On May 9, 2024 Century Therapeutics, Inc. (NASDAQ: IPSC), an innovative biotechnology company developing induced pluripotent stem cell (iPSC)-derived cell therapies in immuno-oncology and autoimmune diseases, reported financial results and business highlights for the first quarter ended March 31, 2024 (Press release, Century Therapeutics, MAY 9, 2024, View Source [SID1234642982]).

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"We have made significant clinical, operational and research-oriented progress so far this year, and I am incredibly excited by the momentum we’ve been able to achieve in such a short period of time," said Brent Pfeiffenberger, Pharm.D., Chief Executive Officer of Century Therapeutics. "Looking ahead, we will be sharing additional clinical data from the ELiPSE-1 trial of CNTY-101 in R/R NHL at the upcoming ASCO (Free ASCO Whitepaper) Annual Meeting in June. In addition, we are on track to initiate the Phase 1 CALiPSO-1 trial of CNTY-101 in SLE in the first half of 2024, while also continuing to progress the multiple planned regulatory filings for CNTY-101 in additional autoimmune disease indications later this year. We believe Century’s position as a leader in allogeneic, iPSC-derived cell therapy is fortified by the recent expansion of our pipeline and platform capabilities through the acquisition of Clade Therapeutics, and we are looking forward to continued execution across our robust portfolio of novel assets providing us with multiple near- and long-term potential value drivers."

Research and Development Highlights and Upcoming Milestones

CNTY-101

CNTY-101 is a CD19 targeting allogeneic iNK cell therapy with 6 precision gene edits powered by Century’s Allo-Evasion technology, which enables repeat dosing without the need for continued lymphodepletion. Century’s core Allo-Evasion edits are designed to overcome the three major pathways of host versus graft rejection: CD8+ T cells, CD4+ T cells and NK cells.

· In April 2024, the Company announced plans to expand clinical development of CNTY-101 into additional autoimmune disease indications beyond SLE. In the second half of 2024, Century intends to submit additional regulatory filings for CNTY-101 in autoimmune disease indications with limited current treatment options and high unmet need. Additionally, the Company is planning to evaluate CNTY-101 for SLE in the Phase 1 CALiPSO-1 trial, which is on track to be initiated in the first half of 2024 with preliminary data expected by the end of 2024.

· Century plans to share additional data from its Phase 1 ELiPSE-1 trial in R/R non-Hodgkin lymphoma (NHL) at the upcoming ASCO (Free ASCO Whitepaper) Annual Meeting, being held May 31-June 4, 2024, in Chicago, IL. As previously announced, in this heavily pretreated and refractory patient population, CNTY-101 has demonstrated a favorable safety profile in the initial seven patients treated with Dose Level 1 (100 million cells) and Dose Level 2 (300 million cells) on a once monthly schedule. In these low dose levels, CNTY-101 demonstrated encouraging early response signals, including two complete responses (CRs) and one partial response (PR). Initial translational data supports the potential for Allo-Evasion to enable a multidose regimen without the need for continued lymphodepletion.

Platform Technology and Additional Pipeline Programs

· In April 2024, Century shared six poster presentations at the 2024 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting showcasing new preclinical data on additional Allo-Evasion edits in addition to the Company’s end-to-end cell therapy capabilities including expertise across iPSC reprogramming, gene editing, protein engineering, Allo-Evasion technology and computational biology. Key abstracts highlighted Century’s novel, dual-targeting CAR for B-cell mediated malignancies demonstrating in vitro and in vivo cytotoxicity and antigen loss resistance; and new data on the Allo-Evasion platform showcasing its potential to evade identification by the host immune system, which would allow for repeat dosing without rejection, enabling increased persistence of the cells during the treatment period and potentially leading to deeper and more durable responses. Across all six posters, the findings highlight Century’s unique gene editing, protein engineering, and manufacturing capabilities that are the foundations of its allogeneic cell therapy pipeline and platform. The presented posters are available at the Scientific Resources page of the Company’s website.

· In April 2024, the Company announced the acquisition of Clade Therapeutics, a privately held biotech company focused on discovering and delivering engineerable, off-the-shelf, scalable, and consistent stem cell-based medicines, with a focus on iPSC-derived αβ T cells. This acquisition brings complementary technology to enhance Century’s Allo-Evasion platform and additional next-generation iT programs spanning targets in cancer, and autoimmune diseases. These programs include CLDE-308, an αβ iT cell program targeting CD19 in autoimmune disease and B-cell malignancies, CLDE-361, an αβ iT cell program targeting BCMA in myasthenia gravis, and an undisclosed iT cell focused research program in solid tumors.

