On November 13, 2018 RadioMedix Inc. (a Texas-based clinical stage biotechnology company) and Orano Med (a nuclear biotechnology company) reported that the United States Food & Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to AlphaMedixTM for the treatment of neuroendocrine tumors (NETs) (Press release, RadioMedix, NOV 13, 2018, View Source [SID1234531347]). AlphaMedixTM is a 212Pb-labeled somatostatin analogue used for the targeted alpha therapy (TAT) of neuroendocrine tumors (NETs).

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"TAT has brought new hope to our patients with cancer, and the range of available therapies in this area will only increase in the near future. We are pleased to conduct the first TAT clinical trial in the U.S. for neuroendocrine cancers, with the aim of registration of the drug," said Dr. Ebrahim S. Delpassand, CEO of RadioMedix and Medical Director of the clinical studies. "We are hoping that AlphaMedixTM will provide a breakthrough therapy that will benefit our patients soon," added Dr. Delpassand. "We are excited about the progress of our dose escalation studies of AlphaMedixTM", echoed Dr. Izabela Tworowska, Ph.D., CSO of RadioMedix.

The mechanism of action of AlphaMedixTM relies on the delivery of a cytotoxic load of alpha-emitters to SSTR-overexpressing neuroendocrine tumors. Alpha- emitters are highly energetic radioactive particles that can cause irreversible damage to the cancer cells. RadioMedix and Orano Med initiated the Phase 1 clinical trial of the safety and preliminary efficacy of AlphaMedixTM in February of 2018 (NCT03466216); the study is currently recruiting patients at the Excel Diagnostics and Nuclear Oncology Center in Houston, TX U.S.

"FDA’s decision to grant Orphan drug Designation is an important regulatory milestone for AlphaMedixTM, a key program in our pipeline of 212Pb-labeled compounds," said Julien Dodet, CEO of Orano Med. "We believe TAT with 212Pb represents a promising new approach and that AlphaMedixTM has the potential to have a meaningful impact on patients".

Orphan designation is granted by the FDA Office of Orphan Products Development to advance the evaluation and development of safe and effective therapies for the treatment of rare diseases or conditions affecting fewer than 200,000 people in the U.S. The designation provides certain benefits to drug developers such as seven-year marketing exclusivity upon FDA approval, tax credits for qualified clinical testing or prescription drug user fee exemption.

About Neuroendocrine Tumors

Neuroendocrine tumors (NETs) are a heterogeneous group of rare neoplasms that originate from neuroendocrine cells. These neoplasms occur mostly in the gastrointestinal tract and pancreas, but can also occur in other tissues including thymus, lung, and other uncommon sites such as ovaries, heart, and prostate. Most NETs strongly express somatostatin receptors (SSTRs).

About AlphaMedixTM

AlphaMedixTM is a radiolabeled SSTR-targeting therapeutic investigational drug for the treatment of NETs patients. The product consists of SSTR-targeting peptide complex radiolabeled with 212Pb and serve as an in vivo generator of alpha-emitting particles. 212Pb isotope is particularly suitable for SSTR therapy applications based upon its half-life, energy and decay properties.

On November 13, 2018 Mustang Bio, Inc. ("Mustang") (NASDAQ: MBIO), a company focused on the
development of novel immunotherapies based on proprietary chimeric antigen receptor engineered T cell ("CAR T")
technology and gene therapies for rare diseases, reported financial results and recent corporate highlights for the third quarter ended September 30, 2018 (Press release, Mustang Bio, NOV 13, 2018, View Source [SID1234531346]).

