Innovative phase 2 trial targets advanced liver cancer



The UCL study will evaluate the safety and effectiveness of MTL-CEBPA in combination with second line standard of care sorafenib versus the use of sorafenib in isolation

The first patient has been dosed in a clinical trial evaluating the use of a new therapy for an advanced type of liver cancer. MTL-EBPA with sorafenib is being analysed with a view to treating advanced hepatocellular carcinoma (HCC).

HCC is the most common type of primary liver cancer in adults and is the third leading cause of cancer-related deaths worldwide. It is also the most common cause of death in people with end of stage liver disease.

The global phase 2 OUTREACH-2 clinical trial will evaluate the safety and effectiveness of MTL-CEBPA in combination with second line standard of care sorafenib, against sorafenib alone. Up to 150 patients will participate in the trial.

“Advanced liver cancer remains a significant unmet medical need, in particular for those patients who are resistant to frontline systematic therapy,” Professor Tim Meyer, Professor of Experimental Cancer Medicine at University College London and chief investigator of the study, commented.

“This combination treatment demonstrated intriguing signals of activity in a phase 1b trial, including durable and complete tumour responses. We believe that MTL-CEBPA’s immunological activity in the tumour microenvironment enables a greater effectiveness of sorafenib and we are excited to seek to validate those early findings in this phase 2 clinical trial,” he added.

As the first ever RNA therapy to enter the clinic, MTL-EBPA is being studied as a combination therapy in cancer. It works by reducing or removing one of the main defence mechanisms tumours use to resist attack by the immune system. This ensures the tumour can be attacked by tumour-targeting drugs as well as the immune system.

An RNA therapy is designed to correct the mutation in the RNA of someone with a genetic disease. In doing this, the RNA can be used to create missing proteins a cell needs and take away underlying causes of diseases.


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BenchSci raises $50m to grow AI-powered R&D platform –

Canadian firm BenchSci has secured around $50 million in third-round financing that will be used to speed up the adoption of its artificial intelligence-powered platform for biomedical research internationally.

The Toronto-based company already counts many of the world’s largest pharma companies among its customers, using its platform for a range of tasks such as improving reagent and antibody selection to help scientists run more successful experiments, drawing on data from published studies and organisations’ internal databases.

The cash injection will “expedite the expansion” of the BenchSci platform, which it says is already used by around 49,000 scientists at more than 4,500 research centres worldwide, including 16 of the top 20 pharma companies.

The company is tapping into longstanding concerns in the life science industry about R&D productivity. In the past decade drug developers have doubled their R&D spend, but the number of drugs getting approved has stayed roughly the same, so the cost of bringing a new medicine to market has been rising.

Revenue per drug has declined, and the result is that R&D returns have fallen by 80% since 2010, to 1.9% in 2018, according to Deloitte data.

BenchSci’s ambition is to tackle inefficiencies in preclinical scientific research, speeding up drug discovery efforts and reducing costs. Ultimately, it aims to trim 50% off the time it takes to bring a drug to market.

“We’re using breakthrough machine learning technology to shape the future of how life science companies conduct research, from identifying targets, to planning experiments, to determining clinical trial risks,” said Liran Belenzon, BenchSci’s chief executive.

The new funding round takes the total raised by the company to $97 million since it was set up in 201, and was led once again by Inovia, which has been investing in it since its series A in 2018, as well as TCV.

“We strongly believe that the preclinical R&D market remains largely untapped and that BenchSci can become a category-defining leader to bring life-saving drugs to market faster,” said Dennis Kavelman, partner at Inovia.


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FDA bans use of Lilly, Regeneron COVID drugs due to Omicron –

The FDA has limited the use of COVID-19 antibody therapies developed by Eli Lilly and Regeneron, on the back of data suggesting they have are “highly unlikely” to be effective against the now-dominant Omicron variant.

