Exhibit 10.1
 
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Pursuant to 17 CFR 20.24b-2, confidential information has been omitted in places
marked “***” and has been filed separately with the Securities and Exchange
Commission pursuant to a Confidential Treatment Application with the Commission.
 
ATTACHMENT 2
MILESTONE AND DELIVERABLES CHART
May 7, 2015
HHSO100201500007C
WBS
Milestone
Deliverable
Success Criteria
Timing
Go/No-Go
for initiation
CLIN 0001 - MANUFACTURE OF CLINICAL TRIAL MATERIAL
1.2.1
Process Improvements Report
Report on Process Development
Process Developed
***
 
1.2.2
Determination of Sufficient Process for Commercial Scale up
Evaluation report
BARDA approval of developed process
***
N/A
1.3.1
Manufacture ***(Batch ***)
***
Acceptable quality and yield
***
N/A
1.3.2
Manufacture ***(Batch ***)
***
Acceptable quality and yield
***
N/A
1.4.1
Manufacture cGMP BCX4430 (***campaign DS Batch ***)
BCX4430 DS
 
CofA,
 
Acceptable quality and yield
***
N/A
1.4.2
Manufacture cGMP BCX4430 (*** campaign DS Batch ***)
BCX4430 DS
 
CofA,
 
Acceptable quality and yield
***
N/A
1.4.3
Prepare a Campaign Summary Reports
Campaign Reports (DS Batches ***)
Completion of DS Campaigns
***
N/A
1.4.4
Drug substance stability study
Initial Report on stability activities
Stability data
***
 
1.5
Drug Product Development
DP Process Development Report (WBS 1.5.4)
 
Pre-formulation and Physicochemical Report (WBS1.5.5)
 
Extractable/Leachable Report (WBS 1.5.7)
 
 
Completion of Studies
***
N/A
1.5.8
Excipient Compatibility Report for IV Formulation
Compatibility Report
IV formulation completed
***
 
1.6.1
Manufacture cGMP DP (CTM Batch *** ***)
BCX4430 DP
 
CofA,
Acceptable quality and yield
***
Accepted GMP DS
1.6.2
Manufacture cGMP DP (CTM Batch *** ***)
BCX4430 DP
 
CofA,
Acceptable quality and yield
***
Accepted GMP DS

 
 
 

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Pursuant to 17 CFR 20.24b-2, confidential information has been omitted in places
marked “***” and has been filed separately with the Securities and Exchange
Commission pursuant to a Confidential Treatment Application with the Commission.
 
1.6.3
Prepare a Campaign Summary Reports
Campaign Reports (CTM Batches ***)
Completion of DP Campaigns
***
N/A
1.6.4
Drug Product stability study
Initial Report on stability activities
Stability Data
***
 
1.6.5
Comparability Study
Comparability Protocol and Report
Completion of DS and DP Campaigns
***
 
1.7.1
Manufacture cGMP BCX4340 (*** campaign DS Batch ***)
BCX4430 DS
 
CofA
Acceptable DS process
***
N/A
1.7.2
Prepare a Campaign Summary Report
Campaign Reports (DS Batch***)
Completion of DS Campaign
***
N/A
1.7.2
Manufacture cGMP DP (CTM Batch *** ***)
BCX4430 DP
 
CofA,
Acceptable quality and yield
***
Accepted GMP DS
1.7.4
Prepare a Campaign Summary Report
Campaign Report (CTM Batches ***)
Completion of DS Campaigns
***
N/A
1.7.5
Drug Substance and Drug Product stability study
Initial report on stability activities
Stability Data
***
Manufacture of 1.7.1 drug substance and 1.7.2 drug product
1.7.6
Comparability Study
Comparability Protocol and Report
Comparable DS and DP profiles
***
N/A
CLIN 0002 – COMMERCIAL SCALE UP AND NDA REGISTRATION BATCHES
Go/No Go Criteria to Initiate: WBS 1.2.2 BARDA approval of process developed
 
2.2
Drug Substance Process Scale-up
Process Development Report (WBS 2.2.4)
Selection of the optimized manufacturing process
***
*** process
2.3.1
 
