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The FDA rejected an application to approve MDMA, also known as ecstasy. Now the agency treatment for PTSD. Next up among the psychedelics vying for an FDA approval: psilocybin for treatment resistant depression.

Long Strange Trip Continues for Psychedelics in PTSD, TRD

This is the first study to investigate the effectiveness of this combination to treat vitiligo. 

Adding a Statin to Light Therapy Significantly Improves Vitiligo Treatment Outcomes

Express Scripts alleges in its suit that the FTC followed “prejudice and politics, not evidence or sound economics,” and that there is no support for the assertion that the power of PBMs has increased over time.


Express Scripts Sues FTC to Withdraw its PBM Report

Peter Wehrwein has been the lead editor of Managed Healthcare Executive since February 2020.

Boehringer Ingelheim said that it will submit a new drug application for nerandomilast based on the positive results.

IPF Drug Meets Primary End Point

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The FDA has approved a subcutaneous form of Genentech’s Ocrevus to treat patients with relapsing multiple sclerosis (RMS) and primary progressive multiple sclerosis (PPMS). Ocrevus Zunovo (ocrelizumab and hyaluronidase-ocsq) approved as a twice-a-year, approximately 10-minute subcutaneous injection to be administered by a healthcare practitioner.

Ocrevus Zunovo will be available in a few weeks and be priced at parity with the Ocrevus IV.

This is the second approval in two days for subcutaneous formulations Genentech’s previously approved products.

“People are living longer with chronic illnesses and with fewer disabilities because of the extensive progress that has been made in the development of medicines that can slow their progression,” Natalie Blake, executive director of the MS Foundation, said in a 

. “But still, not everyone has access to these medicines. It’s crucial to acknowledge each experience with MS is as unique as the individual navigating it, so providing choices to address each person’s needs is essential.”

Ocrevus was first approved as an intravenous injection. It is a humanized monoclonal antibody designed to target CD20-positive B cells, a specific type of immune cell thought to be a key contributor to myelin and nerve cell damage. The 

Genentech offers a copay program for $0 a treatment for eligible commercially insured patients with a limit of $20,000 per calendar year. 

 say that Genentech may cap the assistance if “Genentech determines that a third party has implemented a program that adjusts patient cost-sharing obligations based on the availability of support under the Program and/or excludes the assistance provided under the program from counting towards the patient’s deductible or out-of-pocket cost limitations.”

Ocrevus Zunovo combines Ocrevus with Therapeutics’ Enhanze drug delivery technology. There is no word yet on Ocrevus Zunovo will be available or what the price will be.

The FDA approval of Ocrevus Zunovo is based on pivotal data from the phase 3 OCARINA II trial, which enrolled 236 patients and showed no clinically significant difference in the levels of Ocrevus in the blood when administered subcutaneously. The safety and efficacy profile are consistent to the IV formulation in people with RMS and PPMS.

Out of the exploratory outcomes measured, Ocrevus Zunovo was consistent with IV, demonstrating suppression of relapse activity (97%) and MRI lesions (97%) through 48 weeks. Additionally, more than 92% of trial participants reported being satisfied or very satisfied with the subcutaneous administration of Ocrevus Zunovo.

“Patients treated with subcutaneous Ocrevus experienced appropriate B-cell suppression and impressive near-complete suppression of new inflammatory disease activity,” Scott Newsome, D.O., lead author, Johns Hopkins University School of Medicine, said in a previous news release. These results demonstrate the potential of subcutaneous Ocrevus as a treatment option that can be matched to the individual needs of people with MS and healthcare professionals.”

Ocrevus Zunovo can be administrated by providers twice a year with a 10-minute subcutaneous injection. Ocrevus Zunovo will be available in a few weeks and be priced at parity with Ocrevus IV.

Formulary Watch

Clinical

FDA Approves Subcutaneous Ocrevus for Multiple Sclerosis

 Genentech’s Tecentriq Hybreza (atezolizumab and hyaluronidase-tqjs), the first PD-(L)1 inhibitor for subcutaneous (SC) administration. Tecentriq Hybreza can be injected subcutaneously over approximately seven minutes, compared with 30 minutes to 60 minutes for standard intravenous (IV) infusion of Tecentriq (atezolizumab). Genentech is a member of the Roche Group.

