neuronano update august 2013
DESCRIPTION
ÂTRANSCRIPT
neuronano pharmaCompany and operational activities
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© 2013 Neuro10-9 Pharma, Inc. All rights reserved.
Company overview and business model
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© 2013 Neuro10-9 Pharma, Inc. All rights reserved.
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ACADEMIA INDUSTRY
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ACADEMIA INDUSTRY
commerciability
inno
vatio
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MAR
KET
idea
platform concept
platform technology
lead compounds
drug candidatedrug
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© 2013 Neuro10-9 Pharma, Inc. All rights reserved.
Co-founder &
lead inventor
Kabanov
Co-founder &
acting CEO
Gaymalov
7YPUJPWHS�ZJPLU[PZ[Z�(for
each plafrom
technology)
Yi BatrakovaManickam
Board of KPYLJ[VYZ
Kabanov Polinsky RomarMalkov
Board of advisors
Banks FarrellMcCall Zalipsky
Alakhova
=PJL�7YLZPKLU[�for BD &
Licensing
De Ridder
Bloom
6\Y�VWLYH[PVUZ
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© 2013 Neuro10-9 Pharma, Inc. All rights reserved.
4+ delivery platforms20+ lead compounds10+ indications
Academia
Industry key activities
ReCoPE: reverse concept.
product engineeriing
IP generation
& management
business development -
partnering & licensing
securing grants
& equity investment
translational research key outcomes
Spin-off portfolio
companies
Partnerships
Out-licensing deals
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Lean :OHYLK�HUK�SV^LYLK�YPZR *HWP[HSPaPUN�VU�LZ[HISPZOLK�HUK�LTLYNPUN�YLZV\YJLZ�PU�5*
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pluroMAC™Platform Delivery Technology
Binary:\WLYHJ[P]LPLD™Platform Delivery Technology
nanoZYME™Platform Delivery Technology
*LSS�4LKPH[LK�DeliveryTechnology
Macromolecules
Small molecules
Macromolecules
Small molecules
Antibodies Genes
����WYVK\J[�JHUKPKH[LZ 12+ disease models ����WH[LU[Z�HWWSPJH[PVUZ
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LUa`TLZ! SOD *H[HSHZL OPH BCHEHRP
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BDNF
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NNP-bdnfNZ Rett syndrome
UveitisNNP-sodNZ
NNP-sodNZ Amyotrophic
lateral sclerosis
leptiPOL-LM Obesity
Discovery CandidateSelection
Pre-clinicalResearch
Pre-clinical Development
Clinical Phase 1,2Product Indications
CancerNNP-D773c 505(b)(2)
leptiPOL-HM Alzheimer's disease
6\Y�JVYL�[LJOUVSVNPJHS�HZZL[Z
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pluroMAC™Platform Delivery Technology
Binary:\WLYHJ[P]LPLD™Platform Delivery Technology
nanoZYME™Platform Delivery Technology
*LSS�4LKPH[LK�DeliveryTechnology
Macromolecules
Small molecules
Macromolecules
Small molecules
Antibodies Genes
����WYVK\J[�JHUKPKH[LZ 12+ disease models ����WH[LU[Z�HWWSPJH[PVUZ
June 18, 2013;OL�(TLYPJHU�4LKPJHS�(ZZVJPH[PVU�OHZ�VMÄJPHS-ly recognized obesity as a disease, a move
that could induce WO`ZPJPHUZ� [V� WH`�TVYL�H[[LU[PVU� and ZW\Y� TVYL� PUZ\YLYZ� [V� WH`�MVY�[YLH[TLU[Z.
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In obese patients, the transport of
gut hormones to the brain is impaired
(gut hormone desensitization)
Its possible to treat obesity by de-
livering gut hormones directly to the
brain and restore natural sensitivity
to the hormone observed in health
people.
,MMLJ[P]L�HUK�ZHML�KLSP]LY`�VM�N\[�OVYTVULZ�HJYVZZ�ISVVK�IYHPU�IHYYPLY
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group of hormones secreted by enteroendocrine cells in the stomach,
pancreas, and small intestine that play a role of neurotransmitters
and neuromodulators in the central and peripheral nervous systems.
