1st Order Pharmaceuticals Inc: 1OP-2198

[Autumnly]

Xenon Announces Final Results of XEN1101 Phase 1 Clinical Trial and Update on XEN901 Phase 1 Clinical Trial at the American Epilepsy Society (AES) Annual Meeting

• XEN1101 is a Kv7 potassium channel modulator being developed for the treatment of epilepsy and potentially other neurological disorders
• Xenon today outlined plans for a XEN1101 Phase 2 clinical trial, with patient enrollment expected to begin early in the new year
• Pharmacokinetics: The PK profile of XEN1101 (including an effective half-life greater than 24 hours) supports a once-per-day dosing schedule with expected steady state in approximately one week without the need for titration
• Safety: The majority of adverse events (AEs) were mild or moderate, resolved spontaneously and were consistent with antiepileptic drugs of this class. Sedation (including somnolence, drowsiness) and dizziness (including light-headedness and presyncope) were the most common AEs, while mild cognitive effects (memory and speech impairment) and blurred vision were also observed in a dose dependent manner. There were no SAEs, deaths, or clinically significant ECG or laboratory findings. Phase 1 results suggest that that XEN1101 is generally safe and well tolerated in the doses examined (single doses of up to 30 mg and multiple doses of up to 25 mg once daily).

About the XEN1101 Phase 2 Clinical Trial

• patient enrollment expected to begin early in 2019
• randomized, double-blind, placebo-controlled, multicenter study to evaluate the clinical efficacy, safety and tolerability of XEN1101 administered as adjunctive treatment in adult patients aged 18 to 75 years diagnosed with focal epilepsy
• Approximately 300 patients
• After screening, patients will have 8 weeks of baseline to assess frequency of seizures, followed by 8 weeks of treatment and a 4-week post treatment follow-up period

 

[Deamon22]

@Autumnly thank you for providing updates

 

[friend74]

Are there any (hopefully good) news regarding the latest trials or the drug itself?

 

[Autumnly]

Are there any (hopefully good) news regarding the latest trials or the drug itself?

XEN1101 (in Phase 2)

• Dr. Pimstone continued, "Within our XEN1101 'X-TOLE' Phase 2b clinical trial, as with other trials ongoing in our industry, we are seeing a significant reduction in new patient enrollment for numerous reasons related to the COVID-19 pandemic; therefore, we have adjusted guidance with topline data now anticipated in the first half of 2021, which is, of course, dependent upon the rate of patient enrollment throughout the remainder of this year. Despite this shift in guidance, we are pleased with our progress in the X-TOLE study to date.
• We are particularly encouraged by our review of safety data on a blinded basis, which suggests that XEN1101 is currently being well tolerated. The rate of discontinuations to date in the study is lower than modeled. Of note, to date, more than 90% of subjects from the double-blind portion of the trial have entered the open-label extension phase. Based on this review of blinded safety data to date, we believe tolerability is well within the modeled parameters, and, therefore, we do not believe an interim analysis is required. Our goal is to complete the study, as planned, in the shortest possible time frame.
• source

XEN496 (Phase 3 anticipated to start in 2020)

• U.S. Food and Drug Administration (FDA) recently granted Fast Track designation for the investigation of XEN496 for the treatment of seizures related to KCNQ2 developmental and epileptic encephalopathy (KCNQ2-DEE)
  
• Feedback from the FDA regarding the Phase 3 clinical trial design is expected in the second quarter of 2020, and the Phase 3 clinical trial in KCNQ2-DEE is anticipated to start in 2020, dependent upon the ability to initiate clinical sites and patient enrollment given the ongoing COVID-19 pandemic.
• Dr. Pimstone added, "We are also pleased to report that we recently completed a pharmacokinetic, or PK, study testing XEN496, our proprietary pediatric formulation of ezogabine, in healthy adult volunteers. The PK profile observed for XEN496 appears to be comparable to historical PK data for immediate-release ezogabine tablets, with XEN496 showing similar absorption and elimination curves, and we are excited that these data support Xenon's planned Phase 3 clinical trial of XEN496 in patients with KCNQ2-DEE.
  
• source

 

[UHPTS]

Let's assume that FDA approves XEN-496. Does that approval apply to European countries as well? What is the mechanism?

