On August 14, 2024 bluebird bio, Inc. (NASDAQ: BLUE) ("bluebird bio" or the "Company") reported second quarter results and business highlights for the quarter ended June 30, 2024, including recent commercial and operational progress (Press release, bluebird bio, AUG 14, 2024, View Source [SID1234645891]).

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"We are seeing clear evidence that our commercial launch is accelerating, with over 20 cell collections completed in sickle cell disease and beta-thalassemia to date in 2024, and more than 40 additional patients already scheduled to initiate the treatment journey for a bluebird gene therapy by the end of this year," said Andrew Obenshain, chief executive officer, bluebird bio. "We are further encouraged by the commitment to provide patient access across both commercial and government payers, most recently conveyed through multiple positive Medicaid decisions and the growing number of published coverage policies for LYFGENIA, and we expect approximately 85 patient starts across our portfolio this year."

COMMERCIAL LAUNCH UPDATES

LYFGENIA launch building (lovotibeglogene autotemcel); continued commercial momentum for ZYNTEGLO (betibeglogene autotemcel) and SKYSONA (elivaldogene autotemcel)

27 patient starts completed across portfolio to date in 2024 (19 ZYNTEGLO, 4 LYFGENIA, 4 SKYSONA).
Launch momentum building with more than 40 additional patients scheduled for cell collection across commercial portfolio through year end.
Successfully completed manufacturing and release testing for first commercial LYFGENIA patient and the first infusion is being scheduled.
Rapid acceleration projected for ZYNTEGLO following the recent approval of additional manufacturing capacity.
Validated access and reimbursement strategy is driving favorable coverage landscape

Significant progress with formalized paths to market access. To date, more than half of Medicaid-insured individuals with sickle cell disease in the U.S. live in a state that has affirmed coverage for LYFGENIA through a preferred drug list or published coverage criteria.
Nearly 20% 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.
Timely access to ZYNTEGLO and SKYSONA has continued, with zero ultimate denials to date for either therapy across both Medicaid and commercial payers.
Substantial QTC footprint established

bluebird has activated more than 70 total QTCs for LYFGENIA and ZYNTEGLO (defined as a signed MSA).
Six centers are activated to administer SKYSONA for patients with cerebral adrenoleukodystrophy (CALD).
RECENT COMPANY UPDATES

James Sterling appointed as Chief Financial Officer

Effective June 10, 2024, James Sterling became Chief Financial Officer of bluebird bio. Mr. Sterling most recently served as chief financial officer of Renalytix plc, a diagnostics company focused on clinical management of kidney disease. Mr. Sterling was previously managing partner at Renwick Capital LLC, and managing director at investment banks Brock Capital Group LLC and Aleutian Capital Group. He also serves as a board director for a fund managed by Star Mountain Capital. Mr. Sterling has experience as a management consultant at Booz Allen Hamilton. He received his B.A. from Boston University and an M.B.A. from Columbia Business School.
Michael Cloonan appointed to bluebird bio’s Board of Directors

On June 20, 2024, Michael Cloonan was appointed to bluebird bio’s Board of Directors. Mr. Cloonan is currently President and Chief Executive Officer of Sionna Therapeutics. He was previously Chief Operating Officer at Sage Therapeutics leading all business and G&A functions. During his four years with Sage, he helped lead the growth of the organization through multiple capital raises, the launch of the company’s first product, and execution of a transformational, multi-billion-dollar collaboration. An experienced and respected global biotech leader, Mr. Cloonan has over 20 years of biopharma experience across global organizations in various business and commercial roles.
2024 GUIDANCE

The Company anticipates approximately 85 patient starts (cell collections) combined across its portfolio of three FDA approved therapies (LYFGENIA, ZYNTEGLO, SKYSONA) in 2024. Consistent with previous quarters, bluebird plans to provide quarterly updates on patient starts by indication for each of its therapies.
The Company continues to anticipate gross-to-net discounts across all three products to be in the range of 20% to 25% of gross revenue in 2024 and expects they 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 or fourth quarter of 2024.
SECOND QUARTER FINANCIAL HIGHLIGHTS

Cash Position: The Company’s cash, cash equivalents and restricted cash balance was approximately $193 million, including restricted cash of approximately $49 million, as of June 30, 2024.