The emergency use authorisations (EUAs) for Lilly’s bamlanivimab/etesevimab combination and Regeneron’s REGEN-COV (casirivimab and imdevimab) issued earlier on in the pandemic – when other variants were more common – has been revised to reflect the loss of efficacy against the new strain.

“These treatments are not authorised for use in any US states, territories, and jurisdictions at this time,” said Patrizia Cavazzoni, director of the FDA’s Center for Drug Evaluation and Research (CDER).

“In the future, if patients in certain geographic regions are likely to be infected or exposed to a variant that is susceptible to these treatments, then use of these treatments may be authorised in these regions,” she added.

That effectively closes out use of the drugs, as it is not possible at the moment to tell if a newly-presenting patient has Omicron or another strain of SARS-CoV-2. Moreover, recent data from the Centres for Diseases Control and Prevention (CDC) estimated that almost all (99%) of new cases are being caused by Omicron.

It also switches off a source of sizeable windfall revenue for the two companies. Lilly recorded more than $1.1 billion sales for its COVID-19 antibodies in the first nine months of 2021, while Regeneron booked a whopping $4.7 billion from its drug in the same period.

“It’s highly unlikely that COVID-19 patients seeking care in the US at this time are infected with a variant other than Omicron,” said Cavazzoni.

Avoiding use of the Lilly and Regeneron antibodies “avoids exposing patients to side effects, such as injection site reactions or allergic reactions, which can be potentially serious, from specific treatment agents that are not expected to provide benefit,” she continued.

The block on the use of the two antibodies comes as other drugs are coming to the fore in the treatment of COVID-19, notably Pfizer’s oral antiviral Paxlovid (nirmatrelvir/ritonavir), GlaxoSmithKline/Vir Biotech’s antibody Xevudy (sotrovimab), Gilead’s intravenous antiviral Veklury (remdesivir) and Merck & Co/Ridgeback Bio’s Lagevrio (molnupiravir).

All of these are expected to work against Omicron in patients with mild-to-moderate COVID-19 who are at high risk for progression to severe disease, according to the FDA.

Last week, the US National Institutes of Health (NIH) recommended that patients with COVID-19 needing drug treatment receive the four drugs in the following order of priority: Paxlovid, Xevudy, Veklury, then Lagevrio.

It also said the Lilly and Regeneron antibodies should be avoided “because real-time testing to identify rare, non-Omicron variants is not routinely available.”


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Digital therapeutics: a new frontier of medicine

The area of digital therapeutics is growing rapidly, and in this piece Ben Hargreaves examines why this is the case, how broadly these therapies can be applied and why investment in the area is mounting.

To demonstrate how quickly the digital therapeutics space is developing, it is easiest to state that the first such therapy was approved in 2018 and since that point the annual investment in the space has more than doubled in the intervening years. During the opening three quarters of investment in 2021, more funding was raised than in the entirety of 2020, according to Rock Health. This equated to $21.3 billion of venture funding provided to companies working on digital therapeutics, compared with $14.7 billion during the entirety of 2020.

The pace of development is expected to keep growing, as further digital therapeutics receive approval and the benefits are demonstrated to all stakeholders. Already, this process is being accelerated by the impact of COVID-19 – as broader digital solutions became necessary in the treatment of patients globally.

A spokesperson for the Digital Therapeutics Alliance (DTA) explained how digital therapeutics are easier to deliver to patients in a situation, such as the pandemic: “Since most digital therapeutic interventions are delivered at least in part through patient-owned devices, few technical barriers exist to the implementation and scalability of [such] products. And these solutions can provide patients with continuous support and therapy when they are actively experiencing symptoms or are unable to immediately access their healthcare providers.”

As with many other aspects of life during the pandemic, digital technology has become even closer entwined with everyday life, and, according to the spokesperson for DTA, the demand for a digital solution to health problems is also increasing – not only from patients, but from clinicians and payers as well.

