On August 14, 2024 Celcuity Inc. (Nasdaq: CELC), a clinical-stage biotechnology company pursuing development of targeted therapies for oncology, reported financial results for the second quarter ended June 30, 2024 and other recent business developments (Press release, Celcuity, AUG 14, 2024, View Source [SID1234645893]).

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"We made significant strides advancing the clinical development of gedatolisib this quarter. Overall enrollment in VIKTORIA-1 remains robust and on-track relative to our previous projections," said Brian Sullivan, CEO and co-founder of Celcuity. "We also initiated efforts to launch VIKTORIA-2, a Phase 3 study to evaluate gedatolisib as a first-line treatment option for patients with HR+, HER2- advanced breast cancer."

"In our VIKTORIA-1 study, while overall enrollment is on track, the proportion of patients who have PIK3CA wild-type tumors, versus those with PIK3CA mutations, has recently shifted lower than our original estimates. As a result, we expect to reach the enrollment target for the PIK3CA wild-type cohort in the fourth quarter, rather than the third quarter, as we originally forecasted. In light of this, we expect topline data for the PIK3CA WT cohort to shift to sometime between late Q4 2024 and Q1 2025."

Second Quarter 2024 Business Highlights and Other Recent Developments

● The VIKTORIA-1 Phase 3 trial expects to provide topline data for the PIK3CA wild-type cohort in late Q4 2024 or Q1 2025 and for the PIK3CA mutant cohort in the first half of 2025.

○ VIKTORIA-1 is evaluating gedatolisib in combination with fulvestrant with and without palbociclib in adults with HR+, HER2- advanced breast cancer who have received prior treatment with a CDK4/6 inhibitor.
○ Enrollment of the PIK3CA wild-type cohort is more than 80% complete and expected to reach the enrollment target during Q4 2024. The PIK3CA wild-type cohort represents approximately 60% of the total patients enrolled to date in VIKTORIA-1.

● In May, the Company announced its plan to initiate VIKTORIA-2, a Phase 3 study to evaluate the efficacy and safety of gedatolisib in combination with fulvestrant plus a CDK4/6 inhibitor, either ribociclib or palbociclib, in comparison to fulvestrant plus a CDK4/6 inhibitor as a first-line treatment for patients with HR+/HER2- advanced breast cancer.

○ A safety run-in study to evaluate the safety of gedatolisib combined with ribociclib and fulvestrant will precede initiation of the Phase 3 portion of the study.
○ The Phase 3 portion of the study is expected to enroll approximately 638 patients at up to 200 sites across North America, Europe, Latin America, and Asia.
○ First patient enrollment is expected in the second quarter of 2025.

● During the quarter, the Company secured a combined total of $129 million in gross proceeds from equity and debt financings, which extended the cash runway for current clinical development program activities through 2026.

● The Phase 1b/2 trial, evaluating gedatolisib in combination with darolutamide for the treatment of patients with metastatic castration resistant prostate cancer (mCRPC), remains on track to report preliminary data in the first half of 2025.

○ Enrollment is ongoing and the trial is expected to enroll up to 54 patients with mCRPC whose disease progressed after treatment with an androgen receptor signaling inhibitor.

● Three manuscripts reporting clinical and nonclinical results for gedatolisib were published recently.

○ In April, The Lancet Oncology published results from the dose expansion groups of its Phase 1b study evaluating gedatolisib in combination with palbociclib and endocrine therapy in HR+/HER2- advanced breast cancer. The published manuscript is available online and on the publications section of Celcuity’s website.
○ In June, npj Breast Cancer published results of nonclinical studies showing gedatolisib’s superior potency and efficacy versus single-node PI3K/AKT/mTOR inhibitors in breast cancer models. The article is available online and on the publications section of Celcuity’s website.
○ In August, Molecular Oncology published results of nonclinical studies in prostate cancer models showing gedatolisib’s superior potency and efficacy versus single-node PI3K/AKT/mTOR inhibitors. The article is available online and will soon be available on the publications section of Celcuity’s website.