Based on current operating assumptions, bluebird expects its cash and cash equivalents as of June 30, 2024 will be sufficient to fund its operations into the second quarter of 2025i, not accounting for the cash minimums required under the Company’s loan agreement with Hercules Capital, Inc. and excluding receipt of any future tranches under the agreement.

The Company has also renegotiated certain terms of its loan agreement with Hercules Capital, Inc. and is eligible to receive two future tranches totaling $50 million, contingent upon achievement of patient start and product delivery milestones and completion of additional financing. These tranches take the place of previously agreed tranches tied to patient starts and gross profit.
Revenue, net: Total revenue, net was $16.1 million for the three months ended June 30, 2024, compared to $6.9 million for the three months ended June 30, 2023. The increase of $9.2 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 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 Reports on Form 10-Q for the quarters ended March 31, 2024 (the "Q1 2024 Form 10-Q") and June 30, 2024 (the "Q2 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 Q2 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 June 30, 2024.

CONFERENCE CALL DETAILS

bluebird will hold a conference call to discuss its second quarter 2024 results and business updates today, Wednesday, August 14, 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 2776050.

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 August 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 second quarter ended June 30, 2024, and highlighted recent Company progress (Press release, ArriVent Biopharma, AUG 14, 2024, View Source [SID1234645887]).

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"We made strong progress this quarter further advancing and expanding our global oncology pipeline. At the annual WCLC we plan to present interim first-line firmonertinib monotherapy data from our FURTHER study in patients with NSCLC harboring EGFR PACC mutations, which to our knowledge, will be the first data from a prospectively designed clinical trial of an EGFR tyrosine kinase inhibitor in this patient population," said Bing Yao, Chairman and Chief Executive Officer of ArriVent. "Earlier this quarter we also expanded our ADC portfolio with a multi-target ADC collaboration agreement with Alphamab, combining their discovery capabilities and know-how with our global drug development and commercialization expertise. Collectively, these programs are designed to deliver new targeted therapies with the potential to change the treatment paradigm for millions of patients with tough to treat cancers and high unmet needs."

Second Quarter 2024 and Recent Highlights

Firmonertinib

● Presentation of preclinical data for firmonertinib at the 2024 AACR (Free AACR Whitepaper) Annual Meeting. In April, ArriVent presented preclinical data evaluating firmonertinib in NSCLC with EGFR exon 20 insertion mutations and PACC mutations at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting. In the preclinical study, firmonertinib, a highly brain penetrant mutant-selective EGFR inhibitor, was observed to be broadly active across a wide range of uncommon mutations including PACC and exon 20 insertion mutations.
Pipeline

● Entered into an ADC collaboration agreement with Alphamab. In June, ArriVent entered into a collaboration agreement with Jiangsu Alphamab Biopharmaceuticals Co., Ltd. (Alphamab), a
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wholly owned subsidiary of Alphamab Oncology, to discover, develop and commercialize novel ADCs for the treatment of cancers.

Upcoming Milestones

● Proof-of-concept data to be presented as late-breaker at 2024 WCLC. In August, ArriVent announced that interim FURTHER Phase 1b clinical data for first-line firmonertinib monotherapy in patients with NSCLC and EGFR PACC mutations will be presented as part of a presidential symposium at the 2024 WCLC on September 9, 2024. ArriVent plans to host a virtual webinar event on the Phase 1b firmonertinib EGFR PACC data on September 9, 2024 at 4:30pm ET in conjunction with 2024 WCLC. Please register for the event here: View Source

● Selection of next-generation ADC candidate. ArriVent and its partner, Aarvik Therapeutics, Inc., continue to make progress on selecting a multi-target multivalent ADC clinical candidate, and expect to complete selection 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.

