On February 13, 2018 Myovant Sciences (NYSE: MYOV), a clinical-stage biopharmaceutical company focused on developing and commercializing innovative therapies for women’s health and endocrine diseases, reported corporate updates and reported financial results for the third fiscal quarter ended December 31, 2017 (Press release, Myovant Sciences, FEB 13, 2018, http://investors.myovant.com/news-releases/2018/02-13-2018-210634955 [SID1234523951]).

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"We continue to execute on each of our ongoing global Phase 3 development programs of relugolix for the treatment of endometriosis-associated pain, heavy menstrual bleeding associated with uterine fibroids, and advanced prostate cancer with the goal of completing enrollment in each program this year," stated Lynn Seely, M.D., President and Chief Executive Officer of Myovant Sciences. "In addition, in October, we secured flexible financing commitments of up to $140 million, which will help support the continued advancement of our Phase 3 programs."

Third Fiscal Quarter 2017 Business Highlights

Positive results in two Phase 3 clinical studies conducted by Takeda Pharmaceutical Company Limited ("Takeda") to evaluate the efficacy and safety of relugolix for the treatment of uterine fibroids.

On October 2, 2017, Myovant announced that Takeda reported positive top-line results from a Phase 3 study in Japan evaluating the efficacy and safety of relugolix compared with leuprorelin for the treatment of women with heavy menstrual bleeding associated with uterine fibroids. Relugolix met the study’s primary endpoint, the proportion of patients achieving a pre-defined reduction in menstrual bleeding, demonstrating an 82.2% response rate, and was observed to be statistically non-inferior to leuprorelin (p = 0.0013). The incidence of adverse events in the study was generally similar between treatment groups and consistent with the mechanism of action of the study medications.
On November 9, 2017, Myovant announced that Takeda reported positive top-line results from a Phase 3 study in Japan evaluating the efficacy and safety of relugolix compared with placebo for the treatment of pain associated with uterine fibroids. Of the women treated with relugolix, 57.6% achieved a marked improvement in pain symptoms compared to 3.1% treated with placebo (p < 0.0001). Adverse events in the study were consistent with the mechanism of action of relugolix and adverse events observed in previous clinical studies.
Takeda plans to submit the data from both studies to regulatory authorities in Japan for marketing authorization of relugolix for the treatment of uterine fibroids. Myovant will be solely responsible for obtaining FDA approval for relugolix in the United States.
Secured flexible financing commitments of up to $140 million. On October 16, 2017, Myovant announced that it had secured up to $140 million in flexible financing commitments from NovaQuest Capital Management ("NovaQuest") and Hercules Capital, Inc. ("Hercules"). The NovaQuest financing is comprised of a note purchase commitment of up to $60 million and an equity purchase commitment of up to $40 million. An additional $40 million of debt financing capacity is committed in the form of a term loan facility from Hercules. Myovant plans to use the net proceeds from both financings to fund the ongoing Phase 3 development of relugolix in uterine fibroids, endometriosis and advanced prostate cancer. Pursuant to the agreements, upon closing, Myovant issued notes and shares of common stock of approximately $33 million under the financing commitments.

Third Fiscal Quarter 2017 Financial Summary

Research and development (R&D) expenses for the quarter ended December 31, 2017 were $34.9 million, compared to $6.2 million for the comparable period in 2016. The increase over the prior year period is primarily due to costs associated with the progress in our five ongoing Phase 3 clinical trials of relugolix which were initiated in 2017. R&D expenses for the three months ended December 31, 2017 consisted primarily of clinical trial-related costs of $28.4 million, personnel expenses of $3.2 million, share-based compensation expense of $1.0 million, and costs billed to us under the services agreements with Roivant Sciences Ltd. and Roivant Sciences, Inc. ("the Services Agreements") of $1.9 million, including personnel expenses and third-party costs associated with our ongoing clinical trials and other research programs.

General and administrative (G&A) expenses for the quarter ended December 31, 2017 were $6.6 million, compared to $2.9 million for the same period in 2016. G&A expenses for the three months ended December 31, 2017 consisted primarily of personnel-related and general overhead expenses of $2.7 million, share-based compensation expense of $2.3 million, legal and professional fees of $0.6 million and costs of $1.0 million billed to us under the Services Agreements, including personnel expenses, overhead allocations and third-party costs.