 

[Lucifer]

XEN1101 (in Phase 2)

• Dr. Pimstone continued, "Within our XEN1101 'X-TOLE' Phase 2b clinical trial, as with other trials ongoing in our industry, we are seeing a significant reduction in new patient enrollment for numerous reasons related to the COVID-19 pandemic; therefore, we have adjusted guidance with topline data now anticipated in the first half of 2021, which is, of course, dependent upon the rate of patient enrollment throughout the remainder of this year. Despite this shift in guidance, we are pleased with our progress in the X-TOLE study to date.
• We are particularly encouraged by our review of safety data on a blinded basis, which suggests that XEN1101 is currently being well tolerated. The rate of discontinuations to date in the study is lower than modeled. Of note, to date, more than 90% of subjects from the double-blind portion of the trial have entered the open-label extension phase. Based on this review of blinded safety data to date, we believe tolerability is well within the modeled parameters, and, therefore, we do not believe an interim analysis is required. Our goal is to complete the study, as planned, in the shortest possible time frame.
• source

XEN496 (Phase 3 anticipated to start in 2020)

• U.S. Food and Drug Administration (FDA) recently granted Fast Track designation for the investigation of XEN496 for the treatment of seizures related to KCNQ2 developmental and epileptic encephalopathy (KCNQ2-DEE)
  
• Feedback from the FDA regarding the Phase 3 clinical trial design is expected in the second quarter of 2020, and the Phase 3 clinical trial in KCNQ2-DEE is anticipated to start in 2020, dependent upon the ability to initiate clinical sites and patient enrollment given the ongoing COVID-19 pandemic.
• Dr. Pimstone added, "We are also pleased to report that we recently completed a pharmacokinetic, or PK, study testing XEN496, our proprietary pediatric formulation of ezogabine, in healthy adult volunteers. The PK profile observed for XEN496 appears to be comparable to historical PK data for immediate-release ezogabine tablets, with XEN496 showing similar absorption and elimination curves, and we are excited that these data support Xenon's planned Phase 3 clinical trial of XEN496 in patients with KCNQ2-DEE.
  
• source

Out of those two which one was the Trobalt reformulation?

 

[Greg Sacramento]

There's about 600 bio tech public trading companies that are working on conditions that relate to a cause of tinnitus. Many bio tech companies have associated researchers where some their names have been mentioned here. The tinnitus space is well managed by money management.

I like Xenon. Bio tech managers and their medical researchers have become a little concerned with blurry vision. The stock price has dropped a bit.

I also like VistaGen. The stock price is under a dollar. It they hit goals, stock price will rocket. If not, then it will probably be worth less. https://www.vistagen.com/pipeline

 

[Mathew Gould]

There's about 600 bio tech public trading companies that are working on conditions that relate to a cause of tinnitus. Many bio tech companies have associated researchers where some their names have been mentioned here. The tinnitus space is well managed by money management.

I like Xenon. Bio tech managers and their medical researchers have become a little concerned with blurry vision. The stock price has dropped a bit.

I also like VistaGen. The stock price is under a dollar. It they hit goals, stock price will rocket. If not, then it will probably be worth less. https://www.vistagen.com/pipeline

600 companies... You would think 1 could get lucky with tinnitus in the next 2-3 years or sooner.

 

[UHPTS]

Out of those two which one was the Trobalt reformulation?

Both, I think.

 

[Greg Sacramento]

You would think 1 could get lucky with tinnitus in the next 2-3 years or sooner.

They have FDA fast track.
Read the information in the dark blue area within this link. The publication research support section will take hours to read.

Their research on ion channels and receptors is amazing.

AV -101 might just replace benzos, antidepressants and anticonvulsants (such as gabapentin and pregabalin). No taper is needed.
https://www.vistagen.com/pipeline/av-101/neuropathic-pain

I expect that they will offer more stock shares and then money management will take positions.

 

[UHPTS]

They have FDA fast track.
Read the information in the dark blue area within this link. The publication research support section will take hours to read.

Their research on ion channels and receptors is amazing.

AV -101 might just replace benzos, antidepressants and anticonvulsants (such as gabapentin and pregabalin). No taper is needed.
https://www.vistagen.com/pipeline/av-101/neuropathic-pain

I expect that they will offer more stock shares and then money management will take positions.