Business Highlights

· In April 2024, the Company entered into a securities purchase agreement with a select group of institutional investors for an approximately $60 million private placement of its common stock. The private placement closed on April 15, 2024, and was led by new investors Bain Capital Life Sciences, Adage Capital Partners LP, Octagon Capital, and Superstring Capital, and existing investors including Casdin Capital, Boxer Capital, Venrock Healthcare Capital Partners and DAFNA Capital Management, LLC.

First Quarter 2024 Financial Results

· Cash Position: Cash, cash equivalents, and marketable securities were $249.9 million as of March 31, 2024, as compared to $261.8 million as of December 31, 2023. Net cash used in operations was $30.2 million for the three months ended March 31, 2024, compared to net cash used in operations of $29.2 million for the three months ended March 31, 2023.

· Collaboration Revenue: Collaboration revenue generated through the Company’s collaboration, option and license agreement with Bristol-Myers Squibb (BMS) was $0.9 million for the three months ended March 31, 2024, compared to $1.7 million for the same period in 2023.

· Research and Development (R&D) expenses: R&D expenses were $23.4 million for the three months ended March 31, 2024, compared to $24.9 million for the same period in 2023. The decrease in R&D expenses was primarily due to the reduction in force that occurred in January of 2023.

· General and Administrative (G&A) expenses: G&A expenses were $8.7 million for the three months ended March 31, 2024, compared to $8.9 million for the same period in 2023.

· Net loss: Net loss was $28.1 million for the three months ended March 31, 2024, compared to $31.3 million for the three months ended March 31, 2023.

Financial Guidance

· The Company expects full year generally accepted accounting principles (GAAP) operating expenses to be between $150 million and $160 million as compared to the previous guidance range of $135 million to $145 million. The increase is driven by additional operating expenses related to the integration of Clade Therapeutics, as well as the Company’s planned expanded clinical development of CNTY-101 into additional autoimmune disease indications. The Company is currently in the process of completing its accounting analysis of the acquisition of Clade. The revised guidance does not include any potential non-cash charges related to the acquisition.

· The Company estimates its cash, cash equivalents, and investments will support operations into 2026.

On May 9, 2024 Lisata Therapeutics, Inc. (Nasdaq: LSTA) ("Lisata" or the "Company"), a clinical-stage pharmaceutical company developing innovative therapies for the treatment of advanced solid tumors and other serious diseases, reported a business update and announced financial results for the three months ended March 31, 2024 (Press release, Lisata Therapeutics, MAY 9, 2024, View Source [SID1234642981]).

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"2024, a pivotal year for Lisata, is off to a very strong start," stated David J. Mazzo, Ph.D., President and Chief Executive Officer of Lisata. "Although we project multiple data readouts over the next 18 months, topline results from the Phase 2b ASCEND trial later this year have transformative potential for the Company. These results will be instrumental in determining the future of Lisata, and we plan to use them to explore conditional approvals with various regulatory agencies and/or to design an optimized Phase 3 program in pancreatic ductal adenocarcinoma. Since the start of the year, we have received both U.S. FDA Orphan Drug and Rare Pediatric Disease designations for certepetide, previously known as LSTA1, in osteosarcoma, further validating the broad therapeutic potential of this innovative therapy. We are energized by the progress we are making and excited about Lisata’s prospects."

Dr. Mazzo added, "Our continued prudent financial management allows us to reaffirm our projection that currently available cash will fund operations into early 2026, providing a solid foundation to fund all ongoing and planned trials through to completion. More than ever, we remain confident in our ability to execute our development activities with the goal of reaching critical milestones at the earliest possible juncture."

