Email response from Leo:
On Wed, Jun 17, 2015 at 4:16 PM, Xxxx X Xxxxxxxx wrote:
Aloha Leo,
I have a few questions, mostly just to corroborate discussion board stuff of the last week or two. Please feel free to not answer anything and to only answer what you can.
1) According to a purported email from you, there are now 4 trial sites, though only one reported as enrolling, Oregon and South Dakota. Can you confirm that there are 2 or more sites added or that will be added?
3 sites are now active and we have reached agreements with another 5 sites. We are screening additional sites as well. Additional sites will be coming on line regularly as we want to complete this trial ASAP.
2) Some of us recall your stating somewhere that the decision to drop MD Anderson as a trial site was due to patient competition among trials there as well as competition from a current trial for another OM treatment. Can you confirm this and/or elaborate?
Correct but that is only part of the reason.
I responded yesterday to a shareholder with a similar question as follows.
MD Anderson is an excellent institution. I too was upset that MD Anderson did not work out and that led to the delay of the start of the OM trial.
The CRO we engaged to help organize the clinical trial sites contacted MD Anderson and they were very interested in doing the study. We were told they could provide a substantial number of patients to the trial.
As we engaged in discussions and visited the hospital, we learned that most patients with Head and Neck cancer had been previously treated elsewhere (ineligible patients). We always need untreated patients for this study and those patients often first go to a local hospital. In addition for the few newly diagnosed patients there was a competing trial ongoing at the hospital. To learn all this took some time.
We spent the last few months negotiating and signing up new sites. Hopefully the trial will now proceed expeditiously.
3) Regarding Brilacidin ABSSSI and the upcoming FDA meeting: I’m of the belief that the meeting will only give guidance for the Fast Track designations—rolling review, advanced trial Phase 3/4, surrogate endpoints for the trial outcomes, etc.—defined by the QIDP designation assigned last December. Others think that the FDA will also decide if there is a Phase 3. Can you briefly discuss the main talking points for the meeting, or at least some of the main topics?
The meeting will be an end of Phase 2 meeting. As such the discussions will include our request for permission to conduct a Phase 3 study . We will present the extensive data the FDA required when we made the original request for the meeting. Safety and efficacy will be discussed. I can't begin to describe the amount of work that went into this briefing book. We feel real good about our preparations and will shortly be meeting with them. I figure by mid -late July i'll be able to update shareholders with the results of the meeting.
4) I noted that the completion dates for Kevetrin trial have moved forward to October 2015 for the primary completion and February for the study completion. I take this to mean the expanded protocol for the final up to 20 patients has been determined:
a. Will there be a 12th cohort? If so, will it be 3 patients?
b. Will that be the last cohort, which will mean if no DLT then expanded up to 17 more, or if DLTs are encountered, then cohort 11 @ 750 mg/m2 expands up to 17 more patients?
c. Is there an update coming from the University of Bologna. Have they begun treating patients? Can you elaborate on, or will you be updating us on, the protocol and start for that trial?
I can't comment on these questions as we haven't yet issued a press release to shareholders. However I will say our optimism on Kevetrin is unchanged and we want to move into Phase 2 ASAP. When I update on Brilacidin I will do a complete corporate update as well.
5) One last one just for fun, some have speculated that a buyout might mean $1.5 billion for CTIX, or $10 a share. Would you accept a $1.5 billion offer at this point?
Wow, what a tough question for me as CEO to answer. I have to be real careful with this one.
Cancer- We definitely see potential in Kevetrin. Great cancer drugs can have even greater valuations. We all are waiting for the next update.
Antibiotics- Only after a successful Phase 3 study can $$$ numbers be discussed. Remember ABSSSI is the gateway for many add on Phase 3 trials such as diabetic foot infections, etc.
Psoriasis- A successful Phase 2 study with very good efficacy and low toxicity can become a 0.5 billion dollar value. A successful Phase 3 with good efficacy and low toxicity can become a 2+ billion dollar value.
GI diseases- This is potentially the Jackpot play. If we get good results in the OM trial we would expect it to do very well in GI diseases. Of course we will then begin on formulation work. The potential in GI for an effective drug is in the many billions.
OM- If we are successful in preventing severe OM, medicare and the insurance companies will have to allow for reimbursement. The market size then can be enormous.