Second Quarter 2024 Financial Results

Unless otherwise stated, all comparisons are for the second quarter ended June 30, 2024, compared to the second quarter ended June 30, 2023.

Total operating expenses were $24.3 million for the second quarter of 2024, compared to $15.1 million for the second quarter of 2023.

Research and development (R&D) expenses were $22.5 million for the second quarter of 2024, compared to $13.8 million for the prior-year period. Of the approximately $8.7 million increase in R&D expenses, $6.6 million primarily related to activities supporting the VIKTORIA-1 Phase 3 trial and the initiation of the CELC-G-201 Phase 1b/2 clinical trial, and $2.1 million was related to increased employee and consulting expenses.

General and administrative (G&A) expenses were $1.8 million for the second quarter of 2024, compared to $1.3 million for the prior-year period. Employee and consulting related expenses accounted for $0.3 million of the increase. Professional fees and other administrative expenses accounted for the remaining increase of approximately $0.2 million.

Net loss for the second quarter of 2024 was $23.7 million, or $0.62 loss per share, compared to a net loss of $14.6 million, or $0.66 loss per share, for the second quarter of 2023. Non-GAAP adjusted net loss for the second quarter of 2024 was $22.2 million, or $0.58 loss per share, compared to non-GAAP adjusted net loss of $11.1 million, or $0.51 loss per share, for the second quarter of 2023. Non-GAAP adjusted net loss excludes stock-based compensation expense, non-cash interest expense, and non-cash interest income. Because these items have no impact on Celcuity’s cash position, management believes non-GAAP adjusted net loss better enables Celcuity to focus on cash used in operations. For a reconciliation of financial measures calculated in accordance with generally accepted accounting principles in the United States (GAAP) to non-GAAP financial measures, please see the financial tables at the end of this press release.

Net cash used in operating activities for the second quarter of 2024 was $18.1 million, compared to $9.7 million for the second quarter of 2023.

At June 30, 2024, Celcuity reported cash, cash equivalents and short-term investments of $283.1 million.

Webcast and Conference Call Information

The Celcuity management team will host a webcast/conference call at 4:30 p.m. ET today to discuss the second quarter 2024 financial results and provide a corporate update. To participate in the teleconference, domestic callers should dial 1-800-717-1738 or 1-646-307-1865. A live webcast presentation can also be accessed using this weblink: View Source;tp_key=c55c86e8c3. A replay of the webcast will be available on the Celcuity website following the live event.

On August 14, 2024 C4 Therapeutics, Inc. (C4T) (Nasdaq: CCCC), a clinical-stage biopharmaceutical company dedicated to advancing targeted protein degradation science, reported the ESMO (Free ESMO Whitepaper) Congress decided to move C4T’s previously accepted preliminary monotherapy Phase 1 abstract for CFT1946, a novel BiDAC degrader in mutant BRAF V600 solid tumors, to an oral presentation (Press release, C4 Therapeutics, AUG 14, 2024, View Source [SID1234645892]). This oral presentation session at the ESMO (Free ESMO Whitepaper) Congress 2024 is scheduled for Friday, September 13, 2024, at 4:00 pm to 5:30 pm CEST. Additionally, C4T announced it will host an investor webcast on Friday, September 13, 2024.

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Updated Details for ESMO (Free ESMO Whitepaper) Congress 2024 Presentation
Title: Preliminary Results from a Phase 1 Study of CFT1946, a Novel BiDAC Degrader in Mutant BRAF V600 Solid Tumors
Presentation Date and Time: Friday, September 13, 2024, 4:00 – 5:30 pm CEST
Final Publication Number: 612O
Presenter: Maria Vieito, M.D., Msc (Barcelona, Spain)

C4T Webcast for Analysts and Investors
C4T will host an investor webcast on Friday, September 13, 2024 to discuss the CFT1946 monotherapy data from ongoing CFT1946 Phase 1 trial in BRAF V600 solid tumors. The time of the webcast and access information will be shared closer to the webcast event.

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.