The Quiet Revolution in Central Nervous System Therapies (Part 2): Outlook for 2021 and Beyond
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The Quiet Revolution in
Central Nervous System
Therapies (Part 2):
Outlook for 2021 and Beyond
By Varun Renjen, M.D. and Jose Quejada, Ph.D.
White Paper
Blue Matter, Copyright © 2021White Paper
The Quiet Revolution in Central
Nervous System Therapies (Part 2):
Outlook for 2021 and Beyond
In Part 1, we spent a great deal of to fuel those advancements. The CNS Market Now
time looking backward. We discussed Here, we turn our gaze forward. Top Companies
the “quiet revolution” that has been 2021 promises to be a big year for the • In 2019, Biogen was the CNS mar-
taking place in CNS over the past broader CNS market, and we outline ket leader with 10% market share,
several years. Interesting advances some of our thoughts on why we bolstered by Tecfidera (for multiple
are being made in a range of CNS think that’s the case. Developments sclerosis), the leading CNS product
therapies while other areas, such as during the year ahead are likely to of 2019 with $4.4 billion in sales.
oncology, tend to get more attention. have far-reaching implications, so let’s • By 2022, Roche could overtake
We also shared how investment activ- jump in and see what might happen. Biogen with Ocrevus (also for MS),
ity has been building in CNS, helping with forecasted sales of $7.7 billion
by 2025.
Top Products
Predictions for 2021 Onward: Looking at the • MS therapies held the top spot
with 7 of the top 15 best-selling
CNS Space Through a Disease Specific-Lens CNS products in 2019, with 25% of
the CNS market. Biogen’s Tecfidera
and Roche’s Ocrevus led the way.
• Antipsychotics were in second
A Word About COVID-19 The FDA had used remote inspec-
place with 13% market share.
2021 will be an interesting year in tions due to COVID-19 restrictions.
• Antiepileptics were in third place
CNS but there is the added complexity Even though an FDA advisory panel
with a 12% share.
of COVID-19. As everyone is aware, had backed Alkermes, the agency
the FDA issued dramatically accelerat- decided to take the more conserva- Source: “A view into the central nervous system disor-
ed approvals for the current COVID-19 tive approach.1 ders market.” Biopharma Dealmakers. Sept. 1, 2020
vaccines. As a result of those acceler- • The FDA rejected Supernus for two
ated approvals, the FDA is now under applications (SPN-812 for ADHD and
greater pressure to display strength SPN-830 for Parkinson’s disease). could lead to larger deals in areas with
and rigor by increasing the level of Although no reason was given by greater manufacturing complexity,
scrutiny on subsequent drug approvals. the FDA, Supernus voiced that it such as gene therapies. This trend has
This is complicated by travel restric- had to do with chemistry, manufac- already started in December with Eli
tions that are barring inspectors from turing, and controls (CMC) issues.2 Lilly acquiring Prevail Therapeutics.
visiting manufacturing facilities, result-
ing in some recent rejections due We expect this trend of heightened Alzheimer’s Disease
to manufacturing concerns. Some FDA scrutiny (often related to manu- Amyloid Plaques
notable recent examples include: facturing) to continue through 2021. It is hard to overstate the impor-
• The FDA rejected Alkermes’ Given that this will tend to affect tance of the FDA’s upcoming deci-
schizophrenia and bipolar 1 drug smaller companies, those companies sion on Biogen/Eisai’s aducanumab.
(ALKS3831) due to manufacturing might need to focus more resources Aducanumab has had a rocky clinical
concerns. There were no problems on manufacturing or partner with larg- trial history with Biogen originally
with the data, but the FDA was con- er companies that have the needed discontinuing its development due
cerned with tablet coating issues. expertise already fully developed. This to a statistics-based futility analysis.
