RegulationPriority Review (United States), Accelerated Approval (United States), Orphan Drug (United States), PRIME (European Union), Regenerative Medicine Advanced Therapy (United States)

Structure/Sequence

Sequence Code 1277314924

Source: *****

Clinical Trials associated with Afamitresgene Autoleucel

NCT05642455

/ RecruitingPhase 1/2

A Phase 1/2 Open Label, Basket Study to Assess the Safety, Tolerability and Anti-Tumor Activity of Afamitresgene Autoleucel in Pediatric Subjects With MAGE-A4 Positive Tumors

This is a pediatric basket study to investigate the safety and efficacy of afamitresgene autoleucel in HLA-A*02 eligible and MAGE-A4 positive subjects aged 2-17 years of age with advanced cancers.

Start Date01 Sep 2023

Sponsor / Collaborator-

NCT04408898

/ WithdrawnPhase 2

A Phase 2 Pilot Study of ADP-A2M4 in Combination With Pembrolizumab in Subjects With Recurrent or Metastatic Head and Neck Cancer

This is a study to investigate the efficacy and safety of ADP-A2M4 in combination with pembrolizumab in HLA-A*02 eligible and MAGE-A4 positive subjects with recurrent or metastatic Head and Neck cancer.

Start Date02 Jul 2020

Sponsor / Collaborator

Adaptimmune Ltd.

NCT04044768

/ Active, not recruitingPhase 2

A Phase 2 Single Arm Open-Label Clinical Trial of ADP-A2M4 SPEAR™ T Cells in Subjects With Advanced Synovial Sarcoma or Myxoid/Round Cell Liposarcoma

This is a study to investigate the efficacy and safety of ADP-A2M4 in HLA-A*02 eligible and MAGE-A4 positive subjects with metastatic or inoperable (advanced) Synovial Sarcoma (Cohort 1, 2 and 3 ) or MRCLS (Cohort 1) .

Start Date13 Aug 2019

Sponsor / Collaborator-

100 Clinical Results associated with Afamitresgene Autoleucel

100 Translational Medicine associated with Afamitresgene Autoleucel

100 Patents (Medical) associated with Afamitresgene Autoleucel

Literatures (Medical) associated with Afamitresgene Autoleucel

01 Feb 2026·CURRENT OPINION IN PEDIATRICS

Update on pediatric soft tissue sarcomas

Review

Author: Crane, Jacquelyn ; Aye, Jamie ; Oberoi, Sapna

Purpose of review:

The purpose of this review is to highlight recent findings in the diagnosis, biology, risk-stratification, and treatment of soft tissue sarcomas (STS) in children.

Recent findings:

In rhabdomyosarcoma (RMS), FOXO1 fusion status has been confirmed as an important prognostic factor. Among fusion-negative RMS, TP53 and MYOD1 mutations and detectable circulating tumor DNA at diagnosis are associated with inferior event-free survival in intermediate-risk disease. Delayed primary excision is associated with a reduced risk of local failure whereas radiotherapy dose escalation in large tumors has not improved local control. Maintenance therapy with vinorelbine and oral cyclophosphamide following induction chemotherapy in the RMS2005 trial led to improved survival. In non-rhabdomyosarcoma soft tissue sarcomas, the addition of pazopanib, a multitargeted receptor tyrosine kinase inhibitor, to upfront therapy did not improve survival. Atezolizumab is approved for alveolar soft part sarcoma, larotrectinib for NTRK fusion–positive STS, and afamitresgene autoleucel remains under evaluation in children with synovial sarcoma. Encouraging early results have been reported with tazemetostat and immune checkpoint inhibitors in epithelioid sarcoma and trastuzumab in desmoplastic small round cell tumor, respectively.

Summary:

Pediatric STS are rare and biologically heterogeneous. Genomic advances have refined risk stratification and uncovered therapeutic targets; further progress relies on international collaboration and trials.

01 Jan 2026·Journal for ImmunoTherapy of Cancer

Cell therapy in sarcoma: current landscape and future directions

Review

Author: Sahin, Taha Koray ; Guven, Deniz Can ; Dizdar, Omer ; Thistlethwaite, Fiona ; Connolly, Elizabeth A ; Lim, Kok Haw Jonathan ; Germetaki, Theodora ; Akin, Serkan

