment securities were $217.3 million as of June 30, 2024, which excludes any increase in cash associated with the new $250 million credit facility. We anticipate that our cash, cash equivalents and investment securities as of June 30, 2024, combined with the projected revenues from BRIUMVI, will be sufficient to fund our business based on our current operating plan.

CONFERENCE CALL INFORMATION
The Company will host a conference call today, August 6, 2024 at 8:30 AM ET to discuss the Company’s financial results from the second quarter ended June 30, 2024.

To participate in the conference call, please call 1-877-407-8029 (U.S.), 1-201-689-8029 (outside the U.S.), Conference Title: TG Therapeutics. A live audio webcast will be available on the Events page, located within the Investors & Media section, of the Company’s website at View Source An audio recording of the conference call will also be available for a period of 30 days after the call.

ABOUT BRIUMVI (ublituximab-xiiy) 150 mg/6 mL Injection for IV
BRIUMVI is a novel monoclonal antibody that targets a unique epitope on CD20-expressing B-cells. Targeting CD20 using monoclonal antibodies has proven to be an important therapeutic approach for the management of autoimmune disorders, such as RMS. BRIUMVI is uniquely designed to lack certain sugar molecules normally expressed on the antibody. Removal of these sugar molecules, a process called glycoengineering, allows for efficient B-cell depletion at low doses.

BRIUMVI is indicated in the U.S. for the treatment of adults with RMS, including clinically isolated syndrome, relapsing-remitting disease, and active secondary progressive disease and in the EU and UK for the treatment of adult patients with RMS with active disease defined by clinical or imaging features.

A list of authorized specialty distributors can be found at www.briumvi.com.

IMPORTANT SAFETY INFORMATION

Contraindications: BRIUMVI is contraindicated in patients with:

Active Hepatitis B Virus infection

A history of life-threatening infusion reaction to BRIUMVI

WARNINGS AND PRECAUTIONS

Infusion Reactions: BRIUMVI can cause infusion reactions, which can include pyrexia, chills, headache, influenza-like illness, tachycardia, nausea, throat irritation, erythema, and an anaphylactic reaction. In MS clinical trials, the incidence of infusion reactions in BRIUMVI-treated patients who received infusion reaction-limiting premedication prior to each infusion was 48%, with the highest incidence within 24 hours of the first infusion. 0.6% of BRIUMVI-treated patients experienced infusion reactions that were serious, some requiring hospitalization.

Observe treated patients for infusion reactions during the infusion and for at least one hour after the completion of the first two infusions unless infusion reaction and/or hypersensitivity has been observed in association with the current or any prior infusion. Inform patients that infusion reactions can occur up to 24 hours after the infusion. Administer the recommended pre-medication to reduce the frequency and severity of infusion reactions. If life-threatening, stop the infusion immediately, permanently discontinue BRIUMVI, and administer appropriate supportive treatment. Less severe infusion reactions may involve temporarily stopping the infusion, reducing the infusion rate, and/or administering symptomatic treatment.

Infections: Serious, life-threatening or fatal, bacterial and viral infections have been reported in BRIUMVI-treated patients. In MS clinical trials, the overall rate of infections in BRIUMVI-treated patients was 56%, compared to 54% in teriflunomide-treated patients. The rate of serious infections was 5% compared to 3%, respectively. There were 3 infection-related deaths in BRIUMVI-treated patients. The most common infections in BRIUMVI-treated patients included upper respiratory tract infection (45%) and urinary tract infection (10%). Delay BRIUMVI administration in patients with an active infection until the infection is resolved.

Consider the potential for increased immunosuppressive effects when initiating BRIUMVI after immunosuppressive therapy or initiating an immunosuppressive therapy after BRIUMVI.

Hepatitis B Virus (HBV) Reactivation: HBV reactivation occurred in an MS patient treated with BRIUMVI in clinical trials. Fulminant hepatitis, hepatic failure, and death caused by HBV reactivation have occurred in patients treated with anti-CD20 antibodies. Perform HBV screening in all patients before initiation of treatment with BRIUMVI. Do not start treatment with BRIUMVI in patients with active HBV confirmed by positive results for HBsAg and anti-HB tests. For patients who are negative for surface antigen [HBsAg] and positive for HB core antibody [HBcAb+] or are carriers of HBV [HBsAg+], consult a liver disease expert before starting and during treatment.

