On November 14, 2024 bluebird bio, Inc. (NASDAQ: BLUE) ("bluebird bio" or the "Company") reported third quarter results and business highlights for the quarter ended September 30, 2024, including recent commercial and operational progress (Press release, bluebird bio, NOV 14, 2024, View Source [SID1234648391]).

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"Patient starts more than doubled from our second to third quarter update, providing clear evidence that our commercial launches continued to accelerate," said Andrew Obenshain, chief executive officer. "This momentum, coupled with steps we took in the third quarter to increase manufacturing capacity for ZYNTEGLO and optimize our cost structure, is propelling bluebird forward on our path to becoming a sustainable commercial gene therapy company. We remain focused on securing additional cash resources to extend our runway, which we believe would enable us to achieve this vision and reach cash flow break-even in the second half of 2025."

COMMERCIAL LAUNCH UPDATES

Continued commercial momentum across the portfolio

57 patient starts completed to date in 2024 (35 ZYNTEGLO, 17 LYFGENIA, 5 SKYSONA).
17 additional starts scheduled through the remainder of 2024.
Evidence of strong commercial demand, with 30 patient starts already scheduled in 2025, supporting the potential for cash flow breakeven in the second half of 2025.
More than 70 activated QTCs, with 40% having initiated or completed treatment for at least one patient.
Validated access and reimbursement strategy is driving favorable coverage landscape

To date, more than half of all states have affirmed coverage for LYFGENIA through a preferred drug list or published coverage criteria.
Nearly 50% of Medicaid-insured individuals with sickle cell disease in the U.S. live in a state that has already completed prior authorization approval for the use of LYFGENIA for at least one patient.
Multiple outcomes-based agreements are published and in place for LYFGENIA with national commercial payer organizations, representing more than 200 million U.S. lives.
DATA PRESENTATIONS AT ASH (Free ASH Whitepaper) 2024

Updated data from the Company’s lentiviral vector (LVV) gene addition programs in patients with sickle cell disease who have a history of vaso-occlusive events and patients with beta-thalassemia who require regular blood transfusions will be presented at the 66th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition. The meeting will take place December 7-10, 2024 at the San Diego Convention Center and online.

SICKLE CELL DISEASE DATA

Oral Presentation [#511]: An Update on Lovotibeglogene Autotemcel (lovo-cel) Clinical Trials for Sickle Cell Disease (SCD) and Analysis of Early Predictors of Response to Lovo-cel
Presenting Author: Dr. Stacey Rifkin-Zenenberg (Hackensack)
Date/Time: Sunday, December 8, 2024, 9:30 a.m. – 11:00 a.m. PT
Poster Presentation [#3576]: Participants with a History of Stroke in Lovotibeglogene Autotemcel (lovo-cel) Clinical Trials
Presenting Author: Dr. Jen Jaroscak (The Medical University of South Carolina)
Date/Time: Sunday, December 8, 2024, 6:00 p.m. – 8:00 p.m. PT
BETA-THALASSEMIA DATA

Poster Presentation [#2194]: Betibeglogene Autotemcel (beti-cel) Gene Addition Therapy results in durable Hemoglobin A (HbA) Production with up to 10 Years of Follow-Up in Participants with Transfusion-Dependent β-Thalassemia
Presenting Author: Dr. Alexis A Thompson (Children’s Hospital of Philadelphia)
Date/Time: Saturday, December 7, 2024, 5:30 p.m. – 7:30 p.m. PT
Abstracts outlining bluebird bio’s accepted data at ASH (Free ASH Whitepaper) 2024 are available on the ASH (Free ASH Whitepaper) conference website.

THIRD QUARTER FINANCIAL HIGHLIGHTS

Cash Position: The Company’s cash, cash equivalents and restricted cash balance was approximately $118.7 million, including restricted cash of approximately $48.0 million, as of September 30, 2024.

bluebird and Hercules are engaging collaboratively as bluebird works to secure adequate cash runway to obtain additional financing and reach cash flow break-even. Based on current forecasts, which assume continued cost-saving initiatives, successfully renegotiating key contracts, and continued collaborative engagement from Hercules, we expect our existing cash and cash equivalents will enable us to fund our operations into the first quarter of 2025.

