Halozyme to Participate in the BofA Securities 2025 Healthcare Conference

On May 5, 2025 Halozyme Therapeutics, Inc. (NASDAQ: HALO) ("Halozyme") reported that Dr. Helen Torley, president and chief executive officer, will present and host investor meetings at the BofA Securities 2025 Healthcare Conference (Press release, Halozyme, MAY 5, 2025, View Source [SID1234652523]).

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The presentation is scheduled for Tuesday, May 13 at 4:20pm PT / 7:20pm ET.

A live audio webcast will be available on the Investor Relations section of the Company’s website. Replays of the audio webcasts will be available for 90 days following the conference.

CORCEPT THERAPEUTICS ANNOUNCES FIRST QUARTER FINANCIAL RESULTS AND PROVIDES CORPORATE UPDATE

On May 5, 2025 Corcept Therapeutics Incorporated (NASDAQ: CORT), a commercial-stage company engaged in the discovery and development of medications to treat serious endocrinologic, oncologic, metabolic and neurologic disorders by modulating the effects of the hormone cortisol, reported its results for the quarter ended March 31, 2025 (Press release, Corcept Therapeutics, MAY 5, 2025, https://ir.corcept.com/news-releases/news-release-details/corcept-therapeutics-announces-first-quarter-financial-results-2 [SID1234652506]).

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Financial Results

"In the first quarter, we had another record number of prescriptions from new and existing prescribers, broadly distributed throughout the country. Growing physician awareness of hypercortisolism has resulted in increased screening and treatment of patients with this devastating disease. Our specialty pharmacy vendor began the quarter unable to fulfill this surge in demand, which negatively affected our first quarter financial results. Pharmacy operations improved substantially in March and April, with each month setting a record for tablets dispensed. We are reiterating our 2025 revenue guidance of $900 – $950 million," said Joseph K. Belanoff, M.D., Corcept’s Chief Executive Officer.

Corcept’s first quarter 2025 revenue was $157.2 million, compared to $146.8 million in the first quarter of 2024. First quarter 2025 operating expenses were $153.8 million, compared to $117.3 million in the same period last year. Net income was $20.5 million in the first quarter of 2025, compared to $27.8 million in the first quarter of 2024.

Cash and investments were $570.8 million at March 31, 2025, compared to $603.2 million at December 31, 2024. The balance at March 31, 2025 reflects the acquisition of $43.3 million of common stock in the first quarter pursuant to the company’s stock repurchase program, net exercise of employee stock options and net vesting of restricted stock grants.

Clinical Development

"Our New Drug Application (NDA) for relacorilant in hypercortisolism is progressing towards approval by the end of this year. We will submit our NDA next quarter for relacorilant in platinum-resistant ovarian cancer. We expect that relacorilant will have a role in helping treat earlier stages of ovarian cancer and other tumors that express the glucocorticoid receptor and have already begun our next clinical trial, BELLA. Meanwhile, we are making progress in understanding the role of cortisol modulation to treat a broad range of other serious disorders, including ALS and Metabolic Dysfunction-Associated Steatohepatitis (MASH)," added Dr. Belanoff.

Hypercortisolism (Cushing’s Syndrome)