Progressive Multifocal Leukoencephalopathy (PML): Although no cases of PML have occurred in BRIUMVI-treated MS patients, JC virus infection resulting in PML has been observed in patients treated with other anti-CD20 antibodies and other MS therapies.

If PML is suspected, withhold BRIUMVI and perform an appropriate diagnostic evaluation. Typical symptoms associated with PML are diverse, progress over days to weeks, and include progressive weakness on one side of the body or clumsiness of limbs, disturbance of vision, and changes in thinking, memory, and orientation leading to confusion and personality changes.

MRI findings may be apparent before clinical signs or symptoms; monitoring for signs consistent with PML may be useful. Further investigate suspicious findings to allow for an early diagnosis of PML, if present. Following discontinuation of another MS medication associated with PML, lower PML-related mortality and morbidity have been reported in patients who were initially asymptomatic at diagnosis compared to patients who had characteristic clinical signs and symptoms at diagnosis.

If PML is confirmed, treatment with BRIUMVI should be discontinued.

Vaccinations: Administer all immunizations according to immunization guidelines: for live or live-attenuated vaccines, at least 4 weeks and, whenever possible, at least 2 weeks prior to initiation of BRIUMVI for non-live vaccines. BRIUMVI may interfere with the effectiveness of non-live vaccines. The safety of immunization with live or live-attenuated vaccines during or following administration of BRIUMVI has not been studied. Vaccination with live virus vaccines is not recommended during treatment and until B-cell repletion.

Vaccination of Infants Born to Mothers Treated with BRIUMVI During Pregnancy: In infants of mothers exposed to BRIUMVI during pregnancy, assess B-cell counts prior to administration of live or live-attenuated vaccines as measured by CD19+ B-cells. Depletion of B-cells in these infants may increase the risks from live or live-attenuated vaccines. Inactivated or non-live vaccines may be administered prior to B-cell recovery. Assessment of vaccine immune responses, including consultation with a qualified specialist, should be considered to determine whether a protective immune response was mounted.

Fetal Risk: Based on data from animal studies, BRIUMVI may cause fetal harm when administered to a pregnant woman. Transient peripheral B-cell depletion and lymphocytopenia have been reported in infants born to mothers exposed to other anti-CD20 B-cell depleting antibodies during pregnancy. A pregnancy test is recommended in females of reproductive potential prior to each infusion. Advise females of reproductive potential to use effective contraception during BRIUMVI treatment and for 6 months after the last dose.

Reduction in Immunoglobulins: As expected with any B-cell depleting therapy, decreased immunoglobulin levels were observed. Decrease in immunoglobulin M (IgM) was reported in 0.6% of BRIUMVI-treated patients, compared to none of the patients treated with teriflunomide in RMS clinical trials. Monitor the levels of quantitative serum immunoglobulins during treatment, especially in patients with opportunistic or recurrent infections, and after discontinuation of therapy, until B-cell repletion. Consider discontinuing BRIUMVI therapy if a patient with low immunoglobulins develops a serious opportunistic infection or recurrent infections, or if prolonged hypogammaglobulinemia requires treatment with intravenous immunoglobulins.

Most Common Adverse Reactions: The most common adverse reactions in RMS trials (incidence of at least 10%) were infusion reactions and upper respiratory tract infections.

Physicians, pharmacists, or other healthcare professionals with questions about BRIUMVI should visit www.briumvi.com.

The full Summary of Product Characteristics approved in the European Union (EU) for BRIUMVI can be found here Briumvi | European Medicines Agency (europa.eu).

ABOUT BRIUMVI PATIENT SUPPORT in the U.S.
BRIUMVI Patient Support is a flexible program designed by TG Therapeutics to support U.S. patients through their treatment journey in a way that works best for them. More information about the BRIUMVI Patient Support program can be accessed at www.briumvipatientsupport.com.

