On November 12, 2024 OmniAb, Inc. (NASDAQ: OABI) reported financial results for the three and nine months ended September 30, 2024, and provided operating and partner program updates (Press release, OmniAb, NOV 12, 2024, View Source [SID1234648200]).

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

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

                  Schedule Your 30 min Free Demo!

"We are pleased to report another successful quarter demonstrating our continued commitment to innovation with the expansion of our technology platform and a growing partnership portfolio. Third quarter results reflect consistent growth across key metrics, driven by our planned initiatives and the dedication of our team," said Matt Foehr, Chief Executive Officer of OmniAb. "Our business is well capitalized, and we have a highly scalable model with cutting-edge technologies that are driving significant opportunities. As we move forward, we remain focused on delivering value to our stakeholders and creating sustainable, profitable growth."

Third Quarter 2024 Financial Results

Revenue for the third quarter of 2024 was $4.2 million, compared with $5.5 million for the same period in 2023, with the decrease primarily due to timing of milestones and lower ion channel service revenue.

Research and development expense was $13.3 million for the third quarter of 2024, compared with $13.9 million for the same period in 2023, with the decrease primarily due to lower share-based compensation expense. General and administrative expense was $7.1 million for the third quarter of 2024, compared with $8.5 million for the same period in 2023, with the decrease primarily due to lower legal and share-based compensation expense.

Net loss for the third quarter of 2024 was $16.4 million, or $0.16 per share, compared with a net loss of $15.7 million, or $0.16 per share, for the same period in 2023.

Year-to-Date Financial Results

Revenue for the nine months ended September 30, 2024 was $15.6 million, compared with $29.3 million for the same period in 2023, due to lower milestone revenue primarily related to a $10 million milestone in the prior year period for the first commercial sale of TECVAYLI (teclistamab) in the European Union.

Research and development expense was $41.8 million for the nine months ended September 30, 2024, which was unchanged from the same period in 2023. General and administrative expense was $23.4 million for the nine months ended September 30, 2024, compared with $25.4 million for the same period in 2023, with the decrease primarily due to lower outside service expenses. Amortization of intangible assets increased for the nine months ended September 30, 2024, primarily due to a $1.2 million impairment related to assets associated with two legacy unpartnered Ab Initio programs. Other operating income, net increased for the nine months ended September 30, 2024, primarily due to a $2.4 million reduction in contingent liabilities attributed to changes in ion channel programs.

Net loss for the nine months ended September 30, 2024 was $49.0 million, or $0.48 per share, compared with a net loss of $36.6 million, or $0.37 per share, for the same period in 2023.

As of September 30, 2024, OmniAb had cash, cash equivalents and short-term investments of $59.4 million.

During the third quarter of 2024, OmniAb sold 2.0 million shares of common stock through its at-the-market (ATM) program pursuant to the sales agreement with Jefferies. The net proceeds from these sales were $8.5 million.

Subsequent to the end of the third quarter, OmniAb sold 0.7 million shares of common stock through its ATM program for net proceeds of $2.9 million, which will be reflected in its December 31, 2024 financial statements.

2024 Financial Guidance

OmniAb continues to expect total operating expenses in 2024 to be slightly less than total operating expenses in 2023.

OmniAb now expects to end 2024 with a cash balance in the range of $50 million to $60 million, which includes proceeds from its ATM program. Given the current expected progression of the existing partnered pipeline, OmniAb expects cash use in 2025 to be lower than in 2024 excluding recent ATM program proceeds.

Third Quarter 2024 and Recent Business Highlights

During the third quarter of 2024, OmniAb entered into three new platform license agreements including with 92Bio, Memorial Sloan Kettering Cancer Center and Queen Mary University of London. In addition, OmniAb recently expanded its ion channel screening relationship with Syngenta that was initially established last year. As of September 30, 2024, the Company had 86 active partners and 352 active programs, including 33 OmniAb-derived programs in clinical development or being commercialized. OmniAb also recently entered into a new platform license agreement with Incyte Corporation.