Developing a digital therapy

Despite the potential for digital therapeutic adoption, unlike digital technology companies that have experienced rapid growth during the pandemic, companies focused on such treatments have a development cycle that means overnight commercial success is unlikely. CEO of Pear Therapeutics, Corey McCann told pharmaphorum that the development process for a prescription digital therapeutic can take between three and five years. As the area of bringing a digital therapeutic to market means passing the same level of regulatory scrutiny as a traditional, pharmaceutical therapy, this means “prescription digital therapeutics must undergo extensive clinical trials to demonstrate their safety and effectiveness before they ever reach patients,” said McCann.

However, the digital element does make some aspects of development more streamlined compared to traditional treatments. For example, there is no need for in vivo animal or in vitro pre-clinical or non-clinical studies. In turn, this means that there is no translational risk associated with the work carried out by pharma and biotech companies, as they attempt to replicate positive results from animals into humans.

Another element that sets digital therapeutics apart was outlined by Ben Lewis, CEO of Limbix, a developer of prescription digital therapeutics, who told pharmaphorum that the company has a ‘structured process’ for engaging adolescents as ‘creative partners’ in creating product features. “In the development of SparkRx, Limbix leveraged significant input from a teen advisory council to test features and provide feedback to inform digital systems design for teens and young adults with depression,” added Lewis.

The wider team at Limbix also incorporates a broad range of professionals with different expertise areas. Lewis outlined that the overall design of its SparkRx prescription digital therapeutic, which is a therapy for teens and young adults experiencing symptoms of depression, involved healthcare providers, product designers, clinical researchers, and engineers.

The future use cases for digital therapies

One question that will be posed for digital therapeutics is how many different viable treatments areas could be helped or managed by such therapies. The spokesperson for DTA suggested that already digital therapeutics have found a niche in “addressing critical gaps…for underserved populations,” such as Limbix’s treatment for depression in adolescents – where there are few treatment options.

“We are hearing that healthcare organisations are looking to build models of care to cover a large number of therapeutic areas which would potentially include multiple digital therapeutic products to cover a number of diseases and disorders. This type of model would better help patients and clinicians manage expected co-morbid health conditions and would be well-positioned to address population needs, and integrate into clinician workflows, by matching patients with products to bridge the gap before and between clinic visits and empower them with the tools to feel more comfortable and confident in managing their condition,” the spokesperson continued.

For Pear Therapeutics, its pipeline is focused on psychiatric and neurological conditions, which are the company’s “two initial areas of focus to support our self-commercialisation strategy,” McCann explained. Wider than these two therapeutic areas, the company is also working to provide prescription digital therapies in specialty gastroenterology, oncology, and cardiovascular conditions, as well as for irritable bowel syndrome.

Why turn to digital therapeutics

Many of the advantages of digital therapeutics are self-evident due to the way they are delivered, in the form of a smartphone or device. All of the benefits associated with smartphones then are also applicable to this form of therapy: the treatments are easy to access, they offer convenience and privacy, and, since the devices are usually already owned, can be delivered at a lower cost. The ability for patients to engage with their own healthcare on an active basis and for the clinician to be able to follow the potential progress of the treatment should also lead to better health outcomes for those receiving digital therapeutics.

More than this, McCann referred to digital therapeutics’ potential to collect real world data as ‘unique’, in the possibility for improving the connection between patients and healthcare providers. “This patient monitoring capability afforded by prescription digital therapeutics may be the difference in a patient’s success during a course of treatment,” he said.

The next step in developing the potential of digital therapeutics will be continuing to establish a number of approved and successful treatments. For both Limbix and Pear Therapeutics, investors are seeing enough evidence of the potential to provide financial backing. In December 2021, both companies raised capital to invest in the development of therapies – with Limbix closing a $15 million series A2 financing round while Pear Therapeutics went public and raised $175 million.

With investment flowing into the sector, the likelihood of further treatments and companies emerging should see the area continue to grow. The potential benefits of this type of treatment are clear, though they will not be applicable to all treatment areas, there is the opportunity that in certain conditions, with an unmet need, they could offer solutions where currently none exist.

