Key Takeaways after Neurology Seminar CNS

Redeye Research Note timeStamp 2020/10/16

Redeye gives a note with key takeaways following its seminar on 'Drug Development in the Central Nervous system (CNS).'

Introduction to the Theme
It was the third year in a row Redeye hosted a seminar on this topic, this year in a digital format. We started this seminar by giving an introduction to CNS drug development. Drug sales in CNS is a USD > 80 billion market. Still, some indications only scratch on the true potential’s surface, given the medical needs and prevalence.
The introduction slides can be found here.


Company Presentations
After the introduction, seven biotech/-pharma companies presented their business and how they are active in this space. Redeye has coverage of six of those; Alzecure, Alzinova, Bioarctic, Irlab Therapeutics, Saniona, and Asarina Pharma. In addition to the covered companies, Herantis Pharma (non-Redeye coverage) participated.


Participating companies active in the Alzheimer’s field were Alzecure, Alzinova, and Bioarctic. What is interesting in this cohort is that they all target Amyloid Beta (Aβ), but with different approaches, with a vaccine (Alzinova), small molecules (Alzecure), as well as with biologics/monoclonal antibodies (Bioarctic). It is enjoyable to have three similar but yet so different companies in this field, which stem from Scandinavian science.

Alzheimer is as ever a vigorously debated field. There has been quite a turmoil in recent year. Just when the community was to abandon its last hope in the amyloid-cascade hypothesis, aducanumab resurrected. Aducanumab is an Aβ-monoclonal antibody with preferential binding to Aβ-fibrils (the protein aggregation before it turns into Aβ-plaques). Biogen develops it in partnership with Eisai. Their phase 3 trials were halted by futility analysis in early 2019, with a comeback in the fall of 2019 after further analysis.

Investors with interest in this field should mark 6th November in their calendar. An FDA committee panel meeting will be held, which will offer a glimpse of how they resonate with regards to Biogen’s Biologics License Application (BLA) for aducanumab. What we can say at this point is that it would be sensational if a drug gets approved based on a terminated phase 3 trial.

Some key takeaways on the corporate level include:

  • CEO Martin Jönsson sees potential to out-license projects already at the preclinical stage (Alzecure)
  • CEO Jönsson reiterated its goal to initiate two phase I studies at the end of 2020 (Alzecure)
  • Alzinova received, on the same day as the CNS event occurred (15th October), positive feedback from Finnish regulators, such as the preclinical package and key design features. CEO Kristina Torfgård reiterated its communication on starting phase Ib trial in Q2’21 (Alzinova)
  • Further, it was encouraging to hear CEO Torfgårds confidence in finding a partner with ALZ-101, already after phase Ib completion (Alzinova)
  • The day before the CNS seminar, Bioarctic release its Q3 earnings report. Redeye’s analyst note following the interim report here (Bioarctic)
  • The blood-brain barrier technology remains highly interesting, but CEO Gunilla Osswald emphasized that this is a competitive area. CEO Osswald reiterates that this area holds the potential for both its own projects, but also external biologics outside of the neurodegenerative area (Bioarctic)


In the Parkinson area, Irlab Therapeutics and Herantis Pharma presented its businesses. In addition, previously mentioned Bioarctic has a strong commitment to Parkinson as well.

Parkinson is, next to Alzheimer’s, the most common neurodegenerative disease. It tends earlier-onset, sometimes in individuals younger than 50 years of age, thereby disrupting many of the years where the individual should peak in productivity. There is a great medical need for both disease-modifying as well as more effective symptomatic drug therapies.

Some key takeaways on the corporate level include:

  • October 2020 marks a milestone month for Irlab Therapeutics, with an uplisting to OMX Stockholm main market and IND-approval by the FDA for mesdopetam. CEO Nicholas Waters was satisfied with both milestones, with the uplisting entails more stock-caring, and with the IND entails recruitment of US-patients in clinical trials with mesdopetam (Irlab Therapeutics)
  • Finnish biotech company Herantis Pharma was presented by its newly appointed CEO, Craig Cook. CEO Cook emphasized the fascinating science in why joining the company, and what competencies he hopes to bring to the company (Herantis Pharma)


Other Neurology
Asarina Pharma and Saniona represented the last section in this seminar. Asarina Pharma has programs within menstrual migraine and Tourettes Syndrome, whereas Saniona is a rare disease company within CNS.

Some key takeaways on the corporate level include:

  • CSO and Co-founder Jorgen Drejer gave his perspective on the transformational 2020 for the company (Saniona)
  • Saniona recently received pre-IND feedback from the FDA (our note here). CSO Jorgen Drejer confirmed a clear pathway in Prader-Willi syndrome (PWS), but some regulatory challenges the company needs to address within Hypothalamic Obesity (HO). On our side, we got confirmation that the addressed issues within HO are rather common when addressing a rare disease (Saniona)
  • There’s been a breakthrough of prophylactic treatment in migraine in recent years, with antibodies targeting the calcitonin gene-related peptide (CGRP)/receptor. This is a neuropeptide/receptor expressed in trigeminal ganglia nerves, released after nerve activation. CEO Peter Nordkild explained why these antibodies have poor efficacy in menstrual migraine (MM), or at least haven’t been evaluated solely in this subset of migraine. CEO Nordkild further elaborated on why existing migraine-players might have a business interest in MM (Asarina Pharma)

Final Remarks
This was an encouraging day, with great company presentations across the seminar. After decades of an increased understanding of disease biology and genetics within CNS, we see that this field advances. Still, the real breakthroughs should be ahead of us. For investors, it calls for a high-risk, high-reward area where we would like to conclude this takeaway note with a quote from Roche Pharmaceuticals (the worlds largest pharmaceutical company) CEO at the beginning of 2020:

“We think neuroscience has the potential to be in the ’20s what oncology has been in the last decade.”

Mentioned companies