For AV-101 I only found one mention on tinnitus in this document:

https://ir.vistagen.com/all-sec-filings/content/0001654954-20-007107/0001654954-20-007107.pdf

They received fast track for AV-101 back in 2018, whatever that means for the drug to hit the market.

 

[Utopia]

There's about 600 bio tech public trading companies that are working on conditions that relate to a cause of tinnitus.

@Greg Sacramento
Where can I find a list of these 600 companies? Would love to read more about them. Thank you.

 

[Greg Sacramento]

@Utopia There is no official list. These are companies that I have found by researching. It would be hard to keep an updated list as that would be too time consuming. Some companies are working on medical conditions such as jaw and neck diseases that may cause a very small percentage tinnitus. Some others are working on medical conditions that may have effect from having tinnitus such as anxiety and eye problems.

Above, less than two weeks ago, I mentioned VistaGen, a company that has been around for a long time. The volume started to increase and price has climbed from 44 cents to 75 cents. Today a wealth management firm said that the stock should reach 3.00 a share. The price for a few minutes increased to 95 cents, but soon came back down to 75 cents. It appears that this firm had preloaded clients over the last ten days and did a pump and dump as large sell asks were already into the system. VistaGen has the FDA go ahead for a Phase III study and if they are successful, then price will ready rocket.

Wealth management is deep into the tinnitus and medical space as their goal is to get clients and make money. Most in the tinnitus space are wealth management players, and this makes it difficult to know what treatments and advice may be for real and not just the wind blowing.

 

[tommyd87]

@Utopia There is no official list. These are companies that I have found by researching. It would be hard to keep an updated list as that would be too time consuming. Some companies are working on medical conditions such as jaw and neck diseases that may cause a very small percentage tinnitus. Some others are working on medical conditions that may have effect from having tinnitus such as anxiety and eye problems.

Above, less than two weeks ago, I mentioned VistaGen, a company that has been around for a long time. The volume started to increase and price has climbed from 44 cents to 75 cents. Today a wealth management firm said that the stock should reach 3.00 a share. The price for a few minutes increased to 95 cents, but soon came back down to 75 cents. It appears that this firm had preloaded clients over the last ten days and did a pump and dump as large sell asks were already into the system. VistaGen has the FDA go ahead for a Phase III study and if they are successful, then price will ready rocket.

Wealth management is deep into the tinnitus and medical space as their goal is to get clients and make money. Most in the tinnitus space are wealth management players, and this makes it difficult to know what treatments and advice may be for real and not just the wind blowing.

There will be some success soon with this treatment. This is inevitably why we are seeing wealth firms invest in this industry in droves. It will lead to big benefits financially when something is found particularly in the restoration of hearing arm.

 

[Greg Sacramento]

@tommyd87

There will be some success soon with this treatment. This is inevitably why we are seeing wealth firms invest in this industry in droves.

Not sure why you gave me a creative rating for my post above, but I agree that lots of medical discoveries have been made as I have posted about that many times. The company that I mentioned above has seen a nice price increase since my first mention, but today in after hours it took a little hit because of a recent share offering that was previously known to seasonal investors. This company has a sales and research agreement with another firm that's not a good profit deal. What I care about is their research for anxiety and pain and hoping for products to come to market..

Been doing medical research and investing in medical since the 70's. I have degrees in both areas, but that is worth about two cents. Caution is always needed with the smaller bio techs. For smaller bio techs, reverse splits, increased creative financing and issue of more shares is almost always hurtful to investors. Everyday of the trading week, within minutes, there's several bio techs that increase 50%, 100% or more in price. Some continue to go up and others will fall back down and sometimes very quickly.

 

[tommyd87]

@tommyd87

Not sure why you gave me a creative rating for my post above, but I agree that lots of medical discoveries have been made as I have posted about that many times. The company that I mentioned above has seen a nice price increase since my first mention, but today in after hours it took a little hit because of a recent share offering that was previously known to seasonal investors. This company has a sales and research agreement with another firm that's not a good profit deal. What I care about is their research for anxiety and pain and hoping for products to come to market..

Been doing medical research and investing in medical since the 70's. I have degrees in both areas, but that is worth about two cents. Caution is always needed with the smaller bio techs. For smaller bio techs, reverse splits, increased creative financing and issue of more shares is almost always hurtful to investors. Everyday of the trading week, within minutes, there's several bio techs that increase 50%, 100% or more in price. Some continue to go up and others will fall back down and sometimes very quickly.