Corporate Updates

● Strengthened board leadership. In May 2024, ArriVent appointed John Hohneker, M.D. to its Board of Directors. Dr. Hohneker brings over 30 years of experience in biopharmaceutical leadership and drug development, previously serving as President and CEO of Anokion SA and holding leadership roles at Forma Therapeutics and Novartis AG. He currently serves on the boards of public companies Carisma Therapeutics and Curis, and private companies Sonata Therapeutics and Trishula Therapeutics. Dr. Hohneker serves on ArriVent’s Nominating and Corporate Governance Committee and Compensation Committee. In April 2024, ArriVent appointed Kristine Peterson to its Board of Directors. Ms. Peterson has served on the boards of multiple public biopharmaceutical companies, including Immunocore and ImmunoGen (recently acquired by Abbvie), and brings over 30 years of industry leadership experience having previously served as CEO of Valeritas and Company Group Chair of Johnson and Johnson for their worldwide biotech and oncology groups. Ms. Peterson serves on ArriVent’s Compensation Committee.

Second Quarter 2024 Financial Results

● As of June 30, 2024, the Company had cash and cash equivalents of $298.7 million, which is expected to fund operations into 2026. Net cash used in operations was $37.7 million and $25.5 million for the six months ended June 30, 2024 and 2023, respectively.
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● Research and development expenses were $38.8 million and $30.6 million for the six months ended June 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 $7.6 million and $4.2 million for the six months ended June 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 $39.3 million and $33.7 million for the six months ended June 30, 2024 and 2023, respectively.

On August 14, 2024 AnaptysBio, Inc. (Nasdaq: ANAB), a clinical-stage biotechnology company focused on delivering innovative immunology therapeutics, reported the pricing of an underwritten offering of 2,750,498 shares of its common stock at a price of $36.50 per share, representing a premium of approximately 10% to Anaptys’ closing price on Aug. 13, 2024 (Press release, AnaptysBio, AUG 14, 2024, View Source [SID1234645886]). The gross proceeds from this offering are expected to be approximately $100 million, before deducting underwriting discounts, commissions and other offering expenses payable by Anaptys. All of the shares of common stock are being offered by Anaptys. The offering is expected to close on or about Aug. 15, 2024, subject to the satisfaction of customary closing conditions.

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The offering was led by current investor, EcoR1 Capital, and included participation from both new and existing investors, including Cormorant Asset Management, Farallon Capital Management, First Light Asset Management, Woodline Partners LP, multiple large investment management firms and Sanofi.

"We are excited to announce this focused equity offering, with proceeds intended to be used primarily to accelerate and support the enablement of Phase 3 trials for ANB032, our BTLA agonist, in Phase 2b development in atopic dermatitis, as well as rosnilimab, our PD-1 agonist, in Phase 2b development in rheumatoid arthritis and Phase 2 development in ulcerative colitis," said Daniel Faga, president and chief executive officer of Anaptys. "We are pleased with the quality of our existing and new long-term investors, who share our enthusiasm for the potential of checkpoint agonists to bring the immune system back to homeostasis and durably modify autoimmune and inflammatory diseases."

Anaptys intends to use the net proceeds of this offering primarily for general corporate purposes, which may include funding Phase 3 enabling activities for ANB032 and rosnilimab, working capital and general corporate purposes. Sanofi did not receive rights to any of Anaptys’ programs as a part of their equity investment.

TD Cowen, Leerink Partners, Piper Sandler and Guggenheim Securities are acting as joint book-running managers for the offering.

The shares are being offered by Anaptys pursuant to a registration statement previously filed and declared effective by the U.S. Securities and Exchange Commission ("SEC"). A prospectus supplement and accompanying prospectus relating to and describing the terms of the offering will be filed with the SEC and will be available on the SEC’s website at www.sec.gov. Copies of the prospectus supplement and accompanying prospectus may also be obtained, when available, from: TD Securities (USA) LLC, 1 Vanderbilt Ave., New York, NY 10017, by telephone at (855) 495-9846, or by email at [email protected] or from Leerink Partners LLC, Attention: Syndicate Department, 53 State Street, 40th Floor, Boston, MA 02109, by telephone at (800) 808-7525, ext. 6105, or by email at [email protected].

This press release shall not constitute an offer to sell or the solicitation of an offer to buy any securities of Anaptys, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation, or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

On August 14, 2024 Aileron Therapeutics, Inc. ("Aileron" or the "Company") (NASDAQ: ALRN), a biopharmaceutical company advancing a pipeline of potential first-in-class medicines to address significant unmet medical needs in orphan pulmonary and fibrosis indications, reported financial results for the second quarter ended June 30, 2024, and provided a business update (Press release, Aileron Therapeutics, AUG 14, 2024, View Source [SID1234645885]).