“This approval represents a significant option to improve the patient experience,” Ann Fish-Steagall, RN, senior vice president of Patient Services at the LUNGevity Foundation, said in a 

. “When patients have options, they feel empowered to be vital participants in their own care and choose their preferred treatment option.”

Tecentriq Hybreza combines Tecentriq with Halozyme Therapeutics’ Enhanze drug delivery technology. Tecentriq Hybreza will be available in a few weeks and be priced at parity with the IV Tecentriq 1200 mg vial.

Tecentriq is available to treat patients with early-stage non-small-cell lung cancer, small cell lung cancer and hepatocellular carcinoma. It is a monoclonal antibody designed to bind with PD-L1 that is expressed on tumor cells and tumor-infiltrating immune cells. This enables the reactivation of T cells.

The IV formulation of Tecentriq has an annual wholesale acquisition cost of $185,710. Genentech offers a $0 copay assistance program for eligible patients with commercial insurance with a limit of $25,000 a year. The 

The approval of Tecentriq Hybreza was based on data from the phase 1b/3 IMscin001 study, which enrolled 371 patients and showed comparable levels of Tecentriq in the blood, when administered subcutaneously. The safety and efficacy profile were consistent with the IV formulation. 

The phase 2 IMscin002 study enrolled 179 patients and showed that 71% of patients preferred Tecentriq Hybreza over intravenous atezolizumab. The most common reasons were less time in the clinic, increased comfort during treatment and reduced emotional distress.

Tecentriq Hybreza will be available in a few weeks and be priced at parity with the IV Tecentriq 1200 mg vial.

FDA Approves Subcutaneous Tecentriq to Treat Multiple Cancers

In what might be a potential breakthrough for a significant unmet need, Lexeo Therapeutics announced last month that their gene therapy candidate, LX2006, had achieved positive interim 

 clinical data for treating Friedreich Ataxia (FA) cardiomyopathy. This marks a significant milestone in developing new therapies for this devastating condition.

Given the lack of approved treatments for FA cardiomyopathy, LX2006's potential could represent a breakthrough for patients suffering from a disease characterized by severe cardiac complications.

FA is an exceptionally rare, autosomal recessive disease caused by a mutation in the frataxin gene. The absence of an approved therapy that can slow down the progression of cardiomyopathy in FA, which is responsible for a significant portion of FA-related deaths, underscores the urgent need for new treatments.

FA cardiomyopathy is usually identified by an increase in left ventricular mass index (LVMI) that can lead to heart failure. ​ Elevated LVMI in adults with FA cardiomyopathy is a sign of left ventricular hypertrophy and is associated with mortality in various cardiovascular conditions, including FA cardiomyopathy. ​

Given as a one-time infusion, LX2006 is an adeno-associated virus gene therapy that delivers a functional frataxin gene to cardiac cells to promote the production of normal frataxin protein and restore mitochondrial function in heart muscle cells. Without a working copy of the frataxin gene, the heart is unable to sustain energy production, and it cannot work correctly.

The interim results shared from the SUNRISE-FA Phase 1/2 clinical trial and the Weill Cornell Medicine investigator-initiated 

 suggest that LX2006 is well-tolerated, with no serious adverse events reported. The evidence of clinically meaningful improvements across various cardiac biomarkers, particularly concerning left ventricular hypertrophy, is noteworthy. Since left ventricular hypertrophy is a hallmark of FA cardiomyopathy and correlates with mortality, these findings could pave the way for improved patient outcomes.

Data from eight participants with more than six months of follow-up demonstrated an average reduction in LVMI of 11.4% at 12 months and 18.3% at 18 months. Seventy-five percent of participants with elevated LVMI at the start of the study experienced a reduction in LVMI of over 10% at 12 months.Most participants showed sustained and consistent improvements in other necessary measures of cardiac health, such as left ventricular wall thickness and troponin I, at 12 months.

The restoration of frataxin protein expression observed in cardiac biopsies further underscores the therapeutic potential of LX2006. As the disease progresses with low levels of frataxin leading to heart failure, restoring this protein could be crucial for improving cardiac function and enhancing the overall quality of life for affected individuals.

The fact that LX2006 has received Rare Pediatric Disease designation, Fast Track designation, and Orphan Drug designation from the FDA indicates a strong regulatory interest and support for this treatment. These designations can facilitate a more expedited development and review process, potentially leading to accelerated approval.

