Watch this space..., page-36

Anyways, I head the full briefing and here are my thoughts.

Dr Treagus did not comment when asked whether there was a trail phase 3. he constantly alluded that we will be looking for FDA guidance on our next stage of development.

He clearly mentions that there will be 2 separate applications.
1) Orphan drug designation.
2) breakthrough therapy.

Given the results that we have achieved, he are quite confident that NNZ-2566 should be granted the orphan drug designation. He mentioned that breakthrough therapy will be a separate application and he said we cannot guarantee that we will be granted the BT.

Following is the chart re the approval if BT is granted. We do not need a phase 3.


Source

The U.S. Food and Drug Administration’s (FDA) new breakthrough therapy designation represents perhaps the most important effort to date to reduce the time required to bring new drugs to market. According to the FDA a breakthrough therapy is a drug that is:

• Intended alone or in combination with one or more other drugs to treat a serious or life threatening disease or condition and
• That may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development, as indicated by preliminary clinical evidence.

The breakthrough therapy designation allows for early and positive awareness of a drug’s therapeutic value, signaling that the product delivers substantial improvement on at least one clinical endpoint.

My impression was that Dr Treagus, Joe and Larry were hopeful of BT grant allowing us to avoid the phase 3 and thus did not comment on the whether there will be phase 3 of RETT or not.

Dr Treagus also said that a large number of Pharmas are increasingly monitoring our success but he is fully committed to completing all the trials at hand.

A full update of all the remaining trial and progress will be given before the end of the year.

Table of BT approval and parent companies.

	Column 1	Column 2	Column 3	Column 4
0	Drug Name	FDA Approval Date	Sponsor Company	Indication
1	Gazyva   (Obinutuzumab)	11.01.13	Genentech	Chronic Lymphocytic Leukemia (CLL)
2	Imbruvica   (Ibrutinib)	11.13.13	Pharmacyclics	Mantle Cell Lymphoma (MCL)
3	Sovaldi   (Sofosbuvir)	12.06.13	Gilead   Sciences	Hepatitis C
4	Kalydeco (Ivacaftor)**	02.21.14	Vertex Pharmaceuticals	8 additional mutations in CFTR gene for Cystic Fibrosis (CF)
5	Arzerra (Ofatumumab) ***	04.17.14	GlaxoSmithKline	In combination with Chlorambucil for previously untreated Patients with CLL for whom fludarabine-based therapy is considered inappropriate
6	Zykadia (Ceritinib)	04.29.14	Novartis	Metastatic ALK+ NSCLC
7	Zydelig (Idelalisib)	07.23.14	Gilead Sciences	Chronic Lymphocytic Leukemia (CLL)
8	Imbruvica (Ibrutinib)*	07.28.14	Pharmacyclics	Chronic Lymphocytic Leukemia (CLL) who carry a deletion in chromosome 17 (17p deletion)
9	Promacta (Eltrombopag)**	08.26.14	GlaxoSmithKline	Severe Aplastic Anemia (SAA) with insufficient response to Immunosupporessive Therapy (IST)
10	Keytruda (Pembrolizumab) ****	09.04.14	Merck	Unresectable/ Metastatic Melanoma, disease progression, & have BRAF V600 mutation, following treatment with Yervoy & a BRAF inhibitor
11	Harvoni (Ledipasvir /Sofosbuvir)	10.10.14	Gilead Sciences	Genotype 1 Chronic HCV
12	Esbriet (Pirfenidone)	10.15.14	InterMune	Idiopathic Pulmonary Fibrosis
13	Ofev (Nintedanib)	10.15.14	Boehringer Ingelheim Pharmaceuticals	Idiopathic Pulmonary Fibrosis
14	Trumenba (Meningococcal Group B Vaccine) *****	10.29.14	Pfizer	Meningococcal B Disease