Sarcomas are rare malignancies of mesenchymal origin, characterized by significant biological and clinical heterogeneity. Many subtypes demonstrate limited sensitivity to standard systemic treatments, including immune checkpoint inhibitors. Cell therapy has emerged as a promising strategy, with the potential of durable clinical responses seen with genetically-engineered T-cell receptor T-cell therapies (TCR-T) such as those targeting the cancer-testis antigen MAGE-A4 in synovial sarcoma, leading to the US Food and Drug Administration approval of afamitresgene autoleucel in 2024. This constituted only the second approval of a cell therapy in a solid tumor following lifileucel in melanoma and demonstrated the potential of cell therapies in sarcomas. This review provides the current landscape and growing potential of cell therapies in sarcomas, including TCR-T, chimeric antigen receptor-T cells, tumor-infiltrating lymphocytes, natural killer (NK) cells, and mesenchymal stromal cells. However, the broader application of these therapies is hindered by the lack of targetable sarcoma-restricted immunogenic epitopes, spatiotemporal intratumoral heterogeneity, and a profoundly immunosuppressive tumor microenvironment that impedes effector-cell trafficking, expansion and persistence. While cell therapies hold promise for integration into precision medicine approaches for sarcomas, their successful implementation will require careful evaluation of clinical feasibility, logistical considerations and cost-effectiveness to optimize patient outcomes.

01 Oct 2025·Trends in Cancer

Advancing human leukocyte antigen-based cancer immunotherapy: from personalized to broad-spectrum strategies for genetically heterogeneous populations

Review

Author: Wang, Yunfei ; Oseni, Saheed Oluwasina ; Hwu, Patrick

Human leukocyte antigen (HLA)-based immunotherapeutics, such as tebentafusp-tebn and afamitresgene autoleucel, have expanded the treatment options for HLA-A*02-positive patients with rare solid tumors such as uveal melanoma, synovial sarcoma, and myxoid liposarcoma. Unfortunately, many patients of European, Latino/Hispanic, African, Asian, and Native American ancestry who carry non-HLA-A*02 alleles remain largely ineligible for most current HLA-based immunotherapies. This comprehensive review introduces HLA allotype-driven cancer health disparities (HACHD) as an emerging research focus, and examines how past and current HLA-targeted immunotherapeutic strategies may have inadvertently contributed to cancer health disparities. We discuss several preclinical and clinical strategies, including the incorporation of artificial intelligence (AI), to address HACHD. Last, we emphasize the urgent need for further research to better understand HLA allotype heterogeneity and its influence on tumor immunopeptidome-driven immune responses. We anticipate that these strategies will accelerate the development and implementation of both personalized and broad-spectrum HLA-based immunotherapies, and will ultimately improve cancer treatment across genetically heterogeneous patient populations worldwide.