Third quarter 2024 and recent partner and business highlights include the following:

IMVT-1402

Immunovant announced that the Investigational New Drug (IND) application has been cleared for IMVT-1402 in rheumatoid arthritis (RA), with a potential best-in-class profile in difficult-to-treat (D2T) RA.
Immunovant announced that five IND applications for IMVT-1402 have been cleared across a range of therapeutic areas and U.S. Food & Drug Administration divisions. The company also announced that it is on track to initiate potentially registrational trials with IMVT-1402 in four to five indications, including Graves’ disease (GD) and D2T RA, by March 31, 2025.
Batoclimab

Immunovant reported positive results from the Phase 2a trial of batoclimab in GD. High-dose batoclimab achieved a 76% response rate and a 56% antithyroid drugs (ATD)-free response rate in patients uncontrolled on ATDs at week 12.
Immunovant announced that batoclimab trials in myasthenia gravis and chronic inflammatory demyelinating polyneuropathy are fully enrolled to support data disclosures by March 31, 2025. Data from batoclimab trials in thyroid eye disease are now expected in the second half of calendar year 2025. All batoclimab data will inform future trials with IMVT-1402.
Acasunlimab

Genmab announced that based on encouraging data from the Phase 2 trial in non-small cell lung cancer (NSCLC), a Phase 3 trial is expected to start before the end date of 2024.
TEV-53408

Teva recently disclosed Phase 1 data for TEV-53408 showing a potential best-in-class profile noting high affinity for IL-15, prolonged suppression of free IL-15, and potential for a low dosing frequency. TEV-53408 was well tolerated in a first-in-human study, and a proof-of-concept study in celiac disease is in progress. Additionally, Teva disclosed the initiation of a clinical study in vitiligo, an autoimmune disease.
Sugemalimab

CStone announced that the UK Medicines and Healthcare products Regulatory Agency (MHRA) has approved sugemalimab in combination with platinum-based chemotherapy as a first-line treatment for adult patients with metastatic NSCLC without EGFR-sensitive mutations or ALK, ROS1, RET genomic alterations.
CStone announced long-term survival data for sugemalimab in combination with platinum-based chemotherapy as first-line treatment of stage IV NSCLC at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress 2024. Four-year follow-up data showed that sugemalimab plus platinum-based chemotherapy continues to demonstrate a significant benefit in progression-free survival and overall survival compared with placebo plus platinum-based chemotherapy. The four-year survival rate was 32.1% versus 17.3%.
BC3195

BioCity presented interim clinical results on the safety and efficacy of its first-in-class antibody-drug conjugate BC3195, which targets CDH3 (P-Cadherin), in a Phase 1 clinical trial at ESMO (Free ESMO Whitepaper) 2024.
As of the data cut-off date (August 10, 2024), BC3195 demonstrated impressive antitumor activity in patients with advanced NSCLC with an overall response rate (ORR) of 36.4% (4 of 11 patients). The ORR was 80% (4 of 5 patients) in NSCLC with epidermal growth factor receptor mutations. BC3195 demonstrated manageable safety and tolerability, as well as favorable pharmacokinetic characteristics.
BC3195 is currently undergoing concurrent Phase 1 dose-optimization and dose-expansion studies in China.
Conference Call and Webcast

OmniAb management will host a conference call with accompanying slides today beginning at 4:30 p.m. Eastern time (1:30 p.m. Pacific time) to discuss this announcement and answer questions. To participate via telephone, please dial (800) 549 8228 using the conference ID 94399. Slides, as well as the live and replay webcast of the call, are available at View Source

On November 12, 2024 Olema Pharmaceuticals, Inc. ("Olema", "Olema Oncology", Nasdaq: OLMA), a clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of targeted therapies for breast cancer and beyond, reported financial results for the third quarter ended September 30, 2024, and announced a corporate update (Press release, Olema Oncology, NOV 12, 2024, View Source [SID1234648198]).

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

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

                  Schedule Your 30 min Free Demo!

"We look forward to presenting updated data from our ongoing Phase 2 clinical study of palazestrant in combination with ribociclib in frontline metastatic breast cancer patients at SABCS in December. The OPERA-01 Phase 3 clinical trial of palazestrant as a monotherapy in second/third-line patients continues to advance and we remain on track for top-line readout in 2026," said Sean P. Bohen, M.D., Ph.D., President and Chief Executive Officer of Olema Oncology. "At ENA 2024, we presented three new, robust preclinical data sets. Palazestrant demonstrated combinability and enhanced tumor suppression with both everolimus and capivasertib. OP-3136, our potent and selective KAT6 inhibitor, demonstrated robust anti-tumor activity as a single agent, as well as synergy and enhanced anti-tumor activity in combination with palazestrant. These data reinforce our belief in the potential of OP-3136 as an exciting new therapy for breast and other cancers, and we remain on track to submit the IND application before year end."