The post Digital therapeutics: a new frontier of medicine appeared first on .


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Team behind RECOVERY trial of COVID drugs casts its net wider –

The UK scientists that ran one of the largest trials of experimental COVID-19 drugs have formed a non-profit company that will apply the methodology to other disease areas – with $6.8 million in funding from French drugmaker Sanofi.

The non-profit – called Protas – is led by Sir Martin Landray,  professor of medicine and epidemiology at Oxford University and one of the chief investigators of the RECOVERY trial, which showed that dexamethasone was an effective treatment for COVD-19 and that hydroxychloroquine was not.

RECOVERY was unusual in that it tested several different therapies and had an adaptive design, so new drug candidates could be added to the protocol as needed. It also used a digital approach to patient recruitment, informed consent and randomisation, and enrolled tens of thousands of patients.

Now, that ‘smart trial’ approach will be used to find therapies for a host of other diseases, including common chronic conditions like heart and lung disease, arthritis, depression and dementia, according to Landray.

Sir Martin Landray

He told the BBC today that without a trial like RECOVERY it would not have been possible to establish the value of dexamethasone – an immune-suppressing steroid that some argued might actually be harmful to administer to COVID-19 patients.

“The pandemic isn’t special,” he said. “We have many common conditions that cause ill health for individual patients and real tress on health systems – heart disease, cancer, depression, dementia and so on – and we need better treatment for those conditions too.”

Those trials also need to be large, involve a diverse range of individuals, and able to be run at reduced cost, said Landray.

Protas will design and run smart clinical trials through collaborations with charities, foundations, academic research teams and industry partners, and will take on both repurposed and experimental drugs.

“The cost of developing a new drug is extortionately high,” said Landray, adding that a very large proportion of those costs is in phase 3, and that means many promising therapies may never be taken forward by pharma companies.

In fact, the economics for developing new treatments for chronic diseases is so challenging that fewer therapies are being developed for the conditions that place the greatest burden on patients and the health systems which care for them.

“It’s not about making money from the trials, it’s about driving public health benefit,” he told the BBC.

Sanofi’s chief medical officer Dietmar Berger said that the collaboration will give the company a way to significantly reduce the cost of some of its trials, and to focus on what matters the most for patients, doctors, regulators and payers.

“Protas offers a unique opportunity to anchor clinical research at the heart of patient care across the NHS, making participation as easy as possible and ensuring all health and care staff feel empowered to support research,” he added.

Protas will be building its organisation, technology and collaborations throughout 2022 and said it expects to begin designing its first clinical trials in 2023.


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AbbVie’s Skyrizi nabs psoriatic arthritis nod, its second, shortly after Rinvoq

After Rinvoq won two new indications in as many months, AbbVie’s immunology compatriot Skyrizi is getting in on the approval action. 

Skyrizi clinched a second nod, this time to treat adults with active psoriatic arthritis, AbbVie said late last week. The green light positions Skyrizi to treat an inflammatory disease that affects roughly 30% of psoriasis patients, the Illinois-based drugmaker noted. Last year, BMO Capital Markets analyst Gary Nachman forecast $350 million in psoriatic arthritis sales for the drug by 2025. 

“Patients often do not suspect a connection between their psoriasis skin symptoms and the joint pain, swelling and stiffness they may be experiencing, potentially leading to a delay in diagnosis and treatment of psoriatic arthritis,” Thomas Hudson, M.D., senior vice president of research and development and chief scientific officer at AbbVie, said in a statement.

The FDA gave its thumbs up on data from the pivotal Keepsake-1 and Keepsake-2 studies, in which Skyrizi trumped placebo at reducing a composite symptoms measure, ACR20, after 24 weeks. Specifically, 57.3% of Skyrizi patients in Keepsake-1 and 51.3% of patients on the AbbVie drug in Keepsake-2 saw at least a 20% reduction in their symptoms at the six-month mark. Comparatively, just 33.5% and 26.5% of placebo patients hit the primary endpoint in Keepsake-1 and -2, respectively.