2 Blue Matter, Copyright © 2021White Paper
The Quiet Revolution in Central Nervous System Therapies (Part 2): Outlook for 2021 and Beyond
After re-analyzing their clinical trial an impact in Alzheimer’s is masitinib • Eli Lilly has a P-Tau biomarker assay
data collected at the end of phase 3 by AB Science. In December of 2020, in initial development for early-stage
trials, Biogen decided to pursue FDA the company reported promising re- Alzheimer’s Disease. Again, one
approval. In early November, an FDA sults from a phase IIb/III clinical trial in wonders if payers will cover the test
advisory panel reviewed the drug and confirmed mild to moderate Alzheim- if there is no effective treatment nor
clinical trial data negatively despite the er’s disease. Masitinib is a selective preventative measures available.7
FDA being more positive about it. oral tyrosine kinase inhibitor that is
Given the inconsistent data and the thought to act by either abating neuro- Predictions:
potentially tougher stand of the FDA, inflammation or by targeting amyloid- 1. If aducanumab is not approved,
we think there’s a very good chance beta signaling and tau phosphorylation the novel diagnostics will be less
the drug will be rejected. Moving for- through Fyn Kinase.4 likely to be adopted and payers
ward, Biogen and Eisai may continue Also in December 2020, Novo Nord- may find it less compelling to
to look into aducanumab as a potential isk announced plans to initiate a phase cover.
therapy for smaller sub-sections of 3a program to investigate the safety 2. We will see an acceleration of
Alzheimer’s disease, focusing treat- and efficacy of once-daily oral sema- alternative approaches to treat
ment and limiting its overall market glutide in early Alzheimer’s disease. Alzheimer’s focusing on metabo-
potential. Regardless, if aducanumab Interestingly, semaglutide (a GLP-1 lism (as the notion that Alzheim-
fails to garner FDA approval, we think receptor agonist) is an anti-diabetic er’s is like “type 3 diabetes” gains
there are a couple of implications. medication used to treat type 2 diabe- more traction), preventing neu-
tes but has been shown to have the ronal senescence, and a stronger
Predictions: potential to decrease the occurrence focus on Alzheimer’s as a Tauopa-
1. Biogen will need to do more of developing dementia. If impaired in- thy rather than (or in addition to)
deals. Biogen recently teamed up sulin signaling can be found to contrib- pathogenic amyloid beta accu-
with Sage therapeutics to develop ute to the development of dementia, mulation. These will spur M&A
therapies for depression in a deal it would bolster the hypothesis that activity and development in these
potentially worth over $3B. They Alzheimer’s disease may be “type 3 new areas.
will jointly develop and commer- diabetes” and open a new therapeutic 3. New government focus will help
cialize zuranolone (SAGE-217) for avenue that can be targeted to treat bolster research into Alzheimer’s
major depressive disorder (MDD) the disease.5 disease as these failures may
and postpartum depression (PPD) make pharmaceutical companies
as well as SAGE-324 for tremors.3 Diagnostics more hesitant to find a cure or
2. The amyloid hypothesis will fail, There is also work underway on therapy.
even if Biogen and Eisai con- diagnostics for Alzheimer’s disease.
tinue development of their other However, the success of these tests Duchenne Muscular Dystrophy
amyloid antibody; Ban2401. For will be dependent on the availability of The Duchenne muscular dystrophy
others in the field, there may be an effective Alzheimer’s therapy: (DMD) space has been getting more
a shift to other Alzheimer’s drug • For example, C2N Diagnostics has crowded with Solid Biosciences and
targets or an exit of the mar- released the first blood test for Pfizer in the mix. There is now more
ket altogether. Aducanumab is Alzheimer’s disease in the US, pressure on companies like Sarepta
only the most recent miss in the targeting patients 60 years old or that focus so much on DMD to bring
amyloid field, following bap- older. The test is not covered by therapies to market.
ineuzumab (Pfizer and J&J) and payers, costs $1,250, and has not Sarepta gained approval of Exondys
solanezumab (Eli Lilly). been approved by the FDA. This is 51 on an open label 12-person study.