I didn't mean to give that rating, it wasn't intentional lol. That makes sense about the investment information. It is I guess why when we see shares for certain products fall somewhat significantly it can often be just for market related moves like changes in management, deals and profits/losses and nothing else.

 

[Paulmanlike]

@tommyd87

Not sure why you gave me a creative rating for my post above, but I agree that lots of medical discoveries have been made as I have posted about that many times. The company that I mentioned above has seen a nice price increase since my first mention, but today in after hours it took a little hit because of a recent share offering that was previously known to seasonal investors. This company has a sales and research agreement with another firm that's not a good profit deal. What I care about is their research for anxiety and pain and hoping for products to come to market..

Been doing medical research and investing in medical since the 70's. I have degrees in both areas, but that is worth about two cents. Caution is always needed with the smaller bio techs. For smaller bio techs, reverse splits, increased creative financing and issue of more shares is almost always hurtful to investors. Everyday of the trading week, within minutes, there's several bio techs that increase 50%, 100% or more in price. Some continue to go up and others will fall back down and sometimes very quickly.

Would you personally buy into this company giving what you know?

I saw their pipeline and the statement that it could replace antidepressants and benzos is amazing (if it happens).

Very cautious with these penny stocks, it's not investing, it's more like gambling.

 

[Greg Sacramento]

Would you personally buy into this company giving what you know?

I saw their pipeline and the statement that it could replace antidepressants and benzos is amazing (if it happens).

No, not yet. They announced a share offering after the close on Friday. A share offering was filed with the SEC in October of last year, but only corporate, their underwriter and the SEC knew of date for share offering. I believe it will be 14 million shares. This would amount to 25% of the shares outstanding. Existing shareholders will be diluted by 25%. The offering probably will be at $0.69 or $0.70 which is a 25% discount from Friday's close of $0.92. The stock traded as low as $0.70 in Friday's after hours trading, which is independent from where it will open in Monday's pre market. The first hour of pre market trading for any stock is for investment funds only, although some stocks now trade 24 hours.

Many that will grab these shares will be hedgers who already have put options. It will take a month before a wash is completed.

After that, a reverse spilt may be needed to get a Nasdaq listing. The reserve spilt may be one share for every ten shares owned. Converted price in the area of 10 to 11 dollars per share. Dollar to dollar, one's total investment will remain the same - meaning no investment loss.

After all this, then yes, I probably would consider buying. Their meds if approved for anxiety may replace antidepressants and benzos. No taper, not even a day is said to be needed, even with neuro disease. They have FDA fast track on one, and were given the go ahead for phase III. Their neuro brain research is incredible. I read their research papers with patent discovery of a pathway or gateway to the brain in attempt to cure neuro diseases. They are using this pathway with their present phase medications. They are also working on a pain med that will also use the same pathway to the brain.

I can give you other bio techs that are still very low in price, some under a dollar that may really explode in price.

 

[grate_biff]

AV -101 might just replace benzos, antidepressants and anticonvulsants (such as gabapentin and pregabalin). No taper is needed.

Wow, this is really interesting and relevant to me as I´m taking two ADs, Gabapentin and suffer from benzo withdrawal.

Do you mean one does not have to taper these drugs and switch to AV-101 without withdrawal or do you mean you can taper off of AV-101 without tapering?

Thank you @Greg Sacramento for having so much knowledge about this med

 

[Paulmanlike]

No, not yet. They announced a share offering after the close on Friday. A share offering was filed with the SEC in October of last year, but only corporate, their underwriter and the SEC knew of date for share offering. I believe it will be 14 million shares. This would amount to 25% of the shares outstanding. Existing shareholders will be diluted by 25%. The offering probably will be at $0.69 or $0.70 which is a 25% discount from Friday's close of $0.92. The stock traded as low as $0.70 in Friday's after hours trading, which is independent from where it will open in Monday's pre market. The first hour of pre market trading for any stock is for investment funds only, although some stocks now trade 24 hours.

Many that will grab these shares will be hedgers who already have put options. It will take a month before a wash is completed.

