Fidanacogene elaparvovec

Takeda\'s oncology leaders are focused on building off their own \"institutional knowledge.\"\n Takeda’s revamped R&D strategy means the drugmaker’s oncology leaders are prioritizing work in tumors across thoracic, gastrointestinal (GI) and hematology, while pushing the Japanese company’s cell therapy work focus to the back burner.“We\'re not doing this in a prescriptive way that would never consider anything outside of that,” Teresa Bitetti, president of Takeda’s global oncology business unit, told Fierce Biotech. “But we are looking at areas that we\'ve defined because of the white space, in terms of the unmet need, because of where the science is moving and where our strength lies.”The oncology approach meshes with the drugmaker’s new overall strategy, P.K. Morrow, M.D., head of Takeda’s oncology therapeutic area unit, told Fierce in a joint interview.In January, Takeda\'s R&D head Andy Plump, M.D., Ph.D., explained that the pharma had narrowed its focus down from 10 or 12 modalities to just four: small molecules, biologics, antibody-drug conjugates (ADCs) and allogeneic cell therapies.  Of those core priorities, Takeda’s oncology leaders are taking a deep interest in ADCs, small molecules and complex biologics, Morrow said. So, everything but cell therapy.“It\'s unlikely we\'re going to do any cell therapy deals in the near future,” she said about the fourth modality.“Anytime we\'re dealing with gene therapy or cell therapy, there\'s the potential risk for a durable negative effect,” said Morrow, who joined Takeda last year after serving as Crispr Therapeutics’ chief medical officer. “It doesn\'t happen most of the time, but there\'s always the potential, right?”To protect the patient, cell and gene therapies often require extended follow-up, Morrow explained, noting that the component is an important consideration in drug development.   The shift isn’t shocking—Bitetti told Fierce that Takeda wasn’t “actively pursuing” autologous cell therapies at last year’s American Society of Clinical Oncology (ASCO) conference.The sentiment shadows a broader industry trend as cell and gene companies fight to remain relevant. Most recently, Pfizer discontinued hemophilia product Beqvez, emptying its gene therapy portfolio, while bluebird bio sold for just $29 million after having at one time carried a value of $10 billion.Back at Takeda, the team still touts GDX012, also called TAK-012-1501, an allogeneic gamma delta T cell therapy the company gained from buying GammaDelta Therapeutics in 2021. The asset is currently in phase 1/2 testing for adults with acute myeloid leukemia (AML).The team will “continue for now” to pursue early-stage work in cell therapy, Bitetti said.After a wide-ranging $900 million restructure in 2024, Takeda now touts its “most robust late-stage pipeline” in the pharma’s history, consisting of six late-stage programs across 14 studies, Plump told Fierce at the beginning of the year.Developing a more mature pipeline meant that Takeda had to raise its standards, Plump said. Lifting the bar resulted in several programs being left behind, including a midstage CD19 CAR-NK cell therapy in B-cell malignancies. The asset is still being studied in a phase 1 autoimmune disease program.Momentum amid streamlining?  “We did do some streamlining, but I sort of feel like we\'ve come out of the back end with a lot of energy [and] focus,” Bitetti said. “I don\'t feel like we\'ve lost momentum with it.”The restructure included implementing a more rigorous approach when considering benefit and safety thresholds that determine what programs should progress, according to Morrow.    One of those programs is rusfertide, an injectable hepcidin mimetic designed to treat a rare chronic blood cancer called polycythemia vera (PV). Last year, Takeda paid out $300 million to license the asset from Protagonist Therapeutics.Just this month, the partners reported a phase 3 win, finding that rusfertide reduced blood procedures among patients with PV.Takeda’s plans to file for approval with regulatory authorities are currently “on track,” with a submission expected toward the end of this year, according to Bitetti.If rusfertide passes muster with regulators, the hepcidin mimetic peptide could go on to generate $1 billion to $2 billion in peak sales. The candidate is one of six pipeline drugs that Takeda is hoping will collectively bring in between $10 billion and $20 billion, CEO Christophe Weber said during the company’s J.P. Morgan presentation in January.Takeda isn\'t counting out China When asked about geopolitical changes, such as the BIOSECURE Act and President Donald Trump’s “America First Investment Policy,” Bitetti said Takeda isn’t counting out science from China.