In a major shift away from decades of stigma, the FDA has granted Breakthrough Therapy Designation to MindMed‘s MM120, an LSD-based treatment for Generalized Anxiety Disorder (GAD). This milestone not only underscores the growing recognition of the therapeutic potential of psychedelics but also could point to a potential turning point for the struggling psychedelic sector and specifically bolsters the future of MindMed.
[Related: Could LSD change the game in anxiety treatment?]
Psychedelic stocks: A long, strange trip continues
As with many psychedelic companies, MindMed’s stock (NASDAQ: MNMD) has been on a rollercoaster ride in recent years, briefly reaching close to $60 per share in early and mid-2021 amid peaking investor enthusiasm for the potential of psychedelic-based treatments. But the stock, like many others in the sector, has faced significant declines as investor enthusiasm waned later in 2021 and 2022.
The breakthrough therapy designation could reignite interest and confidence in MindMed and the broader field.As of March 8, 2024, the stock is up 54.35% over the past 5 days. Despite the promising developments for these substances, psilocybin, LSD, and MDMA remain classified as Schedule 1 drugs by the Drug Enforcement Administration (DEA), a designation implying high abuse potential and no currently accepted medical use. This reality highlights the ongoing tension between the therapeutic potential of these compounds and the challenges of regulatory change. If the drugs succeed in winning FDA approval, they are likely to continue to face strict oversight, a prospect that could be complicated by growing efforts to decriminalize naturally-occurring psychedelics in some parts of the U.S.
Despite the promising therapeutic potential of psychedelics, major pharmaceutical companies have largely steered clear of this field owing to the limited patent protection for these compounds and the high-risk nature of developing drugs that the DEA deems to have no medical value and a high abuse potential.
Promising results from the phase 2b trial for an LSD-based anxiety treatment
MindMed’s Phase 2b trial demonstrated promising results for MM120. The study, the largest well-controlled clinical trial of LSD ever conducted in the U.S., showed significant and sustained reductions in anxiety symptoms for up to 12 weeks after a single dose. The data suggest MM120’s potential to provide rapid and lasting relief, in contrast to current GAD treatments that often require daily use and numerous medication changes. SSRIs like escitalopram and paroxetine are commonly used first-line treatments, while benzodiazepines provide fast-acting relief but are habit-forming so are often reserved for short-term use.
“I’ve conducted clinical research studies in psychiatry for over two decades and have seen studies of many drugs under development for the treatment of anxiety,” David Feifel, MD, PhD, an investigator in the study, said in a news release. “That MM120 exhibited rapid and robust efficacy, solidly sustained for 12 weeks after a single dose, is truly remarkable. These results suggest the potential MM120 has in the treatment of anxiety, and those of us who struggle every day to alleviate anxiety in our patients look forward to seeing results from future Phase 3 trials.”
“GAD is an indication that has been severely overlooked for the past several decades, resulting in a current prevalence of approximately 10% of US adults, a number that has tripled in the last two decades,” said Dr. Rakesh Jain, a psychiatriast affiliated with Texas Tech University School of Medicine, in a call with analysts.
“MM120 100 µg demonstrated a 7.7-point improvement over placebo at Week 12 (-21.9 MM120 vs. -14.2 placebo; p<0.003 Cohen’s d=0.81), with a 65% clinical response rate and a 48% clinical remission rate sustained to Week 12,” noted Dr. Daniel Karlin, MindMed’s chief medical officer.
Could an LSD-based anxiety treatment gain legitimacy?
GAD is a prevalent condition, affecting approximately 10% of US adults, and this number has tripled in the last two decades. Current treatments often require daily use and numerous medication changes, highlighting the need for novel, rapid-acting, and long-lasting treatment options. MM120 could potentially fill this critical gap in the treatment landscape.
Potential care delivery model
“Patients are evaluated by LCL party practitioner with prescribing rates often of psychiatrist and if appropriate, prescribe the treatment. The medicine is distributed by specialty pharmacy and the treatment is then administered by the patient at the start of the treatment session. That explained takes place in the clinic or office. The patient is monitored by licensed healthcare providers for the entire duration of the session before being safely released,” explained Dr. Francoise Lilienthal, Mindmed’s chief commercial officer.
“We believe a care delivery model which does not rely on psychotherapy, preparation, integration or other intensive non-drug intervention could enable broader accessibility and a wider range of providers. And delivery sites to potentially adopt MM120 compared to others in our world to our class,” added Dr. Lilienthal.
Next steps for MindMed’s LSD-based anxiety treatment
“We’ve now achieved the goals of our Phase Two development program which included establishing the dose response relationship for MM120 and demonstrating rapid and durable clinical activity gap. We believe the data support dose selection and advancement of MM120 into Phase Three development and are excited to kick off our program later this year,” said Robert Barrow, CEO of MindMed.
MindMed plans to hold an End-of-Phase 2 meeting with the FDA in the first half of 2024 and initiate a phase 3 clinical program in the second half of 2024. The Breakthrough Therapy Designation, coupled with the significant unmet need in GAD treatment, highlights the potential for MM120 to fill a critical gap in the current treatment landscape. If approved, MM120 could offer a novel, rapid-acting, and long-lasting treatment option for the millions of individuals living with GAD who have not found relief with existing medications.
Changing regulatory climate
This decision marks a departure from the Schedule I classification that has hindered scientific exploration of LSD since the 1970s. MindMed’s rigorous clinical trials and positive data have played a pivotal role in demonstrating the potential therapeutic value of LSD within a controlled medical setting. This follows a similar Breakthrough Therapy Designation for psilocybin, and amidst broader efforts to reschedule cannabis, signifying a rapidly changing cultural and regulatory climate surrounding substances once considered strictly illicit.
Looking forward
The Breakthrough Therapy Designation for MM120 offers significant hope for patients suffering from Generalized Anxiety Disorder (GAD), particularly those who experience limited benefit or intolerable side effects from current treatments. While this breakthrough signals a potential shift in the treatment landscape, MM120 still faces regulatory hurdles. The FDA is currently reviewing MDMA for PTSD, and its eventual decision will offer crucial insights into the agency’s stance on psychedelic-assisted therapies. If approved, it suggests LSD and psilocybin could follow, likely with strict controls given their history and potential for abuse.