C2N’s only marketed product and, If the FDA takes a tougher stand, as
There is hope in Alzheimer’s though, without a treatment, there may be we speculate, it may make things dif-
as other treatment approaches are be- little reason for patients to use the ficult for Sarepta’s SRP-9001. Because
ing developed. A great example of a diagnostic and learn they have the of COVID-19, Sarepta is proposing
therapy that has the potential to make disease.6 SRP-9001-103 (study 103), which is an
3 Blue Matter, Copyright © 2021White Paper
The Quiet Revolution in Central Nervous System Therapies (Part 2): Outlook for 2021 and Beyond
open label study in up to 10 patients rooms”) is intended to help patients results in major depressive
to evaluate the safety and expression with treatment-resistant depression. disorder (MDD) from its phase
of SRP-9001 and accelerate Sarepta’s It’s in Phase 2b clinical trials, which 3 trial of AXS-05 (an experi-
validation of it.8 It remains to be seen should end in December 2021. COM- mental non-competitive NMDA
whether a tougher FDA will accept PASS Pathways has been successful antagonist). The NDA for both
this study design. Incidentally, results in raising money to fund its research, AXS-05 and AXS-07 are expect-
from SRP-9001-102 (study 102), a including interest from Otsuka.9 ed in the first quarter of 2021.13
double-blind placebo controlled phase In 2018, COMP360 gained break- b. ACADIA pharmaceuticals has
2 study of SRP-9001 with 41 patients, through designation by the FDA.10 Its submitted an NDA for Nuplazid
are expected in 2021. success or failure will help set the (already approved for Parkin-
tone for “taboo” drugs moving for- son’s disease psychosis) for
Predictions: ward. Already, clinical trial successes dementia-related psychosis.
1. Early success leveraging small of psychedelic treatments has led The expected PDUFA date is
clinical trials in DMD have led to to the launch of investment funds April 3, 2021.14
the continuation of these types of like Neo Kuma Ventures which is c. Cerevance’s CVN058, a small
studies. But, although smaller tri- completely dedicated to psychedelic molecule therapy for cognitive
als in the CNS space are feasible, healthcare investment and is the first impairment associated with
they need to be well-designed of its kind in Britain.11 Schizophrenia (CIAS), may
as the FDA grows more skeptical More traditional treatments have move into a phase 2 study next
about the data generated. also seen recent successes. For year. If approved, it will be the
2. The FDA’s response to small clini- example, in March of 2019, Sage first therapy to treat CIAS in
cal trials—especially with adeno- Therapeutics’ Zulresso was approved. Schizophrenia.15
associated viruses (AAV)—will set It is a GABAA agonist for women with d. In November, Praxis had a
the tone for other genetic treat- postpartum depression. The IV medi- clinical hold imposed for its
ments and the FDA may not be as cation costs about $34,000 per treat- lead MDD therapy. A week
positive as before. ment course that has been shown to later, Praxis announced it has
3. As the competition for DMD be effective for up to 30-days. a path to lifting the hold. The
therapies is heating up, there is necessary animal tox studies
increased pressure for Sarepta to Predictions: should be completed in the first
succeed in developing SRP-9001. If 1. We will see continued investment quarter of 2021 with a phase
this fails, they may lose their foot- growth in treatments for psychiat- 2/3 study in MDD starting in the
hold in the market to Pfizer and/or ric disorders, with resources go- first half of 2021.16
Solid Biosciences, affecting Sarep- ing into both “taboo” treatments, 3. The use of digital therapeutics
ta’s overall success and valuation. like those to be funded by Neo supporting psychiatric disorders
Kuma Ventures, and more tradi- will continue to grow. This is
Psychiatric Disorders tional medications (albeit with driven in part by the COVID-19
The psychiatric illness space has newer treatment paradigms and pandemic, which simultaneously
some important readouts coming in different mechanisms of action in increases the need for support
2021. It will be a “make or break” many cases). and drives patients to more digi-
year for some. There is interest- 2. Smaller companies that are heav- tal interactions.
ing movement in traditional areas of ily invested in CNS could sink or
therapy for mental illness, as well as swim during 2021. Some exam- Pain
with therapies that are considered ples include: Given the much-publicized opioid
more “taboo” such as ketamine-based a. Axsome Therapeutics, in addi- crisis in the US, it’s no surprise that
treatments like Spravato and others. tion to recent success in phase most of the action in this space will
COMPASS Pathways’ COMP360, a 3 clinical trials for AXS-07 for focus on either non-opioid treatments
synthetic derivative of psilocybin (the treatment of acute migraine12, for pain or therapies for opioid use
active ingredient in “magic mush- shows positive long-term dependency (OUD).