Despite the promising data, it is essential to acknowledge the inherent risks and uncertainties associated with clinical trials and the development of new therapies. Lexeo's cautionary note regarding forward-looking statements underscores the unpredictable nature of drug development, where new challenges could arise that may affect trial outcomes or regulatory processes.

Although the initial results are promising, LX2006's ultimate success will depend on ongoing safety evaluations, long-term efficacy data, and the ability to navigate the regulatory landscape.

Lexeo Therapeutics' interim clinical data for LX2006 represents a potentially transformative advancement in treating Friedreich Ataxia cardiomyopathy. While the early results are encouraging, ongoing clinical evaluations and regulatory engagements will be vital in determining the future of this promising gene therapy. 

Lexeo Therapeutics' LX2006 Shows Promise in Treating Friedreich's Ataxia Cardiomyopathy

A number of drugmakers are racing to win the first FDA approval of a Bruton’s kinase (BTK) inhibitor to treat multiple sclerosis (MS). Roche showed it is in the thick of the competition with an announcement of positive results for its BTK candidate, fenebrutinib. In an open-label extension of the phase 2 FENopta study. According to the company's announcement,  96% of participants who received fenebrutinib had no relapses after one year and no change in Expanded Disability Status Scale (EDSS) score over 48 weeks. The overall annualized relapse rate was 0.04.

Furthermore, MRI scans showed that 99% of patients had no new T1 lesions, a measure of active inflammation, according to the Roche news release. T2 lesions, which indicate chronic disease burden, were reduced by three times during the 48-week open-label period compared to the end of the blinded trial. The safety profile was comparable to previously reported results, with the most common adverse effects being urinary tract infection, COVID-19 and pharyngitis.

The FENopta study randomized 109 adults ages 18 to 55 years with relapsing forms of MS to receive oral fenebrutinib or placebo twice daily for 12 weeks. After the three-month period, 99 participants chose to enroll in the open-label extension study, in which all participants will continue to receive daily fenebrutinib for up to three and a half years.

Roche is scheduled to present data from the extension study at the 

40th Congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS)

 in Copenhagen, Denmark, on September 18, 2024.

Fenebrutinib is an investigational oral, noncovalent (reversible) BTK inhibitor being developed by Genentech, a Roche subsidiary, for treating relapsing forms of MS and primary progressive MS (PPMS). In preclinical studies, fenebrutinib demonstrated 130 times more selectivity for BTK versus other kinases, a feature that, along with its reversibility, that Roche claims may help reduce off-target drug effects and potentially contribute to a favorable safety profile for fenebrutinib.

The investigational molecule is currently in three phase 3 trials for the potential treatment of various forms of MS. The FENhance and FENhance 2 trials are evaluating the use of fenebrutinib compared with Sanofi’s Aubagio (teriflunomide) in adults with relapsing forms of MS. The FENtrepid trial compares fenebrutinib with Roche’s Ocrevus (ocrelizumab) in adults with PPMS.

Other investigational BTK inhibitors in phase 3 studies for treating MS include Sanofi’s tolebrutinib, Merck KGaA’s evobrutinib, and Novartis’ remibrutinib. Trials for the four agents had previously been slammed by the FDA with patrial clinical holds because of problems with liver toxicity.  

Roche's drug is one of a number competing to become the first Bruton's kinase inhibitors approved as a treatment for multiple sclerosis.

Fenebrutinib Suppresses and Disability Progression in Majority of MS Patients in Extension Study

The severity of ulcerative colitis (UC) is classified as mild, moderate or severe based on symptoms and clinical findings. The main goal of treatment remains to induce and maintain remission from the mucosal inflammation symptoms associated with the disease. Currently, there are several advanced therapies approved to treat moderate-to-severe UC. These include biologics, such as tumor necrosis factor (TNF) inhibitors, interleukin (IL)-12/23 and IL-23 inhibitors, and an anti-integrin agent. Small molecules, such as Janus kinase (JAK) inhibitors and sphingosine 1-phosphate receptor modulators, are also in the picture.

Although randomized controlled trials are the gold standard for establishing the safety and efficacy of treatments and gaining regulatory approval, results are often not generalizable given the rigid inclusion criteria necessary for minimizing potential confounders in clinical trials. More and more, real-world studies are becoming useful in clinical practice, as they are more likely to represent a generalized population.