The Company anticipates quarterly cash flow break-even in the second half of 2025, assuming it scales to approximately 40 drug product deliveries per quarter and obtains additional cash resources to extend its runway.
Revenue, net: Total revenue, net was $10.6 million for the three months ended September 30, 2024, compared to $12.3 million for the three months ended September 30, 2023, driven by quarter-to-quarter variability in drug product infusions.

Revenue for the third quarter includes revenue from LYFGENIA, following the completion of the first infusion for sickle cell disease.

bluebird previously guided to an anticipated reduction of net revenue in the third quarter; the Company now anticipates net revenue of at least $25 million in the fourth quarter 2024, as previously reported patient starts are infused.
Cost of Product Revenue: Cost of product revenue was $11.8 million for the three months ended September 30, 2024, compared to $9.1 million for the three months ended September 30, 2023.
SG&A Expenses: Selling, general and administrative expenses were $39.8 million for the three months ended September 30, 2024, compared to $40.8 million for the three months ended September 30, 2023. The decrease of $1.0 million was primarily driven by decrease in employee compensation, benefit, and other headcount related expenses, commercial expenses, and facility fees, partially offset by increased professional services fees.
R&D Expenses: Research and development expenses were $23.2 million for the three months ended September 30, 2024, compared to $58.5 million for the three months ended September 30, 2023. The decrease of $35.3 million was primarily driven by material production shift to inventory and cost of product revenue as well as decreased employee compensation, benefit, and other headcount related expenses, consulting fees, and facility and information technology fees.
Net income (loss): Net loss was $60.8 million for the three months ended September 30, 2024, compared to a net loss of $87.2 million for the three months ended September 30, 2023.
CONFERENCE CALL DETAILS

bluebird will hold a conference call to discuss its third quarter 2024 results and business updates today, Wednesday, November 14, 2024, at 8:00 am ET.

To access the live conference call via telephone, please register at this link to receive a dial in number and unique PIN.

To access the live webcast, please visit the "Events & Presentations" page within the Investors & Media section of the bluebird bio website at View Source A replay of the webcast will be available on the bluebird bio website for 90 days following the event.

On November 14, 2024 bioAffinity Technologies, Inc. (Nasdaq: BIAF; BIAFW), a biotechnology company focused on the need for noninvasive, accurate tests for the detection of early-stage lung cancer and other lung diseases, reported financial results for the three months ended September 30, 2024 (Press release, BioAffinity Technologies, NOV 14, 2024, View Source [SID1234648390]).

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Key Highlights

● Generated quarterly revenue of $2.4 million in the third quarter of 2024.
● More than 1,300% growth rate for CyPath Lung orders in first nine months of 2024 over full-year 2023.
● Number of physician offices signed increased by 75% compared to the second quarter of 2024, setting the stage for acceleration of CyPath Lung sales in the quarters ahead.
● In October 2024, CyPath Lung was added to the U.S. Federal Supply Schedule (FSS), a procurement system that provides the Veterans Health Administration and the Military Health System streamlined access to state-of-the-art healthcare products and services. Under the FSS contract, Veterans at high risk for lung cancer will have easy access to CyPath Lung through 1,380 government health care facilities. Approximately 8,000 Veterans are treated for lung cancer annually, according to the VA.
● Referrals and word-of-mouth from physicians, including key opinion leaders (KOLs), continues to be a key driver for expanding CyPath Lung in states beyond Texas; now receiving CyPath Lung orders from physicians in 11 states, up from eight in the second quarter of 2024. In addition to previously reported orders from Pennsylvania, New Jersey, North Carolina, Arizona, Michigan, California, and Ohio, physicians in Alabama, Louisiana and Illinois have also begun ordering CyPath Lung tests.
● Added new sales representative to target increasing opportunities in Texas.

● Continued to advance new product development initiatives in collaboration with Brooke Army Medical Center, the U.S Department of Defense’s largest military health organization, focusing on tests that use the Company’s artificial intelligence and flow cytometry platform for diagnosing COPD and a companion test with bronchoscopy.
● Economic study published in Journal of Health Economics and Outcomes Research, a peer-reviewed journal, concludes that adding CyPath Lung to the standard of care for Medicare patients with a positive lung cancer screening could have saved an average of $2,773 per patient for total cost savings of $379 million in 2022.
● Awarded a Certificate of Grant of Patent from the Japan Patent Office for the Company’s unique method using flow cytometry to predict the likelihood of lung disease, including the CyPath Lung diagnostic test for early-stage lung cancer.
● Appointed William Bauta, Ph.D., as Chief Science Officer following the retirement of Vivienne I. Rebel, M.D., Ph.D. Dr. Bauta joined bioAffinity in 2016 as Senior Vice President. Previously, he was Associate Director of science at Genzyme.
● Successfully closed a $2.7 million registered direct offering and concurrent private placement to fund continued growth.