Recent Progress

•
Continued enrollment of patients in OPERA-01, the pivotal Phase 3 clinical trial of palazestrant as a monotherapy in second/third-line ER+/HER2- metastatic breast cancer.
•
Presented preclinical data for OP-3136 and palazestrant at the 36th EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) Symposium on Molecular Targets and Cancer Therapeutics (ENA 2024) in Barcelona, Spain.
•
Initiated Phase 1b/2 clinical study of palazestrant in combination with everolimus.
•
Successfully completed Investigational New Drug (IND)-enabling studies for OP-3136.

Anticipated Upcoming Milestones

•
Present updated Phase 2 data showing palazestrant in combination with ribociclib at the San Antonio Breast Cancer Symposium (SABCS) in December 2024.
•
Submit the IND application for OP-3136 to the U.S. Food and Drug Administration (FDA) before year-end; initiate the Phase 1 clinical study for OP-3136 in early 2025.
Third Quarter 2024 Financial Results

Cash, cash equivalents, and marketable securities as of September 30, 2024, were $214.8 million.

Net loss for the quarter ended September 30, 2024, was $34.6 million, as compared to $21.5 million for the quarter ended September 30, 2023. The increase in net loss for the third quarter was primarily related to increased spending on clinical development and research activities as a result of late-stage clinical trials for palazestrant and the advancement of our KAT6 inhibitor program, as well as general and administrative (G&A) activities. The increase was partially offset by higher interest income earned from marketable securities.

GAAP research and development (R&D) expenses were $33.2 million for the quarter ended September 30, 2024, as compared to $19.5 million for the quarter ended September 30, 2023. The increase in R&D expenses was primarily related to increased spending on clinical operations and development-related activities as we continue to advance palazestrant through late-stage clinical trials, research-related activities associated with the advancement of our KAT6 inhibitor program, and personnel related costs, including an increase in non-cash stock-based compensation expense of $1.5 million.

Non-GAAP R&D expenses were $28.9 million for the quarter ended September 30, 2024, which excluded $4.3 million non-cash stock-based compensation expense. Non-GAAP R&D expenses were $16.7 million for the quarter ended September 30, 2023, excluding $2.8 million non-cash stock-based compensation expense. A reconciliation of GAAP to non-GAAP financial measures used in this press release can be found at the end of this press release.

GAAP G&A expenses were $4.4 million for the quarter ended September 30, 2024, as compared to $3.9 million for the quarter ended September 30, 2023. The increase in G&A expenses was primarily due to increased spending on corporate-related costs and an increase in non-cash stock-based compensation expense of less than $0.1 million.

Non-GAAP G&A expenses were $3.0 million for the quarter ended September 30, 2024, excluding $1.3 million non-cash stock-based compensation expense. Non-GAAP G&A expenses were $2.6 million for the quarter ended September 30, 2023, excluding $1.3 million non-cash stock-based compensation expense. A reconciliation of GAAP to non-GAAP financial measures used in this press release can be found at the end of this press release.

About Palazestrant (OP-1250)

Palazestrant (OP-1250) is a novel, orally available small molecule with dual activity as both a complete estrogen receptor (ER) antagonist (CERAN) and selective ER degrader (SERD). It is currently being investigated in patients with recurrent, locally advanced or metastatic ER-positive (ER+), human epidermal growth factor receptor 2-negative (HER2-) breast cancer. In clinical studies, palazestrant completely blocks ER-driven transcriptional activity in both wild-type and mutant forms of metastatic ER+ breast cancer and has demonstrated anti-tumor efficacy along with attractive pharmacokinetics and exposure, favorable tolerability, CNS penetration, and combinability with CDK4/6 inhibitors. Palazestrant has been granted U.S. Food and Drug Administration (FDA) Fast Track designation for the treatment of ER+/HER2- metastatic breast cancer that has progressed following one or more lines of endocrine therapy with at least one line given in combination with a CDK4/6 inhibitor. It is being evaluated both as a single agent in an ongoing Phase 3 clinical trial, OPERA-01, and in Phase 1/2 combination studies with CDK4/6 inhibitors (palbociclib and ribociclib), a PI3Ka inhibitor (alpelisib), and an mTOR inhibitor (everolimus). For more information on OPERA-01, please visit www.opera01study.com.

