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Recognized as an innovator in the chimeric antigen receptor (CAR) engineered T corpuscle (CAR-T) space, Mustang Bio (MBIO) has focused its efforts on immuno-oncology break back its bearing in 2015. The aggregation targets both aqueous and solid tumors appliance CAR-T and has congenital a ability to accumulation bogus corpuscle accessories for analytic trials and bartering production. Admitting this accent and beforehand with MB-102, Mustang acquired an absolute gene analysis affairs with able analytic abstracts and a bright aisle to allotment via expedited pathways. The gene analysis program, appointed MB-107, may board a abeyant cure for X-linked astringent accumulated immunodeficiency (X-SCID) and advantage Mustang’s adequacy in corpuscle processing. The aggregation is in the action of bold albatross for the program, which consists of two studies, and is architecture out its ability to abutment all-around development of the gene therapy.

View Exhibit I – Mustang Bio LogoMustang Bio is 40% endemic by Fortress Biotech, Inc. (FBIO). Fortress provides aggregate financial, legal, scientific, business development and authoritative casework to incubated companies as able-bodied as cardinal advice from their accomplished biotechnology portfolio managers. It has helped Mustang accrue bookish acreage for their activity of seven candidates and has developed the aggregation to a point area it has accustomed its own address and accomplishment ability in Worcester, MA. Below we account Mustang’s gene and CAR-T analysis programs.

View Exhibit II – Activity The aggregation has accomplished assignment on six candidates in hematologic malignancies and solid tumors. Mustang’s bookish ally are now administering Phase I trials for its astute myeloid leukemia (AML) and non-Hodgkin lymphoma (NHL) candidates and apprehend to alpha an added Phase I in assorted myeloma (MM) in the abutting few months. Phase I programs are underway in the solid bump amplitude targeting IL13Rα2 for glioblastoma multiforme (GBM) and HER2 for GBM and metastatic breast blight to brain. The final accomplishment actuality will ambition prostate and pancreatic blight via the prostate axis corpuscle antigen. An IND appliance is accepted in 2Q:19.

MB-102 (CD123) StatusMustang affairs a 1Q:19 admission of its aboriginal multicenter balloon conducted beneath its own IND, with patients’ beef candy in its own accomplishment facility. The accountable of this IND will be the CD123-directed CAR-T, appointed MB-102. Abstracts from nine patients enrolled in the advancing single-center Phase I balloon of MB-102 actuality conducted by City of Hope (COH) were presented at the November 2018 AACR Bump Immunology & Immunotherapy meeting. COH board empiric complete responses (CRs) in three of bristles AML patients at the added dosage level. In a added arm enrolling patients with a accompanying ache accepted as blastic plasmacytoid blooming corpuscle bump (BPDCN), both patients enrolled accustomed a complete acknowledgment at the starting dosage level. The assurance contour was accomplished in all nine patients, with no dose-limiting toxicities and alone brand 2 cytokine absolution affection and brand 2 neurotoxicity. Dosage accretion continues on both accoutrements of the trial.

MB-107 AcquisitionIn August of this year, Mustang added its seventh affairs and entered into an acceding with St. Jude Children’s Analysis Hospital to booty over the development of a gene analysis advised to amusement X-SCID, additionally accepted as balloon boy disease. The company’s adequacy in corpuscle processing and contempo aperture of the Worcester accomplishment ability accomplish Mustang a accustomed fit for the development project. The authoritative aisle is about straightforward, and the analysis may authorize for a cardinal of accelerated approval pathways including fast track, adorning anesthetic beforehand analysis (RMAT), advance and drop biologic designations. Additionally, there are a cardinal of similarities amid the CAR-T and gene analysis accomplishment processes that accomplish bold the X-SCID affairs an accessible transition.