Management Commentary

"We are pleased with the continued progress we achieved in the third quarter, highlighted by a 75% growth in the number of physician offices signing on to offer CyPath Lung. This significant expansion not only reflects the increasing recognition of our test’s value in early lung cancer detection but also lays a strong foundation for accelerating sales growth in the coming quarters," bioAffinity President and Chief Executive Officer Maria Zannes said. "With CyPath Lung now being used in 11 states and its recent addition to the U.S. Federal Supply Schedule, we are making meaningful strides in broadening access to this innovative diagnostic tool.

"Our focus remains on expanding our operations and strengthening our foothold in this rapidly growing market," Zannes continued. "Our strategic approach in Texas has resulted in a robust sales and support infrastructure that has us well-equipped to meet rising demand and accelerate our nationwide growth. As we look toward the future, we are confident that these efforts will not only fuel our success but also advance our mission to enhance patient outcomes through groundbreaking, noninvasive cancer diagnostics."

Third Quarter Financial Results

Revenue for the third quarter of 2024 was $2.4 million, compared with $298,000 revenue for the prior-year period. The majority of the year-over-year increase is through the acquisition of Precision Pathology Laboratory Services, LLC (PPLS). Revenue is primarily generated from patient service fees, including billing for CyPath Lung tests, with additional revenues generated from histology service fees and medical director fees.

Research and development expenses were $274,000 for the third quarter of 2024, compared with $330,000 for the comparable period in 2023. The decrease was primarily due to higher R&D laboratory supply and equipment costs following the acquisition of PPLS in the prior year period.

Clinical development expenses were $94,000 for the third quarter of 2024, compared with $106,000 for the third quarter of 2023. The decrease was primarily attributable to higher professional fees in the prior year period related to evaluating the clinical strategy for the Company’s Food and Drug Administration (FDA) pivotal CyPath Lung clinical trial.

Selling, general and administrative expenses were $2.4 million for the third quarter of 2024, compared with $2.0 million for the comparable period in 2023. The increase was primarily attributed to an increase in sales personnel and services to support the launch of CyPath Lung, together with acquired general and administrative costs from PPLS.

Net loss for the third quarter of 2024 was $2.0 million, or $0.16 per share, a $0.3 million improvement from a net loss of $2.3 million, or $0.26 per share, for the comparable period in 2023.

Cash and cash equivalents as of September 30, 2024, were $0.8 million, compared with $2.8 million as of December 31, 2023. Subsequent to the end of the third quarter of 2024, bioAffinity Technologies raised aggregate gross proceeds of $2.7 million in a registered direct offering and concurrent private placement closed on October 21, 2024.

On November 14, 2024 BeiGene, Ltd. (NASDAQ: BGNE; HKEX: 06160; SSE: 688235), a global oncology company, reported its intent to change the Company’s name to BeOne Medicines Ltd., confirming its commitment to develop innovative medicines to eliminate cancer by partnering with the global community to serve as many patients as possible (Press release, BeiGene, NOV 14, 2024, View Source [SID1234648389]).

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"Cancer, a leading cause of death worldwide, exacts an immense toll on individuals, families, and communities. No person, family, scientist, clinician, hospital, policy maker, company or country can or should face this devastating disease alone. We all must work together to win, which is why we are committed to playing a critical role and unifying the global community in the fight against cancer. Our focus is to not only bring innovative medicines to as many people as possible, but also to identify and address the challenges that impede access, making treatments more accessible and affordable," said John V. Oyler, Co-Founder, Chairman and CEO at BeiGene. "We have already helped more than 1.4 million patients, and with one of the most prolific oncology pipelines, this year we will bring more than 10 new potential medicines into the clinic. I look forward to our next chapter of growth as BeOne."

