On May 10, 2024 Instil Bio, Inc. ("Instil") (Nasdaq: TIL), a clinical-stage biopharmaceutical company focused on developing a pipeline of novel therapies, reported its first quarter 2024 financial results and provided a corporate update (Press release, Instil Bio, MAY 10, 2024, View Source [SID1234643083]).

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Recent Highlights:

With successful completion of feasibility studies, preparations are underway with collaborator for a potential investigator-initiated trial (IIT) in non-small cell lung cancer with folate receptor α (FRα)-CoStAR TIL
Exploring opportunities to in-license/acquire and develop novel therapeutic candidates in diseases with significant unmet medical need
Cash runway expected beyond 2026
First Quarter 2024 Financial and Operating Results:

As of March 31, 2024, Instil had cash, cash equivalents, marketable securities and long-term investments of $161.5 million, which consisted of $5.5 million in cash and cash equivalents, $148.3 million in marketable securities and $7.7 million in long-term investments, compared to $175.0 million in total cash, cash equivalents, restricted cash, marketable securities and long-term investments, which consisted of $9.2 million in cash and cash equivalents, $1.5 million in restricted cash, $141.2 million marketable securities and $23.2 million in long-term investments as of December 31, 2023. Instil expects that its cash, cash equivalents, marketable securities and long-term investments as of March 31, 2024 will enable it to fund its current operating plan beyond 2026.

Research and development expenses were $7.3 million for the three months ended March 31, 2024, compared to $20.7 million for the three months ended March 31, 2023.

General and administrative expenses were $12.4 million for the three months ended March 31, 2024, compared to $13.2 million for the three months ended March 31, 2023.

Restructuring and impairment charges were $4.3 million for the three months ended March 31, 2024, compared to $24.6 million for the three months ended March 31, 2023.

Net loss per share, basic and diluted were $3.74 for the three months ended March 31, 2024, compared to $8.77 for the three months ended March 31, 2023. Non-GAAP net loss per share, basic and diluted were $2.39 for the three months ended March 31, 2024, compared to $4.29 for the three months ended March 31, 2023.

Note Regarding Use of Non-GAAP Financial Measures

In this press release, Instil has presented certain financial information that has not been prepared in accordance with U.S. generally accepted accounting principles ("GAAP"). These non-GAAP financial measures include non-GAAP net loss and non-GAAP net loss per share, which are defined as net loss and net loss per share, respectively, excluding non-cash stock-based compensation expense and restructuring and impairment charges. Instil believes that these non-GAAP financial measures, when considered together with the GAAP figures, can enhance an overall understanding of Instil’s financial performance. The non-GAAP financial measures are included with the intent of providing investors with a more complete understanding of Instil’s operating results. In addition, these non-GAAP financial measures are among the indicators Instil’s management uses for planning purposes and to measure Instil’s performance. These non-GAAP financial measures should be considered in addition to, and not as a substitute for, or superior to, financial measures calculated in accordance with GAAP. The non-GAAP financial measures used by Instil may be calculated differently from, and therefore may not be comparable to, non-GAAP financial measures used by other companies. Please refer to the below reconciliation of these non-GAAP financial measures to the comparable GAAP financial measures.

On May 10, 2024 Novartis reported that the FDA has granted Breakthrough Therapy designation to Scemblix (asciminib) for the treatment of adult patients with newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP) (Press release, Novartis, MAY 10, 2024, View Source [SID1234643084]). This marks the third1 Breakthrough Therapy designation for Scemblix.