“One can\'t afford to ignore the innovation that\'s available today coming out of China,” she said. “It\'s not just me-toos that are coming out of China—there\'s some really interesting science.”Bitetti also cited the country’s rapid pace and ability to get the proof of concept very quickly.While she noted that companies still need to be “judicious” given the changing dynamics, she said one should “proceed at your own risk to ignore what\'s happening in China.”Meanwhile, Morrow pointed to the Japanese drugmaker’s successful partnership with Hong Kong-based Hutchmed, in which Takeda paid $400 million upfront for commercial rights outside of China to a colorectal cancer drug called fruquintinib. The oral vascular endothelial growth factor receptors (VEGFR) 1/2/3 tyrosine kinase inhibitor received FDA approval at the end of 2023 and is now marketed as Fruzaqla. Last summer, Takeda also inked a deal worth up to $1.3 billion biobucks that provides the opportunity to license Ascentage Pharma’s olverembatinib, which is being developed for chronic myeloid leukemia (CML), along with other hematological cancers. The Chinese biotech\'s third-generation BCR-ABL tyrosine kinase inhibitor is currently approved in China for certain patients with CML.As for future deals with biotechs, the leaders are looking for validated modalities in disease areas Takeda believes it can make the greatest impact in.“That\'s what you\'ll see in particular in the deals that we\'ve done in the very recent past,” Morrow said, pointing to a December deal with Keros Therapeutics that included $200 million upfront to secure ex-China rights to a molecule that could compete with Bristol Myers Squibb’s Reblozyl in blood cancer indications.The deal covers Keros’ activin inhibitor elritercept that’s in development to treat anemia tied to blood cancers such as myelodysplastic syndromes and myelofibrosis. Keros sees elritercept as a molecule that can act on all stages of platelet and red blood cell production, leading it to identify opportunities across a broad patient population.Building on strengths Takeda’s pipeline decisions also take the company’s strengths into account, Bitetti said.“We have an enormous strength on the [hematology] side,” she explained. “We have very deep relationships that go back a very long time from the early days of Velcade.”The “institutional knowledge” regarding myeloid cancers and commercializing drugs in the area is something Takeda wants to continue to build on.This is exemplified by Takeda’s purchase of oncology biotech Millennium way back in 2008, according to Morrow.The “Millennium legacy” is vital to the oncology unit because it helped Takeda better understand how to develop therapies that could become backbones to other regimens and helped draw in key talent that has expertise in precision medicine. When asked how diversity, equity and inclusion play a role in the department’s hiring practices, Bitetti said DEI considerations continue to be important.“Our position as a company hasn’t changed on diversity, in terms of doing what we think is right for the business and ensuring that we have the right breadth of perspective, not only in our clinical trials, but also how we manage the business,” Bitetti said.  

Pfizer is ending the development and commercialization of its hemophilia B gene therapy Beqvez, the company confirmed in an emailed statement to Endpoints News . The company cited “limited interest patients and their doctors have demonstrated in hemophilia gene therapies to date.” It marks an abrupt end for the gene therapy for the rare, inherited bleeding disorder. Pfizer gained FDA approval for the one-time treatment last April, and the New York pharma giant rolled it out at a price of $3.5 million. The drugmaker didn’t disclose any updates on the gene therapy during its recent earnings. A decade ago, it had licensed Beqvez from Spark Therapeutics, which is now owned by Roche. Kim Phelan, CEO of the Coalition for Hemophilia B, said the nonprofit was “disappointed to hear of this discontinuation,” but that they “respect Pfizer’s decision as they evaluate their portfolio and future direction.” “Importantly, we remain hopeful and reassured that individuals with hemophilia B still have access to an approved gene therapy option,” Phelan said in a statement, referring to CSL Behring’s Hemgenix. CSL has struggled to gain traction for the multimillion-dollar treatment. The pullback casts a new pall over the blood disorder landscape. Two months ago, Pfizer axed its partnership on another gene therapy, a hemophilia A treatment from Sangamo Therapeutics that cleared a Phase 3 last summer. The drugmaker also pulled another blood disorder drug , the sickle cell treatment Oxbryta, last fall after saying the “totality of clinical data” showed the overall benefit of the drug “no longer outweighs the risk.” “We will communicate to those Beqvez patients who are in the qualification process and their providers who have initiated the qualification process and follow their desired pathway through our decision,” the Pfizer spokesperson said in an email. “For any patient who has been treated with Beqvez in any clinical trial, Pfizer remains committed to supporting the clinical trial sites and patients with regard to clinical trial follow-up and patient and trial support.” Pfizer markets another drug for hemophilia A and B. In October, the FDA greenlit the company’s once-weekly injectable, branded as Hympavzi .