BCMA

Results from the dose-escalation portion of the Phase 1 clinical study of ISB 2001 in patients with heavily pretreated multiple myeloma to be presented at the 2025 ASCO Annual Meeting NEW YORK, May 5, 2025 /PRNewswire/ -- IGI, a global, fully integrated clinical-stage biotechnology company focused on developing multispecifics™ in oncology, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for ISB 2001. 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. "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 Multispecific™ 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) 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) 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. About IGI IGI is a global, fully integrated clinical-stage biotechnology company focused on developing innovative biologics in oncology. Headquartered in New York, NY, IGI is advancing a robust pipeline of novel, first-in-class multispecifics™ aimed at addressing complex diseases and treating patients holistically. Powered by its proprietary BEAT technology platform, IGI is committed to delivering breakthrough, curative therapies to improve and extend the lives of patients battling hematological malignancies and solid tumors. For more information, visit . SOURCE Ichnos Glenmark Innovation WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

The first shards of human data on in vivo CAR-T cell therapy look promising, based on recent separate reports from two biotech companies’ academic-led studies in China. Last week, a Chinese startup called Genocury became the second company to share initial clinical data on a CAR-T treatment that doesn’t require a patient’s cells to be extracted from their body. Genocury said that an advanced large B cell lymphoma patient went into complete remission after one month of in vivo CAR-T therapy, and has remained in remission for over three months. Notably, the in vivo CAR-T therapy does not require lymphodepletion, a course of chemotherapy that conventional CAR-T therapies require before treatment and that comes with toxicities. “This is good news for the field and demonstrates the increasing momentum around in vivo CAR-T approaches,” said University of Pennsylvania CAR-T pioneer Bruce Levine, who isn’t involved in Genocury’s studies but is a scientific founder of another in vivo CAR-T company called Capstan. Last week’s results follow Belgian startup EsoBiotec’s announcement in January that a multiple myeloma patient who received the lowest dose of its in vivo CAR-T therapy had no detectable cancer cells in their bone marrow at one month, without any significant adverse events during treatment. That patient also did not receive prior lymphodepletion. The data from both companies come from investigator-initiated studies in China, which biotech startups have been leveraging more frequently to quickly generate early clinical data on their experimental treatments. EsoBiotec is working with Pregene Biopharma which, like Genocury, is based in Shenzhen. In March, AstraZeneca announced plans to acquire EsoBiotec for $425 million, plus additional payments. At least five groups have begun clinical trials for in vivo CAR cell therapies. Aside from EsoBiotec and Genocury, Myeloid Therapeutics, Interius BioTherapeutics and Umoja Biopharma are all studying experimental in vivo cell therapies in patients. In conventional CAR-T therapy, a patient’s cells are extracted, engineered, and then reinfused — a complex, expensive process that can take weeks to months. The therapy, while transformative for certain blood cancers, comes with safety risks from the lymphodepletion before treatment and immune overreaction (cytokine release syndrome and ICANS) afterwards. And so far, comparatively less complex “off-the-shelf” treatments using donor cells have yet to take off. Makers of in vivo cell therapy hope that their treatments can become a different kind of “off-the-shelf” option. Their therapies deliver genetic material to turn the patient’s immune cells into cancer-fighting ones directly in the body. The companies are using different technologies to deliver their treatments and are also going after a range of diseases. The delivery technologies fall into two camps: viral and non-viral. Researchers have more concerns around the potential risks of a virus-based therapy, including of secondary cancer, but it’s an older and more proven approach. Non-viral delivery is newer and more challenging, and fewer companies have reached clinical studies. It is unclear based on Genocury’s disclosures whether it is using a viral or non-viral approach. A photo on its website mentions both viral and non-viral methods, and the company could not be reached for comment. EsoBiotec is developing a lentiviral-based cell therapy that goes after BCMA, a well-known target for multiple myeloma. Interius, which expects to report clinical data later this year, and Umoja are likewise studying lentiviral-based therapies. Umoja, like Genocury, is developing a CD19-targeted CAR-T therapy for blood cancer, and it plans to study the treatment in autoimmune disease as well. AbbVie has an option to license that therapy, and in January the startup raised $100 million to jumpstart its clinical studies. Interius’ therapy targets another immune cell protein called CD20, and it’s studying its B cell cancer therapy in both Australia and Germany. The Philadelphia biotech’s therapy is meant to make both ​​CAR-T and NK cells directly in the body. On the other hand, Sid Mukherjee’s startup Myeloid is using mRNA delivered non-virally. It uses fatty acid envelopes called lipid nanoparticles — similar to what’s used in the Covid vaccines. However, as its name suggests, it’s targeting a type of immune cell called myeloid cells. It has two programs in the clinic going after solid tumors — an area of cancer that conventional CAR-T therapies have yet to crack.