News (Medical) associated with Afamitresgene Autoleucel

25 Feb 2026

·www.biospace.com

Cashing In on ‘Creative Fundamentals’ To Fund Rare Disease R&D

iStock, phive2015 Rare disease drug developers struggle to survive in a biopharma investment market that prioritizes large patient populations. Initiatives like the Orphan Therapeutics Accelerator are attempting to solve what CEO Craig Martin says is not a science problem, but a math problem. The rare disease space got a huge boost last month when the FDA’s rare pediatric disease priority review voucher program was reauthorized after more than a year in limbo. While a step in the right direction, rare and orphan drug leaders say more needs to be done to attract investment for these often-niche programs. “That was, I think, a great legislative victory,” MediciNova Chief Business Officer David Crean said of rare pediatric disease priority review vouchers (PRVs). Craig Martin, founder and CEO of the Orphan Therapeutics Accelerator (OTXL), agreed. “There’s some progress at least,” he told BioSpace at Phacilitate’s Advanced Therapies Week earlier this month, referring to PRVs and recent updates to the U.S. newborn screening program . The rare disease space has also seen a number of regulatory wins recently. This week, the FDA provided highly anticipated guidance for its plausible mechanism pathway for personalized therapies, first announced in November 2025. Two months before that, the agency unveiled its new Rare Disease Evidence Principles framework, intended to streamline the approval of therapies for ultra-rare diseases. But while the regulatory piece is rapidly evolving, the very nature of rare—and particularly ultrarare—diseases, poses a significant investment quagmire for the traditional biotech model, which assumes access to deep public equity markets, Crean said. This is largely due to the small—and sometimes minuscule—number of people affected with these diseases. There are more than 10,000 rare diseases, defined as those affecting fewer than 200,000 people, according to the Rare Disease Company Coalition. Ultrarare conditions , afflicting less than 1,000, offer an even smaller market opportunity. OTXL estimates more than 9,000 indications fit this definition. “Small patient populations mean lower peak revenue potential per indication and that changes the whole risk-return calculus for traditional VCs,” Crean said. Kristen Hege, former SVP of Early Clinical Development, Hematology/Oncology & Cell Therapy at Bristol Myers Squibb, agreed, saying, “Right now, as a rule of thumb, if your product isn’t going to bring in a billion dollars a year, it’s not going to be developed.” Alternative Models This dilemma was the impetus behind OTXL. Prior to 2022, the rare disease drug development space had already begun to stall from both a market and a policy perspective, Martin said. Provisions from the Inflation Reduction Act, signed into law in August 2022, further eroded confidence and commercial viability of these treatments. “A number of these programs were ones that had advanced into trials, were helping children in particular, and yet weren’t moving forward,” he said. “I was haunted by the fact that there were so many of these treatments that could be helping patients, and it wasn’t a science problem; it was a math problem.” OTXL obtains , funds and completes development of these shelved assets, leveraging a network of partners and its tax-exempt status as a nonprofit to lower costs. This tax exemption “incentivize[s] companies that were maybe sitting on an asset to liberate it and allow us to move it forward and to reduce or eliminate upfront costs for licensing or obtaining that asset,” he explained. The company has built relationships with a network of CROs and CDMOs, with whom it works on a deferred or reduced cost basis to further decrease the funding required for late-stage drug development in particular, Martin said. Since its launch in 2024, OTXL has considered more than 60 programs focused on ultrarare diseases, according to Martin, many of which are cell and gene therapies for pediatric conditions. OTXL can’t adopt them all, of course, so the organization is working on an AI-based platform that would allow it to ingest data rooms, vet investigational therapies, perform predictive modeling and conduct development planning, all intended to attractively package the assets for companies or investors to take on. Once a therapy wins regulatory approval, OTXL works with partners and employs alternative approaches—including its tax-exempt subsidiary Orphan Therapies —to ensure access. Recently, OTXL partnered with Italy-based Fondazione Telethon, which in December 2025 won FDA approval for Waskyra, a gene therapy for Wiskott-Aldrich syndrome. Fondazione Telethon, the first non-profit sponsor to secure FDA approval for a gene therapy, was looking for a U.S.