Development Portfolio Highlights

Certepetide as a treatment for solid tumors in combination with other anti-cancer agents
Certepetide is an investigational drug designed to activate the CendR uptake pathway that allows co-administered or molecularly bound anti-cancer drugs to target and penetrate solid tumors more effectively. Certepetide is designed to actuate this active transport system in a tumor-specific manner, resulting in systemically co-administered anti-cancer drugs more efficiently penetrating and accumulating in the tumor, to the exclusion of normal tissues. In preclinical models, certepetide has also shown the ability to modify the tumor microenvironment, leading to the expectation that tumors will become more susceptible to immunotherapies and inhibiting the metastatic cascade (i.e., the spread of cancer to other parts of the body). Lisata and its development partners have amassed significant non-clinical data demonstrating enhanced delivery of a range of existing and emerging anti-cancer therapies, including chemotherapeutics, immunotherapies, and RNA-based therapeutics. To date, certepetide has also demonstrated favorable safety, tolerability and activity in completed and ongoing clinical trials designed to test its ability to enhance delivery of standard-of-care ("SOC") chemotherapy for metastatic pancreatic cancer ("mPDAC"). Certepetide has been granted orphan drug designation for pancreatic cancer in the U.S. and Europe as well as for glioblastoma multiforme ("GBM") and osteosarcoma in the U.S. It also received a Fast Track designation from the FDA for pancreatic cancer and, just recently, a Rare Pediatric Disease designation from the FDA for osteosarcoma. Currently, certepetide is the subject of multiple ongoing or planned Phase 2a and 2b clinical studies being conducted globally in a variety of solid tumor types in combination with a variety of anti-cancer regimens:

•ASCEND: Phase 2b double-blind, randomized, placebo-controlled clinical trial evaluating two dosing regimens of certepetide in combination with gemcitabine/nab-paclitaxel SOC chemotherapy in patients with mPDAC. Cohort A of the study receives a single dose of 3.2 mg/kg certepetide essentially simultaneously with SOC, while Cohort B is identical to Cohort A, but with a second dose of 3.2mg/kg of certepetide given four hours after the first. The trial is being conducted at 25 sites in Australia and New Zealand led by the Australasian Gastro-Intestinal Trials Group in collaboration with the University of Sydney and with the National Health and Medical Research Council Clinical Trial Centre at the University of Sydney as the Coordinating Centre. The conclusion of a planned interim futility analysis in 2023 by the Independent Data Safety Monitoring Committee was that the conditions for futility were not met and that the study should proceed to completion. With trial enrollment completed in the fourth quarter of 2023, Lisata expects topline data from the 95 patients assigned to Cohort A of the study to be reported in the fourth quarter of 2024 and the complete data set of all 158 patients from the study to be available by mid-2025.
•BOLSTER: Phase 2a double-blind, placebo-controlled, multi-center, randomized trial in the U.S. evaluating certepetide in combination with SOC in first-line cholangiocarcinoma. The trial is actively enrolling with enrollment completion expected by the end of the third quarter of 2024.
•CENDIFOX: Phase 1b/2a open-label trial in the U.S. of certepetide in combination with neoadjuvant FOLFIRINOX based therapies in pancreatic, colon and appendiceal cancers. The trial continues to make steady progress with enrollment completion for all three arms expected by the end of 2024.
•Qilu Pharmaceutical, the licensee of certepetide in the Greater China territory, is currently evaluating certepetide in combination with gemcitabine and nab-paclitaxel as a treatment for mPDAC. During the 2023 ASCO (Free ASCO Whitepaper) Annual Meeting, Qilu Pharmaceutical presented an abstract sharing preliminary data from the study which corroborated previously reported findings from the Phase 1b/2a trial of certepetide plus gemcitabine and nab-paclitaxel conducted in Australia in patients with mPDAC. As recently announced, Qilu has begun treating patients in their Phase 2 placebo-controlled trial in mPDAC. The study is planned to take approximately 18 months to complete enrollment and another 13 months for patient follow-up and data analysis and reporting.
•iLSTA: Phase 1b/2a randomized, single-blind, single-center, safety and pharmacodynamic trial in Australia evaluating certepetide in combination with the checkpoint inhibitor, durvalumab, plus standard-of-care gemcitabine and nab-paclitaxel chemotherapy versus standard-of-care alone in patients with locally advanced non-resectable PDAC. Enrollment completion is expected in the second half of 2024.
•A Lisata-funded Phase 2a, double-blind, placebo-controlled, randomized, proof-of-concept study evaluating certepetide in combination with standard-of-care temozolomide versus temozolomide alone in patients with newly diagnosed GBM is being conducted across multiple sites in Estonia and Latvia and is targeted to enroll 30 patients with a randomization of 2:1 in favor of the certepetide treatment group.
First Quarter 2024 Financial Highlights
For the three months ended March 31, 2024, operating expenses totaled $6.6 million, compared to $6.8 million for the three months ended March 31, 2023, representing a decrease of $0.2 million or 3.6%.
Research and development expenses were approximately $3.2 million for the three months ended March 31, 2024, compared to $3.2 million for the three months ended March 31, 2023, representing an essentially unchanged spend. The minor increase of $62,000 or 2.0% was primarily due to an increase in expenses associated with enrollment activities in the current year for the BOLSTER trial, partially offset by a reduction in expenses associated with the Phase 2b ASCEND trial which completed enrollment in the prior year.
General and administrative expenses were approximately $3.4 million for the three months ended March 31, 2024, compared to $3.7 million for the three months ended March 31, 2023, representing a decrease of $0.3 million or 8.3%. This was primarily due to a decrease in staffing costs associated with the elimination of the Chief Business Officer position on May 1, 2023, a reduction in option assumption equity expense in connection with the Company’s merger with Cend Therapeutics, Inc., a decrease in directors and officers insurance premiums, and a reduction in spend on consulting and legal fees partially offset by one-off settlement-related costs.