4 Blue Matter, Copyright © 2021White Paper The Quiet Revolution in Central Nervous System Therapies (Part 2): Outlook for 2021 and Beyond Non-Opioid Treatments for Pain successful, will cause others to follow pharmaceutical companies have been Two high-profile monoclonal antibod- in their example. advancing in the gene therapy space, ies serving as nerve growth factor working to bolster their capabilities. (NGF) inhibitors have important mile- Government interest in OUD During 2021, we will see how suc- stones in early 2021. The NGF drug Related to the fight against OUD, cessful many of these efforts will be. class itself has been shelved for nearly BioXcel Therapeutics announced a If things work out well for these larger a decade due to skeletal side effects grant by the US Department of De- companies, it may encourage others but the opioid crisis pushed the FDA fense (DOD) for BXCL501 for PTSD to follow their leads. Some notable to lift its five-year clinical hold in 2015. related to alcohol and substance examples include: One NGF therapy being developed abuse disorder (ASUD). BXCL501 is • UCB acquired Handl Therapeutics, is tanezumab, a joint effort by Eli Lilly an orally dissolving film formulation of which focuses on AAV-based gene and Pfizer. In March 2020, the FDA dexmedetomidine (dex) in late stage therapies for neurodegenerative dis- accepted a Biologics License Applica- development for agitation and opioid ease. UCB has also partnered with tion (BLA) for subcutaneous use of 2.5 withdrawal symptoms. Lacerta Therapeutics to study AAV- mg tanezumab in patients with chronic based therapies for CNS diseases pain due to moderate-to-severe os- Prediction: with high unmet needs. teoarthritis with inadequate pain relief 1. We will see continued investment • Bayer is working on gene therapies, with other analgesics. The PDUFA date growth related to: stem cell therapies, gene augmen- is December 2020.17 Tanezumab has a. Non-opioid treatments for pain. tation, gene editing and allogeneic not been without controversy, how- b. Treatments for opioid use de- cell therapies. They acquired Ask- ever as Tanezumab-treated patients pendency. Bio and BlueRock therapeutics in have experienced more joint safety 2019 for $600M. AskBio has signed events and total joint replacements Gene Therapies deals with Pfizer in DMD. BlueRock than those treated with placebo.18 In 2006, Dr. Shinya Yamanaka and has its own Parkinson’s program to The other NGF therapy, fasinumab, his team discovered a method to re- regenerate lost dopamine-producing is being brought to market through program mature cells to become plu- cells. partnership between Teva Pharmaceu- ripotent, which means they can give • In December 2020, Eli Lilly bought ticals and Regeneron. Phase 3 results rise to multiple different cell types. Prevail, a neurodegenerative dis- showed that 1mg fasinumab induced This finding led to the establishment ease player, for $1.04 billion to help statistically significant improvement in of induced Pluripotent Stem Cells reinforce its early-stage pipeline.23 the measurement of pain and physical (iPSC) and his Nobel Prize in 2012.21 Prevail has AAV9 therapies in the function among osteoarthritis patients. With its discovery, the scientific pipeline, both in phase 1/2: 1.) However, there are joint issues as- community was elated to find an alter- PR001 is targeted towards Parkin- sociated with fasinumab, as well. This native to embryonic stem cells (ESC). son’s disease with GBA1 mutations has caused the developers to convert However, 14 years since, there are still and neuropathic Gaucher disease a phase 3 trial to a long-term safety no iPSC therapies approved in the US. (nGD); 2.) PR006 is a single-dose study.19 The scientific community is coming to AAV9 for patients with frontotem- Another interesting situation grips with the shortcomings of iPSC poral dementia with GRN mutations involves Trevena. That company technology as a treatment including (FTD-GRN). received approval for OLINVYK in cost of production, genomic instabil- August of 2020. OLINVYK targets the ity of the cells, and the potential for Although big moves have already Mu opioid receptor. Trevena hopes to immunogenicity.22 As such, the initial occurred in this space, there are still address problems with opioid abuse promise of personalized medicine many smaller biotech companies by limiting OLINVYK use to short-term heralded by the discovery of iPSC has focused on CNS and have found IV administration in hospitals or other been tampered down, at least until success in garnering funding. A controlled clinical settings.20 Data these shortcomings are solved. prime example of this is Taysha Gene from 2021 will tell if OLINVYK will be Similarly, gene therapies have held a Therapies. Recently launched, in April widely adopted and if it will help stem lot of initial promise and which may be of 2020, Taysha raised $95 million in the potential for opioid abuse which, if tested in 2021. In recent years, large series B funding in July, had a $100M 5 Blue Matter, Copyright © 2021
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The Quiet Revolution in Central Nervous System Therapies (Part 2): Outlook for 2021 and Beyond
IPO in September, and plans to invest Predictions: 2. Early successes will lead to new
$75 million to build a commercial-scale 1. Several big pharmaceutical com- or continued investment into
manufacturing facility in North Caroli- pany players will initiate investi- smaller biotech companies like
na. All of this was achieved with a lead gation into gene therapies, shift- Taysha Gene Therapies as the
program that was just recently cleared ing the spotlight away from the pharmaceutical industry realizes
by Health Canada for phase 1/2 clinical smaller biotech firms that have the current and near-future poten-
trials in infantile GM2 gangliosidosis.24 been largely running the show. tial of the field of gene therapy.