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"Throughout the first half of the year, we focused on strengthening our balance sheet and advancing the development of inhaled LTI-03 in IPF," said Brian Windsor, Ph.D., President and Chief Executive Officer of Aileron. "We are extremely pleased with the positive data from Cohort 1 of the ongoing Phase 1b clinical trial announced in May, particularly the achievement of statistical significance in three out of eight biomarkers which is a testament to the potential of LTI-03 to inhibit fibrosis and improve lung function. Additionally, in May, we raised approximately $18.2 million in net proceeds in an underwritten registered direct offering, which provides us with the resources to further validate LTI-03 in the ongoing Phase 1b trial. We look forward to reporting topline results from the high-dose cohort in the third quarter of this year."

Second Quarter 2024 Highlights and Recent Updates

Corporate Updates

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In May 2024, the Company completed an underwritten registered direct offering of 4,273,505 shares of its common stock and accompanying warrants to purchase an aggregate of 4,273,505 shares of its common stock. Net proceeds from the offering were approximately $18.2 million, after deducting underwriting discounts and commissions and other offering expenses. The Company has the potential to receive approximately $20.0 million in additional proceeds from the exercise of the warrants issued in the offering.

Pipeline

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Announced positive Cohort 1 data from the ongoing Phase 1b clinical trial evaluating the safety and tolerability of inhaled LTI-03 in patients diagnosed with IPF.

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Following inhaled administration of low dose LTI-03 (2.5 mg BID, or twice daily) in twelve patients, a positive trend was observed in seven out of eight biomarkers. The findings included:

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Evidence of LTI-03 reducing expression of multiple profibrotic proteins produced in both basal-like cells and fibroblasts that contribute to the progression of IPF, with statistically significant decreases in three biomarkers, reinforcing the potential of LTI-03 to inhibit fibrosis, inflammation and associated changes in the lungs.

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LTI-03 stimulated production of solRAGE, a factor indicative of type I epithelial cell health, a critically important aspect of IPF that has gone largely unaddressed.

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LTI-03 was generally well-tolerated with no serious adverse events ("SAEs") reported.

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The Phase 1b trial is ongoing, with topline results from the high-dose Cohort 2 expected in the third quarter of 2024.

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On May 1, 2024, the Company hosted a pulmonary care expert call to discuss the Cohort 1 Phase 1b results of LTI-03, featuring pulmonary care expert Andreas Günther, M.D., Head of the Center for Interstitial and Rare Lung Diseases at the Justus Liebig University in Giessen, Germany. A replay of the event can be accessed at View Source
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LTI-01 is in development for loculated pleural effusion ("LPE"), a serious consequence of pneumonia with significant unmet medical need.

Second Quarter 2024 Financial Results
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Cash Position: Cash and cash equivalents as of June 30, 2024, were $21.9 million, compared to $12.0 million as of March 31, 2024. After including the net proceeds raised from the May 2024 offering and based on its current operating plan, the Company expects its existing cash and cash equivalents to be sufficient to fund the completion of the Phase 1b clinical trial and its operations into the second half of 2025.

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Research and Development ("R&D") Expenses: R&D expenses for the quarter ended June 30, 2024, were $3.7 million, compared to $0.2 million for the quarter ended June 30, 2023. The increase of $3.5 million was primarily a result of the clinical programs acquired as part of the Company’s acquisition of Lung Therapeutics, Inc. in October 2023 (the "Lung Acquisition"). During the quarter ended June 30, 2024, Aileron incurred expenses of $1.1 million on clinical trials, $2.0 million on manufacturing, and $0.1 million on regulatory and development consulting as well as $0.5 million on employee and related expenses associated with clinical programs acquired in the Lung Acquisition.

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General and Administrative ("G&A") Expenses: G&A expenses for the quarter ended June 30, 2024, were $5.3 million, compared to $1.9 million for the quarter ended June 30, 2023. The increase of $3.4 million was primarily due to increased professional fees of $1.0 million and increased employee and related expenses of $1.8 million as a result of increased business activity and headcount associated with the Lung Acquisition, and increased facilities and other expenses of $0.5 million during the quarter ended June 30, 2024 as compared to the quarter ended June 30, 2023.