Overall, net losses were $5.4 million for the three months ended March 31, 2024, compared to $6.2 million for the three months ended March 31, 2023.
Balance Sheet Highlights
As of March 31, 2024, Lisata had cash, cash equivalents, and marketable securities of approximately $43.3 million. Based on its current expected capital needs, the Company believes that its projected capital will fund its current proposed operations into early 2026, encompassing anticipated data milestones from all its ongoing and planned clinical trials.
Conference Call Information
Lisata will hold a live conference call today, May 9, 2024, at 4:30 p.m. Eastern Time to discuss financial results, provide a business update and answer questions.
Those wishing to participate must register for the conference call by way of the following link: CLICK HERE TO REGISTER. Registered participants will receive an email containing conference call details with dial-in options. To avoid delays, we encourage participants to dial into the conference call 15 minutes ahead of the scheduled start time.
A live webcast of the call will also be accessible under the Investors & News section of Lisata’s website and will be available for replay beginning two hours after the conclusion of the call for 12 months.
About Lisata Therapeutics
Lisata Therapeutics is a clinical-stage pharmaceutical company dedicated to the discovery, development and commercialization of innovative therapies for the treatment of advanced solid tumors and other major diseases. Lisata’s lead product candidate, certepetide, is an investigational drug designed to activate a novel uptake pathway that allows co-administered or tethered anti-cancer drugs to target and penetrate solid tumors more effectively. Based on Lisata’s CendR Platform Technology, Lisata has already established noteworthy commercial and R&D partnerships. The Company expects to announce numerous clinical study and business milestones over the next two years and has projected that its current business and development plan is funded with available capital through these milestones and into early 2026. For more information on the Company, please visit www.lisata.com.

On May 9, 2024 bluebird bio, Inc. (NASDAQ: BLUE) ("bluebird bio" or the "Company") reported first quarter results and business highlights for the quarter ended March 31, 2024, including recent commercial and operational progress (Press release, bluebird bio, MAY 9, 2024, View Source [SID1234642980]).

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"bluebird has built a solid commercial gene therapy foundation, with an unparalleled network of qualified treatment centers (QTCs), proven access and reimbursement for patients, and demonstrated demand from both patients and providers," said Andrew Obenshain, chief executive officer, bluebird bio. "Following the completion of the first LYFGENIA patient start earlier this month, and with the continued momentum behind our ongoing launches, we believe we are poised for accelerated growth through the remainder of 2024."

COMMERCIAL LAUNCH UPDATES

Continued commercial momentum for LYFGENIA (lovotibeglogene autotemcel), ZYNTEGLO (betibeglogene autotemcel) and SKYSONA (elivaldogene autotemcel)

First LYFGENIA patient start completed in May 2024.
14 patient starts completed for ZYNTEGLO and SKYSONA since the beginning of 2024 (11 ZYNTEGLO, 3 SKYSONA).
Validated access and reimbursement strategy is driving favorable coverage landscape