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Manuel Litchman, M.D., President and Chief Executive Officer of Mustang, said, "The third quarter of 2018 and recent months have been marked by clinical progress and a key addition to our management team with the appointment of Martina A. Sersch, M.D., Ph.D., as Chief Medical Officer. Notably, we expanded our pipeline into gene therapy by securing an exclusive worldwide license agreement with St. Jude Children’s Research Hospital ("St. Jude") for the development of a first-in-class, ex vivo, clinical-stage lentiviral gene therapy for the treatment of X-linked severe combined immunodeficiency ("X-SCID"). Data from a multicenter Phase 1/2 trial led by St. Jude in infants under the age of two years old are extremely encouraging. Eight patients under the age of two with X-SCID have been treated to date, with results presented at the 21st Annual Meeting of the American Society of Gene & Cell Therapy in May 2018. The therapy was well tolerated. In addition, six patients achieved reconstituted immune systems within three to four months following treatment, with the remaining two patients continuing to progress favorably in earlier stages of recovery. Four of these six patients have discontinued monthly infusions of intravenous immunoglobulin, and the remaining patients, at earlier stages of recovery, continue to progress favorably. In three patients who had disseminated infections prior to therapy, all infections resolved completely. In addition, the therapy is being investigated in patients over the age of two in a second Phase 1/2 trial at the National Institutes of Health ("NIH"), with equally encouraging data and an excellent safety profile to date. The two patients with the longest follow-up have seen sustained restoration of antibody production after immunization, and all five patients treated experienced a decrease in viral infections and overall clinical improvement."

Dr. Litchman continued, "We also recently announced updates on two Phase 1 clinical trials at City of Hope using our
HER2-specific CAR T cell therapy, including a first-of-its-kind trial using intraventricular delivery of CAR T cells to brains of patients with HER2-positive breast cancer with brain metastases. As we look ahead to the fourth quarter of 2018, we look forward to filing our first Investigational New Drug (IND) application to support a Phase 1/2 trial of MB-102 in acute myeloid leukemia ("AML"), blastic plasmacytoid dendritic cell neoplasm and high-risk myelodysplastic syndrome."

Recent Corporate Highlights:
• In July 2018, Mustang completed a pre-IND meeting with the U.S. Food and Drug Administration ("FDA") for MB102 (CD123 CAR T). Based on the meeting, Mustang expects to file an IND in the fourth quarter of 2018 to support a Phase 1/2 trial of MB-102 in AML, blastic plasmacytoid dendritic cell neoplasm and high-risk myelodysplastic
syndrome.
• In August 2018, Mustang announced that it entered into an exclusive worldwide license agreement with St. Jude
for the development of a first-in-class ex vivo lentiviral gene therapy for the treatment of X-SCID, also known as
bubble boy disease. The therapy is currently being evaluated in a Phase 1/2 multicenter trial in infants under the
age of two. This study is the world’s first lentiviral gene therapy trial for infants with X-SCID. The therapy is also
being investigated in patients over the age of two in a second Phase 1/2 trial at the National Institutes of Health
("NIH").
• In October 2018, Mustang appointed Martina A. Sersch, M.D., Ph.D., as Chief Medical Officer.
• Also in October 2018, Mustang announced that a first-of-its-kind Phase 1 clinical trial evaluating the safety and
effectiveness of intraventricular delivery of CAR T cells to the brains of patients with HER2-positive breast cancer
with brain metastases has been initiated at City of Hope. In addition, Mustang announced that City of Hope dosed
the first patient in a Phase 1 clinical trial of HER2-specific CAR T cells in treating recurrent or refractory grade IIIIV
glioma. The trial will evaluate the side effects and best dose of HER2-specific CAR T cells in treating patients
with grade III-IV glioma that has come back or does not respond to treatment.
Financial Results:
• As of September 30, 2018, Mustang’s consolidated cash, cash equivalents, short-term investments (certificates of
deposit) and restricted cash totaled $41.3 million, compared to $47.2 million as of June 30, 2018, and $61.5 million
as of December 31, 2017, a decrease of $5.9 million for the quarter and a decrease of $20.2 million year-to-date.
• Research and development expenses were $5.3 million for the third quarter of 2018, compared to $2.2 million for
the third quarter of 2017. Non-cash, stock-based compensation expenses included in research and development
were $0.7 million for third quarter of 2018, compared to $0.3 million for the third quarter of 2017.
• Research and development expenses from license acquisitions were $1.0 million for the third quarter of 2018,
compared to $0.3 million for the third quarter of 2017.
• General and administrative expenses were $1.3 million for the third quarter of 2018, compared to $4.6 million for
the third quarter of 2017. Non-cash, stock-based compensation expenses included in general and administrative
expenses were $0.2 million for the third quarter of 2018, compared to $0.5 million for the third quarter of 2017.
• Net loss attributable to common stockholders was $7.5 million, or $0.28 per share, for the third quarter of 2018,
compared to $6.9 million, or $0.27 per share, for the third quarter of 2017. Net loss attributable to common
stockholders was $18.9 million, or $0.70 per share, for the first nine months of 2018, compared to $15.7 million,
or $0.63 per share, for the first nine months of 2017.