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According to FDA guidelines, treatments that receive Breakthrough Therapy designation must target a serious or life-threatening disease and demonstrate a potential substantial improvement over existing therapies on one or more clinically significant endpoints.
Breakthrough Therapy designation was granted based on positive data from the Phase III ASC4FIRST study, in which Scemblix met both primary endpoints with superior MMR rates at week 48 compared to investigator-selected TKIs (imatinib, nilotinib, dasatinib and bosutinib) and compared to imatinib alone. Scemblix also demonstrated a favorable safety and tolerability profile with fewer adverse events (AEs) and treatment discontinuations vs. standard-of-care TKIs.
With current standard-of-care TKIs, approximately half2 of newly diagnosed patients with CML fail to meet molecular response goals at one year, and many discontinue or change treatment due to intolerance.
Full results of the ASC4FIRST study will be presented at ASCO (Free ASCO Whitepaper) on Friday, May 31. Novartis is also hosting an in-person investor event in Chicago on Sunday, June 2, to delve deeper into the results and the potential commercial opportunity for Scemblix in 1L CML upon regulatory approval.

Overall, Novartis has received 30 approvals for Breakthrough Therapy designated drugs, reflecting our track record of innovation.

On May 10, 2024 Soligenix, Inc. (Nasdaq: SNGX) (Soligenix or the Company), a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases where there is an unmet medical need, reported its recent accomplishments and financial results for the quarter ended March 31, 2024 (Press release, Soligenix, MAY 10, 2024, View Source [SID1234643085]).

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"Recently we completed a public offering with gross proceeds of approximately $4.75 million, which will allow us to continue to move our rare disease pipeline forward," stated Christopher J. Schaber, PhD, President and Chief Executive Officer of Soligenix. "While we continue our ongoing discussions with the United States (U.S.) Food and Drug Administration (FDA), we shared that the European Medicines Agency (EMA) has agreed to the study design of a confirmatory Phase 3 placebo-controlled study evaluating the safety and efficacy of HyBryte (synthetic hypericin) in the treatment of cutaneous T-cell lymphoma (CTCL) patients with early-stage disease. This study will enroll approximately 80 patients across the U.S. and Europe, starting before the end of 2024, with top-line results expected in the second half of 2026. We also will be initiating a Phase 2 study with SGX945 (dusquetide) in Behçet’s disease later this year with top-line results expected in the first half of 2025, along with top-line results expected during the same timeframe from our ongoing SGX302 (synthetic hypericin) Phase 2 study in mild-to-moderate psoriasis. Additionally, we were recently granted orphan drug designations from the FDA’s Office of Orphan Products Development for the active ingredients in both SuVax, for "the prevention and post-exposure prophylaxis against Sudan ebolavirus (SUDV) infection and MarVax, for "the prevention and post-exposure prophylaxis against Marburg marburgvirus (MARV) infection."

Dr. Schaber continued, "With approximately $7.1 million in cash at March 31, 2024, exclusive of the approximate $4.3 million in net proceeds from our recent financing and our non-dilutive government funding, we continue to manage cash burn very carefully to achieve our near-term milestones. We have a clear vision for the future, and we are actively pursuing new opportunities to create long-term value for our shareholders including but not limited to, partnership and merger and acquisition opportunities."

Soligenix Recent Accomplishments

On April 25, 2024, the Company announced it had received notice of intent to grant additional patents based on its patent application titled "Compositions and Methods of Manufacturing Trivalent Filovirus Vaccines" in the United Kingdom and South Africa, with other international jurisdictions pending. To view this press release, please click here.
On April 18, 2024, the Company announced the pricing of its public offering of 11,875,000 shares of common stock (or common stock equivalents in lieu thereof) and warrants to purchase up to 11,875,000 shares of common stock at a combined public offering price of $0.40 per share and accompanying warrants for aggregate gross proceeds of approximately $4.75 million, before deducting placement agent fees and other offering expenses. To view this press release, please click here.
On April 15, 2024, the Company announced the Office of Orphan Products Development of the U.S. FDA had granted orphan drug designation to the active ingredient in SuVax, the subunit protein vaccine of recombinantly expressed SUDV glycoprotein, for "the prevention and post-exposure prophylaxis against SUDV infection." To view this press release, please click here.
On April 11, 2024, the Company announced the Office of Orphan Products Development of the U.S. FDA had granted orphan drug designation to the active ingredient in MarVax, the subunit protein vaccine of recombinantly expressed MARV glycoprotein, for "the prevention and post-exposure prophylaxis against MARV infection." To view this press release, please click here.
On April 3, 2024, the Company announced it had received agreement from the EMA on the key design components of a confirmatory Phase 3 placebo-controlled study evaluating the safety and efficacy of HyBryte (synthetic hypericin) in the treatment of CTCL patients with early-stage disease. To view this press release, please click here.
Financial Results – Quarter Ended March 31, 2024