Relacorilant for patients with hypercortisolism – U.S. Food and Drug Administration (FDA) accepted NDA for filing; Prescription Drug User Fee Act (PDUFA) target action date of December 30, 2025
GRACE – Pivotal Phase 3 trial of relacorilant in 152 patients with all etiologies of hypercortisolism – Results presented at Endocrine Society (ENDO) annual meeting and Heart in Diabetes (HiD) conference in June 2024
GRADIENT – Randomized, double-blind, placebo-controlled, Phase 3 trial of relacorilant in 137 patients with hypercortisolism caused by adrenal gland pathology – Results will be presented at American Association of Clinical Endocrinology (AACE) annual meeting in May 2025
Phase 3 long-term extension study of 116 patients who completed the GRACE, GRADIENT or Phase 2 relacorilant studies – Results presented at World Congress on Insulin Resistance, Diabetes and Cardiovascular Disease (WCIRDC) in December 2024
CATALYST Part 1 – Prevalence of hypercortisolism in patients with difficult-to-control type 2 diabetes – Results published in Diabetes Care in April 2025
CATALYST Part 2 – Randomized, double-blind, placebo-controlled study of Korlym in 136 patients with hypercortisolism – Results to be presented at the American Diabetes Association’s 85th Scientific Sessions (ADA) in June 2025
MOMENTUM – Enrollment continues in 1,000-patient trial examining the prevalence of hypercortisolism in patients with resistant hypertension – Results expected by year-end
"The positive results from our pivotal GRACE, GRADIENT, long-term extension and Phase 2 studies provide powerful support for the NDA for relacorilant in hypercortisolism. Patients in these studies experienced clinically significant improvements in a wide array of the signs and symptoms of hypercortisolism, without the off-target effects and toxicities that accompany currently available treatments. Relacorilant has the potential to become the new standard of care for patients with hypercortisolism," said Bill Guyer, PharmD, Corcept’s Chief Development Officer.

"CATALYST is a landmark study that will change the way physicians treat some of their sickest patients. Its findings are striking: One in four patients whose diabetes resists treatment with the best available medications have hypercortisolism; hyperglycemia in these patients responds powerfully to treatment with a cortisol modulator. We reached an important milestone with the publication of CATALYST’s prevalence phase results in Diabetes Care and look forward to presenting the full results of the study’s treatment phase at ADA next month," added Dr. Guyer. "Building on the insights from CATALYST, our MOMENTUM study will deepen physicians’ understanding of hypercortisolism as a cause of resistant hypertension."

Oncology

Relacorilant for patients with platinum-resistant ovarian cancer – NDA submission expected in the third quarter with submission of Marketing Authorization Application (MAA) in Europe shortly thereafter
ROSELLA – Primary endpoint of improved progression-free survival (PFS) met in pivotal Phase 3 trial of relacorilant plus nab-paclitaxel in 381 patients with platinum-resistant ovarian cancer (hazard ratio: 0.70; p-value: 0.008; median PFS of 6.5 versus 5.5 months); interim evaluation of overall survival (OS) (hazard ratio: 0.69; p-value: 0.01; median OS of 16.0 versus 11.5 months), with no increase in side effect burden – Results to be presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting in June 2025
BELLA – Enrollment underway in Phase 2 trial of relacorilant plus nab-paclitaxel and bevacizumab in 90 patients with platinum-resistant ovarian cancer
Early-stage prostate cancer – Enrollment continues in randomized, placebo-controlled, Phase 2 trial of relacorilant plus enzalutamide in patients with early-stage prostate cancer, conducted in collaboration with the University of Chicago
"The ROSELLA results are an important advance for patients with platinum-resistant ovarian cancer, a disease with few treatment options. The PFS and OS improvements demonstrated in ROSELLA, with no increase in safety burden, bring us closer to delivering a new standard of care for these patients. We look forward to presenting the full results from ROSELLA in a late-breaker session at ASCO (Free ASCO Whitepaper) and submitting our NDA next quarter," said Dr. Guyer. "We are building on the findings from ROSELLA with our BELLA study, which will examine whether combining relacorilant with two medications – nab-paclitaxel and bevacizumab – will offer patients with platinum-resistant ovarian cancer another potent treatment option."

Amyotrophic Lateral Sclerosis (ALS)

DAZALS – In a randomized, double-blind, placebo-controlled Phase 2 study in 249 patients with ALS, dazucorilant did not meet the primary endpoint of improvement in the ALS Functional Rating Scale-Revised (ALSFRS-R)
DAZALS – An exploratory analysis at the one-year mark shows that patients who received 300 mg of dazucorilant at baseline demonstrate significantly improved overall survival, compared to patients who received placebo and did not switch to dazucorilant in the long-term extension study (hazard ratio of 0.16, p-value: 0.0009)
DAZALS – Results to be presented at European Network to Cure ALS (ENCALS) annual meeting in June 2025
"ALS is a devastating disease. Patients who received dazucorilant did not show improvement in the ALS Functional Rating Scale-Revised (ALSFRS-R), which was DAZALS’ primary endpoint. An improvement in overall survival, first seen at the six-month mark, was also observed at year one of the study. An exploratory analysis determined that patients who received 300 mg of dazucorilant at the start of the study lived significantly longer than patients who received placebo and did not switch to dazucorilant in the long-term extension study, with a hazard ratio of 0.16 (p-value: 0.0009). This long-term extension study is on-going. We will immediately seek input from U.S. and European regulatory authorities on the next steps with dazucorilant," said Dr. Guyer.