-based commercial partner to support Waskyra’s launch this year when OTXL stepped up. With Waskyra,“We kind of got to commercial stage a little faster than we thought we would,” Martin said. Next up, the nonprofit is finalizing terms of an agreement for its first clinical-stage asset, an AAV gene therapy. While Martin declined to reveal the exact program or disease area, he said that compared to Waskyra—which might have a market reach of five to seven patients per year—the condition “is a little bit closer to the cusp of what a biotech might be willing to take on once we further de-risk it.” Rare diseases FDA Greenlights First Gene Therapy for Rare Pediatric Disease Backed by Italy-based Fondazione Telethon ETS, Waskyra, for Wiskott-Aldrich syndrome, is the first gene therapy from a non-profit sponsor to win FDA approval. December 10, 2025 · 1 min read · Tristan Manalac The challenges faced by rare disease drug developers sometimes mean a company is forced to sell off multiple assets or fold entirely. Hege sat on the board of Adaptimmune until the biotech sold its cell therapy portfolio, including synovial sarcoma cell therapy Tecelra, to US WorldMeds in July 2025. Approved by the FDA in August 2024, Tecelra was the first engineered T cell therapy approved for solid tumor cancers in the U.S. “That was a major accomplishment for the field of cell therapy,” Hege said. “Unfortunately, Adaptimmune wasn’t really able to make it commercially viable enough to continue as a standalone company. “Synovial sarcoma is a pretty rare indication, so you couldn’t get a lot of Big Pharma or venture investor interest in that small market opportunity,” Hege continued, calling this a “fundamental problem with our drug development system.” Hege is working with the nonprofit Friends of Cancer Research, which aims to drive collaboration among industry, scientists and policy makers, to devise a strategy to advance breakthrough therapies for “uncommon” cancer indications, including sarcoma, small cell lung cancer, pancreatic cancer and “pretty much every” pediatric cancer. These indications “are still not commercially attractive to drive investment in our current paradigm of cancer R&D,” Hege said. For companies intrepid enough to attempt to traverse the complex rare disease market, the experts who spoke to BioSpace offered advice. “Capital efficiency is super critical here,” said Crean, whose company, MediciNova, faces the additional hurdle of developing treatments in the notoriously challenging neurodegenerative disease space. “It almost becomes a part of the strategic imperative.” Crean recommended employing shared infrastructure across rare disease assets and staged development strategies aimed at reaching meaningful clinical milestones with less capital deployed per stage. In terms of fundraising options for rare disease-focused companies, Crean noted the “creative fundamentals,” including royalty financing, which he is becoming more prevalent. Deals Royalty Financing Serves as Lifeline for Some Biopharma Companies in Uncertain Market As traditional fundraising methods falter for smaller firms, the rise of royalty deals is reshaping how companies access capital, offering an alternative that bypasses equity dilution and debt. October 2, 2024 · 5 min read · Ben Hargreaves Read more Revolution Medicines last month signed a $2 billion deal with Royalty Pharma to support its portfolio of treatments for RAS-addicted malignancies, including pancreatic cancer. Royalty, which buys royalties on future sales in exchange for non-dilutive capital, counts Roche’s Evrysdi for spinal muscular atrophy and Denali’s tividenofusp alfa for Hunter syndrome—which has an FDA action date in April—among its portfolio. Regulatory incentives, such as FDA orphan drug and breakthrough therapy designations, along with PRVs, also need to be incorporated into the strategic and capital financing strategy, Crean said. Part of the solution could actually be on the regulatory side, Hege said. “What does it take in these uncommon cancers to get a product approved? Because in our country, until a product is approved, payers don’t pay for the product, so the cost of development is all borne by the sponsor.” These costs include product manufacturing and clinical trials. Hege suggested a model of shared risk between sponsor and payer, where the payer covers the cost for a product in development, or a regulatory process where the asset is granted accelerated approval earlier in its development course, “at which point it is an FDA-approved product, and the payers have to pay.” This would be followed by a post-marketing agreement to run larger, more expensive trials. “I think when the unmet need is high enough,” Hege said, “we just have to become more risk tolerant.”