According to Peter M. Irving, M.D., from the gastroenterology department at Guy’s and Thomas’ Hospital in London and his colleagues, there is scant comprehensive research assessing the safety and efficacy of currently available advanced therapies for moderate-to-severe UC in a real-world setting. To address this gap, Irving and his colleagues conducted a systematic review of real-world studies evaluating the safety and efficacy of advanced therapies in adults with moderate-to-severe UC.

The review results were published in the September 2024 issue of the 

Journal of Managed Care and Specialty Pharmacy

The review included 139 studies published between 2005 and 2022. The following treatments were assessed in the included studies: the integrin receptor inhibitor Entyvio (vedolizumab), the JAK inhibitor Xeljanz (tofacitinib), the IL-12/23 inhibitor Stelara (ustekinumab), and the TNF inhibitors Humira (adalimumab), Simponi (golimumab), and Remicade (infliximab).

Results showed Entyvio and Xeljanz as the most commonly studied therapies. When the two were compared, Xeljanz showed higher remission rates. When comparing Entyvio with TNF inhibitors, Entyvio  came out on top in terms of remission rates. When looking at steroid-free clinical remission rates, Xeljanz and Stelara had similar outcomes. Within the group of TNF inhibitors, Remicade was the most effective. Lastly, the safety profiles were similar across all therapies.

The authors noted that study limitations, such as small sample sizes and variations in definitions of clinical remission, study design, and patient characteristics, warrant further research exploring these gaps. However, the current findings can be valuable in guiding clinicians treating patients with moderate-to-severe ulcerative colitis.

This study was sponsored by Pfizer, maker of Xeljanz .

Entyvio (vedolizumab) and Xeljanz (tofacitinib) are the most commonly studied therapies, according to the Pfizer-sponsored review, and when the two were compared, Xeljanz showed higher remission rates. 

Results From Review of Real-World Studies of Advanced Therapies in Moderate-to-Severe Ulcerative Colitis

The once-weekly insulin efsitora alfa has been shown to be noninferior to once-daily insulin degludec in a phase 3 trial of adults with type 1 diabetes, according to new 

. Eli Lilly’s insulin efsitora is a basal insulin that is administered subcutaneously.

In the trial, efsitora met the primary endpoint of noninferior A1C reduction at week 26, reducing A1C by 0.53% compared with 0.59% for insulin degludec. But severe hypoglycemic events were higher in the efsitora arm compared with the insulin degludec arm. Hypoglycemia, in which blood sugar is too low, is a side effect of medications used to treat diabetes.

“People with type 1 diabetes need insulin every day. Currently, they can deliver the insulin using an automated insulin delivery system or by taking a daily basal insulin injection and multiple mealtime insulin injections each day,” Richard Bergenstal, M.D., executive director of the International Diabetes Center, HealthPartners Institute, said in a 

. "This new data shows that with one dose a week of basal insulin, efsitora was able to achieve a similar A1C reduction as taking an injection of one of the most used background insulins every day.”

Lilly’s efsitora is being studied in a global phase 3 program. In the open-label QWINT-5, the once-weekly efsitora is being compared with the once-daily insulin degludec in 692 patients with type 1 diabetes who randomly assigned to treatment. The trial consisted of a 52-week treatment period with the primary endpoint measured at 26 weeks. The primary objective was non-inferiority in reducing A1C compared with insulin degludec.

In the trial, however the estimated combined rates of patient-reported clinically significant or severe hypoglycemic events per patient-year of exposure through week 52 were higher in the efsitora arm compared with the insulin degludec arm. About 10% of patients in the efsitoria arm experienced severe hypoglycemia compared with 3% in the degludec arm.

Researchers suggested that there may be a need for additional evaluation of efsitora dose initiation and optimization in people with type 1 diabetes.

Insulin degludec, developed by Novo Nordisk, is a once-daily basal insulin that first received approval in 2016 with the brand name Tresiba to improve glycemic control in patients with type 1 and type 2 diabetes. The FDA approved in July 2022 Novo Nordisk’s unbranded insulin degludec, which launched at a discount of 65% off the list price of Tresiba.

Insulin efsitora was able to lower A1C in patients with type 1 diabetes but more patients experienced severe hypoglycemic events than patients taking insulin degludec.

Calum Yacoubian, M.D.

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