About OP-3136

OP-3136 is a novel, orally available small molecule that potently and selectively inhibits KAT6, an epigenetic target that is dysregulated in breast and other cancers. In preclinical studies, OP-3136 has demonstrated significant anti-proliferative activity in ER+ breast cancer models and is combinable and synergistic with endocrine therapies including palazestrant and CDK4/6 inhibitors. Olema has successfully completed IND-enabling studies in support of a potential Investigational New Drug (IND) application with the FDA and expects to initiate Phase 1 clinical trials for OP-3136 in early 2025.

On November 12, 2024 Nuvalent, Inc. (Nasdaq: NUVL), a clinical-stage biopharmaceutical company focused on creating precisely targeted therapies for clinically proven kinase targets in cancer, reported pipeline progress, reiterated key anticipated milestones, and announced third quarter 2024 financial results (Press release, Nuvalent, NOV 12, 2024, View Source [SID1234648196]).

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

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

                  Schedule Your 30 min Free Demo!

"Throughout 2024, the Nuvalent team has made significant strides in advancing our pipeline of novel kinase inhibitors, exemplified by the achievement of all of the 2024 milestones originally laid out in our OnTarget 2026 operating plan. We believe our recent upsized public offering reflects the shared excitement around these achievements and the potential for multiple value-creating catalysts ahead," said Alexandra Balcom, Chief Financial Officer at Nuvalent. "We believe we are well-positioned to execute on our mid- and long-term goals and remain sharply focused on moving our programs forward as efficiently as possible for patients."

"Our portfolio achievements and the acceleration of our development timelines have stemmed from a foundational tenet of our approach to drug discovery and development: collaboration with leading physician-scientists from the outset of each program," said James Porter, Ph.D., Chief Executive Officer at Nuvalent. "Today, we are thrilled to welcome Dr. Alice Shaw, Chief of Strategic Partnerships at Dana-Farber Cancer Institute, to our Scientific Advisory Board in recognition of her invaluable contributions to our ROS1 and ALK programs since our company’s formation. A leading expert in targeted oncology, Dr. Shaw’s research has deepened the scientific understanding of oncogene-driven lung cancers and their mechanisms of resistance, and contributed to numerous new therapeutic options for patients. We look forward to continuing to leverage her insights and expertise as we advance our clinical programs and discovery pipeline."

"Targeted kinase inhibitors are important treatment options for cancer patients, but key challenges, including drug resistance and off-target side effects, can limit their therapeutic impact," said Dr. Shaw. "I am excited by Nuvalent’s pursuit of new therapeutic approaches to overcome the limitations of existing therapies. The clinical proof-of-concept data from both the ROS1 and ALK programs are encouraging and support the approach of designing highly selective and brain-penetrant inhibitors that retain potency against known resistance mutations. I look forward to working with the team to advance the current pipeline of novel therapeutic candidates and identify additional areas of medical need where Nuvalent could potentially make a meaningful impact for patients."

Recent Pipeline Progress and Anticipated Milestones

ROS1 Program

•
Nuvalent recently reported updated data from the fully enrolled Phase 1 portion of its ongoing ARROS-1 Phase 1/2 clinical trial in patients with advanced ROS1-positive NSCLC during an oral presentation at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress 2024. Data presented showed that treatment with zidesamtinib resulted in durable clinical responses in heavily pre-treated patients with ROS1-positive NSCLC, including in subgroups of patients who had likely exhausted all available therapies including lorlatinib and/or repotrectinib, had a history of brain metastases, or had the G2032R resistance mutation. Additionally, zidesamtinib was well-tolerated with a preliminary safety profile that was favorable and consistent with its ROS1-selective, TRK-sparing design.
•
Additionally, during a poster session at the ESMO (Free ESMO Whitepaper) Congress 2024, the company presented new preclinical data further characterizing the intracranial activity of zidesamtinib.
•
Enrollment is ongoing in the Phase 2 portion of the ARROS-1 Phase 1/2 trial of zidesamtinib for patients with advanced ROS1-positive NSCLC and other solid tumors. Between September 2023 and September 1, 2024, 227 patients were enrolled in the ongoing single-arm, multi-cohort Phase 2 portion of the ARROS-1 trial, which is designed with registrational intent. The company expects to report pivotal data from this trial in 2025.