Orphan DrugDrug ApprovalPriority ReviewBreakthrough TherapyCell Therapy

02 Dec 2025

·endpoints.news

Exclusive: ARCH, Parker Institute build next big oncology bet with suite of programs and startup acquisition

Some of the biggest names in oncology R&D have been quietly consolidating assets, money and programs under a new startup called Medici Therapeutics, Endpoints News has learned. The stealthy startup’s backers include two of the biggest names in biotech and cancer: ARCH Venture Partners and the Parker Institute for Cancer Research (PICI), which is finalizing its investment plans in the startup, according to a source familiar with its plans. Two sources say Medici recently acquired Nutcracker Therapeutics, which is developing personalized RNA-based medicines and has raised more than $200 million from ARCH and other investors. Medici’s existence has been mentioned briefly in a handful of regulatory and scientific documents, but the scope of its actions and ambitions hasn’t been previously reported. A spokesperson for the startup declined to say how much money it’s raised. But it has already built a portfolio of programs, including personalized cancer vaccines, tumor-infiltrating lymphocyte (TIL) therapies, in vivo TCR therapies, cellular rejuvenation approaches and mRNA manufacturing capabilities. Its chief scientific officer is Ira Mellman , the former Genentech VP of cancer immunology who is now president of research at PICI . Nicholas Restifo , a 30-year principal investigator at NIH, is the startup’s chief medical officer. The technology chief is David Thompson, who’s also co-founded 64x Bio, a startup in the viral gene therapy manufacturing field. The board is led by Matthew Rabinowitz, the former longtime CEO of Natera. It has also brought on major figures, including Bob Langer, Carl June, Drew Weissman, George Church and Michael Snyder as advisors. Its CEO is Denitsa Milanova, a mechanical engineer who did fellowships in Snyder and Church’s labs. While it’s named after the powerful Italian Renaissance dynasty, Medici’s origins are more humble. It began life several years ago as Marble Therapeutics, which was working on anti-aging. But it began to point its cellular rejuvenation platform and other technologies toward oncology, with the goal of developing cures rather than step changes, according to a source familiar with the biotech’s evolution. ARCH is known for taking large swings with loads of cash, sometimes leading to multibillion-dollar successes such as obesity drug developer Metsera. Co-founder Bob Nelsen has previously said the firm tries to “hit home runs all the time.” At Medici, it would need a manufacturer to help speed up development of its therapies and get into the clinic, potentially as soon as next year. That drove the purchase of Nutcracker, after talks this summer. Nutcracker’s microfluidic chip-based process can help make medicines in a box that is similar in size to an office printer. Nutcracker’s shareholders will become Medici shareholders, according to a source familiar with the matter. The combined company has about 60 employees, according to another source. All of the sources cited here spoke on condition of anonymity, and they also didn’t share many details on the startup’s scientific programs. Like most of ARCH’s other major bets, Medici will likely face competition along the way. BioNTech, Moderna and its partner Merck, and multiple other biopharmas have been trying to create personalized cancer vaccines for years. Meanwhile, the first TCR drug was approved by the FDA, with Adaptimmune’s sarcoma therapy Tecelra. But Adaptimmune struggled financially and eventually sold Tecelra , along with other cell therapy assets, for $55 million in cash earlier this year. ARCH is willing to take on those risks, though. Preventive medicine has become a key piece of the firm’s investing mission. “Why would we take a really shitty healthcare system that wastes $3 trillion a year, probably, of the $4 trillion, on a reactive healthcare system instead of reinventing it to be more preventive and more curative and that we don’t need all these big, giant hospitals and a bunch of other stuff?” Nelsen said in a September 2024 interview . Editor’s note: This story was updated to clarify that PICI is finalizing its investment plans in Medici.

