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The months follow each other and are not the same for the Bordeaux biopharmaceutical company Aelis Farmawhich has been developing a new family of drugs since 2013, CB1-SSi (1).
In September, a study on the effectiveness of its treatment for cannabis addiction did not achieve its main objective, precipitating its stock market plunge (read below). Two months later, the biotech announced results “exceeding all expectations” concerning the safety and effectiveness of another compound, this time for cognitive disorders linked to Down syndrome.
“The CB1-SSi AEF02017 [c’est son nom, NDLR] is a drug that can be used to treat several cognitive deficits”, explains Doctor Pier-Vincenzo Piazza, general director of Aelis, former research director of Inserm and founder of Neurocentre Magendie.
“The first indication that we explored is Down syndrome because there are very significant needs. If physical medical advances have made it possible to increase the life expectancy of Down syndrome carriers from nineteen years (in the 1970s) to 66 years, the subject of cognitive problems remains unresolved. And we find ourselves with families who are anxious about the idea that their children will survive them. »
Unexpected
This phase 1/2 study, lasting four weeks, was carried out in Madrid and Barcelona among 29 adults aged 18 to 35. “The first objective was to evaluate the safety and tolerability of the drug – a primary concern for families,” specifies Dr. Piazza, according to whom “the results are excellent.”
The other objective: to evaluate its effectiveness on three criteria. “We first measured the patients’ brain activity during a working memory task. For them, it is higher because these tasks tire them. They need to mobilize more resources. However, we observed that their activity was reduced to the level of a “neurotypical” person. »
Second criterion, cognitive flexibility (the ability to use information learned in a different context to solve a new problem). “We also see a trend of improvement. » And the icing on the cake: “Where we obtained the most significant effects, and against all expectations, it was on adaptive behavior,” notes Stéphanie Monlezun, director of operations.
New study
“All the experts with whom we worked told us: ‘Don’t get false hopes, acting via a drug on the most integrated aspects is unheard of,’” smiles Dr. Piazza. “However, we noticed an improvement in expression and the ability to write. And also an improvement in social skills. »
“All of this affects us a lot on an emotional level, because we have been working with families for a long time”
Dr. Piazza believes that these results are “the best ever observed for Down syndrome. They indicate that we have in our hands a treatment capable of making a difference to the quality of life of these people. All of this affects us enormously on an emotional level, because we have been working with families for a long time. »
The next step is a larger-scale study, lasting six months, in around ten clinical centers spread across three countries, with 200 to 250 people. It should start in mid-2025. “Laboratories are closely monitoring our results,” slips Dr. Piazza.
At a less advanced stage, Aelis Farma is exploring applications of this drug to treat cognitive deficits associated with other pathologies: autism, aging, even schizophrenia and Parkinson’s disease.
(1) Specific inhibitors of CB1 receptor signaling of the endocannabinoid system.
Shaken on the stock market
This result comes at the right time for Aelis, which fell sharply on the stock market in September. Listed at €11.40 on the 3rd, the biotech fell to €4.54 on the 4th. At the end of November, the stock was around €2.90. Less 75% compared to spring.
“The results were poorly received by the markets, which reacted as if the compound was dead,” comments Dr. Piazza. However, if our main criterion has not been achieved, the result is not really negative. We are starting another phase 2, which delays a possible market launch by two years. » In truth, what the market didn’t like was that a direct move to phase 3 of its treatment for cannabis addiction would have unlocked a 100 million euro option. from the Indivior laboratory.
What is there to regret about being listed on the stock exchange? “No, the correction was certainly severe, but we are a forward-thinking team. We have money to finance our next studies, with visibility until the end of 2026 in order to develop new compounds and strengthen our value. »
Proof of this confidence in the future, Aelis is inaugurating a new laboratory on November 28 in the European Institute of Chemistry and Biology (IECB) in Pessac.
## Interview: Hope for Down Syndrome and the Volatility of Biotech Innovation
**Welcome to World Today News. Today, we’re diving into the fascinating world of biotechnology and the hopeful potential for breakthroughs in treating cognitive disorders. Joining us are two distinguished guests:**
* **Dr. Pier-Vincenzo Piazza**, General Director of Aelis Farma and former research director of Inserm.
* **Stéphanie Monlezun**, Director of Operations at Aelis Farma.
**Thank you both for being here.**
**(Section 1: A Breakthrough for Down Syndrome)**
**Interviewer:** Dr. Piazza, the recent results regarding AEF02017 for Down syndrome have been described as “exceeding all expectations.” Can you elaborate on these findings and what they mean for individuals with Down syndrome and their families?
**Dr. Piazza:**
*
**Interviewer:**
Stéphanie, you mentioned “unexpected” improvements in adaptive behavior. Can you elaborate on what these improvements look like in real-world scenarios and how significant they are for individuals with Down syndrome?
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**Interviewer:** This research offers hope for substantial improvement in the quality of life for individuals with Down syndrome. How do you see this treatment potentially transforming their daily experiences and future aspirations?
**
Section 2: The Challenges and Rewards of Biotech Innovation **
**Interviewer:** Aelis Farma faced a significant setback in September when its cannabis addiction treatment trial didn’t meet its primary objective, leading to a sharp drop in stock prices. Dr. Piazza, how did the company navigate this challenge, and what lessons did you learn from this experience?
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**Interviewer:** Stéphanie, the volatility of the biotech sector is well-known. How does Aelis Farma balance the pursuit of groundbreaking research with the financial realities of the market?
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**(Section 3: Looking Towards the Future)**
**Interviewer:** Dr. Piazza, what are the next steps for AEF02017, and what timeline are you looking at for potential regulatory approval and wider availability?
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**Interviewer:** Beyond Down syndrome, Aelis Farma is exploring applications for this drug in other cognitive disorders. Can you share some insights into these explorations and the potential impact on other patient populations?
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**Interviewer:**
This research is clearly driven by a deep commitment to improving lives. What message would you like to leave with our viewers about the importance of continued investment and support for biotech innovation?
**Both Guests:**
**Thank you, Dr. Piazza and Stéphanie Monlezun, for sharing your insights and passion with us today. This is a truly promising field with the potential to transform lives, and we eagerly anticipate further developments from Aelis Farma.**
