Notable Clinical Trials that Shaped December 2023

Clinical trials play a crucial role in advancing medical knowledge and enhancing patient outcomes within the pharmaceutical and MedTech fields. As per EMR estimations, the clinical trials market size is poised to attain a value of USD 92.56 billion by 2032, with a steady CAGR of 7.5%. These studies are fundamental in ensuring the effectiveness and safety of new treatments, interventions, and medications. Each trial represents a commitment to develop highly efficient therapies, underscoring the significance of exploring notable clinical trials aimed at improving patient care.

1. VX-548 Displayed Promising Results in Treating Painful Diabetic Peripheral Neuropathy

In December 2023, Vertex Pharmaceuticals published the details of a Phase 2 clinical trial that evaluated the efficacy of VX-548, a selective NaV1.8 inhibitor, for treating painful diabetic peripheral neuropathy (DPN) .The study exhibited that VX-548 was the only medication that showed a significant reduction in pain intensity on a Numeric Pain Rating Scale (NPRS) at Week 12 across all doses administered. An active reference arm using pregabalin was used to gauge the effectiveness of the VX-548 treatment. The trial also indicated that VX-548 had good tolerability at all doses tested, and the adverse events (AEs) reported were mostly mild to moderate, with no serious adverse events (SAEs) linked to the medication.

Vertex plans to advance VX-548 into pivotal development after consulting with regulatory authorities. Additionally, a second Phase 2 trial for VX-548 in peripheral neuropathic pain has been initiated, targeting patients experiencing painful lumbosacral radiculopathy (LSR). This is a type of pain caused by nerve root damage or injury in the lumbar spine. Moreover, the progress of three Phase 3 studies evaluating VX-548 in acute pain is on track, and the results are expected in the first quarter of 2024.

2. BI 690517 Demonstrates Positive Effect on Treating Chronic Cancer Disease

A study published in December 2023, examined the effects of a medication called BI 690517 on the urinary albumin-to-creatinine ratio (UACR) and its potential impact on managing kidney disease. The result indicated that administering BI 690517 at different doses (3 mg: -22%, 10 mg: -39%, 20 mg: -37%) led to significant reductions in UACR percentages compared to those who received a placebo (-3%). These reductions suggest that BI 690517 could be effective in decreasing the levels of urinary protein, which can be indicative of kidney damage or disease progression. It was also found that when combined with empagliflozin, BI 690517 showed a similar reduction in UACR, indicating its potential in combination with other medications.

3. Selective Inhibitor Nanatinostat Well Tolerated against EPV-Positive Nasopharyngeal Carcinoma

A clinical study conducted in December 2023, showed that combining nanatinostat with the ganciclovir prodrug, valganciclovir, was safe and effective in treating recurrent EBV-positive lymphoid malignancies.

NPC (nasopharyngeal carcinoma ) has the highest incidence rates in the Asian region. Although there have been advances in treatment options over the last three decades, there is still a need for potent agents that specifically target EBV-positive disease. The result of the trial displayed that the combination of nanatinostat and valganciclovir was well-tolerated at doses higher than the recommended phase II dose (RP2D) previously identified in the lymphoma study (nanatinostat 20 mg once daily for 4 days/week, along with valganciclovir 900 mg once daily).

4. Tarlatamab Shows Potential Against Small Cell Lung Cancer

A trial, currently in the early stages, evaluated the effectiveness of two different doses of a new experimental drug named tarlatamab against small cell lung cancer. (SCLC) This trial particularly focused on individuals with significant cancer progression, even after undergoing at least two types of treatment. Several participants in the study had already undergone at least three distinct treatments before participating in this trial.

Results from the clinical trial revealed that the novel targeted immunotherapy drug was capable of shrinking tumours by 40% in about one out of every three individuals diagnosed with small cell lung cancer.