AcquisitionDrug ApprovalCell Therapy

21 Nov 2025

·endpoints.news

Jeff Bluestone, Stephen Dilly talk CEO handoff, what’s next for Sonoma; Scott Gottlieb's next move

Endpoints News sat down with Jeff Bluestone and Stephen Dilly after Sonoma Biotherapeutics announced earlier this week that Bluestone was stepping down from his CEO role and Dilly would be taking his place. Bluestone, a prominent academic and the first head of the Parker Institute for Cancer Immunotherapy, founded Sonoma six years ago to develop regulatory T cell therapies. On Monday, he handed the reins to Sonoma over to Dilly, the former CEO of Codexis and Sierra Oncology . This conversation has been substantially edited for length and clarity. Lei Lei Wu: Jeff, I’ll start with you. Not too long ago, we met to speak about Sonoma’s first clinical data. How long has this transition been in the works for? And why now? Jeff Bluestone: We needed to go find a great CEO. It took me a little bit more than 24 hours to do that, so we started the search a little while ago. But, very excited to have Stephen come on board. I just felt it was the right time to turn the reins over, because I think the company is a really great place right now. Someone emailed me this morning and said it’s a little bit like [Los Angeles Dodgers Hall of Famer] Sandy Koufax . In his last year, he was an All-Star pitcher, shutouts every day. And I figured, might as well leave on a good note. Plus my grandkids, my wife and the rest of my life had a big impact as well. Wu: Are you a Dodgers fan? Bluestone: No, not at all. My father grew up a New York Giants fan, and we hated the Yankees and we hated the Dodgers. Wu: What’s next for you? Bluestone: It’s hard to use the word “retire.” It feels like a definitive ending, which it won’t be. I’m really lucky that Stephen is interested in having me continue to advise the company, which I will, and I’ll be on the board. Aside from that, I’m on the Gilead board, which I’ll continue to do, and then I want to spend some time thinking about how I can help young people. The idea of being able to help young student scientists is exciting to me. I’ll probably go back to UCSF for journal clubs and lab meetings and the like. And then finally, just do some of the things in life that I sacrificed over these many years. Wu: Stephen, turning to you now. What made you interested in this role? Stephen Dilly: One of the things that I believe in very strongly is that people are ideally suited to stages of journeys. What Jeff has done over the last six years at Sonoma, something that I would not be able to do, is take basic science and incredible insights and turn that from a concept into something that’s ready for going into people. I’m a drug developer. Dyed-in-the-wool drug developer. It’s a different thought process. The research process is inclusive and exploratory. The development process is very targeted. A lot of it is about the way you look at challenges, the way you address them, the way you move forward. And this company is absolutely in my sweet spot. A lot of the raw material is here to do something quite remarkable. — Lei Lei Wu → T cell receptor specialist Adaptimmune announced on Monday that its CEO of six years, Adrian Rawcliffe , left “in connection with the company’s restructuring.” It’s the end of an era for Adaptimmune, which won approval last year for the first cell therapy based on T cell receptors. Known as Tecelra , the therapy was approved for a rare cancer called synovial sarcoma. The news comes after Adaptimmune sold Tecelra , among other assets, and laid off nearly two-thirds of its workforce. Most of the company’s executives departed with the layoffs, and Rawcliffe is the latest to do so. Chris Hill is now CEO as well as CFO. And the company’s longtime COO William Bertrand also left “in connection with the restructuring,” Adaptimmune said Monday . → Former FDA Commissioner Scott Gottlieb has joined UnitedHealth Group ’s board, the massive health insurer said Tuesday. Gottlieb also sits on the boards of Pfizer and Illumina . The move comes as UnitedHealth has faced increasing pressure on its business with higher medical costs and scrutiny over insurance denials , as well as a ransomware attack on its Change Healthcare business. Former CEO Andrew Witty stepped down in May and was replaced by longtime UnitedHealth chief Stephen Hemsley . Its stock is down 38% this year. “We welcome his deep expertise and thought leadership as we strive to help people live healthier lives and make the health system work better for everyone,” Hemsley said in a news release . → Juby Chandy will have new responsibilities at GSK on Dec. 1: The current GSK India CFO has been named VP of finance for the Asia Pacific region. Scrolling is required to go through Chandy’s 17-year career at GSK, where he has also served as area finance director for Southeast Asia and head of finance, general medicines, global pharma, among other roles. In October, Emma Walmsley conducted her last quarterly report at GSK before commercial chief Luke Miels takes over as CEO. → Philina Lee will be the next CEO at Sydney-based cancer biotech AdvanCell on Jan. 1. She’ll replace Andrew Adamovich , who will have a new role as managing director, Australia. Lee will take over a company that just stocked up on cash with a $112 million Series C in February. She had an 11-year run at Blueprint Medicines and was chief commercial officer until Sanofi bought the Ayvakit maker for more than $9 billion this year. (Incidentally, her former boss Kate Haviland said on LinkedIn this week that she’ll “continue contributing to the life sciences ecosystem” as a senior advisor at Jefferies .) AdvanCell’s lead program 212Pb-ADVC001 is a PSMA-targeting radioligand for patients with prostate cancer. “Philina’s leadership further strengthens our ability to establish the United States as a global hub while continuing to deliver innovation from Australia and cementing our position as a leader in targeted alpha radiotherapies,” Adamovich said in a statement . → At another Australian drugmaker, Mesoblast has tapped Bristol Myers Squibb and Allergan vet James O’Brien as CFO. O’Brien will be based in the US and was CFO at Cognition Therapeutics (2019-23) and Faron Pharmaceuticals (2023-24). Mesoblast’s head of finance Andrew Chaponnel had been interim finance chief since Josh Muntner hit the exit in 2021. At long last, the FDA approved Mesoblast’s mesenchymal stromal cell therapy Ryoncil in December 2024. → Gilead general counsel Deborah Telman will leave on Dec. 5, according to a terse SEC filing . Telman replaced Brett Pletcher as EVP, corporate affairs and general counsel in August 2022. Gilead did not provide any details about Telman’s departure. → As part of a restructuring, Nxera Pharma is trimming its leadership team, going from 