On a call with members of the media Tuesday morning, AstraZeneca CEO Pascal Soriot focused heavily on the need for greater R&D innovation outside the U.S. \n AstraZeneca has axed three MedImmune legacy monoclonal antibody drugs from its neuroscience pipeline in a first-quarter clear-out.The Anglo-Swedish Big Pharma announced in its first-quarter presentation (PDF) Tuesday morning that it had stopped work on a phase 1 Alzheimer’s disease drug as well as two phase 2 therapies. One of the midstage candidates was being tested against migraines and the other was for osteoarthritis pain and painful diabetic neuropathy.The Alzheimer’s drug, MEDI1814, is an amyloid beta mAb that had been developed in collaboration with Eli Lilly in a pact dating back nearly a decade.Lilly, which markets its AD therapy Kisunla and teamed up with AZ on MEDI1814 back in 2016, has taken back the drug.AZ also axed two more clinically advanced prospects. The first, MEDI0618, works as a PAR2 antagonist mAb migraine drug. The market for migraine drugs, both as treatments and prophylactic options, has exploded over the past five years, with a host of meds from Pfizer/Biohaven, Eli Lilly, Amgen, Teva and more fighting for ground. The final midstage cull was for MEDI7352, a non-opioid NGF/TNF bispecific mAb targeting osteoarthritis pain and painful diabetic neuropathy.The decision for this neuro-focused clear-out was strategic, and AZ has formally terminated each of the projects. All three came from MedImmune, its $15.2 billion acquisition in 2007 that has seen multiple experimental drug clear-outs over the years.AZ generated $13.58 billion in revenue during the first quarter, a 10% increase at constant exchange rates from the first quarter in 2024. R&D spend, meanwhile, was up a hefty 16% at constant exchange rates to $3.08 billion and represented 23% of total sales.FDA layoffs, R&D boosts and a U.S. focus On a call with members of the media Tuesday morning, AZ’s chief Pascal Soriot said that despite the Trump administration-directed layoffs at the FDA, the Big Pharma has “so far not seen any delays in the processing of our applications [or] any delays to our meetings we regularly have with FDA staff.\" A big theme for Soriot was AZ\'s U.S. investment and focus, following a common trend for Big Pharmas working in the country amid pharmaceutical tariff threats from the U.S. government.He reiterated the multibillion-dollar investment AZ announced last November in manufacturing and R&D, saying that “we have even greater U.S. investment in manufacturing and R&D planned, leveraging our two large R&D sites\" in Gaithersburg, Maryland, and Cambridge, Massachusetts.Details on exactly what that R&D investment would like and specific dollar values were not given. AZ’s chief financial officer Aradhana Sarin said on the call that investments “will be driven by our portfolio” and that AZ will spend money where trials and trial sites are producing positive outcomes worthy of further investment. A spokesperson for AZ confirmed there were no immediate specifics on the R&D investment front as the company plans to review what is working over time and thus what will require deeper investment. One area will, however, be cell therapy, a field Soriot spotlighted during the call and where AZ recently acquired a Belgium cell therapy specialist.AZ also currently has seven cell therapy programs in the clinic, led by a BCMA and CD19 dual-targeting CAR-T for multiple myeloma.In response to questions about U.S. drug research innovation in general, Soriot said the country is “funding innovation in pharmaceuticals for the world” but that this was not fair. He said that Europe must take greater steps to invest more.Likening Europe’s recent decision to spend more of its gross domestic product on defense, he said Europe should take the same tack with pharmaceuticals spending, upping its percentage of GDP and/or healthcare budgets to pay more for R&D innovation.“Innovation has mostly been funded by the United States,” Soriot said on the call. “Europe now has to invest more. Just like Europe has decided to allocate a greater share of GDP to defense, a greater share of GDP has to be allocated to pharmaceutical innovation.”Soriot, when asked, did not give a specific percentage on what he thought that figure of GDP should be, but he noted the percentage of healthcare budgets spent on medicines was 13% to 15% in the U.S., while it was 10% to 11% in Europe and just 7% in the U.K., which has the NICE health technology assessment body to help temper drug prices.