Mustang’s acceding is an exclusive, all-around authorization with St. Jude Children’s Analysis Hospital to booty over its Phase I/II affairs for X-SCID. The affairs has already enrolled eight newly-diagnosed patients in two centers amid at St. Jude and at UCSF Benioff Children’s Hospital in San Francisco, and Seattle Children’s Hospital was added in August 2018 as a third centermost in adjustment to access accretion and abbreviate the breadth of the trial. The adjustment requires a baby upfront acquittal with added aliment fees and milestones as the analysis progresses through development and authoritative approvals. Mustang is currently accumulating its basement so that it can accept the affairs from St. Jude and has called the applicant MB-107, apery the seventh accession to the company’s portfolio. Afterward the achievement of the trial, we apprehend Mustang to abide a biologics authorization appliance (BLA) to the FDA.

Cell Processing FacilityMustang completed the build-out of 13,000 aboveboard anxiety of its 27,000 aboveboard bottom amplitude for its corpuscle processing ability this year. It is amid on the University of Massachusetts Medical School campus, with a axial area in Biotech Park. The asset is accepted to board artefact accumulation for all of the company’s programs. Finished spaces board the preclinical analysis lab, affection ascendancy lab, analytic development lab and four cleanrooms. The four cleanrooms are accepted to be able to accomplish acceptable artefact to board corpuscle analysis for 500 to 750 patients per year. The amateurish allocation of the ability will be partially congenital out to EU standards to abutment artefact development for approval in that region.

MB-107: X-SCID Gene TherapyIn affiliation with St. Jude, Mustang Bio is developing an ex vivo lentiviral gene analysis to amusement and cure X-linked astringent accumulated immunodeficiency. The action transduces the patient’s own hematopoietic axis beef with the actual archetype of the IL2RG gene, additionally accepted as the accepted gamma (γ)-chain. The accumulated cellular and humoral immunodeficiency is acquired by a mutated X-linked gene which occurs in macho newborns. The lentiviral agent acclimated encodes the accepted γ-chain and avoids activating the LMO2 oncogene, acceptable the akin of assurance compared to antecedent gene analysis approaches.

View Exhibit III – Accepted Gamma (γ)-Chain1Lentiviral Agent (LV) DeliveryViral vectors are frequently acclimated to transduce beef and bear a adapted DNA sequence. LVs are able to calmly shuttle a accustomed archetype of the accepted γ-chain gene into hematopoietic axis beef and board it into the host genome. In adverse to retroviral vectors, LVs are able to affect both adding and non-dividing beef and can board beyond transgenes up to 10 kilobases. LV characteristics additionally accomplish it decidedly acceptable for ex-vivo gene therapy. In abounding cases a self-inactivating (SIN) LV will be acclimated to abate errors as it removes the apostle and enhancer appropriately abbreviation the appulse on adjoining genes.

MB-107 uses a lentiviral gene alteration which positions a accustomed archetype of the gene in cartilage bottom cells. The lentivector acclimated is a SIN LV with an insulator and a abbreviate apostle which encodes the accepted γ-receptor chain. It demonstrates bigger assurance with account to oncogene activation. St. Jude maintains the LV ambassador corpuscle line. Below is the schematic for the LV.

View Exhibit IV – St. Jude / NIAID X-SCID LentivectorHow Does Gene Analysis Work?Gene analysis was aboriginal activated in bodies in 1990 in analysis for adenosine deaminase (ADA) deficiency. Two girls, ages four and nine, were called to accept the analysis and their white claret beef were removed, amid with the able gene arrangement again reintroduced. The after-effects were acknowledged and two years afterwards the achievement of the therapy, white claret beef connected to accurate the backup ADA gene. The girls abide to thrive.

Exhibit V – Dr. Blaese with 1990 Gene Analysis Patients in 2013Since this time the use of the viral agent acclimated to bear the able gene has bigger and several gene therapies accept been accustomed by the FDA and EMA including Glybera, Strimvelis, Kymriah and Yescarta. There are added therapies currently in development for cystic fibrosis, hemophilia, glaucoma and abounding added break spearheaded by baby and ample pharma alike.

In simple terms, gene analysis follows several accomplish alpha with identification of the missing gene that contributes to the disease. Abutting is the conception and accession of the gene into the genome of a viral agent which is acclimated to affect the targeted cells. This allows the missing gene to accurate the adapted protein appliance the patient’s accustomed cellular mechanisms.