RELATED: Eyeing its post-Humira future, AbbVie angles for broader Rinvoq use in U.S., Europe

Skyrizi also helped reduce the swelling of fingers and toes as well as swelling of the enthesis—where a tendon or ligament meets a person’s bonein patients who already had the conditions, known as dactylitis and enthesitis, AbbVie said.

In patients with coexistent plaque psoriasis, Skyrizi bested placebo at improving skin lesions, too, as measured by the Psoriasis Area Severity Index (PASI 90) at the 6-month mark.

Finally, Skyrizi charted a statistically significant improvement in physical function over placebo as measured by the Health Assessment Questionnaire-Disability Index over that same period of time. The average difference was 0.20 in Keepsake-1 and 0.16 in Keepsake-2.

The trials included patients who responded poorly to or couldn’t tolerate biologic therapies plus non-biologic disease-modifying antirheumatic drugs, AbbVie said. 

The dosing for the new indication is the same Skyrizi regimen at play in moderate-to-severe plaque psoriasis, AbbVie noted. A patient receives a single 150mg injection below the skin four times a year after two starter doses. Skyrizi can be taken alone or alongside disease-modifying antirheumatic drugs, AbbVie said.

RELATED: Even AbbVie CEO can’t confirm Rinvoq’s sales as FDA mulls JAK label

Skyrizi plus arthritis and atopic dermatitis med Rinvoq are the linchpins in AbbVie’s post-Humira strategy. The company recently reconfirmed its projection that the meds would clinch a combined $15 billion in sales in 2025. Ahead of the 2022 J.P. Morgan Healthcare Conference, the drugmaker reduced Rinvoq’s sales estimate for the middle of the decade by $500 million but boosted Skyrizi’s by the same amount, courtesy of a warning on Rinvoq’s label and Skyrizi’s continued strength in psoriasis, AbbVie said at the time.

Rinvoq, for its part, has been picking up new indications at a rapid clip. The drug in December won approval in psoriatic arthritis. Then, a little less than a week ago, the FDA gave Rinvoq its blessing in moderate-to-severe atopic dermatitis. That nod specifically covers patients who’ve failed on or aren’t suitable for other pills or injections.

In the third quarter of 2021, Skyrizi raked in $796 million in worldwide sales, AbbVie reported in October. Rinvoq’s haul, meanwhile, over the same period was $453 million.


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IBM sells off large parts of Watson Health business –

A private equity group has agreed to take over most of IBM Watson Health, seven years after the business was launched with a pledge to revolutionise healthcare data analysis.

Francisco Partners is picking up a range of databases and analytics tools – including Health Insights, MarketScan, Clinical Development, Social Programme Management, Micromedex and other imaging and radiology tools – for an undisclosed sum estimated to be in the region of $1 billion.

IBM said the sell-off is tagged as “a clear next step” as it focuses on its platform-based hybrid cloud and artificial intelligence strategy, but it’s no secret that Watson Health has failed to live up to its early promise.

The sale also marks a retreat from healthcare for the tech giant, which is remarkable given that it once said it viewed health as second only to financial services market as a market opportunity.

IBM said it “remains committed to Watson, our broader AI business, and to the clients and partners we support in healthcare IT.”

The company reportedly invested billions of dollars in Watson, but according to a Wall Street Journal report last year, the health business – which provided cloud-based access to the supercomputer and  a range of analytics services – has struggled to build market share and reach profitability.

An investigation by Stat meanwhile suggested that Watson Health’s early push into cancer for example was affected by a premature launch, interoperability challenges and over-reliance on human input to generate results.

For its part, IBM has said that the Watson for Oncology product has been improving year-on-year as the AI crunches more and more data.

That is backed up by a meta analysis of its performance published last year in Nature found that the treatment recommendations delivered by the tool were largely in line with human doctors for several cancer types.

However, the study also found that there was less consistency in more advanced cancers, and the authors noted the system “still needs further improvement.”