ALK Program

•
Nuvalent recently reported updated data from the fully enrolled Phase 1 portion of its ongoing ALKOVE-1 Phase 1/2 clinical trial in patients with advanced ALK-positive NSCLC during an oral presentation at the ESMO (Free ESMO Whitepaper) Congress 2024. Data presented showed that treatment with NVL-655 resulted in durable clinical responses in heavily pre-treated patients with ALK-positive NSCLC, including in subgroups of patients who had likely exhausted all available therapies including lorlatinib, had a history of brain metastases, or had single or compound ALK resistance mutations. Additionally, NVL-655 demonstrated a favorable preliminary safety profile consistent with its ALK-selective, TRK-sparing design.
•
Enrollment is ongoing in the Phase 2 portion of the ALKOVE-1 Phase 1/2 trial of NVL-655 for patients with advanced ALK-positive NSCLC and other solid tumors. Between February 2024 and September 1, 2024, 229 patients were enrolled in the ongoing single-arm, multi-cohort Phase 2 portion of the ALKOVE-1 trial, which is designed with registrational intent for TKI pre-treated patients. The company expects to report pivotal data from this trial in 2025.
•
Nuvalent recently announced the ALKAZAR Phase 3 trial, its front-line development strategy for the company’s ALK program. The Phase 3 ALKAZAR trial will be a global, randomized, controlled trial designed to evaluate NVL-655 versus the current standard of care for the treatment of patients with TKI-naïve ALK-positive NSCLC. Patients will be randomized 1:1 to receive NVL-655 monotherapy or ALECENSA (alectinib) monotherapy, reflecting input from collaborating physician-scientists and alignment with the U.S. Food and Drug Administration (FDA). The company plans to initiate the ALKAZAR study in the first half of 2025.
•
A manuscript describing the design and characterization of NVL-655 and detailing Nuvalent’s approach to rationally targeting ALK, was published in Cancer Discovery, a journal of the American Association for Cancer Research (AACR) (Free AACR Whitepaper). The publication, Nuvalent’s second in Cancer Discovery, provides a comprehensive assessment of NVL-655’s preclinical activity and includes preliminary clinical case studies.

HER2 Program

•
Enrollment is ongoing in the HEROEX-1 Phase 1a/1b clinical trial evaluating NVL-330 for pre-treated patients with HER2-altered NSCLC. The trial will evaluate the overall safety and tolerability of NVL-330. Additional objectives include determination of the recommended Phase 2 dose (RP2D), characterization of the pharmacokinetic profile, and preliminary evaluation of anti-tumor activity.

Corporate Highlight

•
Nuvalent has appointed leading medical oncologist Alice T. Shaw, M.D., Ph.D., to its Scientific Advisory Board. Dr. Shaw is the Chief of Strategic Partnerships at Dana-Farber Cancer Institute and oversees the collaborative efforts between researchers and the life sciences industry to help accelerate the development of new and innovative therapies for patients with cancer. Dr. Shaw was previously Vice President and Global Head of Translational Clinical Oncology at Novartis Institutes for BioMedical Research (NIBR) where she led early drug development in oncology for almost five years. Prior to Novartis, Dr. Shaw was the Director of the Center for Thoracic Cancers at Massachusetts General Hospital (MGH), the Paula O’Keeffe Endowed Chair of Thoracic Oncology at MGH, Co-Leader of the Dana-Farber/Harvard Cancer Center Thoracic Oncology Program, and Professor of Medicine at Harvard Medical School. During her time at MGH, Dr. Shaw studied resistance to targeted therapies in the lab and the clinic, and helped develop numerous targeted therapies for patients with oncogene-driven lung cancer. She was the overall principal investigator of multiple Phase 1, 2 and 3 studies, including registrational trials leading to FDA approval of crizotinib for ALK and ROS1-positive NSCLC, ceritinib, alectinib, and lorlatinib. Dr. Shaw was recently elected to the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Board of Directors. She was previously Co-Leader of the SU2C Lung Cancer Dream Team and a standing member of the US FDA Oncologic Drug Advisory Committee (ODAC).

Financing Highlight

•
On September 18, 2024, Nuvalent closed an upsized underwritten public offering of 5,750,000 shares of Class A common stock, which included 750,000 shares of Class A common stock sold pursuant to the exercise in full by the underwriters of their option to purchase additional shares, at a price to the public of $100.00 per share. The gross proceeds of the offering were approximately $575.0 million, before deducting underwriting discounts and commissions and other offering expenses.