On November 13, 2018 ProMIS Neurosciences, Inc. (TSX: PMN); (OTCQB: ARFXF), a biotechnology company focused on the discovery and development of antibody therapeutics targeting toxic oligomers implicated in the development of neurodegenerative diseases, reported its operational and financial results for the three and nine months ended September 30, 2018 (Press release, ProMIS Neurosciences, NOV 13, 2018, View Source [SID1234531345]).

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"Over the first three quarters of 2018, we focused on three key priorities to advance our business", stated ProMIS Executive Chairman, Eugene Williams. "First, to continue to advance PMN310, our lead therapeutic antibody for Alzheimer’s disease, toward the goal of generating initial clinical trial results in 2020. For this purpose, we anticipate using an innovative trial design with evaluation of novel biomarkers to support assessment of signs suggestive of early efficacy; second, to expand our portfolio by developing therapeutic antibodies targeting toxic oligomers of alpha-synuclein for Parkinson’s disease (PD), toxic aggregates of Tar-DNA binding protein (TDP43) for ALS and toxic forms of tau protein, implicated in the development of Alzheimer’s and other dementias; and third, to actively reach out to the pharmaceutical industry with a view to partnering one or more of these programs."

Recent Corporate Highlights

During the nine months ended September 30, 2018, we received proceeds of $1,797,640 related to the exercise of common stock warrants and stock options. The warrants were exercisable at either $0.17, $0.20 or $0.30.
On July 10, the Company presented preclinical data on its lead product candidate for Alzheimer’s disease, PMN310, at the 2018 Alzheimer’s Association International Conference (AAIC). Results indicated that PMN310 shows potential for best-in-class selectivity against toxic oligomers of amyloid beta, considered a root cause of Alzheimer’s disease.
On August 21, we announced that our lead antibody candidate for Alzheimer’s disease, PMN310, showed no binding to amyloid beta (Aβ) plaque in AD brain samples in stark contrast to BAN2401 and aducanumab which both displayed robust Aβ plaque reactivity. These findings extend the results ProMIS announced in January 2018, showing greater selectivity of PMN310 for Aβ oligomers compared to aducanumab. Binding of therapeutic antibodies to Aβ deposits in brain tissue, in particular blood vessels, is believed to underlie the development of ARIA (amyloid-related imaging abnormalities; brain swelling and microhemorrhages) in treated AD patients. Lack of PMN310 binding to amyloid deposits in Alzheimer’s brain tissue may eliminate dose-limiting brain swelling seen in clinical trials with BAN2401 and aducanumab.
On September 13, we announced the appointment of James Kupiec, MD, to the position of Chief Medical Officer. Over the past 17 years Dr. Kupiec has held positions of increasing responsibility at Pfizer, including Vice President, Neuroscience Research Unit, Pfizer Worldwide Research and Development, and Vice President, Global Clinical Leader for Parkinson’s Disease.
On October 11, we announced the identification of several potential antibody therapeutic candidates aimed at selectively targeting toxic oligomers of the protein α-synuclein, considered a root cause of Parkinson’s disease (PD).
Financial Results

Results of Operations – Three months ended September 30, 2018 and 2017

The net loss for the three months ended September 30, 2018, was $2,912,244, compared to a net loss of $1,618,681 for the three months ended September 30, 2017. The increased loss in the current period reflects the costs associated with operating the Company’s AD therapeutics program, increased contract research and consultant salaries and associated costs, supporting its patent portfolio, increased share-based compensation and general corporate expenditures.

Research and development expenses for the three months ended September 30, 2018, were $1,867,648, as compared to $1,233,323 in the three months ended September 30, 2017. Costs were higher in the current period due to higher research program costs for the AD therapeutics program, recruiting expenses and higher costs to support its patent portfolio, offset by lower stock-based compensation.

General and administrative expenses for the three months ended September 30, 2018, were $1,044,596, as compared to $392,103 in the three months ended September 30, 2017. The increase in expenditures in the current period reflects higher consultant salaries and associated costs, other professional fees, investor/public relations, and stock-based compensation.