About Our Brand Evolution: BeOne Medicines

The proposed new name and logo illustrates our focus on coming together against cancer. Key attributes of the new logo design include:

"Be" represents the fundamental goal of any patient with cancer – simply to be free of disease;
"One" emphasizes our unity as a team and focus on bringing together patients, caregivers, scientists, healthcare providers, governments and industry with a shared mission to eliminate cancer together;
The word "Onc" spelled in red within "One" illustrates our redoubled commitment to oncology; and
The power button within the last "e" represents our always "on" approach in pursuing novel medicines that turn cancer "off" by disrupting key drivers of cancer cell growth and survival, harnessing the body’s immune system to attack tumors, and targeting specific biomarkers of cancer. The tilted angle of the button embodies our path which is not always straight-forward as we push the boundaries of science.
The new name is part of a broader strategic growth plan that has enabled the Company’s global oncology leadership since its inception in 2010. The Company recently reported $1 billion in quarterly total revenue driven by strong growth in product revenue in the U.S. and Europe. To support its expansive clinical portfolio and global growth, the Company opened its $800 million flagship clinical R&D and manufacturing facility at the Princeton West Innovation Campus in Hopewell, N.J. in July. Once the name is approved by shareholders, the company’s stock ticker on Nasdaq will change to "ONC".

The Company’s nearly 11,000 colleagues have advanced more than 20 molecules into the clinic and secured regulatory approvals across five continents. Its unique global research and development team, including clinical operations and development, is comprised of more than 3,600 colleagues conducting clinical trials across Europe, North and South America, Australia, and Asia in more than 45 countries. Its portfolio strategy emphasizes rapid generation of early-stage clinical proof-of-concept data enabled by its speed- and cost-advantaged ("Fast to Proof of Concept") approach to global clinical operations. The Company has solidified its leadership in hematology with BRUKINSA (zanubrutinib), which has the broadest label of any BTK inhibitor and, in the U.S., is the leader in new patient starts in both frontline and relapsed/refractory chronic lymphocytic leukemia in addition to all other approved B-cell malignancies. The Company is advancing this impactful therapy, which is approved in more than 70 markets, as the cornerstone of its hematology franchise as a monotherapy and as a backbone for potential best-in-class combinations with late-stage BCL2 inhibitor sonrotoclax and BTK degrader BGB-16673. In addition, the Company is focused on growing its leadership in solid tumors with its PD-1 inhibitor TEVIMBRA (tislelizumab) and by advancing potential best-in-class assets for breast, lung and gastrointestinal cancers across several modalities, including antibody drug conjugates, multi-specific antibodies, targeted protein degraders, and small molecule inhibitors.

On November 14, 2024 ArriVent BioPharma, Inc. (Company or ArriVent) (Nasdaq: AVBP), a clinical-stage company dedicated to accelerating the global development of innovative biopharmaceutical therapeutics, reported financial results for the third quarter ended September 30, 2024, and highlighted recent Company progress (Press release, ArriVent Biopharma, NOV 14, 2024, View Source [SID1234648388]).

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"Our lead clinical program of firmonertinib in non-small cell lung cancer (NSCLC) is strongly advancing with encouraging potential to expand across EGFR mutant types. In September, we announced compelling monotherapy data for firmonertinib from our FURTHER study supporting rapid and robust anti-tumor activity across EGFR PACC mutations building on the strong activity observed in patients with EGFR exon 20 insertion mutations," said Bing Yao, Chairman and Chief Executive Officer of ArriVent. "Importantly, the high activity in tough to treat patients with central nervous system (CNS) metastases or compound mutations points to firmonertinib as a promising candidate for front-line patients with PACC mutations. Collectively, the broad activity in EGFR PACC mutations, the high responses in the CNS, and consistent, manageable safety profile across trials reinforces the promise firmonertinib holds to address unmet needs across the spectrum of EGFR mutant NSCLC."

Dr. Yao continued, "We expect several near-term catalysts for our firmonertinib program including initiating dose expansion for the combination study in classical EGFR mutant NSCLC with SHP2 inhibitor ICP-189 in the fourth quarter of the year, an update on the Phase 1b PACC study and plans for our potential registration study in the first half of 2025, and topline pivotal data from our global Phase 3 FURVENT study in front-line EGFR exon 20 mutant NSCLC in 2025. With our strong balance sheet and operating runway into 2026, we are well-positioned to execute across our near-term catalysts."