Some General / Additional Predictions
Companies Looking to Invest Spaces That Will Be More Active • Viela Bio with inebilizumab, initiating a
• Biogen – Given the situation with for Business Development and phase 3 pivotal clinical trial in 202025
aducanumab, coupled with the Licensing • Argenx with efgartigimod, with
genericization of its main revenue In 2021, we expect the CNS space plans to submit a BLA in 2021
driver Tecfidera, investment activ- to continue to grow overall, but we following positive top-line phase 3
ity will be extremely important to think there will be higher activity in the clinical trial results26
Biogen and could have a strong sub-spaces below.
impact on how the company moves Genome Engineering
forward. It needs to address poten- Psychiatric Illnesses/Mental Health Business development and licens-
tial revenue shortfalls but quick fixes The recent substantial activity in ing in genome engineering has been
can be found, just like the recent mental health in both previously running hot with major deals struck by
move to partner with Sage Thera- ‘taboo’ and traditional medication is Bayer, UCB, and Eli Lilly. As the prom-
peutics. expected to continue into 2021. This ise of genome engineering bears out,
• Amgen – Amgen will look to build market has a distinct need for new there will be continued deals made
upon its migraine portfolio, driven therapies and our understanding of focusing on AAVs and other platform
by Aimovig, a calcitonin gene-relat- the brain is reaching a point where technologies that enable gene therapy.
ed peptide (CGRP) antagonist. help can be delivered. Axome Thera- Companies to watch include Precision
• J&J – J&J has developed a robust peutics, with its clinical trial success in Bio, BioXcel, Neuron23, LocanoBio,
portfolio of drugs that can be further MDD (and also acute migraine treat- and Neurogene.
complemented in CNS. ment) is one company to watch.
• Novartis – Novartis will continue to Novel Approaches to CNS
expand its CNS portfolio. Neuromuscular Disease In 2020, we saw novel approaches
• Otsuka – Otsuka will move to fur- With the recent advances in gene to treating CNS diseases and we ex-
ther bolster its CNS pipeline. therapy, there has been increasing pect that these will continue in 2021.
• Eli Lilly – The recent acquisition of interest in treating neuromuscular Keep an eye on the companies below.
Prevail (see above) makes big in- diseases with defined genetic origins • Rubedo Life Sciences is advanc-
vestments from Eli Lilly a little more (e.g., DMD). Larger pharmaceutical ing therapies towards neurode-
unlikely but, historical strength in companies will continue to shore up generative conditions, age-related
CNS and recent deals may look to expertise neuromuscular disease- diseases, and muscle disorders.
grow even more in this area. related treatment Interestingly, Rubedo developed the
• UCB – UCB will make investment Outside of gene therapy, there has ALEMBIC drug discovery platform
moves to further diversify its portfo- been movement in treatments towards that identifies therapeutic candi-
lio away from epilepsy. myasthenia gravis using antibody treat- dates against senescent cells, pro-
ments. Two companies to watch are: inflammatory, and profibrotic cells.