ABOUT MULTIPLE SCLEROSIS
Relapsing multiple sclerosis (RMS) is a chronic demyelinating disease of the central nervous system (CNS) and includes people with relapsing-remitting multiple sclerosis (RRMS) and people with secondary progressive multiple sclerosis (SPMS) who continue to experience relapses. RRMS is the most common form of multiple sclerosis (MS) and is characterized by episodes of new or worsening signs or symptoms (relapses) followed by periods of recovery. It is estimated that nearly 1 million people are living with MS in the United States and approximately 85% are initially diagnosed with RRMS.1,2 The majority of people who are diagnosed with RRMS will eventually transition to SPMS, in which they experience steadily worsening disability over time. Worldwide, more than 2.3 million people have a diagnosis of MS.1

Servier’s VORANIGO® (vorasidenib) Tablets Receives FDA Approval as First Targeted Therapy for Grade 2 IDH-mutant Glioma

On August 6, 2024 Servier reported that the U.S. Food and Drug Administration (FDA) has approved VORANIGO, an isocitrate dehydrogenase-1 (IDH1) and isocitrate dehydrogenase-2 (IDH2) inhibitor, indicated for the treatment of adult and pediatric patients 12 years and older with Grade 2 astrocytoma or oligodendroglioma with a susceptible IDH1 or IDH2 mutation following surgery including biopsy, sub-total resection, or gross total resection (Press release, Servier, AUG 6, 2024, View Source [SID1234645443]). VORANIGO is available and offers glioma patients the ability to actively manage their disease with the convenience of a once-daily pill.

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Gliomas are types of brain cancer that can hinder normal brain function and cause a variety of symptoms. Diffuse gliomas with IDH mutations represent the most common malignant primary brain tumors diagnosed in adults younger than 50 years of age. They are not curable with current therapies and without treatment they continue to grow and infiltrate normal brain tissue.

"Today’s approval of VORANIGO is an enormous leap forward in cancer care, and a defining moment for people living with Grade 2 IDH-mutant glioma," said Arjun H. Prasad, Chief Commercial Officer, Servier Pharmaceuticals. "VORANIGO, which is the first breakthrough in this specific disease area in nearly 25 years, offers patients unprecedented improvement in progression free survival. We are proud to deliver this first-of-its-kind therapy to patients in need, and we remain committed to bringing innovative targeted therapies to people with cancer."

In healthy human cells, a family of genes called isocitrate dehydrogenases (IDH) help break down nutrients and generate energy for cells. Mutations in IDH1 and IDH2 are associated with a variety of cancers, where they prevent cells from differentiating, or specializing, into the kind of cells they are ultimately supposed to become. When cells cannot differentiate properly, they may begin to grow out of control.4 In IDH-mutant gliomas, VORANIGO works by reducing the activity of the mutant IDH1 and IDH2 enzymes, to help control the disease.

"Patients living with Grade 2 IDH-mutant gliomas have long faced the harsh reality of an incurable disease with very limited post-surgery treatment options," said Ralph DeVitto, President & CEO, of the American Brain Tumor Association. "The FDA approval of VORANIGO marks a monumental breakthrough in glioma treatment, offering renewed hope for patients and their families living with this relentless disease."

The approval of VORANIGO is supported by results from the pivotal Phase 3 INDIGO clinical trial published in The New England Journal of Medicine and presented during the Plenary Session at the 2023 Annual Meeting of the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper), which showed that VORANIGO significantly extended progression free survival and time to next intervention, when compared to placebo. The INDIGO study showed that VORANIGO was well tolerated, and its safety profile was consistent with results from the Phase 1 studies. The most common (≥15%) adverse reactions were fatigue, COVID-19, musculoskeletal pain, diarrhea and seizure.5

"Glioma is a unique cancer. Many of the patients I’ve met are in their 30’s and 40’s and in the prime of their lives. They have small children and are at the height of their careers. A glioma diagnosis is devastating. VORANIGO can offer patients and their families hope for the future," said David K. Lee, CEO, Servier Pharmaceuticals. "As we advance more targeted therapies, identifying mutations and understanding how these mutations impact cancer and its progression are key to helping the right patients find the right treatment, at the right time. We are humbled to lead the field of IDH-mutant inhibition, and we are committed to researching its applicability in glioma and other cancers."

About the INDIGO Phase 3 Trial (NCT04164901)5
INDIGO, the pivotal Phase 3 clinical trial, met its major efficacy outcome of progression free survival (PFS) per a blinded independent review committee (BIRC) and key secondary endpoint of time to next intervention (TTNI) at the prespecified second interim analysis. The major efficacy outcome, PFS was statistically significant and clinically meaningful in favor of the vorasidenib arm. Median PFS was 27.7 months in the vorasidenib group, compared with 11.1 months in the placebo group (Hazard Ratio [HR], 0.39; 95% Confidence Interval [CI], 0.27 to 0.56; 1-sided P<0.001). TTNI was also statistically significant (HR, 0.26; 95% CI, 0.15 to 0.43; 1-sided P<0.001). Median TTNI was not reached for vorasidenib and was 17.8 months for placebo. Vorasidenib was also shown to reduce the tumor volume by a mean of 2.5% (TGR of –2.5%; 95% CI: -4.7% to -0.2%) every 6 months, while tumor volume increased by a mean of 13.9% (TGR of 13.9%; 95% CI: 11.1% to 16.8%) every 6 months for patients randomized to the placebo arm, as measured by a BIRC.