Watson Health offers a range of other services of course, including tools for genomic analysis and running clinical trials that have found favour with a number of pharma companies. Francisco said in a statement that it offers “a market leading team [that] provides its customers with mission critical products and outstanding service.”

The deal is expected to close in the second quarter, with the current management of Watson Health retaining “similar roles” in the new standalone company, according to the investment company.

IBM’s step back from health comes as tech rivals are still piling into the sector.

Last month, arch-rival Oracle announced a $28 billion takeover of electronic health record company Cerner, while 2021 also saw Microsoft’s $19.7 billion play for AI specialist Nuance and a $17 billion takeover of Athenahealth by investment groups Bain Capital and Hellman & Friedman.


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With omicron on the prowl, FDA extends inspection pause into February

The pandemic continues to drag on the FDA’s inspection plans. Now, the regulator is prolonging an intermission that kicked off just before the new year and was pegged to conclude last week.

The extended pause applies to certain inspection activities as the FDA keeps tabs on the COVID-19 pandemic and the spread of omicron, the regulator said Friday. While the agency will continue to perform mission-critical inspections at home and abroad, it is drawing out the pause on domestic surveillance inspections through Feb. 4. The goal is to restart those activities “as soon as safely possible.”

As for oversight overseas, the FDA said it would carry on with previously planned foreign surveillance inspections that have received country clearance and are within the Centers for Disease Control and Prevention’s level 1 or level 2 COVID-19 travel recommendation. If those requirements aren’t met, the inspection will have to be rescheduled, the FDA said.

By April, the regulator says it hopes to “return to a regular cadence for foreign surveillance inspections.”

RELATED: 2022 forecast: Can the FDA whittle down its manufacturing site inspection backlog next year?

The agency continues to leverage tools like remote assessments and import operations surveillance to help with its food and drug oversight.

Earlier this month, the FDA announced a pause on certain inspections through Jan. 19 as it worked “to ensure the safety of its employees and those of the firms it regulates” in the wake of the troubling coronavirus variant. The pause itself was initiated on Dec. 29, the FDA said at the time. 

Before the first omicron-fueled inspection pause, the FDA had been planning to overhaul its overseas oversight mission. But in announcing the temporary halt earlier this month, the FDA said it was “postponing the planning of prioritized surveillance foreign inspection assignments that were scheduled to begin in February 2022.”

Foreign inspections have been a pain point for the agency since before the COVID-19 pandemic, Mary Denigan-Macauley, director of healthcare at the Government Accountability Office (GAO), said last year in an interview.

Two big problems stem from overseas staffing vacancies and language barriers. The FDA in December told Fierce Pharma it was “actively working on these issues.” The agency cited a pilot program planned for 2022 that would strive to “enhance translator capabilities of foreign drug inspections” as well as the recruitment of “foreign cadre staff” who would be based in the U.S. but travel to foreign countries to conduct inspections.


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SMi’s 6th Annual Highly Potent Active Pharmaceutical Ingredients –

SMi’s 6th Annual
Highly Potent Active Pharmaceutical Ingredients
9th and 10th May 2022
London, UK
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Ensuring Risk-Based, Safety-Driven, High-Quality Operation

The Global High Potency API/HPAPI Market is projected to reach 37 billion USD by 2027, growing at an ever-increasing CAGR of 8.43%. The expanding production of high potency APIs, along with the increase in associated toxicities, are driving requirements for more effective containment solutions and much-needed guidelines to ensure worker safety.

SMi Group are delighted to announce the 6th Annual Highly Potent Active Pharmaceutical Ingredients Conference will be taking place on 9th – 10th May 2022 in London, UK. The conference will encompass the critical topics including risk assessment, occupational toxicology, engineering controls for containment, facility design, and the impact of pharmaceutical manufacture on the environment.