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The Quiet Revolution in Central Nervous System Therapies (Part 2): Outlook for 2021 and Beyond
• Apple Tree Partners is funding Pre-clinical models showed re- increase expression of the healthy
the development of Nereid Thera- myelination and neuroprotection copy of SCN1A (the gene associ-
peutics with $50 million to study capabilities of CNM-Au8. Interim ated with Dravet syndrome) and
biomolecular condensates as a new results of CNM-Au8 in phase 2 clini- codon suppression.30 As mentioned
platform to develop treatments for cal trials (REPAIR-MS, REPAIR-PD) above, epilepsy’s market share
neurodegenerative diseases like showed positive results in both MS comes in 3rd in the CNS market,
ALS, Alzheimer’s, and Huntington’s and Parkinson’s disease with bioen- meaning that success in this part-
disease.27 Successful therapies ergetic improvements in NAD and nership can lead to strong interest
coming from the study of biomo- ATP levels.28 In September of 2020, from other pharmaceutical compa-
lecular condensates would bolster Clene Nanomedicine announced nies.
this platform as a viable method to plans to go public through a merger
develop CNS drugs. with Tottenham Acquisition.29 Clearly, the CNS market offers much
• Clene Nanomedicine is develop- • Trevard Biosciences is partnered to track in 2021. The “Quiet Revolu-
ing bioenergetic nanotherapeutics. with Zogenix to develop RNA-based tion” continues, and we will be sure to
Particularly, CNM-Au8 is an add-on therapies to treat Dravet syndrome keep any eye on developments, offer-
treatment for neurodegenerative and other genetic epilepsies. Their ing insights for the biopharma innova-
diseases (Parkinson’s, ALS, MS). aim is to use mRNA stabilization to tors that are operating in this space.
Endnotes:
1
Jensen, K., FDA unexpectedly rejects Alkermes schizophrenia drug, citing manufacturing concerns, Biopharma Dive, Nov. 17, 2020, https://www.bio-
pharmadive.com/news/alkermes-schizophrenia-drug-fda-rejection/589199/
2
Mast, J., FDA hits Supernus with double whammy, handing biotech a CRL and RTF on same day, Endpoints News, Nov. 10, 2020, https://endpts.com/
fda-hits-supernus-with-double-whammy-handing-biotech-a-crl-and-rtf-on-same-day/
3
Taylor, N.P., Biogen bets $1.5B on Sage’s once-failed depression drug, Fierce Biotech, Nov. 30, 2020, https://www.fiercebiotech.com/biotech/biogen-
bets-1-5b-sage-s-once-failed-depression-drug
4
Terry, Mark, AB Science’s Masitinib Shows Unusually Good Data in Late-Stage Alzheimer’s Trial, Biospace, Dec. 18, 2020, https://www.biospace.com/
article/ab-science-s-masitinib-looks-promising-in-phase-iib-iii-alzheimer-s-trial/
5
Company press release, Novo Nordisk, Dec. 16, 2020, https://www.globenewswire.com/news-release/2020/12/16/2146164/0/en/Novo-Nordisk-to-enter-
phase-3-development-in-Alzheimer-s-disease-with-oral-semaglutide.html
6
Hale, C., C2N debuts Alzheimer’s blood test for predicting amyloid plaque deposits in the brain, Fierce Biotech, Oct. 30, 2020, https://www.fiercebio-
tech.com/medtech/c2n-debuts-alzheimer-s-blood-test-for-predicting-amyloid-plaque-deposits-brain
Hale, C., Alzheimer’s blood tests show new promise, giving decades of early warning, Fierce Biotech, Jul. 28, 2020, https://www.fiercebiotech.com/
7
medtech/alzheimer-s-blood-tests-show-new-promise-giving-decades-early-warning
8
Company press release, Sarepta Therapeutics, Nov. 5, 2020, https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-thera-
peutics-commence-dosing-next-study-commercial
9
Company press release, Otsuka, Apr. 25, 2020, https://www.otsuka-us.com/discover/compass-series-b
10
Company press release, COMPASS Pathways, Oct. 23, 2018, https://www.prnewswire.com/news-releases/compass-pathways-receives-fda-break-