Results of Operations – Nine months ended September 30, 2018 and 2017

The net loss for the nine months ended September 30, 2018, was $6,683,714, compared to a net loss of $4,894,280 for the nine months ended September 30, 2017. The increased loss in the current period reflects the costs associated with operating the Company’s AD therapeutics program, increased contracted research and consultant salaries and associated costs, supporting its patent portfolio, increased share-based compensation and general corporate expenditures.

Revenues for the nine months ended September 30, 2018, and 2017 were nominal and relate to legacy technologies.

Research and development expenses for the nine months ended September 30, 2018, were $4,096,729, as compared to $3,084,683 in the nine months ended September 30, 2017. Costs are higher in the current period due to higher research program costs for the AD therapeutics program, recruiting expenses and higher costs to support its patent portfolio, offset by lower stock-based compensation.

General and administrative expenses for the nine months ended September 30, 2018, were $2,587,583, as compared to $1,812,160 in the nine months ended September 30, 2017. The increased expenditures in the current period reflect increased consultant salaries and associated costs, other professional fees, investor/public relations and higher stock-based compensation, offset by foreign exchange gains.

Outlook

The Company plans to further advance its AD portfolio, with a focus on the development of PMN310 with a goal of generating initial clinical trial results in 2020. Based on the highly selective binding of PMN310 to the toxic Aβ oligomers and lack of off-target binding to non-toxic forms of Aβ (monomer, plaque), the ProMIS AD program will continue to develop data further supporting potential best-in-class safety and efficacy versus other Aβ-directed therapies currently in development. The Company also plans to pursue generation of selective antibodies targeting toxic forms of the protein tau for treatment of AD and other tau-related dementias.

Finally, using its unique technology platform, ProMIS will advance work to identify and validate selective antibody therapies for the toxic oligomers of alpha synuclein in PD and TDP43 in ALS and frontotemporal dementia, with a view to partnering these assets

On November 13, 2018 Molecular Templates, Inc. (Nasdaq: MTEM, "Molecular" or "Molecular Templates"), a clinical-stage oncology company focused on the discovery and development of the company’s proprietary engineered toxin bodies (ETBs), which are differentiated, targeted, biologic therapeutics for cancer, reported financial results for the third quarter of 2018 (Press release, Molecular Templates, NOV 13, 2018, View Source [SID1234531340]). As of September 30, 2018, Molecular’s cash and cash equivalents totaled $78.7 million, this does not include $30 million received from Takeda in October 2018. Molecular’s current cash balance is expected to fund operations into 1H 2021.

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"Our recently announced CD38 collaboration with Takeda enables the development of TAK-169, the most potent ETB we have created with our platform to date. Importantly, the upfront payment from Takeda and the equity financing that we closed in September provide the funding to allow us to generate clinical data from multiple pipeline candidates throughout 2019 and beyond," said Eric Poma, Ph.D., Molecular Templates’ Chief Executive and Scientific Officer. "In 2019, our lead program MT-3724 will be in multiple Phase II studies and we expect to start clinical trials for three additional ETBs targeting CD38, HER2, and PD-L1."

Company Highlights and Upcoming Milestones

Corporate

On September 19, 2018, Molecular announced an agreement with Takeda for the joint development of CD38-targeted ETBs for the treatment of multiple myeloma. TAK-169, the lead development candidate, is a CD38-targeted ETB that resulted from a previous discovery collaboration between the two companies. Under the terms of the agreement, Takeda made an upfront payment of $30 million and Molecular is eligible to receive development, regulatory and commercial milestone payments of up to $632.5 million if Molecular exercises its co-development option or $337.5 million if Molecular does not exercise or opts out of its co-development option. Takeda has also agreed to pay royalties on the sales of the commercial products developed through the collaboration. The royalty percentages would range from low double-digits to low twenties if Molecular exercises its option to co-develop, and from high-single digits to low teens if Molecular does not exercise its option to co-develop. Molecular and Takeda will share equally in the development costs.
On September 18, 2018, Molecular entered into a Cancer Research Grant Contract with the Cancer Prevention and Research Institute of Texas (CPRIT), in connection with a grant of approximately $15.2 million awarded by CPRIT to Molecular in November 2016 to fund research of a cancer therapy involving a CD38 targeting ETB (MT-4019). Molecular may also use such funds to develop a replacement CD38 targeting ETB, with or without a partner and expects to use this grant to fund development of TAK-169.
On September 25, 2018, Molecular announced the closing of an underwritten public offering of its common stock, which generated gross proceeds of approximately $52 million.
TAK-169