After that, a reverse spilt may be needed to get a Nasdaq listing. The reserve spilt may be one share for every ten shares owned. Converted price in the area of 10 to 11 dollars per share. Dollar to dollar, one's total investment will remain the same - meaning no investment loss.

After all this, then yes, I probably would consider buying. Their meds if approved for anxiety may replace antidepressants and benzos. No taper, not even a day is said to be needed, even with neuro disease. They have FDA fast track on one, and were given the go ahead for phase III. Their neuro brain research is incredible. I read their research papers with patent discovery of a pathway or gateway to the brain in attempt to cure neuro diseases. They are using this pathway with their present phase medications. They are also working on a pain med that will also use the same pathway to the brain.

I can give you other bio techs that are still very low in price, some under a dollar that may really explode in price.

Greg thank you very much for your well informed posts as usual.
I would be interested in the biotechs you talk about for some high risk stock picks.

I am pretty impressed by the information. This also interests me from a patient view point. I have issues with OCD and had a depressive episode in 2017. I take regular AD's for this and have had to resort to benzos in the past.

I will definitely be keeping a close eye on this company.

 

[Oto]

They are recruiting now for XEN1101 in my country: Spain. Only for patients diagnosed with focal epilepsy.

Link here:
https://www.cun.es/investigacion/en...01-tratamiento-complementario-epilepsia-focal

Sorry Guys, What of these drugs are targeting for tinnitus?

 

[Greg Sacramento]

@Paulmanlike Details are out... 15,265,000 shares at $0.80/share. $12.5 million net. I wasn't far off on the amount of new issued shares, but I believe that they raised the price from $0.72 to $0.80 because they saw too much down pressure in after hours. I still would wait for a reverse split announcement before investment, because when a reverse spilt is announced which is now in review, the price will take another (larger) hit. Then after that, if the FDA gives positive guidance on Phase III along with the consumer medication application format being resolved, then I would consider buying.

@grate_biff Any drug that requires a taper will be needed. It is said that AV-101 will not require a taper, as so far there has been no side effects. We will know more soon.

 

[Paulmanlike]

@Paulmanlike Details are out... 15,265,000 shares at $0.80/share. $12.5 million net. I wasn't far off on the amount of new issued shares, but I believe that they raised the price from $0.72 to $0.80 because they saw too much down pressure in after hours. I still would wait for a reverse split announcement before investment, because when a reverse spilt is announced which is now in review, the price will take another (larger) hit. Then after that, if the FDA gives positive guidance on Phase III along with the consumer medication application format being resolved, then I would consider buying.

@grate_biff Any drug that requires a taper will be needed. It is said that AV-101 will not require a taper, as so far there has been no side effects. We will know more soon.

I'm going to buy after the reverse stock split. Thanks Greg. Did you buy any FREQ stock?

 

[Autumnly]

Xenon Pharmaceuticals Receives FDA Feedback and is on Track to Initiate XEN496 Phase 3 Clinical Trial for the Treatment of KCNQ2-DEE Before Year-End
October 8, 2020

Positive Opinion Received Supporting Orphan Medicinal Product Designation in Europe for XEN496 for the Treatment of KCNQ2-DEE

Spoiler

BURNABY, British Columbia, Oct. 08, 2020 (GLOBE NEWSWIRE) -- Xenon Pharmaceuticals Inc.(Nasdaq:XENE), a neurology-focused biopharmaceutical company, today provided regulatory updates on its proprietary pediatric neurology program, XEN496, a Kv7 potassium channel modulator that is a proprietary pediatric formulation of the active ingredient ezogabine being developed for the treatment of KCNQ2 developmental and epileptic encephalopathy (KCNQ2-DEE). With the U.S. Food and Drug Administration(FDA) having completed its review of the clinical trial protocol, Xenon is on track to initiate the XEN496 Phase 3 clinical trial in pediatric patients with KCNQ2-DEE before year-end.

In addition, Xenon has received a positive opinion from the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA), which recommends the granting of an orphan medicinal product designation for XEN496 for the treatment of KCNQ2-DEE. This European designation is in addition to the FDA granting Fast Track designation for XEN496 for the treatment of seizures associated with KCNQ2-DEE as well as Orphan Drug Designation (ODD) for the treatment of KCNQ2-DEE.