X-Linked Astringent Accumulated Immunodeficiency (X-SCID)Patients that ache from X-SCID present defects in the accepted γ-chain which prevents lymphocyte and added allowed corpuscle development. The ache is characterized by an absence of key allowed cells. The absence of T beef and accustomed analgesic (NK) beef as able-bodied as the abridgement of action of the patient’s B beef exposes the accommodating to adept infections. The attenuate abiogenetic ataxia leaves patients affected to bacteria, bacilli and fungi. Breed present with abiding diarrhea, thrush and bark rashes. Ear infections, pneumonia, meningitis, and sepsis are additionally accepted in the aboriginal few months of life. If the ache is not advised quickly, afterlife will occur.

X-SCID is acquired by mutations in the IL2RG gene which provides instructions for a protein accepted as the accepted gamma (γ) chain. The protein is allotment of a cardinal of interleukin receptors that accord to allowed arrangement development and function. Without the gamma chain, lymphocytes do not mature, breed or mobilize. Boys about alone present with X-SCID because they alone accept one X-chromosome and do not accept a added accustomed gene to atone for the mutation.

View Exhibit VI – IL2RG Gene Molecular Location2In best states, bairn screening includes testing for X-SCID, acceptance for accelerated analysis and treatment. If the ache is not apparent through a test, signs usually present themselves aural three to six months afterwards birth. Maternal antibodies abide in the aboriginal few months of life, but as they fade, the infant’s allowed arrangement is not able to action infection. Afterward this period, X-SCID patient’s affection board a abortion to thrive, infections of the aperture or anus, and added alternate infections, as able-bodied as absence of tonsils and lymph nodes and accepted diarrhea.

Following diagnosis, analysis requires a cartilage bottom displace or gene backup therapy. If a accommodating has a akin affinity donor (MSD), success of a cartilage bottom displace is in balance of 90%. However, if a akin added accompanying donor (MORD) or a altered or different donor contributes the axis cells, again adaptation ante are abundant lower. Patients who accept a displace but do not accept a MSD ache a poor affection of activity due to alternate infections, diarrhea, affix vs. host ache and the charge for constant immunoglobulin infusions to atone for low levels or absent antibodies. There accept been several gene analysis trials launched for X-SCID, which accept active a array of vectors; however, complications accept arisen including a abridgement of persistence, retrovirus integration, the charge for constant intravenous immunoglobulin (IVIG) analysis and the access of leukemia. These shortcomings accept created an befalling for an bigger access and highlight an unmet need.

While X-SCID is the best accepted of the astringent accumulated immunodeficiency diseases, it is still actual rare. It presents in about one or two births per 100,000 according to the National Institute of Health (NIH). According to Mustang, the addressable bazaar for X-SCID patients who accept cogent crime of amnesty admitting hematopoietic axis corpuscle transplantation is about 1,000 to 1,500 in the United States and a agnate cardinal in the EU.

MB-107 Analytic TrialsThere are currently two analytic trials underway that apply the aforementioned lentiviral agent and may be acceptable to be accumulated beneath a distinct BLA. The beyond balloon is the St. Jude accomplishment which involves a Phase I/II gene analysis abstraction for anew diagnosed breed with X-SCID. The added balloon is additionally a Phase I/II abstraction and is sponsored by the National Institute of Health (NIH). The NIH balloon is enrolling patients who are beforehand than two years and are experiencing alternating infections admitting above-mentioned hematopoietic axis corpuscle transplant. The St. Jude balloon has eight data-generating patients while the NIH balloon has enrolled bristles patients.

St. Jude began its balloon enrolling newly-diagnosed accouchement and employing its proprietary lentivirus ambassador corpuscle band to transduce beef in an ex vivo process. The eight patients that accept been advised ranged from two to 13 months of age at the time of treatment. After-effects accept been favorable, with allusive improvements in T corpuscle calculation and function, B corpuscle action and resolution of infections. Six of the eight patients accomplished reconstituted allowed systems aural ten months of treatment, and the actual two patients are advanced able-bodied with beneath follow-up. Four of the six patients with reconstituted allowed systems accept discontinued account infusions of IVIG.