Manufacture BCX4340 DS (DS Registration Batch ***)
BCX4430 Registration
DS
 
CofA
Acceptable quality and yield
***
*** process
2.3.2
 
Manufacture BCX4340 DS (DS Registration Batch ***)
BCX4430 Registration DS
 
CofA,
Acceptable quality and yield
***
*** process
2.3.3
 
Manufacture BCX4340 DS (DS Registration Batch ***)
BCX4430 Registration DS
 
CofA,
Acceptable quality and yield
***
*** process
2.3.4
Prepare a Campaign Summary Report
Campaign Reports (DS Batches ***)
Completion of DS Campaign
***
N/A
2.4.1
 
Manufacture BCX4430 DP (DP Registration Batch ***)
BCX4430 DP
 
CofA,
Acceptable quality and yield
***
Accepted GMP DS

 
 
 

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Pursuant to 17 CFR 20.24b-2, confidential information has been omitted in places
marked “***” and has been filed separately with the Securities and Exchange
Commission pursuant to a Confidential Treatment Application with the Commission.
 
2.4.2
 
Manufacture BCX4430 DP (DP Registration Batch ***)
BCX4430 DP
 
CofA,
Acceptable quality and yield
***
Accepted GMP DS
2.4.3
 
Manufacture BCX4430 DP (DP Registration Batch ***)
BCX4430 DP
 
CofA,
Acceptable quality and yield
***
Accepted GMP DS
2.4.4
Prepare a Campaign Summary Report
Campaign Report (CTM Registration Batches ***)
Completion of DS Campaigns
***
N/A
2.5
Drug substance and Drug Product stability study
Report on stability activities
Stability Data
***
 
2.6
Comparability Study
Comparability Protocol and Report
Comparable DS and DP profiles
***
Accepted GMP DS
CLIN 0003 – NONCLINICAL NDA-ENABLING TOXICOLOGY - IM
Go/No Go Criteria to Initiate: WBS 1.4.1 Completion of Manufacture cGMP BCX4430
(*** campaign DS Batch ***)
3.1.1
Complete GLP *** IM Tox Study - ***
Study Report
Established NOAEL
***
Drug Substance confirming to release criteria
3.1.2
Complete GLP *** IM Tox Study - ***
Study Report
Established NOAEL
***
Drug Substance confirming to release criteria
3.2.1
Conduct *** assessment in *** and ***
Study Report
No significant findings
***
N/A
3.2.2
Conduct *** Dose Range Finding Studies in the ***
Study Report
No significant findings
***
N/A
3.2.3
Conduct Definitive *** toxicology in the ***
Study Report
No significant findings
***
N/A
3.2.4
Conduct *** toxicology ***
Study Report
No significant findings
***
N/A
3.3.1
Conduct Radiolabeled ADME study - ***
Study Report
Characterize drug disposition
***
Acceptable Radiolabel Material
3.3.2
Conduct Radiolabeled ADME – ***
 
Study Report
Characterize drug disposition
***
Acceptable Radiolabel Material
CLIN 0004 – IN VITRO EXPERIMENTS – IV
Go/No Go to Initiate: WBS 1.5.8 Completion of Excipient compatibility studies
for IV formulation
4.1.
Conduct *** Test – IV
Study Report
No effect on ***
***
IV formulation WBS 1.5.8
4.2.
Conduct *** Test – IV
Study Report
No effect on ***
***
N/A

 
 
 

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Pursuant to 17 CFR 20.24b-2, confidential information has been omitted in places
marked “***” and has been filed separately with the Securities and Exchange
Commission pursuant to a Confidential Treatment Application with the Commission.
 
4.3
*** IV experiments
Study report on all *** assays with recommendation to proceed CLIN0005
No toxicology ***
***
 
CLIN 0005 – NONCLINICAL NDA-ENABLING TOXICOLOGY – IV
Go/No Go to Initiate: WBS 4.3 Completion of *** IV toxicology studies
5.1.1
Complete GLP *** IV Tox Study - ***
Study Report
Established NOAEL
***
Drug Substance confirming to release criteria
5.1.2
Complete GLP ***IV Tox Study - ***
Study Report
Established NOAEL
***
Drug Substance confirming to release criteria
5.2.1
Conduct *** assessment in ***
Study Report
No significant findings
***
N/A
5.2.2
Conduct *** Dose Range Finding Studies in the ***
Study Report
No significant findings
***
N/A
5.2.3
Conduct Definitive *** toxicology in the ***
Study Report
No significant findings
***
N/A
5.2.4
Conduct *** toxicology ***
Study Report
No significant findings
***
N/A

 
 
 

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