I leave it for others to determine value
On Wed, Jun 17, 2015 at 4:16 PM, Xxxx X Xxxxxxxx wrote:
Aloha Leo,
I have a few questions, mostly just to corroborate discussion board stuff of the last week or two. Please feel free to not answer anything and to only answer what you can.
1) According to a purported email from you, there are now 4 trial sites, though only one reported as enrolling, Oregon and South Dakota. Can you confirm that there are 2 or more sites added or that will be added?
3 sites are now active and we have reached agreements with another 5 sites. We are screening additional sites as well. Additional sites will be coming on line regularly as we want to complete this trial ASAP.
2) Some of us recall your stating somewhere that the decision to drop MD Anderson as a trial site was due to patient competition among trials there as well as competition from a current trial for another OM treatment. Can you confirm this and/or elaborate?
Correct but that is only part of the reason.
I responded yesterday to a shareholder with a similar question as follows.
MD Anderson is an excellent institution. I too was upset that MD Anderson did not work out and that led to the delay of the start of the OM trial.
The CRO we engaged to help organize the clinical trial sites contacted MD Anderson and they were very interested in doing the study. We were told they could provide a substantial number of patients to the trial.
As we engaged in discussions and visited the hospital, we learned that most patients with Head and Neck cancer had been previously treated elsewhere (ineligible patients). We always need untreated patients for this study and those patients often first go to a local hospital. In addition for the few newly diagnosed patients there was a competing trial ongoing at the hospital. To learn all this took some time.
We spent the last few months negotiating and signing up new sites. Hopefully the trial will now proceed expeditiously.
3) Regarding Brilacidin ABSSSI and the upcoming FDA meeting: I’m of the belief that the meeting will only give guidance for the Fast Track designations—rolling review, advanced trial Phase 3/4, surrogate endpoints for the trial outcomes, etc.—defined by the QIDP designation assigned last December. Others think that the FDA will also decide if there is a Phase 3. Can you briefly discuss the main talking points for the meeting, or at least some of the main topics?
The meeting will be an end of Phase 2 meeting. As such the discussions will include our request for permission to conduct a Phase 3 study . We will present the extensive data the FDA required when we made the original request for the meeting. Safety and efficacy will be discussed. I can't begin to describe the amount of work that went into this briefing book. We feel real good about our preparations and will shortly be meeting with them. I figure by mid -late July i'll be able to update shareholders with the results of the meeting.
4) I noted that the completion dates for Kevetrin trial have moved forward to October 2015 for the primary completion and February for the study completion. I take this to mean the expanded protocol for the final up to 20 patients has been determined:
a. Will there be a 12th cohort? If so, will it be 3 patients?
b. Will that be the last cohort, which will mean if no DLT then expanded up to 17 more, or if DLTs are encountered, then cohort 11 @ 750 mg/m2 expands up to 17 more patients?
c. Is there an update coming from the University of Bologna. Have they begun treating patients? Can you elaborate on, or will you be updating us on, the protocol and start for that trial?
I can't comment on these questions as we haven't yet issued a press release to shareholders. However I will say our optimism on Kevetrin is unchanged and we want to move into Phase 2 ASAP. When I update on Brilacidin I will do a complete corporate update as well.
5) One last one just for fun, some have speculated that a buyout might mean $1.5 billion for CTIX, or $10 a share. Would you accept a $1.5 billion offer at this point?
Wow, what a tough question for me as CEO to answer. I have to be real careful with this one.
Cancer- We definitely see potential in Kevetrin. Great cancer drugs can have even greater valuations. We all are waiting for the next update.
Antibiotics- Only after a successful Phase 3 study can $$$ numbers be discussed. Remember ABSSSI is the gateway for many add on Phase 3 trials such as diabetic foot infections, etc.
Psoriasis- A successful Phase 2 study with very good efficacy and low toxicity can become a 0.5 billion dollar value. A successful Phase 3 with good efficacy and low toxicity can become a 2+ billion dollar value.
GI diseases- This is potentially the Jackpot play. If we get good results in the OM trial we would expect it to do very well in GI diseases. Of course we will then begin on formulation work. The potential in GI for an effective drug is in the many billions.
OM- If we are successful in preventing severe OM, medicare and the insurance companies will have to allow for reimbursement. The market size then can be enormous.
I leave it for others to determine value