Upcoming Events

•
Guggenheim’s Inaugural Healthcare Innovation Conference: Management will be participating in a fireside chat on Wednesday, November 13, 2024, at 9:00 a.m. ET in Boston.
•
Stifel 2024 Healthcare Conference: Management will be participating in a fireside chat on Tuesday, November 19, 2024, at 3:35 p.m. ET in NYC.

A live webcast of each fireside chat will be available in the Investors section of Nuvalent’s website at www.nuvalent.com, and will be archived for 30 days following the conference.

Third Quarter 2024 Financial Results

•
Cash Position: Cash, cash equivalents and marketable securities were $1.2 billion as of September 30, 2024. The company’s cash, cash equivalents and marketable securities as of September 30, 2024, inclusive of the proceeds from the $575 million public offering, are expected to extend the company’s operating runway into 2028.
•
R&D Expenses: Research and development (R&D) expenses were $60.6 million for the third quarter of 2024.
•
G&A Expenses: General and administrative (G&A) expenses were $15.8 million for the third quarter of 2024.
•
Net Loss: Net loss was $84.3 million for the third quarter of 2024.

About OnTarget 2026

OnTarget 2026 delineates Nuvalent’s 3-year operating plan towards bringing new, potential best-in-class medicines to patients with cancer. As part of this plan announced in January 2024, Nuvalent outlined the following anticipated milestones throughout 2024, leading to the company’s first potential pivotal data in 2025 and first potential approved product in 2026:

•
2024: Execute on Global Registrational Strategies
•
2025: First Pivotal Data
•
2026: First Approved Product

On November 12, 2024 2seventy bio, Inc. (Nasdaq: TSVT), reported financial results and recent highlights for the third quarter ended September 30, 2024 (Press release, 2seventy bio, NOV 12, 2024, View Sourcenode/8831/html#q32024earningspr_118244pm.htm" target="_blank" title="View Sourcenode/8831/html#q32024earningspr_118244pm.htm" rel="nofollow">View Source [SID1234648193]).

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

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

                  Schedule Your 30 min Free Demo!

"We are very pleased to report 42% sequential growth in quarterly Abecma sales, which was part of a continued expansion of the CAR-T class into earlier lines for multiple myeloma. When combined with the significant progress our team has made in streamlining our cost structure, 2seventy continues to make meaningful progress on our goal of achieving breakeven operations," said Chip Baird, chief executive officer, 2seventy bio. "Abecma has a differentiated safety profile, as further supported by recent real-world evidence. Physicians familiar with Abecma understand that with effective use of bridging, they can achieve deep and durable responses. With more than 16,000 patients diagnosed annually in the U.S., we believe Abecma will continue to hold a meaningful place in a growing but highly competitive multiple myeloma market, and we remain committed to expanding the reach of Abecma to as many patients as possible."

ABECMA COMMERCIAL AND REGULATORY HIGHLIGHTS
•Third quarter Abecma (idecabtagene vicleucel; ide-cel) U.S. revenues, as reported by Bristol Myers Squibb (BMS), were $77 million. The Company expects Abecma revenues of approximately $240 – $250 million for the full year of 2024.
•2seventy bio and BMS continue to focus on competitively differentiating Abecma’s safety and efficacy profile supported by the strength of the KarMMa-3 and real-world data.
•2seventy bio, in partnership with study sponsor BMS, has discontinued enrollment in its ongoing Phase 3 KarMMa-9 study evaluating Abecma with lenalidomide maintenance versus lenalidomide maintenance alone in patients with newly diagnosed multiple myeloma (NDMM) who have suboptimal response to autologous stem cell transplant. 2seventy and BMS remain committed to expanding the reach of Abecma to as many multiple myeloma patients as possible.
•2seventy bio and BMS share equally in all profits and losses related to development, manufacturing, and commercialization of Abecma in the U.S. The Company reported collaborative arrangement revenue of approximately $11 million related to the collaboration with BMS for the three months ended September 30, 2024.

