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GNMX: Details of New Gene-Subset ADHD Study. Top-Line Data Mid-2018

05/19/2017
By Brian Marckx, CFA

NASDAQ:GNMX

Q1 Results / Update: New ADHD Study Enriched for 9-Genes, Topline Data Expected Mid-2018

GNMX (NASDAQ:GNMX) reported Q1 financial results and provided an update on their clinical programs.  

Relative to the financials, operating expenses continue to largely track our expectations.  Net loss and EPS were $10.9M and ($0.29) versus our $10.5M and ($0.28) estimates.

Cash used in operating activities was $10.7M ($9.9M ex-changes in working capital).  Cash balance at Q1 quarter end was $29.2M which the company expects to be sufficient to fund operations through Q2 2018 – which is also when they expect top-line data from the newly initiated 9-gene mGluR+ADHD study to be available.  

Relative to the operational update, we learned about the initial plan for a new phase II mGluR+ADHD study which will be enriched for patients with mutations to the nine genes which showed a particularly strong response to AEVI-001.  Management also touched on plans for AEVI-001 in Autism Spectrum Disorder and provided a brief update on the anti-LIGHT severe pediatric crohn’s disease program (AEVI-002) as well as the 22Q deletion syndrome trial.  

New Phase II mGluR+Genetic Subset ADHD Study

As a reminder, in March GNMX announced that while secondary data was positive, SAGA failed to meet the primary endpoint.  Then in April the company announced additional data from SAGA which showed that ADHD patients which had mutations to nine of the 273 genes in the mGluR network showed a clinically and statistically significant response to AEVI-001 based on the primary and secondary endpoints.  See below for full details.    

This new phase II study will enrich for this high-responder population – that is, for mutations to CNTN4 and eight specific (still undisclosed) GRMs and neurodevelopmental genes.  While specific details of the study including expected total enrollment still need to be finalized, management provided the following initial details;

➢ N = still to be determined but expected to be smaller than SAGA (N=96)
➢ Multi-center randomized, placebo-controlled
➢ Ages 6 – 17 (i.e. younger population should reduce placebo response)
➢ Enrollment will be staged adaptive with CNTN4 patients (N = ~40-70) enrolling first followed by the other genes 

The staged adaptive design allows for both super-enrichment (our word) as well as for assessing response on the broader 9-gene population.  Given the particularly robust response from CNTN4 patients (again, more detail below) in SAGA, this could be the low-hanging fruit in terms demonstrating clinically significant response in this follow-on study, although also represents about 50% of the prevalence among the general ADHD population as compared to the full 9-gene subset.  Clearly, the objective will be to demonstrate a clinically significant response within a broader population (i.e. 9 genes) in order to increase the potential market size – this follow-on study may do just that given that the 9-gene subset data from SAGA was also quite compelling - similarly demonstrating statistical significance on the primary and key secondary endpoints. 

Management expects enrollment to begin by the end of June 2017 and is shooting to have top-line results by mid-2018, although it is not yet clear whether that will include only the CNTN4 patients or the whole 9-gene set. 

Assuming positive results, the pathway forward is expected to include two phase III studies within the genetic subset, one in patients 6-12 years and one in 6-17 years.    

Autism Spectrum Disorder
 
CNTN4 mutations have also been associated with more severe phenotypes (i.e. disruptive behavior, anger control, etc) and other disorders including Autism Spectrum Disorder.  GNMX is currently working with CHOP to better understand that relationship and related phenotypes and noted that depending on the results, may initiate dialogue with FDA (potentially later this year) regarding a potential CNTN4 orphan indication program in ASD.  
 
22q11.2 Deletion Syndrome

Phase I/II study commenced in October 2016 although enrollment has been progressing slowly due to the characteristically fragile nature of patients with this disorder. Management noted on the Q1 call that they expect to have data on two patients later this year.   
 
Anti-LIGHT Severe Pediatric Crohn’s Phase 1/2 Signal Finding Study

Open label.  Clinical endpoints will be endoscopic evaluation (i.e. healing) and the Pediatric Crohn s
Disease Index.  While commencement of the study has been delayed, the company now expects it to begin in Q2 2017 and to have initial data in 2H 2017.

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