The INDIGO study showed that vorasidenib was well tolerated, and its safety profile was consistent with results from the Phase 1 studies.

INDIGO was a registration-enabling Phase 3 global, randomized, double-blind placebo-controlled study of vorasidenib in patients with residual or recurrent Grade 2 glioma with an isocitrate dehydrogenase 1/2 (IDH1/2) mutation who have undergone surgery as their only treatment.

About Glioma6
Gliomas are tumors that arise from glial or precursor cells within the central nervous system (CNS). The 2021 World Health Organization (WHO) classification recognizes four general groups of gliomas, one of which is adult-type diffuse gliomas. These diffuse gliomas are the most common primary malignant brain tumors in adults. The pathogenesis and prognosis of these tumors are tightly linked to mutations (or lack thereof) in the metabolic enzyme isocitrate dehydrogenase (IDH), and molecular testing is required for proper diagnosis. As of 2021, adult-type diffuse gliomas are sub-divided into only three categories:

Astrocytoma, IDH-mutant (CNS WHO grades 2-4)
Oligodendroglioma, IDH-mutant and1p19q-codeleted (CNS WHO grades 2-3)
Glioblastoma, IDH-wildtype (CNS WHO grade 4)

Black Diamond Therapeutics Reports Second Quarter 2024 Financial Results and Provides Corporate Update

On August 6, 2024 Black Diamond Therapeutics, Inc. (Nasdaq: BDTX), a clinical-stage oncology company developing MasterKey therapies that target families of oncogenic mutations in patients with cancer, reported financial results for the second quarter ended June 30, 2024, and provided a corporate update (Press release, Black Diamond Therapeutics, AUG 6, 2024, View Source [SID1234645411]).

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"We continue to execute on enrollment of patients with EGFR mutant NSCLC into second/third-line and first-line Phase 2 cohorts, and remain on track to announce initial results later in the third quarter of this year and in the first quarter of 2025, respectively", said Mark Velleca, M.D., Ph.D., Chief Executive Officer of Black Diamond Therapeutics. "We also look forward to sharing analyses of real world data at the 2024 ESMO (Free ESMO Whitepaper) Congress in September 2024 on treatment practices and therapeutic outcomes for newly diagnosed NSCLC patients with non-classical EGFR mutations that demonstrate a significant unmet medical need."

Recent Developments & Upcoming Milestones:

BDTX-1535:

In April 2024, Black Diamond described real world evidence of the evolving EGFR mutation landscape in patients with non-small cell lung cancer (NSCLC) and the potential of BDTX-1535 to address a broader range of mutations compared to existing therapies at the 2024 American Association for Cancer Research (AACR) (Free AACR Whitepaper) annual meeting. The analyses revealed a spectrum of previously underappreciated non-classical mutations, as well as an increased prevalence of the acquired resistance mutation C797S. These non-classical EGFR mutations were present in 20-30% of newly diagnosed epidermal growth factor receptor mutation positive (EGFRm) NSCLC patients.
In June 2024, Black Diamond presented additional data from the Phase 1 dose escalation trial of BDTX-1535 in patients with relapsed/recurrent glioblastoma (GBM), and initial intratumoral pharmacokinetic data from a "window of opportunity" (also known as a Phase 0/1 "Trigger") trial sponsored by the Ivy Brain Tumor Center, in patients with recurrent high-grade glioma (HGG), at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting. Safety and tolerability data in the Phase 1 trial were consistent with BDTX-1535 clinical data in patients with NSCLC previously presented in October 2023 at the European Organization for Research and Treatment of Cancer-National Cancer Institute-American Association for Cancer Research (AACR-NCI-EORTC) (Free AACR-NCI-EORTC Whitepaper) AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper). Among 19 efficacy evaluable patients, several experienced stable disease with promising durability. Results from the investigator-sponsored trial demonstrated that BDTX-1535 penetrates the blood brain barrier with clinically meaningful unbound drug concentration in gadolinium non-enhancing regions of the brain and inhibition of corresponding pharmacodynamic markers. Eight out of nine patients exceeded the pre-specified threshold for drug concentration in the brain tumor tissue and continued on study.
Black Diamond anticipates the following upcoming key milestones for BDTX-1535:
Disclosure of initial Phase 2 data in 2L/3L EGFRm NSCLC patients with non-classical mutations or the acquired resistance C797S mutation remains on track for later in Q3 2024.
Disclosure of initial Phase 2 data in 1L EGFRm NSCLC patients with non-classical mutations remains on track for Q1 2025 (NCT05256290).
An abstract has been accepted for presentation at the 2024 European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress titled "Real World Evidence of Treatment Practices and Therapeutic Outcomes for Newly Diagnosed NSCLC Patients with Non-classical EGFR Mutations Demonstrates High Unmet Medical Need", which will detail an analysis of Guardant Inform data on treatment outcomes for newly diagnosed NSCLC patients with tumors expressing non-classical mutations.
BDTX-4933:

BDTX-4933 is a brain-penetrant oral inhibitor of oncogenic alterations in KRAS, NRAS and BRAF.
Enrollment of patients with BRAF and select RAS/MAPK mutation-positive cancers, with an emphasis on patients with KRAS mutant NSCLC, is progressing through escalating doses in a Phase 1 trial (NCT05786924). An update from this trial is on track for Q4 2024.
Corporate

Chief Business Officer & Chief Financial Officer, Fang Ni, Pharm.D, will participate in a panel discussion at the Wedbush PacGrow Healthcare Conference taking place August 13-14, 2024, in New York, NY.
Financial Highlights

Cash Position: Black Diamond ended the second quarter of 2024 with approximately $123.0 million in cash, cash equivalents, and investments compared to $131.4 million as of December 31, 2023. Net cash used in operations was $14.7 million for the second quarter of 2024 compared to $14.4 million for the second quarter of 2023.
Research and Development Expenses: Research and development (R&D) expenses were $12.6 million for the second quarter of 2024, compared to $13.2 million for the same period in 2023. The decrease in R&D expenses was primarily due to workforce efficiencies and reduced spending on early discovery projects.
General and Administrative Expenses: General and administrative (G&A) expenses were $9.6 million for the second quarter of 2024, compared to $6.9 million for the same period in 2023. The increase in G&A expenses was primarily due to an increase in consulting and other professional fees.
Net Loss: Net loss for the second quarter of 2024 was $19.9 million, as compared to $19.2 million for the same period in 2023.
Financial Guidance

Black Diamond ended the second quarter of 2024 with approximately $123.0 million in cash, cash equivalents and investments which the Company believes is sufficient to fund its anticipated operating expenses and capital expenditure requirements into the fourth quarter of 2025.

Myriad Genetics Reports Strong Second Quarter 2024 Financial Results, including 15% Revenue Growth Year-Over-Year; Raises 2024 Financial Guidance and Long-Term Revenue Growth Target to 12%

On August 6, 2024 Myriad Genetics, Inc. (NASDAQ: MYGN), a leader in genetic testing and precision medicine, reported financial results for its second quarter ended June 30, 2024 and raised its previously issued financial guidance on business performance for the full-year 2024 (Press release, Myriad Genetics, AUG 6, 2024, View Source [SID1234645427]).

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"We are very proud to have delivered another quarter of strong double digit year-over-year revenue growth in the second quarter of 2024. Our year-to-date 2024 revenue growth of 13% year-over-year, following our 11% year-over-year revenue growth in calendar year 2023, and our 15% year-over-year revenue growth in the second quarter 2024, demonstrate the sustainability of our organic growth and gives us the confidence to raise our long-term revenue growth target to 12%," said Paul J. Diaz, President and CEO of Myriad Genetics. "In the second quarter, we saw strong performance across our portfolio, highlighted by increasing evidence of market share gains in prenatal testing. We anticipate these trends to continue as we move through the year and into 2025. In addition, second quarter average revenue per test improved across our product portfolio, benefiting from expanded coverage and our ongoing efforts in revenue cycle management. We remain optimistic about the evolution of our product portfolio as we continue to publish additional clinical validation studies and launch new products.