(J[P]L�WOHYTHJL\[PJHS�PUNYLKPLU[Z
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Macro-molecules
;LJOUVSVN`� PU]VS]LZ� Z\YMHJL� TVKPÄJH[PVU� VM� [OLYHWL\[PJ� WYV[LPUZ�with precisely crafted poloxamers and allows active delivery of these
WYV[LPUZ�[V�[OL�KLÄULK�IYHPU�YLNPVUZ�
SLW[P763�34��HK]HU[HNLZ!�� Rapid brain delivery
�� Targeted hypothalamus delivery
�� Immediate effectiveness
�� Independence of diet and exercise
�� Convenience of intranasal delivery
�� Safety
SLW[P763�34�!�Poloxamer conjugated leptin hormone for intranasal
and intravenous hypothalamus delivery
SLW[PU GLP-1 amylin OXM
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�� Tong et al. (2012). Mol Pharm. 10:360-77
�� Tong et al. (2010). Mol Pharm 7:984-92
�� Price et al. (2010). 1�7OHYTHJVS�,_W�;OLY�333:253-63
�� Batrakova et al. (2008). )PVJVUQ\N�*OLT 19:1071-7
�� Batrakova et al (2005). )PVJVUQ\N�*OLT 16:793-802
UNC - NIH RO17VS`WLW[PKL�4VKPÄJH[PVU�MVY�,UOHUJLK�)YHPU�+LSP]LY`������$1,920,000 (J[P]L
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0UK\Z[Y`�JVSSHIVYH[PVUZ!�� Dr. Catherine Farrell, Ph.D. 0UKLWLUKLU[�JVUZ\S[PUN (Amgen)
�� Dr. John 6NLa, Ph.D. 7LHR�[V�7LHR�)PV+L]LSVWTLU[ (Amgen)
�� Dr. David Essayan, M.D. 6UJVYK��0UJ (Amgen)
�� Dr. Samuel AHSPWZR`, Ph.D. SJZ (ALZA)
�� Dr. John McCall, Ph.D. 7OHYTHJ��33*��7ÄaLY�
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5 yearsurvival
45%
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5 yearsurvival
5 yearsurvival
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2008
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7.6 12.7
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5 yearsurvival
5 yearsurvival
68% 68%
T\S[PWSL�T`LSVTH RHWVZP�ZHYJVTH
;YLH[TLU[�HK]HU[HNLZ�� rapid API internalization
�� low immunogenicity
�� improved solubility
�� enhanced biocompatibility
�� increased bioavailability
�� superior API protection
(70�YLSLHZL�MYVT�SPWVZVTLZ�\WVU�HYYP]HS�PU[V�[OL�[\TVY
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This group of possible APIs includes emulsions, micellar solutions,
liposomes, lipid nanoparticles, and drug-lipid conjugates.
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(J[P]L�WOHYTHJL\[PJHS�PUNYLKPLU[Z
+LSP]LY`�WSH[MVYT�[LJOUVSVN`���IPUHY`�ZH73? 7YVK\J[�7YV[V[`WL���557�+���J�MVY�73+
liposomal chemotherapies adjuvation with poloxamers
liposomal chemotherapies
:[LW����3PWVZVTHS�(70�JOLTV[OLYHW` :[LW����4L[H�HKQ\]H[PVU�^P[O�557�73+��
7YVSVUNLK�(70�OHSM�SPML
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Tumor
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the tumor site
Drug release from liposomes
and penetration into tumor site
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UNC Carolina CCNE$55,000(J[P]L
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�� Dr. William Zamboni, PhD. UNC Chapel Hill�� Dr. Victoria Bae-Jump. M.D., PhD., UNC Chapel Hill�� Dr. David Darr, PhD., UNC Chapel Hill�� Dr. Charles Perou, PhD., UNC Chapel Hill�� Dr. Gabriel 3VWLa, M.D., PhD., 4+�(UKLYZVU�*HUJLY�*LU[LY�� Dr. Bulent 6aWVSH[, M.D., PhD., 4+�(UKLYZVU�*HUJLY�*LU[LY
Zhao et al., (2013)�1V\YUHS�VM�JVU[YVSSLK�YLSLHZL����!�����
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0UK\Z[Y`�JVSSHIVYH[PVUZ!�� Dr. Jo *H[V, Ph.D. *H[V�=LU[\YLZ
�� Dr. Ken Phelps, Ph.D. *HTHYNV�7OHYTHJL\[PJHS�:LY]PJLZ
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examination by an ophthalmologist or optometrist—HUK�\YNLU[�[YLH[TLU[�[V�JVU[YVS�[OL�PUÅHTTH[PVU�
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7YV�PUÅHTTH[VY`�JLSSZ�PUÄS[YH[PUN�Z[PT\SH[LK�ZP[LZ��PUK\JPUN�[OL�96:�production and accumulation. This leads to the oxidative stress, cell
death, and ultimately blindness.
