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The Oncology Pipeline Overview: Recent Approvals and Near-Term Drugs

JHOP - December 2021 Vol 11, No 6 - AVBCC 2021 Highlights
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Many innovative therapies in oncology have made it to market recently, with a wealth of agents that are currently on the verge of US Food and Drug Administration (FDA) approval. At the 2021 Summit of the Association for Value-Based Cancer Care, a panel of oncology drugs experts moderated by Doug Long, MBA, Vice President of Industry Relations at IQVIA, discussed the current pharmaceutical trends in oncology drug development, including recent drug approvals in oncology, and the near-term innovative agents coming to market in the near future, noting that 75% of pharmaceutical research and development is focused on oncology.

The US oncology therapeutics market has reached $71 billion, according to Mr Long. Although COVID-19 has slowed the growth of drug sales, the total growth in the oncology market is being driven by the targeted drugs in oncology, he emphasized. COVID-19 has not had an impact on the number of drug launches in the oncology space, he said, and 2020 and 2021 have seen similar rates of drug launches in oncology as was seen in 2019.

Dipti Shah, PharmD, CSP, Senior Clinical Program Manager, CVS Specialty, provided an overview of the oncology drug landscape, highlighting a handful of especially innovative pharmaceuticals, some in niche areas.

Recently Approved Cancer Drugs

In 2021, tivozanib (Fotivda) received FDA approval for the treatment of patients with relapsed or refractory renal-cell carcinoma. Although an improvement in progression-free survival was demonstrated with tivozanib compared with sorafenib (Nexavar) in patients with advanced renal-cell carcinoma, overall survival data favored sorafenib, Dr Shah observed.

Umbralisib (Ukoniq) was granted FDA approval for the treatment of marginal-zone lymphoma. Approximately 7500 patients annually are diagnosed with marginal-zone lymphoma in the United States. “An additional therapy in this space is going to give patients choice, especially with its good side effect profile in clinical trials,” she said.

Tepotinib (Tepmekto) gained approval for the treatment of metastatic non–small-cell lung cancer (NSCLC) harboring MET exon 14 skipping alterations. Only approximately 3% to 4% of patients with NSCLC have such alterations, but their prognosis is poor, Dr Shah said.

Lisocabtagene maraleucel (Breyanzi) is a CAR T-cell genetically modified therapy that was approved by the FDA for the treatment of patients with relapsed or refractory large B-cell lymphoma. In the single-arm, open-label TRANSCEND clinical trial conducted in patients with relapsed or refractory B-cell lymphoma, the overall response rate with lisocabtagene maraleucel was 73%. Of the 104 patients who had a complete response, 65% had remission lasting ≥6 months, and 62% had remission lasting ≥9 months.

The 10-year follow-up of these patients will be very important, and will ultimately be the driver of value behind these therapies, said panelist Cheryl Allen, PharmD, Executive Vice President, Industry Relations and Business Development at Asembia.

Sotorasib (Lumakras) is an oral therapy that targets the KRAS gene; it is the first drug to receive FDA approval for any malignancy associated with KRAS; it was approved for the treatment of patients with NSCLC and a KRAS G12C mutation. “It is important to note that KRAS has multiple biomarkers within itself,” said Dr Shah. “Only those patients with a KRAS G12C mutation will respond to this therapy.” This type of mutation represents less than 3% of patients with NSCLC, she said.

Mobocertinib (Exkivity) is an oral tyrosine kinase inhibitor that was approved for the treatment of metastatic NSCLC and EGFR exon 20 insertion mutation. EGFR exon 20 insertion mutations occur in 4% to 12% of patients with NSCLC and EGFR mutation, and approximately 2% of all patients with NSCLC.

The Near-Term Drug Pipeline

Dr Shah highlighted 3 novel therapies in oncology that are expected to gain FDA approval before the end of 2021 or in 2022—pacritinib, ublituximab, and magrolimab—as well as the immunotherapy nivolumab (Opdivo), which is expected to receive yet another new indication in the near future.

Pacritinib is an oral macrocyclic JAK inhibitor that is being developed for the treatment of myelofibrosis, and may represent a good option in patients with severe thrombocytopenia, Dr Shah said. Its unique mechanism of action, inhibiting JAK2 and IRAK1 without inhibiting JAK1, may reduce the risk of cytopenias as well as immune dysfunction associated with other JAK inhibitors. It was granted a priority review by the FDA in June 2021, and final approval is expected before the end of 2021.

A biologics license application was accepted by the FDA for the CD20-directed monoclonal antibody ublituximab (to be used in combination with umbralisib) for the treatment of patients with chronic lymphocytic leukemia or with small lymphocytic leukemia. Expected approval is March 2022.

An application for the use of nivolumab in combination with ipilimumab (Yervoy) or with chemotherapy in the first-line setting for patients with unresectable advanced, recurrent, or metastatic esophageal squamous-cell carcinoma was accepted by the FDA. This application was based on results from the pivotal phase 3 CheckMate-648 clinical trial, in which the combination of nivolumab plus ipilimumab and nivolumab plus chemotherapy demonstrated a significant and clinically meaningful benefit in overall survival compared with chemotherapy alone in patients with unresectable, advanced or metastatic esophageal squamous-cell carcinoma associated with PD-L1 expression of ≥1%, as well as in the all-randomized patient population. FDA approval is expected in May 2022.

Magrolimab is an anti-CD47 monoclonal antibody that is being studied in combination with azacitidine (Onureg, which was approved in 2021 for myelodysplastic syndrome) as first-line combination regimen for the treatment of patients with intermediate-, high-, or very high-risk myelodysplastic syndrome. In a phase 1b clinical trial, this combination showed a complete response rate of 42% versus 20% with azacitidine monotherapy. FDA approval is expected in the third quarter of 2022.

Unmet Need a Huge Burden

“Despite amazing new drugs, some patients aren’t benefiting sufficiently or at all. There’s still tremendous unmet need in oncology, which is why the huge pipeline of activity,” said Jeffrey Bockman, PhD, Executive Vice President, Oncology Practice Head, Cello Health BioConsulting. That oncology pipeline is filled with a plethora of non-immunotherapy agents, but immune-oncology and precision medicine are driving the pipeline value, Dr Bockman said.

Clinical benefit—or value—and not simply price, is becoming the focus even for early-stage drug developers, said Dr Bockman, especially for multicomponent high-priced immunotherapy regimens.

Dr Allen made a case for value-based oncology determinations with the increase in innovation.

Right now, much of the cost of therapy is the payers’ burden, she said, and the patient is generally not involved. Financial support from the manufacturer is usually limited to commercially insured patients. To make better decisions, with the increasing competition in the oncology space, “head-to-head studies are needed to see what this will do ultimately for the payer base,” Dr Allen said.

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