5. Subcutaneous Semaglutide Demonstrates Promising Results for Combating Obesity

The SELECT clinical trial, published in December 2023, assessed the effectiveness of subcutaneous semaglutide in helping overweight people with weight loss. Typically used for diabetes, this once-a-week drug reduced major heart-related issues by 20% in about 33 months. However, some people discontinued the drug consumption due to stomach issues, which is a common side effect of this type of medication. The benefit of subcutaneous semaglutide was observed in patients with concurrent statin application as well. These findings suggest that subcutaneous semaglutide might help prevent heart issues in people with a history of heart problems, even if they don't have diabetes but are overweight or obese.

6. Hope for Schizophrenia Treatment as Potential Drug Showcases Promising Results

United States-based Vanderbilt University and Neumora Therapeutics entered a research-based collaboration and discovered a new potential treatment, known as NMRA-266, for schizophrenia. According to EMR calculations, the Schizophrenia drugs market size is anticipated to grow at a CAGR of 5.4% and attain a value of USD 12.97 billion by 2032. The development of this novel drug can add significant worth to the rising market value in the future. It is centred around targeting the M4 muscarinic receptor, responsible for regulating dopamine in the brain through a positive allosteric modulation. It has also received the FDA’s green signal to enter Phase I clinical trials.

7. A Leap Forward in Stimulant Use Disorder Treatment as Multiple Drugs Undergo Stringent Trials
 
Researchers at the UT Southwestern Medical Center are leading three research projects to provide potential treatment alternatives for methamphetamine and cocaine addiction. With a USD 30 million investment from the National Institute on Drug Abuse (NIDA), the major drugs under evaluation include test injections of naltrexone, buprenorphine, along with determining the efficacy and safety against intravenously delivered ketamine (an anaesthetic drug) versus midazolam (a sedative) in patients. In addition, transcranial magnetic simulation for patients with either MUD or CUD is under trial as well.

In the United States, over 1.6 million people struggle with methamphetamine use disorder (MUD), and 1.4 million battles cocaine use disorder (CUD), resulting in tens of thousands of deaths annually.

8. A Novel Combination Therapy for Metastatic Triple Negative Breast Cancer

Researchers at Vanderbilt conducted a trial using a combination of atezolizumab (an immunotherapy drug) and carboplatin (a different kind of chemotherapy) for people with metastatic triple-negative breast cancer. They aimed to examine the efficacy and performance of this combo to find biomarkers that exhibit how well the immunotherapy works.

When combined with carboplatin instead of nab-paclitaxel, atezolizumab showed significant improvements in efficacy. Patients on this new combination had improved experiences, prior to cancer worsening (from around 2.2 months to 4.1 months) and lived longer (from about 8.6 months to 12.6 months) compared to those getting just carboplatin. This trial involved 106 patients from different backgrounds and was done at six cancer centers as part of the Translational Breast Cancer Research Consortium. EMR reports that the breast cancer market size is expected to grow at a CAGR of 10.10% with the values likely to reach USD 72.1 billion by 2032.

9. Mantle Cell Lymphoma Receives New Combination Treatment Alternative

Researchers at the American Society of Hematology found that the combination of two drugs, ibrutinib and venetoclax, exhibited a major impact on people with a severe type of lymphoma called mantle cell lymphoma (MCL). In a large-scale study, researchers compared the impact of a drug combo to a placebo when used alongside ibrutinib. The results demonstrated a significant improvement. People on the combo had their disease under control for about 32 months compared to 22 months with only ibrutinib. Approximately 54% of the people treated with the combination experienced complete remission as compared to those on only ibrutinib (32%).

Mantle cell lymphoma is a rare and aggressive type of lymphoma, highly susceptible in the geriatric population. This combo allegedly improved the disease without it getting worse and led to overall remission in 82% of patients with MCL who came back or weren't responding to treatment.

As technological advancements continue to soar and our understanding of human physiology deepens, there is an optimistic expectation for the emergence of new treatments, along with enhancements to existing therapies. Such clinical trials lead to the revelation of unique insights that will contribute to improved patient care and a brighter and healthier future.