Metabolic Dysfunction-Associated Steatohepatitis (MASH)

MONARCH – Enrollment continues in randomized, double-blind, placebo-controlled, Phase 2b trial of miricorilant in patients with biopsy-confirmed or presumed MASH
"In our Phase 1b study, miricorilant reduced liver fat very rapidly, improved liver health and key metabolic and lipid measures and was well-tolerated. We look forward to building on these promising results in our MONARCH study. First results are expected by the end of next year," said Dr. Guyer.

Conference Call

We will hold a conference call on May 5, 2025, at 5:00 p.m. Eastern Time (2:00 p.m. Pacific Time). Participants must register in advance of the conference call by clicking here. Upon registering, each participant will receive a dial-in number and a unique access PIN. Each access PIN will accommodate one caller. A listen-only webcast will be available by clicking here. A replay of the call will be available on the Investors / Events tab of Corcept.com.

Calquence plus chemoimmunotherapy approved in the EU as first and only BTK inhibitor for 1st-line mantle cell lymphoma

On May 5, 2025 AstraZeneca reported that Calquence (acalabrutinib) in combination with bendamustine and rituximab has been approved in the European Union (EU) for the treatment of adult patients with previously untreated mantle cell lymphoma (MCL) who are not eligible for autologous stem cell transplant (Press release, AstraZeneca, MAY 5, 2025, View Source [SID1234652524]).

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The approval by the European Commission follows the positive opinion of the Committee for Medicinal Products for Human Use and was based on positive results from ECHO Phase III trial, presented at the European Haematology Association (EHA) (Free EHA Whitepaper) 2024 Congress and published in The Journal of Clinical Oncology, which demonstrated that Calquence plus bendamustine and rituximab reduced the risk of disease progression or death by 27% compared to standard-of-care chemoimmunotherapy (hazard ratio [HR] 0.73; 95% confidence interval [CI] 0.57-0.94; p=0.016). Median progression-free survival (PFS) was 66.4 months for patients treated with the Calquence combination versus 49.6 with chemoimmunotherapy alone.

MCL is a rare and typically aggressive form of non-Hodgkin lymphoma, often diagnosed at an advanced stage.1,2 An estimated 6,000 patients were diagnosed with MCL in the UK, France, Germany, Spain and Italy in 2024.3

Martin Dreyling, MD, Department of Medicine, University Hospital LMU Munich, and investigator in the trial, said: "This approval provides a new first-line treatment option for patients in the EU with mantle cell lymphoma, an aggressive lymphoma with a dismal long-term outcome still today. With a progression-free survival improvement of more than 16 months for these patients, the acalabrutinib combination is a much-needed advance in this challenging disease."

Dave Fredrickson, Executive Vice President, Oncology Haematology Business Unit, AstraZeneca, said: "Treatment with the Calquence combination in first-line mantle cell lymphoma demonstrated a significant improvement in progression free survival and a consistent safety profile for patients in the pivotal ECHO trial. As the first and only BTK inhibitor approved in this indication in the EU, we are proud to provide a much-needed new option to patients living with this difficult disease."

The safety and tolerability of Calquence was consistent with its known safety profile, and no new safety signals were identified.

Calquence plus bendamustine and rituximab is approved in the US and several other countries in this setting based on the ECHO results. Regulatory applications are currently under review in Japan and several other countries in this indication.

This approval follows the recent approval for Calquence monotherapy for the treatment of adult patients with relapsed or refractory MCL in the EU.