Successfully confirmed prior authorization approval for commercial and Medicaid-insured patients for LYFGENIA.
Multiple outcomes-based agreements in place for LYFGENIA with national commercial payer organizations; published coverage policies are in place for more than 200 million U.S. lives.
Discussions ongoing with Medicaid agencies representing 80% of Medicaid-insured individuals with sickle cell disease in the U.S.
Timely access to ZYNTEGLO and SKYSONA has continued, with zero ultimate denials for either therapy across both Medicaid and commercial payers.
Substantial QTC footprint established

bluebird has activated 64 QTCs for LYFGENIA and ZYNTEGLO (defined as a signed MSA).
Six centers are also activated to administer SKYSONA for patients with cerebral adrenoleukodystrophy (CALD).
2024 GUIDANCE

The Company anticipates 85 to 105 patient starts (cell collections) combined across all three of its FDA approved therapies (LYFGENIA, ZYNTEGLO, SKYSONA) in 2024. Consistent with previous quarters, bluebird plans to provide quarterly updates on patient starts for each of its therapies.
Gross-to-net discounts across all three products are expected to be in the range of 20% to 25% of gross revenue in 2024 and will fluctuate based on product and payer mix, as well as utilization of outcomes-based agreements for LYFGENIA and ZYNTEGLO.
Based on projected timelines from cell collection to infusion, the Company anticipates recognizing revenue from its first infusion of LYFGENIA in the third quarter of 2024.
FIRST QUARTER FINANCIAL HIGHLIGHTS

Cash Position: The Company’s cash, cash equivalents and restricted cash balance was approximately $264 million, including restricted cash of approximately $52 million, as of March 31, 2024

Based on current business plans, which assumes the Company’s ability to achieve certain commercial revenue targets, bluebird expects its cash and cash equivalents, excluding restricted cash and assuming the remaining two tranches totaling $50 million in proceeds from its term loan facility with Hercules Capital are executed, will be sufficient to meet bluebird’s planned operating expenses and capital expenditure requirements through Q1 2026.

Revenue, net: Total revenue, net was $18.6 million for the three months ended March 31, 2024, compared to $2.4 million for the three months ended March 31, 2023. The increase of $16.2 million was due to increased ZYNTEGLO product revenue.
On March 26, 2024, bluebird announced that it will restate its consolidated financial statements as of and for the year ended December 31, 2022, and for each of the first three quarters of 2022 and 2023 in its Annual Report on Form 10-K for the year ended December 31, 2023 (the "2023 Form 10-K"). The restatements relate to the identification of embedded leases and the treatment of non-lease components contained in lease agreements. The restatement is not expected to impact the Company’s cash position or revenue. As a result of the restatement, the Company is delayed in filing its 2023 Form 10-K and its Quarterly Report on Form 10-Q for the quarter ended March 31, 2024 (the "Q1 2024 Form 10-Q"). The Company is continuing to work expeditiously to complete these filings.

The financial results included in this press release represent the most current information available to the Company’s management. The Company expects that its actual results to be reported in its Q1 2024 Form 10-Q will not differ materially from the results included herein, however, these results are subject to change following the completion of the Company’s financial close procedures and the review of its consolidated financial statements for the quarter ended March 31, 2024.

CONFERENCE CALL DETAILS

bluebird will hold a conference call to discuss its first quarter 2024 results and business updates today, Thursday, May 9, 2024, at 8:00 am ET.

To participate in the conference call, please dial +1 (800) 715-9871 (U.S. and Canada) and ask to be joined into the bluebird call or provide the Conference ID 9768329.

The live webcast of the call may be accessed by visiting the "Events & Presentations" page within the Investors & Media section of the bluebird website at View Source A replay of the webcast will be available on the bluebird website for 90 days following the event.

On May 9, 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 first quarter ended March 31, 2024, and provided a corporate update (Press release, Black Diamond Therapeutics, MAY 9, 2024, View Source [SID1234642979]).

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"We reached an important inflection in BDTX-1535 development this past quarter, as we initiated dosing of patients with non-classical EGFR mutant NSCLC in the first-line setting, and are on track to release initial results from the ongoing Phase 2 second/third-line cohorts in the third quarter of this year ", said Mark Velleca, M.D., Ph.D., Chief Executive Officer of Black Diamond Therapeutics. "Our recent AACR (Free AACR Whitepaper) presentation on the evolving EGFR mutation landscape highlights the major unmet need in EGFR mutant NSCLC, and the differentiated BDTX-1535 profile as the potential first- and best-in-class fourth-generation oral TKI in clinical development addressing the full spectrum of classical, non-classical, and C797S resistance mutations in NSCLC."