10 execs to seven by March 2026 in a bid to cut operating expenses. Nxera is also pruning its workforce by about 15% across Japan and the UK, and focusing its sights on obesity, metabolic and endocrine therapies. While the company is cutting jobs elsewhere, it has recruited Patrik Foerch as CSO and president of Nxera Pharma UK , taking over the reins from Matthew Barnes . Foerch was CSO of Peptone , ran preclinical drug discovery at Sitryx , and had a 13-year stint at UCB . Nxera hopes that the cost-cutting measures will help the company reach its net sales forecast of 50 billion yen in 2030. → With ex- NGM Bio CEO David Woodhouse now at the controls, immunomodulatory upstart Nuvig Therapeutics has welcomed Celia Lin as CMO. Lin joined Santa Ana Bio in the same capacity shortly after the Versant biotech’s $125 million Series B last summer, but she stepped down nine months later. Peer Review covered Lin’s appointment as CMO of Vera Therapeutics and her departure in early 2024, when she was replaced by Robert Brenner . Nuvig disclosed a nine-figure Series B of its own last year and is evaluating NVG-2089 in chronic inflammatory demyelinating polyneuropathy (CIDP) and immune thrombocytopenia. → Paolo Tombesi is retiring after CFO posts at multiple drugmakers over the past 25 years. He’s handing the job at Athena Countouriotis ’ Avenzo Therapeutics to Scott Lipman , who was promoted to CBO last year and will retain both duties. Tombesi helped Countouriotis steer their prior biotech, Turning Point Therapeutics , toward a multibillion-dollar exit to Bristol Myers Squibb . Along with Lipman, they reunited for Avenzo, raising more than $440 million over the past few years for small molecules and antibody-drug conjugates licensed from Chinese partners. Prior to that, Tombesi built his CFO chops at Epizyme and Insmed after holding regional CFO posts at Novartis and Bristol Myers. → 4D Molecular Therapeutics has found a new CFO after Uneek Mehra ’s departure in July. Kristian Humer held this job title at Foghorn Therapeutics and Viridian Therapeutics after 11 years with the healthcare investment banking group at Citi . Mehra took the CFO job at BridgeBio Oncology Therapeutics , or BBOT. Shares $FDMT surged this summer when 4DMT notched a Phase 2 win with its gene therapy 4D-150 for diabetic macular edema, and there’s been an upward trend ever since. → Andy Nichols announced on LinkedIn that he’s “finally pulling the plug and disconnecting from corporate life, at least for a while,” after 40 years in the industry and two years at Jazz Pharmaceuticals as SVP of research, nonclinical development and clinical pharmacology. By the way, Zymeworks has named EcoR1 Capital partner Scott Platshon as acting chief investment officer. Jazz and Zymeworks outlined a Phase 3 win this week with their HER2 bispecific Ziihera in gastroesophageal adenocarcinoma, and their stock prices popped as a result. → Abraxane developer José Iglesias ’ first day as chief medical affairs officer at Agenus was Nov. 10, according to a Tuesday press release . Iglesias had a brief turn as CMO at Tim Lu ’s Senti Bio in 2020, and he reemerged as consulting medical chief for Panavance Therapeutics . Agenus is combining its anti-CTLA-4 antibody botensilimab with its PD-1 candidate balstilimab . Known colloquially as BOT/BAL , the combo is in Phase 3 for microsatellite-stable colorectal cancer. → French ADC maker Adcytherix has hired a pair of execs after a host of investors participated in its €105 million Series A . Medical chief Jan Schellens just held the same position at another ADC developer in France called Skymab Biotherapeutics , and he was CMO of Dutch biotech Byondis from 2019-23. Finance chief Jaime Arango had multiple roles over a nine-period at Merck , and he’s been CFO at Medincell and TreeFrog Therapeutics . → Eduardo Dunayevich will help steer the clinical direction of San Francisco-based Neurona Therapeutics , the CNS cell therapy startup that nabbed $ 102 million earlier this year for its epilepsy program. The new CMO was VP of clinical development and leader of the VMAT2 franchise at Neurocrine Biosciences . Dunayevich has been in industry for a quarter-century, making stops at Eli Lilly , Orexigen Therapeutics , Amgen , Takeda , Annexon Biosciences and Greenwich Biosciences along the way. → Elicio Therapeutics , co-founded by former MIT professor Darrell Irvine , has selected Marc Wolfgang as chief technology officer. Wolfgang just finished a stint as SVP of technical development and operations over at Flagship ’s Sail Biomedicines , which cut 30% of its staff in July. Wolfgang’s résumé also includes gigs at BioNTech , Cerulean Pharma , Momenta Pharmaceuticals , Millennium Pharmaceuticals , Biogen and Boehringer Ingelheim . → Kameel Farag helped set up cell therapy maker Aspen Neuroscience for a potential IPO (with a $115 million Series C disclosed Thursday). Now, a few months after leaving the CFO post at Aspen, he’s returning to the role on an interim basis at Akari Therapeutics . Farag built up his finance acumen during a 16-year career at Amgen and a three-year stint at Ionis . Akari has two ADC programs in preclinical development. → After bagging Y-mAbs in a $412 million all-cash deal , specialty drugmaker SERB Pharmaceuticals is bringing its CMO into the fold. Vignesh Rajah will serve in the same role of medical chief at SERB. Besides Y-mAbs, Rajah had roles at Sanofi and Wyeth , as well as a decade-long career at GSK. SERB picked up Y-mAbs and its neuroblastoma treatment Danyelza in August, hoping to further expand upon its own portfolio of more than 70 drugs. → Mosaic Therapeutics CEO Thomas Fuchs is already making an impact in his first month on the job, bringing in Samsara BioCapital venture partner Stephen Shuttleworth as CSO. AstraZeneca vet Barry Davies is retiring from the position and will be “on extended gardening and travel leave,” according to his LinkedIn. Shuttleworth was CSO, COO and executive director at Karus Therapeutics , and he chaired the scientific advisory board at the end of his 12-year career with the company. → To the Dallas metro area we go, where OncoNano Medicine has lassoed Brent Sorrells as COO. Sorrells spent eight years with Allakos , where he was promoted to VP of product development and strategy in mid-2024. Concentra Biosciences scooped up the biotech earlier this year, setting off a chain reaction of similar acquisitions for Kevin Tang . OncoNano is developing a STING agonist called ONM-501 for solid tumors. → Edgewise Therapeutics has welcomed Chris Martin to the board of directors, following the appointment of CFO Michael Nofi . Martin helped launch the COPD treatment Ohtuvayre as chief commercial officer at Verona Pharma . Merck bought Verona this summer for $10 billion. Lydia Ramsey Pflanzer contributed to this edition.