through-therapy-designation-for-psilocybin-therapy-for-treatment-resistant-depression-834088100.html
11
Company press release, Neo Kuma Ventures, Dec. 17, 2020, https://www.prnewswire.co.uk/news-releases/neo-kuma-ventures-britain-s-first-psychedel-
ic-healthcare-fund-launches-882630536.html
Terry, M., Axsome’s Migraine Drug Provides Relief in Phase III Trial, BioSpace, Dec. 31, 2020, https://www.biospace.com/article/axsome-s-migraine-
12
drug-provides-relief-in-phase-iii-trial
13
Keown, A., Hope Shines Through as Axsome Seeks Approval for MDD Drug, BioSpace, Dec. 1, 2020, https://www.biospace.com/article/axsome-plans-
to-seek-regulatory-approval-for-mdd-asset-following-positive-phase-iii-study/
Company press release, ACADIA Pharmaceuticals, Jul. 20, 2020, https://ir.acadia-pharm.com/news-releases/news-release-details/acadia-pharmaceuti-
14
cals-announces-us-fda-accepted-filing
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The Quiet Revolution in Central Nervous System Therapies (Part 2): Outlook for 2021 and Beyond
Company press release, Cerevance, Nov. 16, 2020, https://www.globenewswire.com/news-release/2020/11/16/2127507/0/en/Cerevance-s-CVN058-
15
Achieves-Primary-Endpoint-in-Phase-Ib-Schizophrenia-Cognition-Study.html
Mast, J., BioMarin taps AI upstart Deep Genomics for discovery pact; Praxis says it has a path to lifting clinical hold, Endpoints News, Nov. 17, 2020,
16
https://endpts.com/biomarin-taps-ai-upstart-deep-genomics-for-discovery-pact-praxis-says-it-has-a-path-to-lifting-clinical-hold/
17
Company press release, Eli Lilly, March 2, 2020, https://investor.lilly.com/news-releases/news-release-details/us-fda-accepts-regulatory-submission-
tanezumab-potential-first
18
Cush, J. Safety Concerns Offset the Modest Improvements of Tanezumab in Osteoarthritis, RheumNow, Jul. 10, 2019, https://rheumnow.com/content/
safety-concerns-offset-modest-improvements-tanezumab-osteoarthritis
Tong, A., Regeneron adds more positive Ph. III data for its NGF program — but safety is still a big concern, Endpoints News, August 6, 2020, https://
19
endpts.com/regeneron-adds-more-positive-phiii-data-for-its-ngf-program-but-safety-is-still-a-big-concern/
20
Snyder Bulik, B., Trevena preps low-key launch, responsible marketing for long-awaited opioid Olinvyk, Fierce Biotech, Aug. 17, 2020, https://www.
fiercepharma.com/marketing/trevena-preps-low-key-launch-for-finally-approved-opioid-olinvyk-promises-responsible
21
Nobel Prize, Shinya Yamakana Facts, https://www.nobelprize.org/prizes/medicine/2012/yamanaka/facts/, accessed Dec. 23, 2020
del Carmen Ortuño-Costela, M., et al, The Challenge of Bringing iPSCs to the Patient, International Journal of Molecular Sciences, Dec. 19, 2013,
22
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6940848/
Blankenship, Kyle, Eli Lilly picks up gene therapy player Prevail in deal worth $1B+, making good on bolt-on pledge from earlier this year, Endpoints
23
News, Dec. 15, 2020, https://endpts.com/eli-lilly-picks-up-gene-therapy-player-prevail-in-deal-worth-1b-making-good-on-bolt-on-pledge-from-earlier-this-
year/
Kansteiner, F, Young but ambitious Taysha lays out $76M for commercial gene therapy plant, Fierce Pharma, Dec. 22, 2020, https://www.fiercepharma.
24
com/manufacturing/young-but-mighty-taysha-lays-out-76m-for-commercial-gene-therapy-plant
25
ClinicalTrials.gov, https://clinicaltrials.gov/ct2/show/NCT04524273, accessed Dec. 23, 2020
Company press release, Argenx, Oct. 5, 2020, https://www.argenx.com/news/argenx-presents-additional-efgartigimod-data-global-phase-3-adapt-trial-
26
myasthenia-gravis
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8 Blue Matter, Copyright © 2021White Paper
New Ideas. Better Results.
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This paper authored Varun Renjen, M.D. and Jose Quejada, Ph.D.
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