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 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 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 Immix Biopharma, Inc. ("ImmixBio", "Company", "We" or "Us" or "IMMX"), a clinical-stage biopharmaceutical company trailblazing cell therapies in autoimmune disease, reported new clinical data from its Phase 1b/2a NEXICART-1 (NCT04720313) study of novel, autologous, sterically-optimized BCMA-targeted chimeric antigen receptor T (CAR-T) cell therapy, NXC-201, in patients with relapsed/refractory AL Amyloidosis (R/R ALA) in a late breaking oral presentation at the 27th Annual Meeting of The American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) in Baltimore, MD (Press release, Immix Biopharma, MAY 10, 2024, View Source [SID1234643082]). All patients were relapsed/refractory to standards-of-care Dara-CyBorD (daratumumab combined with cyclophosphamide, bortezomib, and dexamethasone).

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"Relapsed/refractory AL Amyloidosis remains an unmet medical need, with no approved options for treatment," said Polina Stepensky, M.D., Director of the Hadassah Medical Organization’s Department of Bone Marrow Transplantation and Immunotherapy for Adults and Children, and NEXICART-1 principal study investigator. "We continue to be encouraged by NXC-201’s response rates and durable effect in patients without significant pre-existing cardiac damage, exemplified by our 28-month longest responder with response ongoing."

"We are excited to present clinical data from the NEXICART-1 clinical study at ASGCT (Free ASGCT Whitepaper). This study has informed the design of NEXICART-2 clinical trial, expected to open in the U.S. mid-2024," said Ilya Rachman, M.D., Ph.D., Chief Executive Officer of Immix Biopharma. Gabriel Morris, Chief Financial Officer of Immix Biopharma, added: "We believe NXC-201’s tolerability profile, including lack of neurotoxicity, makes it uniquely suitable as a potential new treatment option for relapsed/refractory AL Amyloidosis patients."

At the NXC-201 ASGCT (Free ASGCT Whitepaper) 2024 late-breaking oral presentation, data were presented from 13 relapsed/refractory AL amyloidosis patients (including 3 new patients) in the ongoing Phase 1b/2a NEXICART-1 study. Patients were infused with CAR+T cells at doses of 150 x 106 (n=1), 450 x 106 (n=2), and 800 x 106 (n=10).

Patient characteristics:

85% (11/13) had cardiac involvement
38% (5/13) had New York Heart Association (NYHA) stage 3 or 4 heart failure
38% (5/13) had Mayo stage 3 AL amyloidosis disease
Relapsed/refractory to a median 4 lines of prior therapy (range: 3-10)
1 patient, patient 11, was treated and progressed on a BCMA-targeted bispecific antibody before NXC-201 treatment
Safety and efficacy data:

Overall response rate (ORR) of 92% (12/13) for relapsed/refractory AL Amyloidosis patients enrolled in NEXICART-1:
12 out of 12 patients not exposed to prior BCMA-targeted bispecific responded to NXC-201 (100% ORR), of which 9 out of 12 were complete responders (75% CRs)
1 patient with prior exposure to BCMA-targeted bispecific treatment did not respond
Best responder had a duration of response of 28.0 months as of May 10, 2024, with response ongoing
There were no immune effector cell-associated neurotoxicity syndrome (ICANS) events
Median cytokine release syndrome (CRS) duration was 2 days (range: 1-5):
No grade 4 CRS events
2 experienced no CRS; 3 experienced grade 1 CRS; 6 Experienced grade 2 CRS; 2 experienced grade 3 CRS
The NXC-201 27th Annual Meeting of The American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) late-breaking oral presentation can be accessed on the ImmixBio corporate website at this link: View Source

ASGCT 2024 Presentation Details:

Title
"Academic BCMA-CART cells (HBI0101), a promising approach for the treatment of LC Amyloidosis"
Type
Late Breaking Oral Presentation
Oral Presentation Date/Time
Friday, May 10, 2024, 8:45 am – 9:00 am Eastern Time
Session Title
Late-Breaking Abstracts II
Location
Baltimore Convention Center, Baltimore, MD
About NEXICART-1
NEXICART-1 (NCT04720313) is an ongoing Phase 1b/2a, open-label study evaluating the safety and efficacy of NXC-201 (formerly HBI0101), in adults with relapsed/refractory multiple myeloma and relapsed/refractory AL amyloidosis. The primary objective of the Phase 1b portion of the study is to characterize the safety and confirm the recommended Phase 2 dose (RP2D) of NXC-201. The Phase 1b portion has been successfully completed, with a recommended Phase 2 dose (RP2D) of 800 million CAR+T cells.

About NEXICART-2
NEXICART-2 (NCT06097832) is an open-label, single-arm, multi-site Phase 1b dose expansion clinical trial in relapsed/refractory AL Amyloidosis for CAR-T NXC-201 in the United States. Over a period of approximately 18 months from first patient dosing, NEXICART-2 is expected to enroll 40 patients with adequate cardiac function who have not been exposed to prior BCMA-targeted therapy. The objectives are the safety and efficacy of NXC-201. The expected primary endpoints are complete response rate and overall response rate according to consensus recommendations (Palladini et al. 2012).

About NXC-201
NXC-201 is a sterically-optimized BCMA-targeted chimeric antigen receptor T (CAR-T) cell therapy, which we believe has the potential to be the only "Single-Day CRS" CAR-T, targeting AL Amyloidosis and other autoimmune diseases. It is being studied in a comprehensive clinical development program for the treatment of patients with relapsed/refractory AL amyloidosis, and expanding into other autoimmune indications. These trials build on a robust NXC-201 clinical dataset initiated in February 2021. NXC-201 has been awarded Orphan Drug Designation (ODD) by the FDA in both AL Amyloidosis and multiple myeloma, and awarded EU ODD by the EMA in multiple myeloma and AL Amyloidosis.

On May 10, 2024 FORE Biotherapeutics reported that the Company will be presenting at the 2024 RBC Capital Markets Global Healthcare Conference (Press release, Fore Biotherapeutics, MAY 10, 2024, View Source [SID1234643080]). The presentation will take place on Wednesday, May 15, 2024 at 9:00-9:25 AM ET.

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William Hinshaw, Chief Executive Officer, and Jeffrey Sacher, Interim Chief Financial Officer, will host and participate in one-on-one meetings. Please contact your RBC Capital Markets salesperson or Argot Partners to schedule one-on-one meetings with the management team.