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ROS scavenger enzymes are aimed to reduces the hypoxic stress
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In AD patients, the transport of leptin
to the brain is impaired.
Its possible to slow down the degen-
erative process of AD by delivering
SLW[PU�KPYLJ[S`�[V�[OL�IYHPU��ZWLJPÄJHS-ly to hippocampus.
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© 2013 Neuro10-9 Pharma, Inc. All rights reserved.
Leptin is a 16-kDa protein
hormone that plays a key role
in regulating energy intake
and expenditure, including
appetite and hunger,
metabolism, and behavior.
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Macromolecules
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SLW[P763�/4��HK]HU[HNLZ!�� Rapid brain delivery
�� Targeted hippocampus delivery
�� Immediate effectiveness
�� Convenience of intranasal delivery
�� Safety
SLW[P763�/4�!�Poloxamer conjugated leptin hormone for�PU[YHUHZHS�OPWWVJHTW\Z��KLSP]LY`
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�� In preapration
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0UK\Z[Y`�JVSSHIVYH[PVUZ!�� Dr. Catherine Farrell, Ph.D. 0UKLWLUKLU[�JVUZ\S[PUN (Amgen)
�� Dr. John 6NLa, Ph.D. 7LHR�[V�7LHR�)PV+L]LSVWTLU[ (Amgen)
�� Dr. David Essayan, M.D. 6UJVYK��0UJ (Amgen)
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�� Dr. John McCall, Ph.D. 7OHYTHJ��33*��7ÄaLY�
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The standard of care therapy, Riluzole is modestly-effective and
does not cure ALS. It decreases glutamate-induced excitotoxicity
and extends patient survival by only 3-6 months while they need to
be continuously monitored for possible liver toxicity. There is an \YNLU[�ULLK�[V�KL]LSVW�H�ZHML�HUK�LMMLJ[P]L�[OLYHW`�MVY�(3:��
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half-life for�LUOHUJLK�PU[YHUHZHS�KLSP]LY`�[V�[OL�IYHPU
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�� Dr. Carol 4PSSPNHU, Ph.D. >HRL�-VYLZ[�<UP]LYZP[ �̀�5*
�� Rosenbaugh et al. )PVTH[LYPHSZ. 2010 Jul;31(19):5218-26.
�� Klyachko et al. 5HUVTLKPJPUL. 2012 Jan;8(1):119-29
�� Manickam et al. 1�*VU[YVS�9LSLHZL. 2012 Sep 28;162(3):636-45
�� Rosenbaugh et al. (T�1�7O`ZPVS�9LN\S�0U[LNY�*VTW�7O`ZPVS��2013 Jun
1:R917-28
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brain will ameliorate Rett Syndrome
symptoms. However BDNF has L_[YLTLS`�ZOVY[�OHSM�SPML�HUK�WVVY�WLUL[YH[PVU�[OYV\NO�)))��
Rett syndrome (RTT) is an X-linked postnatal neurodevelopmental
disorder, which is primarily caused by mutations in the gene
encoding methyl-CpG binding protein 2 (MeCP2). A number
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a secreted protein (cytokine)
that, in humans, is encoded
by the BDNF gene.
(J[P]L�WOHYTHJL\[PJHS�PUNYLKPLU[
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Brain-derived UL\YV[YVWOPJ�MHJ[VY
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Macromolecules
Small molecules
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*VU[HJ[�<Z�Zagit Gaymalov, Ph.D.
919.923.7401