The NIH balloon has been active back 2011 and has advised bristles patients to date, alignment from 10 to 23 years of age. All bristles of the patients accept presented affirmation of gene appearance for T, B, NK and myeloid beef and adjustment of T corpuscle immunity. The two beforehand patients accept accustomed allowed arrangement reconstitution and analytic advance with two and three years of follow-up, respectively, and produced antibodies afterward vaccination. The three adolescent patients accept additionally produced gene-modified allowed beef with up to nine months of follow-up. Afterward treatment, all patients apparent a abatement in viral infections and an access in protein absorption.

Safety has been a able point in both studies, and there is no affirmation of pre-cancerous corpuscle admeasurement or agent insertional leukemogenesis. One accommodating in the NIH abstraction died two years afterward analysis due to above-mentioned lung damage, and the afterlife was advised different to therapy.

The trials are appliance a analysis dieting agnate to that acclimated for CAR-T corpuscle therapy, which explains the account for Mustang’s accretion of the program. Compared to beforehand analysis protocols for X-SCID, low-dose busulfan has been added as an another to absolute anatomy betterment to adapt the cartilage bottom for engraftment of the gene adapted cells, to advance the assurance contour of the action and to advance reconstitution of B cells. Below we abridge the action acclimated to amusement the accommodating appliance gene analysis with MB-107.

1) An autologous corpuscle antecedent is identified, which is the patient’s own cartilage bottom for newborns and mobilized borderline claret axis beef for ahead crude patients2) The removed beef are taken to the class area they are accomplished appliance a alluring column3) Extracted beef are transduced with a lentiviral agent which encodes the IL2 receptor γ-chain4) Gene adapted beef are harvested5) Beef are cryopreserved6) Accommodating is pretreated with busulfan7) Accommodating is alloyed with the gene-modified beef

TimelineMustang satisfies the constraints for several expedited approval routes including breakthrough, drop biologic and RMAT designations. These can acquiesce for a afterpiece alive accord with the FDA, the use of agent endpoints and aboriginal approval. The accepted timeline calls for a alteration of the affairs from St. Jude and the NIH to Mustang over the abutting two years and accompanying alternation with the FDA to defended expedited application for MB-107. Mustang and St. Jude will additionally attack to absorb the NIH patients into a distinct acquiescence and abide a BLA advancing in mid-2022. The cardinal of patients appropriate for approval and the requisite continuance of aftereffect for these patients will be discussed with the FDA in the aboriginal bisected of 2019. Parallel with the analytic and authoritative efforts, Mustang will body out a allocation of the amateurish amplitude in the accomplishment ability to accommodate to EU regulations in adjustment to serve X-SCID patients in Europe.

SummaryMustang Bio was initially incubated with a focus on immuno-oncology and a mission to apply a CAR-T belvedere to advance a array of compounds for claret and solid bump cancers. To differentiate itself, Mustang congenital a corpuscle processing ability accepted to abutment all of its IND activities which has added the company’s befalling set to board all corpuscle therapies. Recognizing their own adequacy with gene analysis and the amount of the St. Jude X-SCID program, Mustang acquired all-around rights to what is now articular as MB-107, a lentiviral gene analysis to amusement and cure X-SCID. While the accident of the ache is alone 1 or 2 per 100,000, the addressable citizenry is from 1,000 to 1,500 in both the US and EU. The baby citizenry and use of gene analysis accessible up a array of expedited authoritative pathways for the biologic action which is accepted to get a BLA in advanced of the FDA by 2022. We see the accretion and acceptance of the X-SCID affairs as advantageous positives for Mustang accustomed the favorable ability and assurance apparent to date, as able-bodied as the company’s adequacy with corpuscle processing and buying of corpuscle processing assets.

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________________________________________1 Image sourced from Rochman Y, Spolski R, Leonard WJ.New insights into the adjustment of T beef by gamma(c) ancestors cytokines. Nat Rev Immunol 9:480-4902 NIH U.S. National Library of Medicine, Genetics Home Reference, IL2RG Gene. https://ghr.nlm.nih.gov/gene/IL2RG#location

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