Recent Developments & Upcoming Milestones:
BDTX-1535:
•Following receipt of End of Phase 1 feedback from the U.S. Food and Drug Administration (FDA) in the fourth quarter of 2023, Black Diamond initiated dosing of a Phase 2 first-line cohort in patients with non-classical epidermal growth factor receptor mutation positive (EGFRm) non-small cell lung cancer (NSCLC) during the first quarter of 2024. (NCT05256290)
•In April 2024, Black Diamond described real world evidence of the evolving EGFR mutation landscape in patients with 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 oral presentation titled "BDTX-1535 – A MasterKey EGFR Inhibitor Targeting Classical, Non-Classical and the C797S Resistance Mutation to Address the Evolved Landscape of EGFR Mutant NSCLC," evaluated more than 235,000 sequenced cases of NSCLC sourced from Guardant Health (GuardantINFORM) and Foundation Medicine (FoundationInsights). The analyses revealed a broad spectrum of 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 EGFRm NSCLC patients.

•Black Diamond anticipates the following upcoming key milestones for BDTX-1535:
◦Poster presentations at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting on June 1, 2024, of Phase 1 dose escalation data in patients with relapsed/recurrent GBM, and initial intratumoral pharmacokinetic results 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).
◦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 Q3 2024.
BDTX-4933:
•Enrollment of patients with BRAF and select RAS/MAPK mutation-positive cancers, with an emphasis on patients with KRAS mutant NSCLC is ongoing in a Phase 1 dose escalation trial of BDTX-4933, a brain-penetrant oral inhibitor of oncogenic alterations in KRAS, NRAS and BRAF (NCT05786924). An update from this trial is anticipated in Q4 2024.
Corporate
•In April 2024, the Company announced changes to its Board of Directors. Industry veterans Shannon Campbell and Prakash Raman, Ph.D., have been appointed to the Company’s Board of Directors. In addition, Wendy Dixon, Ph.D., and Alex Mayweg, Ph.D., have stepped down as members of the Board of Directors.
Financial Highlights
•Cash Position: Black Diamond ended the first quarter of 2024 with approximately $115.2 million in cash, cash equivalents, and investments compared to $131.4 million as of December 31, 2023. Net cash used in operations was $21.2 million for the first quarter of 2024 compared to $20.0 million for the first quarter of 2023.
•Research and Development Expenses: Research and development (R&D) expenses were $13.5 million for the first quarter of 2024, compared to $14.8 million for the same period in 2023. The decrease in R&D expenses was primarily due to reduced spending on early discovery projects.
•General and Administrative Expenses: General and administrative (G&A) expenses were $6.7 million for the first quarter of 2024, compared to $6.8 million for the same period in 2023. The decrease in G&A expenses was primarily due to a decrease in personnel related costs and legal and other professional fees.
•Net Loss: Net loss for the first quarter of 2024 was $18.2 million, as compared to $20.9 million for the same period in 2023.
Financial Guidance
•Black Diamond ended the first quarter of 2024 with approximately $115.2 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 third quarter of 2025.

On May 9, 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 first quarter of 2024 (Press release, BioXcel, MAY 9, 2024, View Source [SID1234642978]).

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"The fundamentals of our business are strong as we look to continue advancing and expanding our agitation portfolio," said Vimal Mehta, Ph.D., CEO of BioXcel Therapeutics. "We have designed two Phase 3 registrational programs for BXCL501 in addition to strengthening our balance sheet and intellectual property. We are intensely focused on the BXCL501 journey into the at-home setting and expansion into Alzheimer’s-related agitation with the goal of bringing new treatment options to larger numbers of patients while expanding the market potential of our lead neuroscience asset."

TRANQUILITY and SERENITY Clinical Programs

· Plans for two late-stage programs are advancing following recently announced designs of pivotal Phase 3 trials:

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

o SERENITY At-Home* safety 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.

· Study protocol submitted to FDA.

IGALMI Post-Marketing Requirement (PMR) Study

· Study was designed to evaluate whether tolerance, tachyphylaxis, or withdrawal occur following repeat dosing of IGALMI following seven days of repeated treatment.

o Completed enrollment of approximately 20 patients with frequent episodes of agitation for bipolar disorders or schizophrenia in an open-label study.

o Patients self-administered 180 mcg of IGALMI for repeated agitation episodes over the treatment period.

o Initiated data cleaning to enable database lock.