Benefits of Attending the HPAPI Conference:

•Dissect comprehensive hazard assessment and occupational exposure limit determination
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•Explore the employment of cleaning validation, organisational controls, and facility as key measure to tackle cross contamination

This two-day conference offers you peer-to-peer networking with industry experts including heads and directors of EHS, Occupational Hygiene, Quality Assurance and more. Expand your community and explore the latest developments in the industry at this two-day event, including key regulatory updates, and leading case studies from leading pharmaceutical and biotechnology companies.

Early Birds:
•Register by 31st January to save £400
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Register online at: www.highlypotentapi.com/pharmaphorum

Additional Contact Info:
T: +44 (0) 20 7827 6154
E: [email protected]
Follow us: @SMiPharm – Twitter | SMiPharma – LinkedIn


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Gilead pulls 2 cancer accelerated approvals as changing lymphoma landscape disrupts confirmatory trial

Back in 2014, Gilead Sciences’ Zydelig became the first PI3K inhibitor cleared by the FDA for certain B-cell blood cancers. Fast forward to today, and the company has found itself withdrawing two indications gained under the accelerated approval pathway as more treatments have become available.

Gilead is pulling Zydelig’s uses in relapsed follicular lymphoma (FL) and relapsed small lymphocytic lymphoma (SLL), citing failure to complete an FDA-mandated confirmatory trial. The move doesn’t affect Zydelig’s relapsed chronic lymphocytic leukemia use, which was earned with a full FDA go-ahead, the company said.

The retraction comes as the FDA is paying more careful attention to accelerated approvals that have failed to meet postapproval trial requirements. And as Gilead noted, the treatment landscape for the two indolent non-Hodgkin lymphoma subtypes has changed.

Gilead won accelerated approvals for FL and SLL based on phase 2 data showing tumor responses. But drugs that came after it have posted mature clinical efficacy data.

RELATED: AACR: Bayer’s Aliqopa, in redemption of the PI3K drug class, leaps ahead of Gilead’s Zydelig in lymphoma

Bayer’s rival PI3K inhibitor Aliqopa, used on top of Roche’s Rituxan, cut the risk of disease progression or death by 48% over Rituxan alone in patients with indolent non-Hodgkin’s lymphoma who had already received one prior therapy, Bayer unveiled in April 2021. Bayer hopes to use the new phase 3 data to turn Aliqopa’s accelerated approval for third-line treatment of follicular lymphoma into a full approval.

Zydelig’s own Rituxan combo trials were stopped early after reports of multiple deaths in its clinical programs in 2016. Because of that safety concern, Zydelig’s sales never picked up, with merely $50 million in global sales in the first nine months of 2021.

Other potentially safer PI3K options than Zydelig, such as TG Therapeutics’ Ukoniq, have also won FDA approvals in the late-line setting and are undergoing clinical trials to move earlier in the treatment sequence.

Plus, AbbVie and Johnson & Johnson’s BTK inhibitor, Imbruvica, has been able to treat newly diagnosed CLL and SLL patients thanks to an FDA nod in 2016 based on disease progression data.

RELATED: TG tumbles as FDA still hasn’t found what it’s looking for in U2 leukemia combo, orders advisory committee

Pointing to the evolved treatment landscape, Gilead admitted that enrollment into Zydelig’s confirmatory study has been a challenge.

Gilead’s decision also comes shortly after Secura Bio made a similar move for Verastem Oncology-inherited PI3K med Copiktra. In December, Secura voluntarily pulled Copiktra’s third-line FL indication off the U.S. market. The company at the time said it had determined that “the current treatment landscape for FL patients in the U.S. and the logistics, cost and timing of the postmarketing requirements for Copiktra in FL was no longer merited.”

The FDA recently launched an industrywide campaign targeting accelerated approvals in oncology with unresolved confirmatory trials. The initiative has triggered the withdrawal of several cancer drug indications, most notably for PD-1/L1 inhibitors including Merck’s Keytruda, Bristol Myers Squibb’s Opdivo, AstraZeneca’s Imfinzi and Roche’s Tecentriq.


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