Notes
Mantle cell lymphoma (MCL)
While MCL patients initially respond to treatment, patients do tend to relapse.4 MCL comprises about 3-6% of non-Hodgkin lymphomas, with an annual incidence of 0.5 per 100,000 population in Western countries; It is estimated that there are more than 21,000 patients diagnosed with MCL in the US, UK, France, Germany, Spain, Italy, Japan and China.3,4,5

ECHO
ECHO is a randomised, double-blind, placebo-controlled, multi-centre Phase III trial evaluating the efficacy and safety of Calquence plus bendamustine and rituximab compared to SoC chemoimmunotherapy (bendamustine and rituximab) in adult patients at or over 65 years of age (n=635) with previously untreated MCL.6 Patients were randomised 1:1 to receive either Calquence or placebo administered orally twice per day, continuously, until disease progression or unacceptable toxicity. Additionally, all patients received six 28-day cycles of bendamustine on days 1 and 2 and rituximab on day 1 of each cycle, followed by rituximab maintenance for two years if patients achieved a response after induction therapy.6

The primary endpoint is PFS assessed by an Independent Review Committee; other efficacy endpoints include overall survival (OS), overall response rate, duration of response and time to response.6 The trial was conducted in 27 countries across North and South America, Europe, Asia and Oceania.6

The ECHO trial enrolled patients from May 2017 to March 2023, continuing through the COVID-19 pandemic. Prespecified PFS and OS analyses censoring for COVID-19 deaths were conducted to assess the impact of COVID-19 on the study outcome in alignment with the FDA.

Calquence
Calquence is a second-generation, selective inhibitor of Bruton’s tyrosine kinase (BTK). Calquence binds covalently to BTK, thereby inhibiting its activity.7 In B-cells, BTK signalling results in activation of pathways necessary for B-cell proliferation, trafficking, chemotaxis and adhesion.

Calquence is approved for the treatment of chronic lymphocytic leukaemia (CLL) and small lymphocytic lymphoma (SLL) in the US, Japan and China, and approved for CLL in the EU and many other countries. Calquence is also approved for the treatment of adult patients with previously untreated MCL in the US and other countries. It is also approved for the treatment of adult patients with MCL who have received at least one prior therapy in China and several other countries. Calquence is not currently approved for the treatment of MCL in Japan.

As part of an extensive clinical development programme, Calquence is currently being evaluated as a single treatment and in combination with standard-of-care chemoimmunotherapy for patients with multiple B-cell blood cancers, including CLL, MCL and diffuse large B-cell lymphoma.

AstraZeneca in haematology
AstraZeneca is pushing the boundaries of science to redefine care in haematology. Our goal is to help transform the lives of patients living with malignant, rare and other related haematologic diseases through innovative medicines and approaches that are shaped by insights from patients, caregivers and physicians.

In addition to our marketed products, we are spearheading the development of novel therapies designed to target underlying drivers of disease across multiple scientific platforms. Our acquisitions of Alexion, with expertise in rare, non-malignant blood disorders, and Gracell Biotechnologies Inc., pioneers of autologous cell therapies, expand our haematology pipeline and enable us to reach more patients with high unmet needs through the end-to-end discovery, development and delivery of novel therapies.

Entry into a Material Definitive Agreement

On May 5, 2025, TuHURA Biosciences, Inc. ("TuHURA" or the "Company") and Kineta, Inc., a Delaware corporation ("Kineta"), entered into a First Amendment (the "Amendment") to the previously disclosed Agreement and Plan of Merger, dated December 11, 2024 (as amended from time to time, the "Merger Agreement"), by and among TuHURA, Kineta, Hura Merger Sub I, Inc., a Delaware corporation and a direct wholly-owned subsidiary of TuHURA ("Merger Sub I"), Hura Merger Sub II, LLC, a Delaware limited liability company and direct wholly-owned subsidiary of TuHURA ("Merger Sub II," and together with Merger Sub I, the "Merger Subs"), and Craig Philips, solely in his capacity as the representative, agent and attorney-in-fact of the stockholders of Kineta (the "Stockholders Representative") (Filing, TuHURA Biosciences, MAY 5, 2025, View Source [SID1234652653]). Each capitalized term used but not otherwise defined under Item 1.01 of this Current Report on Form 8-K has the meaning given to it in the Merger Agreement.