Soligenix’s revenues for the quarter ended March 31, 2024 were $0.1 million as compared to $0.3 million for the quarter ended March 31, 2023. Revenues primarily relate to government contracts and grants awarded in support of SGX943 for treatment of emerging and/or antibiotic-resistant infectious diseases; development of CiVax, our vaccine candidate for the prevention of COVID-19, and evaluation of HyBryte for expanded treatment in patients with early-stage CTCL.

Soligenix’s net loss was $1.9 million, or ($0.18) per share, for the quarter ended March 31, 2024, as compared to $1.0 million, or ($0.36) per share, for the quarter ended March 31, 2023. The increase in net loss was primarily due to the recognition of an income tax benefit during the three months ended March 31, 2023 with no corresponding income tax benefit recognized during the three months ended March 31, 2024.

Research and development expenses were $1.1 million as compared to $0.9 million for the quarters ended March 31, 2024 and 2023, respectively. The increase was primarily due to an increase in preliminary costs associated with the anticipated initiation of our Phase 2 study in Behçet’s Disease and the second confirmatory Phase 3 CTCL trial.

General and administrative expenses were $1.0 million and $1.2 million for the quarters ended March 31, 2024 and 2023, respectively. This decrease in general and administrative expenses is primarily attributable to a reduction in legal and professional fees associated with the reverse stock split of our issued and outstanding shares of common stock during the three months ended March 31, 2023.

As of March 31, 2024, the Company’s cash position, exclusive of the approximate $4.3 million in net proceeds from our recent financing, was approximately $7.1 million.

On May 10, 2024 Xenetic Biosciences, Inc. (NASDAQ:XBIO) ("Xenetic" or the "Company"), a biopharmaceutical company focused on advancing innovative immune-oncology technologies addressing hard to treat cancers, reported its financial results for the first quarter of 2024 and provided a business update (Press release, Xenetic Biosciences, MAY 10, 2024, View Source [SID1234643087]).

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"We have initiated several preclinical studies on the influence of DNase in combination with standard treatment paradigms such as chemotherapy or immune checkpoint blockade and expect to present results in the second half of this year. We anticipate that this growing body of data will have an important impact in demonstrating the promise of incorporating DNase into clinical treatment regimens for certain solid tumors, and in guiding our clinical development strategy," commented, Jeffrey Eisenberg, Chief Executive Officer of Xenetic. "We believe we have successfully positioned 2024 to be a year of meaningful preclinical data and remain focused on executing our plans to advance development towards our first in human trial."

Summary of Financial Results for First Quarter 2024

Net loss for the quarter ended March 31, 2024 was approximately $1.2 million. Research & development expenses for the three months ended March 31, 2024 increased by approximately $0.3 million, or 58.6%, to approximately $0.9 million from approximately $0.6 million in the comparable quarter in 2023. The increase was primarily due to the Company’s increased spending in connection with our pre-clinical development efforts related to our DNase platform. General and administrative expenses for the three months ended March 31, 2024 decreased by approximately $0.1 million, or 9.8%, to approximately $0.8 million from approximately $0.9 million in the comparable quarter in 2023. The decrease was primarily due to a decrease in accounting and legal fees during the three months ended March 31, 2024 compared to the same period in 2023.

The Company ended the quarter with approximately $7.8 million of cash.