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Net Loss: Net loss for the quarter ended June 30, 2024, was $8.9 million, compared to $1.8 million for the quarter ended June 30, 2023. The basic and diluted net loss per share for the quarter ended June 30, 2024 was $0.45 compared to $0.39 for the quarter ended June 30, 2023.

On August 14, 2024 Achilles Therapeutics plc (NASDAQ: ACHL), a clinical-stage biopharmaceutical company developing AI-powered precision T cell therapies targeting clonal neoantigens to treat solid tumors, reported its financial results for the second quarter ended June 30, 2024, and recent business highlights (Press release, Achilles Therapeutics, AUG 14, 2024, View Source [SID1234645884]).

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"During the second quarter we shared interim Phase I/IIa data from our ongoing CHIRON and THETIS TIL-based cNeT clinical trials and established an important research collaboration with Arcturus Therapeutics to explore the use of clonal neoantigens in second-generation personalized mRNA cancer vaccines", said Dr Iraj Ali, Chief Executive Officer of Achilles Therapeutics. "Our insights into the factors that drive durable engraftment and immune evasion led us to add an additional cohort in the CHIRON and THETIS trials to evaluate cNeT persistence and clinical activity in patients with enhanced host conditioning (EHC). These findings, along with the cancer vaccine research collaboration, continue to illustrate the potential value of our platform, including the unparalleled capability of PELEUS to select tumor targets with the highest potential for immune system recognition in a variety of modalities including TIL, neoantigen vaccines, ADCs, and TCR-T therapies.

Our financial position remains strong with more than $95 million in cash, which we expect to support operations through 2025, including the completion of the ongoing Phase I/IIa trials and the Arcturus collaboration."

Clinical and Business Updates
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Announced research collaboration with Arcturus Therapeutics to explore second-generation mRNA cancer vaccines

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Combines Achilles’ AI-powered, tumor-targeting technology with Arcturus’ self-amplifying mRNA platform
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Combined technologies have the potential to generate potent and durable T cell responses in pre-clinical IND-enabling studies 
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Provided an interim Phase I/IIa update on the use of clonal neoantigen reactive T cells (cNeT) in advanced NSCLC and melanoma
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Dosed first EHC patients in CHIRON and THETIS, with the first three EHC patients showing improved cNeT persistence and engraftment
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Delivered 10 products containing over 100 million cNeT and five containing over one billion cNeT
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Matilde Saggese, MD, has been appointed as Interim Chief Medical Officer. Dr. Saggese has served as Vice President and Medical Director of Achilles Therapeutics since March 2021.

Financial Highlights
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Cash and cash equivalents: Cash and cash equivalents were $95.1 million as of June 30, 2024, as compared to $131.5 million as of December 31, 2023. The Company believes that its cash and cash equivalents are sufficient to fund its planned operations through 2025.
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Research and development (R&D) expenses: R&D expenses were $13.6 million for the second quarter ended June 30, 2024, compared to $13.8 million for the second quarter ended June 30, 2023. The decrease was primarily driven by decreased activity in THETIS and lower personnel costs, partially offset by increased activity in CHIRON.
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General and administrative (G&A) expenses: G&A expenses were $4.2 million for the second quarter ended June 30, 2024, compared to $4.3 million for second quarter ended June 30, 2023. This decrease was primarily driven by lower personnel costs and lower insurance premiums.
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Net loss: Net loss for the second quarter ended June 30, 2024 was $16.4 million or $0.41 per share compared to $16.8 million or $0.42 per share for the second quarter ended June 30, 2023.

2024 Focus

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Clinical Data: Report clinical activity and translational science data from patients in CHIRON and THETIS, evaluating the benefit of EHC, with a meaningful data update expected in the second half of 2024
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Arcturus Collaboration: Ongoing evaluation of best-in-class, self-amplifying mRNA (sa-mRNA) personalized cancer vaccines (PCVs) targeting clonal neoantigens with the potential to generate IND-enabling data

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Translational Science: Leverage the Company’s unique bioinformatics platform to better understand the drivers associated with clinical responses
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Manufacturing Development: Continue VELOS and PELEUS development to optimize cNeT dose and functionality