Following the announcement of the CD38 joint development agreement in September, Takeda and Molecular are conducting IND enabling studies for TAK-169, which is expected to enter the clinic for the treatment of multiple myeloma in 2019.
MT-3724

Molecular expects to begin enrollment in 4Q18 for a Phase II combination study with MT-3724 and chemotherapy in earlier lines of diffuse large B-cell lymphoma (DLBCL).
In 1Q19 Molecular expects to start a Phase II monotherapy study, which has the potential to be a pivotal study.
Molecular expects to initiate a second Phase II combination study with MT-3724 and Revlimid (lenalidomide) in earlier lines of DLBCL in 1Q19.
Research

Molecular expects to file an IND application for an ETB targeting HER2 in 1Q19.
Molecular expects to file an IND application for an ETB targeting PD-L1 (with antigen seeding) in 2H19.
Several other ETB candidates are in pre-clinical development, targeting both solid and hematological cancers.
Takeda Multi-Target Collaboration

Takeda and Molecular are conducting lead optimization for ETBs against two undisclosed targets selected by Takeda under the collaboration. Should Takeda exercise its option to license ETBs for both targets, Molecular would receive $25 million and would be eligible to receive up to $547 million in milestone payments and tiered royalties on sales.
Financial Results

The net loss attributable to common shareholders for the third quarter of 2018 was $5.2 million, or ($0.19) per basic and diluted share. This compares with a net loss attributable to common shareholders of $11.1 million, or ($0.62) per basic and diluted share, for the same period in 2017.

Revenues for the third quarter of 2018 were $6.8 million, compared to $0.6 million for the same period in 2017. Revenues for the third quarter of 2018 were comprised of grant revenue from the Cancer Prevention & Research Institute of Texas, and revenues from collaborative research and development agreements. Total research and development expenses for the third quarter of 2018 were $8.3 million, compared with $2.5 million for the same period in 2017. Total general and administrative expenses for the third quarter of 2018 were $3.5 million, compared with $4.0 million for the same period in 2017.

The net loss attributable to common shareholders for the nine months ended September 30, 2018 was $23.7 million, or ($0.87) per basic and diluted share. This compares with a net loss attributable to common shareholders of $17.2 million, or ($2.75) per basic and diluted share, for the same period in 2017.

Revenues for the nine months ended September 30, 2018 were $8.6 million, compared to $2.6 million for the same period in 2017. Revenues for the nine months ended September 30, 2018 were comprised of grant revenue from the Cancer Prevention & Research Institute of Texas, and revenues from collaborative research and development agreements. Total research and development expenses for the nine months ended September 30, 2018 were $22.6 million, compared with $4.8 million for the same period in 2017. Total general and administrative expenses for the nine months ended September 30, 2018 were $10.2 million, compared with $8.2 million for the same period in 2017

On November 13, 2018 Rubius Therapeutics, Inc. (Nasdaq: RUBY), a biotechnology company developing an entirely new class of allogeneic cellular therapies, reported third quarter 2018 financial results and operational progress (Press release, Rubius Therapeutics, NOV 13, 2018, View Source [SID1234531334]).

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"During the third quarter, we remained focused on advancing our first program, RTX-134, for the treatment of phenylketonuria. We are on track to file an Investigational New Drug application during the first quarter of 2019," said Pablo J. Cagnoni, M.D., chief executive officer of Rubius Therapeutics. "We are also continuing to advance our earlier pipeline, which we believe holds broad therapeutic potential across cancer, autoimmune disease and additional enzyme deficiencies. In order to successfully bring our medicines to patients, we are ensuring that we will have state-of-the-art manufacturing capabilities in place, as shown through the purchase, in July, of our 135,000-square foot manufacturing facility in Smithfield, RI."