Third Quarter 2024 and Recent Highlights

Firmonertinib

● Positive proof-of-concept data in EGFR PACC mutant NSCLC. In September, ArriVent presented interim FURTHER Phase 1b clinical data for first-line firmonertinib monotherapy in patients with EGFR PACC mutant NSCLC during the Presidential Symposium at the 2024 annual World Conference on Lung Cancer and hosted a virtual webinar. In what we believe to be the first clinical dataset testing an EGFR inhibitor in a randomized defined population of EGFR PACC

mutant NSCLC, firmonertinib demonstrated robust systemic and CNS anti-tumor activity with a manageable safety profile consistent with previous trials.
Upcoming Milestones

● Dose expansion for SHP2 inhibitor combination. Initiation of dose expansion cohort for the ongoing Phase 1b trial evaluating firmonertinib in combination with SHP2 inhibitor ICP-189 by InnoCare in patients with classical EGFR mutations expected in the fourth quarter of 2024.
● EGFR PACC pivotal study plan. Data from the FURTHER Phase 1b (NCT05364043) trial continues to mature for first-line firmonertinib monotherapy in patients with EGFR PACC mutant NSCLC. ArriVent expects to provide an update on EGFR PACC plans in the first half of 2025.
● Selection of next-generation antibody drug conjugate (ADC) candidate. ArriVent and its partner, Aarvik Therapeutics, Inc., continue to make progress, including initiating CMC activities, on selecting a multi-target multivalent ADC candidate for the treatment of solid tumors in the ARR-002 program, and expect to complete selection of a candidate that will be advanced into IND enabling studies in late 2024 or early 2025.
● Top-line pivotal Phase 3 data in 2025. Firmonertinib is currently being studied as a monotherapy in the pivotal, global Phase 3 FURVENT trial (NCT05607550) evaluating firmonertinib in previously untreated NSCLC patients whose tumors contain EGFR exon 20 insertion mutations with topline data expected in 2025.
Third Quarter 2024 Financial Results

● As of September 30, 2024, the Company had cash and cash equivalents of $282.9 million, which is expected to fund operations into 2026. Net cash used in operations was $54.1 million and $40.9 million for the nine months ended September 30, 2024 and 2023, respectively.

● Research and development expenses were $58.9 million and $44.9 million for the nine months ended September 30, 2024 and 2023, respectively. The increase in expense was primarily due to increased headcount and clinical expense related to firmonertinib.

● General and administrative expenses were $11.8 million and $6.6 million for the nine months ended September 30, 2024 and 2023, respectively. The increase in expense was primarily due to expenses related to expanding the infrastructure necessary for operating as a public company.

● Net loss was $59.9 million and $48.1 million for the nine months ended September 30, 2024 and 2023, respectively.

On November 14, 2024 Alector, Inc. (Nasdaq: ALEC), a clinical-stage biotechnology company pioneering immuno-neurology, reported that the Company has entered into a debt financing agreement with Hercules Capital, Inc. (NYSE: HTGC) for up to $50 million (Press release, Alector, NOV 14, 2024, View Source [SID1234648387]).

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"Alector is in a strong cash position with more than $457 million in cash and investments. This credit facility further enhances our financial strength and provides the Company with increased strategic and operational flexibility," said Marc Grasso, M.D., Chief Financial Officer of Alector. "We anticipate transformational data from both the AL002 INVOKE-2 Phase 2 trial and the latozinemab INFRONT-3 pivotal Phase 3 trial within our runway. This credit facility provides additional funding to advance our preclinical pipeline including our proprietary, versatile Alector Brain Carrier blood-brain barrier platform and programs."

Under the terms of the agreement, Alector drew an initial $10 million at closing. An additional $15 million is available at the Company’s request through June 30, 2026, with an additional $25 million available upon lender approval. The Company is under no obligation to draw funds in the future. The credit facility carries a low double-digit cost of capital.

"Hercules is pleased to enter into a strategic relationship with Alector as it advances its portfolio of assets aimed at treating neurodegenerative diseases," said Lake McGuire, Managing Director at Hercules Capital. "This capital commitment from Hercules seeks to help Alector deliver new therapeutic options to patients and further advance their novel and proprietary blood-brain barrier technology."

Alector’s cash, cash equivalents and investments were $457.2 million as of September 30, 2024. Excluding this $50 million credit facility, the Company believes that its current cash, cash equivalents and investments will be sufficient to fund its operations through 2026.