Executive ChangeDrug ApprovalAcquisition

100 Deals associated with Afamitresgene Autoleucel

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Synovial Sarcoma	United States	Adaptimmune LLC	02 Aug 2024

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Esophageal Carcinoma	Phase 3	United Kingdom	Adaptimmune Ltd.	30 Jan 2022
Myxoid Liposarcoma	Phase 3	United Kingdom	Adaptimmune Ltd.	30 Jan 2022
Ovarian Cancer	Phase 3	United Kingdom	Adaptimmune Ltd.	30 Jan 2022
Stomach Cancer	Phase 2	Spain	-	31 Jul 2021
Squamous Cell Carcinoma of Head and Neck	Phase 2	United States	Adaptimmune Ltd.	02 Jul 2020
Malignant neoplasm of gastro-oesophageal junction	Phase 1	United Kingdom	Adaptimmune Ltd.	30 Jan 2022
Bladder Cancer	Phase 1	United States	-	15 May 2017
Bladder Cancer	Phase 1	Canada	-	15 May 2017
MAGEA4-Positive Solid tumor	Phase 1	United States	-	15 May 2017
MAGEA4-Positive Solid tumor	Phase 1	Canada	-	15 May 2017

Clinical Result

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04044768 (SPEARHEAD-1, FDA_CBER) Manual	Phase 2	Synovial Sarcoma HLA-A02:01P \| HLA-A02:02P \| HLA-A*02:03P ... View more	44	TECELRA	dnjjajotyl(phofkvadbq) = omitcztaeg ilfbxbjdjk (afifspokbu, 28.4 - 59.0) View more	Positive	02 Aug 2024
NCT04044768 (SPEARHEAD-1, Literature \| Pubmed \| Lancet) Manual	Phase 2	Synovial Sarcoma \| Myxoid Liposarcoma	52	Afamitresgene autoleucel	qfoeobqwxy(uhonweltlw) = mvhwocbxhu czhvjldjna (xiunbkhfzz, 24–51) View more	Positive	27 Mar 2024
	Phase 2	Synovial Sarcoma \| Myxoid Liposarcoma	52	Afamitresgene autoleucel (synovial sarcoma)	qfoeobqwxy(uhonweltlw) = xmpmdvhizj czhvjldjna (xiunbkhfzz, 24–55)	Positive	27 Mar 2024
NCT04044768 (SPEARHEAD-1, ESMO_SRC2024) Manual	Phase 2	Synovial Sarcoma \| Myxoid Liposarcoma	52	Afamitresgene autoleucel	ivyhmvvicp(abcmcngsoa) = irsegqoqqc txhvjbqxgk (ktuciugloe ) View more	Positive	14 Mar 2024
NCT04044768 \| NCT03132922 (SPEARHEAD-1, Biospace) Manual	Not Applicable	Synovial Sarcoma	-	Afami cel	nihpmqmxpz(sfepqtpukv) = iabuvxgpzj gqnnyjmkpx (zeyuddnglz ) View more	Positive	06 Dec 2023
NCT04044768 (SPEARHEAD-1, ASCO 12023 \| ASCO 22023) Manual	Phase 2	Synovial Sarcoma	44	Afamitresgene Autoleucel (Cohort 1)	znbfhiubbp(vdzhwgfqdu) = cjnqzbhraw tjcrukoqzq (eqipzvxgzo, 2.8 - 5.8) View more	Positive	31 May 2023
NCT04044768 (SPEARHEAD-1, ASCO 12023 \| ASCO 22023) Manual	Phase 2	Synovial Sarcoma	72	Afamitresgene Autoleucel	vcazudulap(vjkvodkbhp) = fcamhmbkxe cwilvuzesw (thncxbewui ) View more	Positive	26 May 2023
NCT03132922 (SPEARHEAD-1, ASGCT2023)	Phase 1	MAGE-A4	-	Afami-cel	xkslvskbqp(sfyrocjpny) = nixqufnznq wfwdjnrhlh (rkbjiljbmg ) View more	Positive	01 May 2023
NCT03132922 (Pubmed \| Nat Med) Manual	Phase 1	MAGEA4-Positive Solid tumor \| Ovarian Cancer \| Synovial Sarcoma ... HLA-A*0201 Positive \| MAGEA4 Positive View more	38	Afamitresgene autoleucel	hyxfwqpvaw(gztauygsjq) = All patients (N = 38, nine tumor types) experienced Grade ≥3 hematologic toxicities; 55% of patients (90% Grade ≤2) experienced cytokine release syndrome. rthtdvjfac (lteremnirb )	Positive	01 Jan 2023
NCT03132922 (Pubmed \| Nat Med) Manual	Phase 1		38	Afamitresgene autoleucel (synovial sarcoma)		Positive	01 Jan 2023
NCT04044768 \| NCT03132922 (SPEARHEAD-1, ASCO2022)	Phase 1/2	Synovial Sarcoma \| Myxoid Liposarcoma HLA-A*02+ \| MAGE-A4+	69	Afami-cel	pdbyrrwvts(jmrpjlkzgu) = nieymjqhvj tmecdvturx (xmvxekukqx ) View more	-	02 Jun 2022
NCT04044768 (SPEARHEAD-1, ASCO 12021 \| ASCO 22021) Manual	Phase 2	Synovial Sarcoma \| Myxoid Liposarcoma HLA-A*02 Positive	32	afamitresgene autoleucel	jvhbwqpezd(tckssewovn) = cqhjhqatrf sufrozznqa (ymfnalcdoa ) View more	Positive	28 May 2021

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