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As previously disclosed, pursuant to the terms of the Merger Agreement and subject to the terms and conditions set forth therein, Merger Sub I will (a) merge with and into Kineta (the "First Merger"), with Kineta being the surviving corporation of the First Merger, also known as the "Surviving Entity"; and (b) immediately following the First Merger and as part of the same overall transaction as the First Merger, the Surviving Entity will merge with and into Merger Sub II (the "Second Merger" and, together with the First Merger, the "Mergers" ), with Merger Sub II being the surviving company of the Second Merger.

Also, as previously disclosed, subject to the terms and conditions of the Merger Agreement, at the effective time of the First Merger (the "Effective Time"), each share (the "Share") of Kineta’s common stock, par value $0.001 per share ("Kineta Common Stock"), issued and outstanding immediately prior to the Effective Time (other than (i) Shares held in treasury by Kineta or held directly by TuHURA or the Merger Subs, which Shares will be cancelled, or (ii) Shares that are held by any holder who is entitled to demand and properly demands appraisal of such Shares of pursuant to, and in compliance with, Section 262 of the General Corporation Law of the State of Delaware) will thereupon be converted automatically into and will thereafter represent the right to receive, without interest, (x) the number of validly issued, fully paid and non-assessable shares of common stock, $0.001 par value per share, of TuHURA ("TuHURA Common Stock") (rounded down to the nearest whole share subject to the payment of any cash in lieu of fractional shares as set forth in the Merger Agreement) equal to (i) the Initial Per Share Stock Consideration plus (ii) the Delayed Per Share Stock Consideration, (y) plus an amount in cash equal to (i) the Per Share Cash Consideration plus (ii) the Disposed Asset Payment Right (collectively, the Initial Per Share Stock Consideration, the Delayed Per Share Stock Consideration, the Per Share Cash Consideration and the Disposed Asset Payment Right are referred to as the "Merger Consideration").

Among other things, the following terms in the Merger Agreement have been revised pursuant to the Amendment as follows:

As amended, "Initial Per Share Stock Consideration" means the number of shares of TuHURA Common Stock being issued for each share of Kineta Common Stock, determined as follows:


the difference of $16,500,000 and any deductions if the Per Share Cash Consideration (as described below) is less than zero;

such difference in the first bullet, divided by $5.7528, such stock price, the "Parent Share Value"; and

with such resulting quotient from the second bullet, divided by the fully diluted Kineta Common Stock, all rounded down to six (6) decimal places.
As amended, "Delayed Per Share Stock Consideration" means the number of shares of TuHURA Common Stock being issued for each share of Kineta Common Stock, determined as follows:


the difference of $6,500,000 and (i) any liabilities incurred by TuHURA due to a breach of the undisclosed liabilities representation made by Kineta in the Merger Agreement; (ii) any and all losses incurred through the six months after the Closing and those losses estimated to be incurred by TuHURA related to any stockholder litigation; and (iii) any amount to which the closing net working capital deficient is greater than $6,000,000;

such difference in the first bullet, divided by the Parent Share Value; and

with such resulting quotient from the second bullet, divided by the fully diluted Kineta Common Stock, all rounded down to six (6) decimal places.
As amended, "Per Share Cash Consideration" means an amount in cash for each share of Kineta Common Stock, determined as follows:


the difference of $12,000,000 and (i) $5,000,000 (as credit for the exclusivity payment already made by TuHURA to Kineta); (ii) $300,000 (as credit for the extension payment already made by TuHURA to Kineta); (iii) $695,000 (which represents advances already made by TuHURA to Kineta in connection with the exclusivity agreement); (iv) the Loaned Amount, if any; and (v) if the net working capital is less than $0, such difference (and if the net working capital is greater than $0, then such difference will be added to the $12,000,000 base cash consideration); and