Dr. Simon Pimstone, Xenon's Chief Executive Officer, said, "This marks an extremely important milestone for Xenon, with the first of our proprietary product candidates now poised to enter a pivotal Phase 3 clinical trial. With feedback from the FDA, pharmacokinetic data supportive of our proprietary pediatric formulation, and considerable progress made in site selection and other preparations, we are excited to move forward with our 'EPIK' Phase 3 clinical trial studying XEN496 in pediatric patients with KCNQ2-DEE. In addition, receiving an orphan medicinal product designation for XEN496 in Europe underscores that KCNQ2-DEE is a severe, early onset epilepsy disorder and further validates Xenon's novel, 'precision medicine' approach to develop treatments for pediatric epilepsies. We continue to work closely with the medical community, genetic testing companies, and patient advocacy groups to identify potential patients for our EPIK study, which we expect to initiate before year-end."

The XEN496 Phase 3 "EPIK" clinical trial is titled "A Phase 3 Study of Adjunctive XEN496 in Pediatric Subjects with KCNQ2 Developmental and Epileptic Encephalopathy." This study is designed as a randomized, double-blind, placebo-controlled, parallel group, multicenter clinical trial to evaluate the efficacy, safety and tolerability of XEN496 administered as adjunctive treatment in approximately 40 pediatric patients aged one month to less than 6 years with KCNQ2-DEE. After screening, patients will enter a baseline period to assess the frequency of seizures. Eligible subjects will be randomized on a 1:1 basis to receive either XEN496 or placebo for approximately 15 weeks (titration and a 12-week maintenance period). At the end of treatment, there will be a period of tapering off of study drug, followed by a 28-day safety monitoring period. Patients may be considered for an open-label extension if they meet all requirements. The primary endpoint is the percent change from baseline in monthly countable motor seizure frequency during the blinded treatment period, as recorded by caregivers in a daily seizure diary. Key secondary endpoints include the proportion of patients experiencing greater than or equal to 50 percent reduction in monthly seizure frequency from baseline, caregiver global impression of change (CaGI-C) scores, and caregiver global impression of severity (CaGI-S) scores.

 

[Turgy]

will / can it help with tinnitus?

 

[Ken219]

I'd love to have normal hearing again but I take a reduction in tinnitus without restored hearing!

 

[Greg Sacramento]

Would you personally buy into this company giving what you know?

I saw their pipeline and the statement that it could replace antidepressants and benzos is amazing (if it happens).

Very cautious with these penny stocks, it's not investing, it's more like gambling.

VTGN - VistaGen --- NO - still won't buy the stock. They will need to do a reverse spilt and then another dulition of shares. Good science does not mean that the company is investor friendy. Xenon - subject in this thread today, took a price hit today on their news. Investors are concerned that the news release will be followed by dulition of shares. This often does happen, but I have not been following Xenon of late other than medical news releases.

I mentioned Novaxax in the stock market investing thread. I bought shares in May @3.95 a share and sold some a few months later @180.00 a share. A couple of weeks ago, I sold the rest. I made a killing, but I can't touch the money because it's in trust. Would not have a use for it anyways, because of my conditions and pain, I never leave my house, except to go the doctors.

 

[xyz]

Xenon Pharmaceuticals Receives FDA Feedback and is on Track to Initiate XEN496 Phase 3 Clinical Trial for the Treatment of KCNQ2-DEE Before Year-End
October 8, 2020

Positive Opinion Received Supporting Orphan Medicinal Product Designation in Europe for XEN496 for the Treatment of KCNQ2-DEE

Spoiler

BURNABY, British Columbia, Oct. 08, 2020 (GLOBE NEWSWIRE) -- Xenon Pharmaceuticals Inc.(Nasdaq:XENE), a neurology-focused biopharmaceutical company, today provided regulatory updates on its proprietary pediatric neurology program, XEN496, a Kv7 potassium channel modulator that is a proprietary pediatric formulation of the active ingredient ezogabine being developed for the treatment of KCNQ2 developmental and epileptic encephalopathy (KCNQ2-DEE). With the U.S. Food and Drug Administration(FDA) having completed its review of the clinical trial protocol, Xenon is on track to initiate the XEN496 Phase 3 clinical trial in pediatric patients with KCNQ2-DEE before year-end.