SELECT THIRD QUARTER FINANCIAL RESULTS
•Total revenues were $13.5 million for the three months ended September 30, 2024, compared to $12.0 million for the three months ended September 30, 2023. Total revenues were $34.9 million for the nine months ended September 30, 2024, compared to $89.7 million for the nine months ended September 30, 2023.
•Research and development expenses were $8.3 million for the three months ended September 30, 2024, compared to $51.3 million for the three months ended September 30, 2023. Research and development expenses were $68.3 million for the nine months ended September 30, 2024, compared to $179.5 million for the nine months ended September 30, 2023.
•Selling, general and administrative expenses were $12.9 million for the three months ended September 30, 2024, compared to $13.0 million for the three months ended September 30, 2023. Selling, general and administrative expenses were $35.4 million for the nine months ended September 30, 2024, compared to $53.2 million for the nine months ended September 30, 2023.
•Net loss was $9.9 million for the three months ended September 30, 2024, compared to $71.6 million for the three months ended September 30, 2023. Net loss was $37.7 million for the nine months ended September 30, 2024, compared to $160.7 million for the nine months ended September 30, 2023.
•The Company reiterates its net cash spend range of $40-60 million for 2024.
•Cash, cash equivalents, and marketable securities totaled $192 million as of September 30, 2024; the Company continues to expect to have cash runway beyond 2027.

Conference Call Information
2seventy bio will host a conference call and live webcast today, November 12 at 8:00 a.m. ET to discuss third quarter 2024 financial results and recent business highlights. Participants can access the conference call live via webcast which is available on the Investors and Media page of the Company’s website at View Source Participants who wish to ask a question may register here to receive dial-in numbers and a unique pin to join the call.

A replay of the webcast may be accessed from the "News and Events" page in the Investors and Media section of our website at View Source and will be available for 30 days following the event.

On November 12, 2024 Moleculin Biotech, Inc., (Nasdaq: MBRX) ("Moleculin" or the "Company"), a late-stage pharmaceutical company with a broad portfolio of drug candidates targeting hard-to-treat tumors and viruses, reported it has received Institutional Review Board (IRB) approval for its Phase 3 pivotal trial protocol evaluating Annamycin in combination with Cytarabine (also known as "Ara-C" and for which the combination of Annamycin and Ara-C is referred to as "AnnAraC") for the treatment of AML patients who are refractory to or relapsed after induction therapy (R/R AML) (MB-108) (Press release, Moleculin, NOV 12, 2024, View Source [SID1234648180]). This Phase 3 "MIRACLE" trial (derived from Moleculin R/R AML AnnAraC Clinical Evaluation) will be a global trial, including sites in the US.

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

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

                  Schedule Your 30 min Free Demo!

"IRB approval marks an important milestone towards the launch of our MIRACLE pivotal Phase 3 trial of Annamycin in AML patients. Our team is focused on getting clinical trial sites up and running, and we believe we will be in a position to commence enrollment in the first quarter of 2025," commented Walter Klemp, Chairman and Chief Executive Officer of Moleculin. "We have been extremely busy meeting with potential investigators for this study and are highly encouraged by these conversations. The positive feedback from the clinicians on the strength of our data generated to date, along with our alignment with FDA on the strategic design of the MIRACLE study, gives us a high level of confidence as we take these next steps toward potentially bringing a much needed solution to AML patients."

The MIRACLE study, subject to appropriate future filings with and potential additional feedback from the FDA and their foreign equivalents, is expected to initially utilize an adaptive design whereby the first 75 to 90 subjects will be randomized in Part A of the trial to receive high dose cytarabine (HiDAC) combined with either placebo, 190 mg/m2 of Annamycin, or 230 mg/m2 of Annamycin, such doses were specifically recommended by the FDA in our end of Phase 1B/2 meeting. At that point, the trial will be unblinded to select the optimum dose for Annamycin. For Part B of the trial, approximately 240 additional subjects will be randomized to receive either HiDAC plus placebo or HiDAC plus the optimum dose of Annamycin. The selection of the optimum dose will be based on the overall balance of safety, pharmacokinetics and efficacy, consistent with the FDA’s new Project Optimus initiative.

Annamycin currently has Fast Track Status and Orphan Drug Designation from the FDA for the treatment of relapsed or refractory acute myeloid leukemia, in addition to Orphan Drug Designation for the treatment of soft tissue sarcoma. Furthermore, Annamycin has Orphan Drug Designation for the treatment of relapsed or refractory acute myeloid leukemia from the European Medicines Agency (EMA).