At the same time, we continue to improve access and ease of use for our customers, as we accelerate electronic medical record (EMR) integrations for new customers and make meaningful progress in our Labs of the Future initiative. Myriad Genetics is growing profitably and delivering improved financial results, including a 17% year-over-year increase in gross profit of $147.1 million, cash flow from operations of $2.6 million, and $16.4 million of adjusted operating cash flow. All while continuing to invest in the innovation required to achieve our mission and vision to reach more patients with life-saving precision medicine."
Financial and Operational Highlights
•Test volumes of 389,000 in the second quarter of 2024 increased 9% year-over-year.
•The following table summarizes year-over-year testing volume changes in the company’s core product categories:
Three months ended Six months ended
(in thousands)
June 30, 2024 June 30, 2023
% Change
June 30, 2024 June 30, 2023
% Change
Product volumes:
Hereditary cancer
73 71 3 % 144 136 6 %
Tumor profiling
14 16 (13) % 28 32 (13) %
Prenatal 173 154 12 % 345 312 11 %
Pharmacogenomics
129 117 10 % 253 227 11 %
Total 389 358 9 % 770 707 9 %

•The following table summarizes year-over-year revenue changes in the company’s core product categories:
Three months ended Six months ended
(in millions)
June 30, 2024 June 30, 2023
% Change
June 30, 2024 June 30, 2023
% Change
Product revenues:
Hereditary cancer
$ 91.5 $ 76.7 19 % $ 179.6 $ 152.4 18 %
Tumor profiling
32.6 36.0 (9) % 63.5 73.3 (13) %
Prenatal 44.4 35.6 25 % 88.7 71.8 24 %
Pharmacogenomics
43.0 35.2 22 % 81.9 67.2 22 %
Total $ 211.5 $ 183.5 15 % $ 413.7 $ 364.7 13 %

•Gross margin of 69.6% in the second quarter of 2024 increased 110 basis points year-over-year, reflecting operating leverage and improved average revenue per test. Adjusted gross margin in the second quarter of 2024 was 70.1%, an increase of 110 basis points year-over-year as the company’s revenue cycle, Labs of the Future and supply chain initiatives begin to take hold.
•Second quarter of 2024 operating expenses were $183.6 million. Adjusted operating expenses were $140.8 million, increasing 6% over the year ago period. This increase was driven by investments in technology, product development and R&D. Adjusted operating expenses accounted for 67% of total revenue in the second quarter of 2024, down from 73% of total revenue in the second quarter of 2023.
2

•Operating loss in the second quarter of 2024 was $36.5 million, improving $77.2 million year-over-year; adjusted operating income in the second quarter of 2024 was $7.4 million, improving $14.2 million year-over-year.

Business Performance and Highlights

Oncology
The Oncology business delivered revenue of $82.2 million in the second quarter of 2024.
•Second quarter 2024 hereditary cancer testing revenue in Oncology grew 11% year-over-year, reflecting ongoing initiatives to improve average revenue per test through payer coverage expansion and revenue cycle process improvements that are reducing the company’s no pay rate.
•Second quarter 2024 tumor profiling revenue of $32.6 million grew 5% compared to first quarter 2024 but decreased 10% year-over-year, reflecting the ongoing challenging biopharma environment, slow ramp of biopharma contracts executed in 2023, and challenges in the international business.
•In July 2024, Myriad Genetics received a patent relating to detecting circulating tumor DNA in patient fluid samples, which is complementary to a patent granted earlier in the year for the company’s methods of preparing cell-free DNA. Both of these patents support advancing commercialization of the company’s high sensitivity tumor informed Molecular Residual Disease (MRD) assay.
•In July 2024, Myriad Genetics entered into an agreement with Personalis, Inc. (Nasdaq: PSNL) to cross-license patent estates covering tumor-informed approaches to detect MRD. The agreement helps solidify each company’s freedom to operate in the MRD market and broadens patient access to the benefits of MRD testing.
•Announced a collaboration with GSK (NYSE: GSK) aimed at improving access to homologous recombination deficiency (HRD) diagnostic testing for high-grade serous ovarian cancer (HGSOC) patients, leveraging Myriad Genetics’ MyChoice HRD Plus and MyChoice CDx Plus tests in nine countries outside the United States.
•Myriad Genetics and QIAGEN (NYSE: QGEN) agreed to develop a globally distributable kit-based test for analyzing HRD status to support research into personalized medicine in multiple solid tumor types, including ovarian cancer.
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•In August 2024, Myriad Genetics announced that it further advanced its international reorganization efforts, including the closing of the sale of its EndoPredict business to Eurobio Scientific. The reorganization of its international operations better aligns company resources to its domestic opportunities while continuing to serve key biopharma partners and patients globally and builds on Myriad Genetics’ efforts this year to accelerate profitable business growth across its portfolio.