Corporate Updates

IGALMI Commercialization

· Net revenue grew 55% in Q1 2024 over the prior quarter driven largely by volume contracting, new customer acquisition, increased utilization among existing customers, and the permanent J-Code for IGALMI that became effective January 1, 2024.

Patent Portfolio

The Company continues to strengthen its intellectual property portfolio with over 30 granted or allowed patents and more than 140 additional patent applications in prosecution as of April 2024.

· Recently granted two new patents for BXCL501, in Japan and the U.S., with patent protection to 2039 and 2043, respectively.

· Eight currently listed U.S. patents for IGALMI in the United States Food and Drug Administration’s ("FDA") Approved Drug Products with Therapeutic Equivalence Evaluations (commonly known as the "Orange Book"), with two additional recently allowed patents that will be submitted for listing in the Orange Book once issued by the USPTO.

OnkosXcel Therapeutics

· Announced late-breaking abstract selected for presentation at 2024 ASCO (Free ASCO Whitepaper) Annual Meeting on preliminary findings from a Phase 2 investigator-sponsored trial of BXCL701 and KEYTRUDA (pembrolizumab) in metastatic pancreatic ductal adenocarcinoma (PDAC).

First Quarter 2024 Financial Results

Net Revenue: Net revenue from IGALMI was $582,000 for the first quarter of 2024, compared to $206,000 for the same period in 2023, representing a 182% increase. Sequential quarterly revenue increased 55% in Q1 2024 from the fourth quarter of 2023. The increased revenue for both periods was primarily attributable to increasing demand with existing customers, new customer orders, and volume-based contracting.

Research and Development (R&D) Expenses: R&D expenses were $11.4 million for the first quarter of 2024, compared to $27.8 million for the same period in 2023. The decreased expenses were primarily attributable to the wind-down of the SERENITY III and TRANQUILITY II and III trials, as well as decreased professional fees, personnel, and related costs.

Selling, General and Administrative (SG&A) Expenses: SG&A expenses were $13.3 million for the first quarter of 2024, compared to $23.6 million for the same period in 2023. The reduced expenses were primarily attributable to a decrease in personnel and costs associated with the commercialization of IGALMI compared to the first quarter of 2023. The reduced expenses were partially offset by increased professional fees in the first quarter of 2024.

Net Loss: BioXcel Therapeutics had a net loss of $26.8 million for the first quarter of 2024, compared to a net loss of $52.8 million for the same period in 2023. The Company used $17.7 million in operating cash during the first quarter of 2024.

Cash and cash equivalents totaled $74.1 million as of March 31, 2024. This includes the $25 million from the registered direct offering announced on March 25, 2024.

Conference Call and Webcast

BioXcel Therapeutics will host a conference call and webcast today, May 9, 2024, at 8:00 a.m. ET to discuss its first quarter 2024 financial results. To access the call, please dial 877-407-5795 or 201-689-8722. A live webcast will be available on the Investors section of the corporate website, bioxceltherapeutics.com, and a replay will be available through August 9, 2024.

BioXcel Therapeutics may use its website as a distribution channel of material information about the Company. Financial and other important information regarding the Company is routinely posted on and accessible through the Investors sections of its website at www.bioxceltherapeutics.com. In addition, you may sign up to automatically receive email alerts and other information about the Company by visiting the "Email Alerts" option under the News/Events section of the Investors & Media website section and submitting your email address.

*SERENITY At-Home represents the redesigned SERENITY III trial.

About IGALMI (dexmedetomidine) sublingual film

INDICATION

IGALMI (dexmedetomidine) sublingual film is a prescription medicine, administered under the supervision of a health care provider, that is placed under the tongue or behind the lower lip and is used for the acute treatment of agitation associated with schizophrenia and bipolar disorder I or II in adults. The safety and effectiveness of IGALMI has not been studied beyond 24 hours from the first dose. It is not known if IGALMI is safe and effective in children.

IMPORTANT SAFETY INFORMATION

IGALMI can cause serious side effects, including:

· Decreased blood pressure, low blood pressure upon standing, and slower than normal heart rate, which may be more likely in patients with low blood volume, diabetes, chronic high blood pressure, and older patients. IGALMI is taken under the supervision of a healthcare provider who will monitor vital signs (like blood pressure and heart rate) and alertness after IGALMI is administered to help prevent falling or fainting. Patients should be adequately hydrated and sit or lie down after taking IGALMI and instructed to tell their healthcare provider if they feel dizzy, lightheaded, or faint.