Interest Expense for the quarter ended December 31, 2017 was $0.9 million and consisted of interest expense accrued and paid under the financing agreements with NovaQuest and Hercules as well as the associated amortization of debt discount and issuance costs. There was no interest expense for the comparable prior year period.

Net loss for the quarter ended December 31, 2017 was $41.8 million, or $0.70 per share, compared to a net loss of $8.1 million or $0.15 per share for the same period in 2016. The increase in net loss was driven by the increase in costs associated with our ongoing LIBERTY 1 and LIBERTY 2, SPIRIT 1 and SPIRIT 2, and HERO Phase 3 clinical studies which were initiated in 2017, as well as increased personnel expenses to support Myovant’s growing operations.

Cash and committed funding totaled $235.9 million at December 31, 2017 consisting of $128.9 million of cash and financing commitments totaling $107.0 million available under our financing agreements with NovaQuest and Hercules.

About Relugolix

Relugolix is an oral, once-daily, small molecule gonadotropin-releasing hormone (GnRH) receptor antagonist that has been evaluated in over 1,600 study participants in Phase 1, Phase 2 and Phase 3 clinical trials. In these trials, relugolix has been shown to be generally well tolerated and to suppress estrogen and progesterone levels in women and testosterone levels in men. Common side effects are consistent with suppression of these hormones. In the ongoing Phase 3 SPIRIT clinical trials in women with endometriosis-associated pain and the ongoing Phase 3 LIBERTY clinical trials in women with heavy menstrual bleeding associated with uterine fibroids, relugolix will be evaluated with and without low-dose hormonal add-back therapy, the addition of which is expected to decrease potential side effects such as bone mineral density loss and hot flashes. The ongoing Phase 3 HERO study is evaluating relugolix in men with advanced prostate cancer.

On February 13, 2018 Cellectis (Paris:ALCLS) (NASDAQ:CLLS), a clinical-stage biopharmaceutical company focused on developing immunotherapies based on gene-edited allogeneic CAR T-cells (UCART), reported the issuance of two U.S. patents – US 9,855,297 and US 9,890,393 – for the invention of certain uses of RNA-guided endonucleases, such as Cas9 or Cpf1, for the genetic engineering of T-cells (Press release, Cellectis, FEB 13, 2018, View Source [SID1234523941]). The patents came into force on January 2nd, 2018 and February 13th, 2018, respectively.

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Both patents claim methods by which T-cells are gene edited using transient expression of CRISPR/Cas9 components. These inventions are based on the early work initiated by inventors at Cellectis when the CRISPR technology first came to light.

These therapeutic-focused patents follow the grant by the European Patent Office of patent No. EP3004337 for similar inventions and previous intellectual property that Cellectis has obtained over the last two decades for major gene editing technologies including meganucleases, TALEN, MegaTAL and CRISPR.

"Cellectis is a pioneering gene editing company that has always been keen to be at the forefront of all gene editing technologies," said Dr. André Choulika, Cellectis Chairman & CEO. "We have been the first to explore the potential of CRISPR in its early days in various applications, including therapeutics and plants. These early findings ultimately led to the grant of this set of new patents. As such, these patents only reinforce Cellectis’ leadership position in the gene editing industry."

Convinced of their strong value for the future development of engineered CAR T-cells, Cellectis will make these patents available for licensing to companies that are willing to use CRISPR technologies in T-cells. The technical knowledge in these patents could, for example, help users engineer allogeneic CAR T-cells while suppressing genes involved in checkpoint inhibitions, such as PD-1, engineer drug resistance, or remove MHC (Major Histocompatibility Complex) related genes. The technology could also be used to insert a DNA CAR construct by gene targeting a specific locus in the genome of T-cells.

The inventors of these patents are Dr. André Choulika, Chairman & CEO of Cellectis and one of the pioneers in the development of nuclease-based genome editing technologies; Dr. Philippe Duchateau, Cellectis Chief Scientific Officer and seasoned gene editing expert; and Dr. Laurent Poirot, Cellectis Head of Early Discovery and expert of gene functions in immune cells.