In addition, Xenon has received a positive opinion from the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA), which recommends the granting of an orphan medicinal product designation for XEN496 for the treatment of KCNQ2-DEE. This European designation is in addition to the FDA granting Fast Track designation for XEN496 for the treatment of seizures associated with KCNQ2-DEE as well as Orphan Drug Designation (ODD) for the treatment of KCNQ2-DEE.

Dr. Simon Pimstone, Xenon's Chief Executive Officer, said, "This marks an extremely important milestone for Xenon, with the first of our proprietary product candidates now poised to enter a pivotal Phase 3 clinical trial. With feedback from the FDA, pharmacokinetic data supportive of our proprietary pediatric formulation, and considerable progress made in site selection and other preparations, we are excited to move forward with our 'EPIK' Phase 3 clinical trial studying XEN496 in pediatric patients with KCNQ2-DEE. In addition, receiving an orphan medicinal product designation for XEN496 in Europe underscores that KCNQ2-DEE is a severe, early onset epilepsy disorder and further validates Xenon's novel, 'precision medicine' approach to develop treatments for pediatric epilepsies. We continue to work closely with the medical community, genetic testing companies, and patient advocacy groups to identify potential patients for our EPIK study, which we expect to initiate before year-end."

The XEN496 Phase 3 "EPIK" clinical trial is titled "A Phase 3 Study of Adjunctive XEN496 in Pediatric Subjects with KCNQ2 Developmental and Epileptic Encephalopathy." This study is designed as a randomized, double-blind, placebo-controlled, parallel group, multicenter clinical trial to evaluate the efficacy, safety and tolerability of XEN496 administered as adjunctive treatment in approximately 40 pediatric patients aged one month to less than 6 years with KCNQ2-DEE. After screening, patients will enter a baseline period to assess the frequency of seizures. Eligible subjects will be randomized on a 1:1 basis to receive either XEN496 or placebo for approximately 15 weeks (titration and a 12-week maintenance period). At the end of treatment, there will be a period of tapering off of study drug, followed by a 28-day safety monitoring period. Patients may be considered for an open-label extension if they meet all requirements. The primary endpoint is the percent change from baseline in monthly countable motor seizure frequency during the blinded treatment period, as recorded by caregivers in a daily seizure diary. Key secondary endpoints include the proportion of patients experiencing greater than or equal to 50 percent reduction in monthly seizure frequency from baseline, caregiver global impression of change (CaGI-C) scores, and caregiver global impression of severity (CaGI-S) scores.

XEN496 administered as adjunctive treatment in approximately 40 pediatric patients aged one month to less than 6 years with KCNQ2-DEE

40 patients for a phase 3 trial seems quite low. Also I am wondering why they test some redesigned Trobalt on children. However if they produced some Trobalt with such few side effects that even babies can take it, this would certainly be great.

 

[tommyd87]

I'd love to have normal hearing again but I take a reduction in tinnitus without restored hearing!

So would I but I'd inevitably think that the ability to relieve tinnitus issues now would make things much more manageable for people in the interim until there are actually hair cell and/or other treatments that deal with that.

XEN496 administered as adjunctive treatment in approximately 40 pediatric patients aged one month to less than 6 years with KCNQ2-DEE

40 patients for a phase 3 trial seems quite low. Also I am wondering why they test some redesigned Trobalt on children. However if they produced some Trobalt with such few side effects that even babies can take it, this would certainly be great.

It might actually have a lot to do with the types of people participating in the trial (being small children) and also about how many unfortunate people have the condition, causing an issue in getting large numbers participating in a trial. There also could be the possibility that the FDA can consequently make a determination on the medicine's performance with these trial numbers. I cannot see the FDA allowing a trial to proceed if they didn't think that it could deliver desirable outcomes.

Although the terrifically positive thing is that if they take this medicine through the trial successfully and also are able to demonstrate it works well with dealing with tinnitus then this will be a big win with their medicine and actually also make it much more in demand and therefore valuable too.

 

[FGG]

XEN496 administered as adjunctive treatment in approximately 40 pediatric patients aged one month to less than 6 years with KCNQ2-DEE

40 patients for a phase 3 trial seems quite low. Also I am wondering why they test some redesigned Trobalt on children. However if they produced some Trobalt with such few side effects that even babies can take it, this would certainly be great.

They are testing this drug for a severe form of childhood epilepsy, that's why it's tested on pediatric patients.