· Heart rhythm changes (QT interval prolongation). IGALMI should not be given to patients with an abnormal heart rhythm, a history of an irregular heartbeat, slow heart rate, low potassium, low magnesium, or taking other drugs that could affect heart rhythm. Taking IGALMI with a history of abnormal heart rhythm can increase the risk of torsades de pointes and sudden death. Patients should be instructed to tell their healthcare provider immediately if they feel faint or have heart palpitations.

· Sleepiness/drowsiness. Patients should not perform activities requiring mental alertness, such as driving or operating hazardous machinery, for at least 8 hours after taking IGALMI.

· Withdrawal reactions, tolerance, and decreased response/efficacy. IGALMI was not studied for longer than 24 hours after the first dose. Physical dependence, withdrawal symptoms (e.g., nausea, vomiting, agitation), and decreased response to IGALMI may occur if IGALMI is used longer than 24 hours.

The most common side effects of IGALMI in clinical studies were sleepiness or drowsiness, a prickling or tingling sensation or numbness of the mouth, dizziness, dry mouth, low blood pressure, and low blood pressure upon standing.

These are not all the possible side effects of IGALMI. Patients should speak with their healthcare provider for medical advice about side effects.

Patients should tell their healthcare provider about their medical history, including if they suffer from any known heart problems, low potassium, low magnesium, low blood pressure, low heart rate, diabetes, high blood pressure, history of fainting, or liver impairment. They should also tell their healthcare provider if they are pregnant or breastfeeding or take any medicines, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Patients should especially tell their healthcare provider if they take any drugs that lower blood pressure, change heart rate, or take anesthetics, sedatives, hypnotics, and opioids.

Everyone is encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088. You can also contact BioXcel Therapeutics, Inc. at 1-833-201- 1088 or [email protected].

Please see full Prescribing Information at igalmi.com.

About BXCL501

In indications other than those approved by the U.S. Food and Drug Administration (FDA) as IGALMI (dexmedetomidine) sublingual film, BXCL501 is an investigational proprietary, orally dissolving film formulation of dexmedetomidine, a selective alpha-2 adrenergic receptor agonist. BioXcel Therapeutics believes that BXCL501 potentially targets an important mediator of agitation, and the Company has observed anti-agitation results in multiple clinical trials across several neuropsychiatric disorders. BXCL501 is under investigation by BioXcel Therapeutics for the acute treatment of agitation associated with dementia due to probable Alzheimer’s disease and for the acute treatment of agitation associated with bipolar I or II disorder or schizophrenia in the at-home setting. The safety and efficacy of BXCL501 for these investigational uses have not been established. BXCL501 has been granted Breakthrough Therapy designation by the FDA for the acute treatment of agitation associated with dementia and Fast Track designation for the acute treatment of agitation associated with schizophrenia, bipolar disorders, and dementia.

About BXCL701

BXCL701 is an investigational, oral innate immune activator designed to initiate inflammation in the tumor microenvironment. Approved and experimental immunotherapies often fail to address cancers that appear "cold." Therefore, BXCL701 is being evaluated to determine if it can render "cold" tumors "hot," making them more detectable by the adaptive immune system and thereby facilitating the development of a strong anticancer immune response. OnkosXcel Therapeutics’ preclinical data support BXCL701’s potential synergy with both current checkpoint inhibitors and emerging immunotherapies directed to activate T-cells. BXCL701 is currently being developed as a potential therapy for the treatment of aggressive forms of prostate cancer and advanced solid tumors that are refractory or treatment naïve to checkpoint inhibitors. BXCL701 has received Orphan Drug Designation from the U.S. Food and Drug Administration in four indications: acute myelogenous leukemia, pancreatic cancer, stage IIb to IV melanoma, and soft tissue sarcoma. The U.S. Food and Drug Administration (FDA) designated as a Fast Track development program the investigation of BXCL701 in combination with a checkpoint inhibitor for treatment of patients with metastatic small cell neuroendocrine prostate cancer (SCNC) with progression on chemotherapy and no evidence of microsatellite instability. An 800+-subject clinical database, with data collected by the Company and others, supports the ongoing development of BXCL701.