ASH 2020: Gilead eyes new Yescarta indication, Vertex/CRISPR make headway in sickle cell and more – PMLiVE

Posted: December 12, 2020 at 7:59 pm

It was a bumper year at the annual American Society of Hematology (ASH) meeting, with a slew of new data presented across the board. Here is a round-up of some of the key data presented.

Gilead eyes new first-line indication for CAR T Yescarta

Gileads Kite division presented new phase 2 data for its CAR T therapy Yescarta at ASH 2020 in relapsed or refractory high-risk large B-cell lymphoma (LBCL), a potential new indication.

In the phase 2 ZUMA-12 study, after a single infusion of Yescarta (axicabtagene ciloleucel), 85% of LBCL patients achieved a response, while 74% of patients achieved a complete response.

After a median follow-up of 9.5 months, the median progression-free survival, median overall survival and median duration of response were not yet reached.

Yescarta has already presented four-year survival data in patients with third-line refractory LBCL and we are now excited for what these ZUMA-12 results signal for its potential in earlier lines of treatment, said Ken Takeshita, global head of clinical development, Kite.

Yescarta was first approved by the US Food and Drug Administration (FDA) for the treatment of adult patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy.

Eli Lilly and Loxos BTK inhibitor shows blood cancer promise

Eli Lillys Loxo Oncology revealed some promising early-stage results for its Brutons tyrosine kinase (BTK) inhibitor LOXO-305 in patients with chronic lymphocytic leukaemia (CLL) and small lymphocytic lymphoma (SLL).

LOXO-305, an oral BTK inhibitor, is designed to address acquired resistance to currently available BTK inhibitors, such as J&Js Imbruvica.

In the phase 1/2 study of the BTK inhibitor, out of 139 CLL/SLL patients who were efficacy-evaluable, 88 responded to treatment, with 19 partial responses with ongoing lymphocytosis, 45 with stable disease and one with progressive disease.

In a subgroup of patients previously treated with a BTK inhibitor, the overall response rate was 62%, which increased to 84% for those with ten months or more follow-up.

It is important to note that the patients included in this study were heavily pre-treated, with all CLL/SLL patients having received a median of three prior lines of therapy.

Vertex/CRISPR Therapeutics make headway in sickle cell and beta thalassaemia

Also presenting data at ASH were Vertex and CRISPR Therapeutics, whose gene-editing therapy CTX001 scored some promising results in transfusion-dependent beta thalassaemia (TDT) and sickle cell disease (SCD) patients.

The CRISPR/Cas9-based gene therapy was investigated in a total of ten patients, seven of which had TDT and three with SCD.

All TDT patients treated with CTX001 were transfusion independent at last follow-up and all SCD patients were free of vaso-occlusive crises (VOCs), the companies announced at the meeting.

These are the first published results from CRISPR/Cas9 therapy in people with a genetic disease and represent an important milestone in medicine and for our collaboration with CRISPR Therapeutics, said Reshma Kewalramani, chief executive officer and president, Vertex.

Most importantly, this data represents a critical step in our effort to bring transformative and potentially curative therapies to patients, he added.

bluebird bios beta thalassaemia gene therapy Zynteglo scores promising long-term data

bluebird bio also posted long-term data for its own beta thalassaemia gene therapy Zynteglo (betibeglogene autotemcel) at ASH 2020.

The new data reflects up to six years of safety and efficacy data for the gene therapy in patients with TDT.

Among 32 patients enrolled in the 13-year, long-term LTF-303 study, 64% of patients treated in the phase 1/2 portion and 90% in the phase 2 portion achieved transfusion independence (TI).

In the LTF-303 study, all patients who achieved TI remained free from transfusions, with the median duration of ongoing TI coming in at 39.4 months.

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ASH 2020: Gilead eyes new Yescarta indication, Vertex/CRISPR make headway in sickle cell and more - PMLiVE

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