On May 10, 2024 Cure Genetics reported the safety and efficacy data of their CAR-NKT product, CGC729, for patients with relapsed and refractory metastatic renal cell carcinoma (R/R mRCC) in an oral presentation at the 27th Annual Meeting of the American Society for Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) in Baltimore (Press release, CureGenetics, MAY 10, 2024, View Source [SID1234643645]). This marked the first-in-human trial using CAR-NKT therapy for R/R mRCC, demonstrating a good safety profile and encouraging anti-tumor activity.

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This phase I dose-escalation clinical trial was conducted at Fudan University Shanghai Cancer Center using a single-arm, 3+3 design to evaluate the safety and efficacy of CGC729 at three dose levels (DL1: 5 x 106/m2; DL2: 1.5 x 107/m2; DL3: 4.5 x 107/m2) in the treatment of R/R mRCC patients who had at least two lines of prior therapy. As of April 2024, five patients were enrolled and received a single infusion of CGC729. All patients underwent lymphodepletion prior to CGC729 infusion.

Safety Evaluation: As of April 11, 2024, all five patients (three in DL1 and two in DL2) experienced no dose-limiting toxicities (DLTs). The most common adverse events were lymphodepletion-related neutropenia, thrombocytopenia, and leukopenia. One patient in DL1 (0102) developed grade 2 cytokine release syndrome (CRS), which resolved within 24 hours; the same patient also developed grade 2 immune effector cell-associated neurotoxicity syndrome (ICANS), which improved rapidly after symptomatic treatment.

Efficacy Evaluation: Among four eligible patients, the overall response rate (ORR) reached 50% (2/4). Among CD70 positive patients (n=3),ORR was 66.7% (2/3), with two patients achieving partial response (PR). CGC729 demonstrated promising efficacy with deep anti-tumor activity. The longest duration of response (DOR) is continuing at nine-month after treatment. Furthermore, pharmacokinetic measurements showed robust expansion of CGC729 in all subjects, regardless of the CD70 expression level in the tumor, with prolonged persistence in the blood up to 20 weeks.

Conclusion: The interim analysis of this first-in-human trial shows that anti-CD70 CAR-NKT (CGC729) has a good safety profile and robust anti-tumor activity. It may provide a promising treatment option for R/R mRCC in the future.

About the ASGCT (Free ASGCT Whitepaper) Presentation

Title：Interim Safety and Efficacy Evaluation of Anti-CD70 CAR-NKT (CGC729) Phase I Study for Patients with Relapsed and Refractory Metastatic Renal Cell Carcinoma (R/R mRCC)
No：Late-breaking Abstract No. 10
Format：Oral presentation
Room：Ballroom 4
Time：2024/5/10 8:30 am – 8:45 am
Presenter：Dr. Yuanyuan Qu, Fudan Univeristy Shanghai Cancer Center

About AIMS CAR-NKT

The challenge of treating solid tumors is compounded by tumor heterogeneity and a suppressive tumor microenvironment (TME), which are major barriers to successful CAR-T cell therapy. Addressing this, Natural Killer T (NKT) cells, a subset of multifactorial innate-like T cells, offer new possibilities in solid tumor therapy due to their multifactorial anti-tumor capabilities and TME modulation properties.

Cure Genetics’ innovative AIMS CAR-NKT platform is based on these NKT cell features, aimed at enhancing solid tumor infiltration, modulating the TME, and prolonging sustained expansion. This platform has demonstrated good safety and promising efficacy in our lead product, CGC729, and is concurrently being expanded to other autologous/allogeneic products.

About CGC729

CGC729 is an autologous anti-CD70 CAR-NKT with a differentiated mechanism of action from T cells and NK cells. First, CGC729 combines adaptive and innate immunity with multiple tumor-killing mechanisms targeting CD70, CD1d, and stress ligands frequently expressed in RCC. Additionally, CGC729 has the capability to modulate the TME through skewing macrophage differentiation and resisting TGF-β inhibition.