such difference in the first bullet, divided by the fully diluted Kineta Common Stock, all rounded down to six (6) decimal places.
As amended, "Loaned Amount" means all principal and interest outstanding under any loan between TuHURA, on the one hand, and Kineta, on the other hand, consisting of (i) $250,000 that was previously advanced by TuHURA to Kineta, (ii) $250,000 to be advanced by TuHURA to Kineta on or before May 15, 2025, and (iii) $250,000 to be advanced by TuHURA to Kineta on or before June 3, 2025 (with the advance in foregoing clause (iii) being contingent upon TuHURA’s receipt of (A) proceeds from the Concurrent Investment or (B) proceeds from TuHURA stockholder warrant exercise payments due on May 30, 2025), in each case, for any expenses incurred by Kineta in the ordinary course of business or expenses incurred in connection with the Program Assets and approved by TuHURA, and such amount shall be paid by TuHURA to Kineta no later than five (5) Business Days after the request is made (and invoice or proof of expense is provided to TuHURA) as long as no event of default has occurred and is continuing under the Merger Agreement as of the date of such request and so long as the parties thereto are then still working in good faith toward a Closing.

In addition to the foregoing, pursuant to the Amendment, the parties have agreed that, as a condition precedent to the obligations of TuHURA and the Merger Subs to effect the Mergers and otherwise consummate the transaction contemplated by the Merger Agreement, the Concurrent Investment shall have been completed and TuHURA shall have received gross proceeds of no less than Twenty Million Dollars ($20,000,000), which gross proceeds shall have been received by TuHURA, or will be received by TuHURA substantially simultaneously with Closing.

Also pursuant to the Amendment, the End Date has been extended from April 30, 2025, to June 30, 2025, subject to possible extension as provided by the Amendment.

Other than as expressly modified by the Amendment, the Merger Agreement, which was filed as Exhibit 2.1 to the Current Report on Form 8-K filed by the Company with the Securities and Exchange Commission (the "SEC") on December 12, 2024, remains in full force and effect. The foregoing description of the Amendment does not purport to be complete and is subject to, and qualified in its entirety by reference to, the full text of the Amendment, which is attached as Exhibit 2.1 hereto and incorporated herein by reference.

Additional Information about the Proposed Mergers and Where to Find It

This communication may be deemed to be solicitation material with respect to the proposed Mergers between TuHURA and Kineta. In connection with the proposed Mergers, TuHURA has filed relevant materials with the SEC, including the preliminary joint proxy statement/prospectus filed on February 7, 2025 and subsequently amended on May 6, 2025 (the "Joint Proxy Statement/Prospectus"). TuHURA will mail a definitive Joint Proxy Statement/Prospectus to the TuHURA stockholders when it becomes available. Investors and securityholders of TuHURA and Kineta are urged to read these materials because they contain important information about TuHURA, Kineta and the Mergers. This communication is not a substitute for the definitive Joint Proxy Statement/Prospectus, when it becomes available, or any other documents that TuHURA may file with the SEC or send to securityholders in connection with the proposed transactions.

Investors and securityholders may obtain free copies of the documents filed with the SEC, once available, on TuHURA’s website at www.tuhurabio.com, on the SEC’s website at www.sec.gov or upon written request to: TuHURA, 10500 University Drive, Suite 110, Tampa, FL 33612.

No Offer or Solicitation

This communication shall not constitute an offer to sell or the solicitation of an offer to sell or the solicitation of an offer to buy any securities, nor shall there be any sale of securities in any jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such jurisdiction. No offering of securities shall be made except by means of a prospectus meeting the requirements of Section 10 of the Securities Act of 1933, as amended (the "Securities Act").

Participants in the Solicitation

Each of TuHURA, Kineta and their respective directors and executive officers may be deemed to be participants in the solicitation of proxies from the stockholders of TuHURA in connection with the proposed Mergers. Information about TuHURA’s directors and executive officers is set forth in TuHURA’s filings with the SEC, including TuHURA’s Form 10-K filed on March 31, 2025. Additional information regarding the direct and indirect interests, by security holdings or otherwise, of those persons and other persons who may be deemed participants in the solicitation of proxies in the Mergers may be obtained by reading the definitive Joint Proxy Statement/Prospectus when it becomes available. You may obtain free copies of these documents as described above under "Additional Information about the Proposed Mergers and Where to Find It".