Third quarter highlights include:

· On track to submit first IND for lead program, RTX-134, during the first quarter of 2019, and an additional three to four INDs during 2019 and 2020

· Closed initial public offering (IPO) in July 2018, raising $254.3 million in net proceeds

· Acquired and initiated renovations on manufacturing facility in Smithfield, RI; facility is expected to be operational by the end of 2020

· Continued to generate promising preclinical data in support of additional pipeline programs; data expected to be published during 2019

· Strengthened internal capabilities in discovery, platform development and manufacturing and grew the organization to 110 employees to predominantly support research and development (R&D) activities

Third Quarter Financial Results

Net loss for the third quarter of 2018 was $26.4 million or $0.42 per common share, compared to $11.9 million or $1.48 per common share in the third quarter of 2017.

In the third quarter of 2018, Rubius invested $14.4 million in R&D related to its novel RED PLATFORM and towards expanding and advancing its product pipeline, compared to $6.1 million in the third quarter of 2017. The year-over-year increase was due to an additional $2.9 million of costs incurred in preparation for the Phase 1/2a clinical trial for RTX-134, and $3.6 million was associated with personnel costs and stock-based compensation driven by increases in R&D headcount to support Rubius’ goal of delivering four to five IND’s during 2019 and 2020.

G&A expenses were $13.2 million during the third quarter of 2018, as compared to $5.8 million for the third quarter of 2017. The higher costs were primarily driven by a $4.6 million increase in stock-based compensation and a $1.9 million increase in personnel costs and professional fees to support the Company’s growth and to operate as a public company.

Nine Month Financial Results

Net loss for the first nine months of 2018 was $62.0 million or $2.33 per common share, compared to $27.0 million or $3.42 per common share in the first nine months of 2017.

In the nine months ended September 30, 2018, Rubius invested $35.2 million in R&D related to its novel RED PLATFORM and towards expanding and advancing its product pipeline, compared to $14.6 million in the first nine months of 2017. The year-over-year increase was due to an increase of $5.8 million of costs incurred in preparation for the Phase 1/2a clinical trial for RTX-134, and $6.6 million in personnel costs and stock-based compensation driven by increases in R&D headcount to support Rubius’ goal of delivering four to five IND’s during 2019 and 2020.

G&A expenses were $27.3 million during the first nine months of 2018, as compared to $11.2 million for the same period in 2017. The higher costs were primarily driven by a $8.7 million increase in stock-based compensation and a $6.0 million increase in personnel costs and professional fees to support the Company’s growth and to operate as a public company.

During the third quarter of 2018, the Company adopted new guidance for the accounting for stock-based payments to nonemployees, effective as of January 1, 2018. As a result of this adoption, previously reported amounts for the six months ended June 30, 2018 for R&D expenses and G&A expenses were reduced by $0.7 million and $8.0 million, respectively.

Cash Position

As of September 30, 2018, cash, cash equivalents and investments grew significantly to $408.9 million as compared to $104.3 million as of December 31, 2017, providing Rubius with a cash runway into 2021. The increase in cash reflects $254.3 million of net proceeds from the company’s IPO during the third quarter of 2018 and $101.0 million of net proceeds received from its Series C preferred stock financing during the first quarter of 2018. The proceeds received from the financings were offset by $38.6 million used in operations during the nine-month period and $11.7 million of capital purchases, including $8.0 million to acquire the manufacturing facility in Smithfield, Rhode Island.

About Phenylketonuria and RTX-134

Phenylketonuria (PKU) is an inherited, rare enzymatic disorder characterized by the body’s inability to effectively metabolize the amino acid phenylalanine. The accumulation of phenylalanine in the blood causes damage to the central nervous system and a range of symptoms, including intellectual disability, delayed development and impaired cognitive function. RTX-134 is an allogeneic cellular therapy for the treatment of PKU, which expresses the enzyme phenylalanine ammonia lyase (PAL) inside the cell. In preclinical studies, phenylalanine was shown to diffuse into RTX-134, where PAL converted phenylalanine into ammonia and trans-cinnamic acid, metabolites that are cleared by the body. Compared to current therapeutic interventions, RCT product candidates may have a longer and more sustained treatment duration given the 120-day half-life of red blood cells and may avoid immune-driven adverse events and reduction in efficacy, which result from antibody formation.