Women’s Health
The Women’s Health business delivered revenue of $86.3 million in the second quarter of 2024.
•Second quarter 2024 hereditary cancer testing revenue in Women’s Health grew 31% year-over-year as more practitioners see the benefit of incorporating MyRisk with RiskScore as part of a comprehensive breast cancer risk assessment program.
•Prenatal testing revenue in the second quarter of 2024 grew 25% year-over-year, reflecting market share gains, expanded coverage by payers, and ongoing initiatives to improve average revenue per test.
•Myriad Genetics launched the Universal Plus Panel for Foresight Carrier Screen, which includes 39 new conditions and screens up to 272 genes associated with serious inherited conditions.
•Ten abstracts, including four on FirstGene, have been accepted to be showcased at the National Society of Genetic Counselors’ 43rd annual meeting, which begins on September 17, 2024, in New Orleans, LA.

Pharmacogenomics
In the pharmacogenomics category, GeneSight test revenue was $43.0 million in the second quarter of 2024.
•Second quarter 2024 GeneSight testing revenue grew 22% year-over-year, reflecting ongoing initiatives to improve average revenue per test.
•Currently, biomarker legislation for state-regulated plans has passed in 15 states. In many of these states, commercial and managed Medicaid payers have modified their coverage policies to include GeneSight and Prolaris. Additionally, there are a number of states that have legislation in process. Myriad Genetics continues to see an increasing number of payors incorporating, or planning to incorporate, GeneSight into their coverage. Notably, this includes Blue Shield of California, a major commercial and managed Medicaid plan, effective July 1, 2024.

Financial Guidance
Myriad Genetics does not provide forward-looking guidance on a GAAP basis for the measures on which it provides forward-looking non-GAAP guidance as the company is unable to provide a quantitative reconciliation of forward-looking non-GAAP measures to the most directly comparable forward-looking GAAP measure, without unreasonable effort, because of the inherent difficulty in accurately forecasting the occurrence and financial impact of the various adjusting items necessary for such reconciliations that have not yet occurred, are dependent on various factors, are out of the company’s control, or cannot be reasonably predicted. Such adjustments include, but are not limited to, real estate optimization and transformation initiatives, certain litigation charges and loss contingencies, costs related to acquisitions/divestitures and the related amortization, impairment and related charges, and other adjustments. For example, stock-based compensation may fluctuate based on the timing of employee stock transactions and unpredictable fluctuations in the company’s stock price. Any associated estimate of these items and its impact on GAAP performance could vary materially.

Below is a table summarizing Myriad Genetics’ fiscal year 2024 financial guidance*:
(in millions, except per share amounts) PRIOR
FY 2024 CURRENT
FY 2024 Expected Year-Over-Year Change
Revenue $820 – $840 $835 – $845 11% – 12%
Gross margin % 69.5% – 70.5% 70.0% – 70.5%
100 – 150 bps
Adjusted OPEX $572 – $582
$575 – $585
6% – 8%
Adjusted EBITDA** $20 – $30
$25 – $35
$36 – $46
Adjusted EPS*** $0.00 – $0.05
$0.08 – $0.12
$0.35 – $0.39
*
Assumes currency rates as of August 6, 2024.
** Adjusted EBITDA is defined as Net Income (loss) plus income tax expense (benefit), total other income (expense), non-cash operating expenses, such as amortization of intangible assets, depreciation, impairment of long-lived assets, and share-based compensation expense, and one-time expenses such as expenses from real estate optimization initiatives, transformation initiatives, legal settlements, and divestitures and acquisitions.
***
Full-year 2024 adjusted EPS is based on a 91 million share count.

These projections are forward-looking statements and are subject to the risks summarized in the safe harbor statement at the end of this press release.

Conference Call and Webcast
A conference call will be held today, Tuesday, August 6, 2024, at 4:30 p.m. EDT to discuss Myriad Genetics’ financial results and business developments for the second quarter 2024. A live webcast of the conference call can be accessed on Myriad Genetics’ Investor Relations website at investor.myriad.com. To participate in the live conference call via telephone, please register at https://register.vevent.com/register/BI620080625d0e42be9b313c17391abf61. Upon registering, a dial-in number and unique PIN will be provided to join the conference call. Following the conference call, an archived webcast of the call will be available at investor.myriad.com.