Ichnos Glenmark Innovation (IGI) Receives U.S. FDA Fast Track Designation for ISB 2001 for Relapsed/Refractory Multiple Myeloma

On May 5, 2025 IGI, a global, fully integrated clinical-stage biotechnology company focused on developing multispecificsTM in oncology, reported that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for ISB 2001 (Press release, Ichnos Sciences, MAY 5, 2025, View Source;utm_medium=rss&utm_campaign=ichnos-glenmark-innovation-igi-receives-usfda-fasttrack-designation-for-isb-2001-for-relapsed-refractory-multiple-myeloma [SID1234652507]). This important designation was granted for the treatment of adult patients with relapsed or refractory multiple myeloma (RRMM) who have received at least three prior lines of therapy including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody. ISB 2001 is an investigational trispecific antibody therapeutic that targets BCMA and CD38 on myeloma cells and CD3 on T cells. ISB 2001 is currently being evaluated in a Phase 1 dose-expansion study.

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"A growing number of patients with multiple myeloma have been heavily pretreated, have exhausted currently approved therapies, and continue to face disease progression," said Cyril Konto, M.D., President and CEO of IGI. "At IGI, we have long recognized the urgent need for novel treatment options – particularly for patients who have already received first-generation bispecifics or CAR T-cell therapies. Our trispecific candidate is designed to enhance tumor targeting while reducing on-target, off-tumor toxicity. We are honored to receive this Fast Track designation and look forward to working closely with the FDA to advance our MultispecificTM T-cell engager, with the goal of delivering a first-in-class therapy for patients with relapsed or refractory multiple myeloma."

IGI recently completed the dose-escalation portion of its Phase 1 clinical study in patients with heavily pretreated multiple myeloma. Initial study results, presented in an oral session at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December 2024, demonstrated a high overall response rate (ORR) with durable responses and a favorable safety profile. Complete results from the dose-escalation portion will be presented in a rapid oral session at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting on Monday, June 2, 2025.

The FDA’s Fast Track designation is designed to enable the development and expedite the review of drugs that treat serious conditions and address unmet medical needs, with the ultimate goal of getting important new drugs to patients earlier. A drug that receives Fast Track designation may be eligible for more frequent meetings and communications with the FDA and rolling review of any application for marketing approval. A drug receiving Fast Track designation also may be eligible for Priority Review if relevant criteria are met. ISB 2001 was previously granted Orphan Drug Designation by the FDA in July 2023.

ASCO Rapid Oral Presentation Details:
Session title: Phase 1, first-in-human study of ISB 2001: A BCMAxCD38xCD3-targeting trispecific antibody for patients with relapsed/refractory multiple myeloma (RRMM)—Dose escalation results. (Abstract # 7514)

Session Name: Hematologic Malignancies—Plasma Cell Dyscrasia

Date & Time: June 2, 2025, 8 AM – 9:30 AM CDT

About ISB 2001 and Relapsed/Refractory Multiple Myeloma

ISB 2001 is a first-in-class trispecific T-cell engager that targets BCMA and CD38 on myeloma cells and CD3 on T cells. Developed using IGI’s proprietary BEAT protein platform, ISB 2001 was engineered with two distinct binders against myeloma-associated antigens to enhance avidity, even at low target expression levels, while aiming to improve safety over first-generation bispecific antibodies. The dose-expansion portion of the ongoing Phase 1 trial in patients with RRMM (NCT05862012) is currently enrolling patients across 9 sites in the United States and Australia.

Nearly all patients with relapsed or refractory multiple myeloma (RRMM) ultimately experience disease progression. With no cure currently available and limited treatment options once approved therapies are exhausted, there remains a significant unmet need. IGI is developing ISB 2001 to address this gap, specifically for patients who have previously received T-cell–directed therapies, including CAR T-cell treatments and bispecific antibodies.