Claims 1 and 2 of US 9,855,297:

1. A method of preparing genetically modified primary T-cells for immunotherapy comprising the steps of: (a) transfecting mRNA encoding an RNA-guided endonuclease into the primary T-cells, wherein the RNA-guided endonuclease is expressed from the transfected m RNA; (b) introducing a DNA vector that encodes a specific guide RNA, wherein the specific guide RNA directs the RNA-guided endonuclease to at least one targeted locus in the T-cell genome into the primary T-cells; (c) cleaving at least one targeted locus in the T-cell genome with the RNA-guided endonuclease; (d) generating a genetic modification at the site of the cleavage; and (e) expanding the resulting genetically modified T-cells.

2. The method of claim 1, wherein the RNA-guided endonuclease is Cas9.

Claim 1 of US 9,890,393:

1. A method of preparing T-cells for immunotherapy comprising the step of:

(a) genetically modifying primary T-cells by introduction and/or expression into the cells of at least:

– a RNA-guided endonuclease; and

– a specific guide RNA that directs said endonuclease to at least one targeted locus in the T-cell genome,

wherein said RNA-guided endonuclease is expressed from transfected mRNA;

wherein said RNA-guided endonuclease comprises the amino acid sequence set forth in SEQ ID NO:1 or SEQ ID NO:2; and

(b) expanding the resulting cells.

On February 13, 2018 Neurocrine Biosciences, Inc. (NASDAQ: NBIX) reported its financial results for the fourth quarter and year-end of 2017, and provided an update on the launch of INGREZZA (valbenazine) and clinical development programs for 2018 (Press release, Neurocrine Biosciences, FEB 13, 2018, View Source;p=RssLanding&cat=news&id=2332208 [SID1234523953]).

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"This past year was a year of tremendous progress for Neurocrine with the approval and successful launch of INGREZZA. I am proud of our team’s dedication and commitment to successfully launching INGREZZA while also advancing key development programs in our pipeline," said Kevin Gorman, Ph.D., Chief Executive Officer of Neurocrine Biosciences. "For 2018, we remain focused on maximizing our opportunity with INGREZZA to aid patients impacted by tardive dyskinesia through further disease state awareness and positive patient outcomes. In addition, we expect to make great strides in our many research and development programs, including a potential FDA approval from our collaboration with AbbVie for elagolix in endometriosis, results from a Phase IIb study of INGREZZA for Tourette syndrome, and progressing opicapone for the treatment of Parkinson’s disease."

Fourth Quarter and Year-End 2017 Sales Results

Neurocrine reported net product sales for INGREZZA of $64.5 million for the fourth quarter ended December 31, 2017. Total Company revenues for the fourth quarter were $94.5 million inclusive of a $30 million milestone payment received from AbbVie for the U.S. Food and Drug Administration (FDA) acceptance of the elagolix endometriosis NDA filing in the fourth quarter.

For the year ended December 31, 2017, net product sales of INGREZZA were $116.6 million and total Company revenues of $161.6 million inclusive of $45 million revenue recognized from our collaboration agreements with AbbVie and Mitsubishi Tanabe Pharma Corporation (Mitsubishi Tanabe).

No similar net product sales were reported for the comparable periods of 2016. INGREZZA was made available for commercial distribution on May 1, 2017, and the Company recognizes revenue using a sell-in methodology when products are delivered to select pharmacies or distributors.

For the fourth quarter of 2017, the Company reported net income of $6.9 million, or $0.07 diluted earnings per share, compared to a net loss of $44.7 million, or $0.51 loss per share for the same period in 2016. For the year ended December 31, 2017, the Company reported a net loss of $142.5 million, or $1.62 loss per share, as compared to a net loss of $141.1 million, or $1.63 loss per share for 2016.

Research and development (R&D) expenses were $25.6 million during the fourth quarter of 2017 compared to $22.6 million for the fourth quarter of 2016. The increase in R&D expense is principally due to increased program activity in R&D. For the year ended December 31, 2017, R&D expenses were $121.8 million, compared to $94.3 million for the same period in 2016. This increase is primarily due to a $30 million payment in the first quarter of 2017 from the Company’s entering into an exclusive licensing agreement with BIAL – Portela & CA, S.A. (BIAL) for the development and commercialization of opicapone in the United States and Canada, which was expensed as in-process R&D.

Sales, general and administrative (SG&A) expenses increased to $56.3 million for the fourth quarter of 2017 from $23.7 million for the fourth quarter of 2016. For the year ended December 31, 2017, SG&A expenses were $169.9 million, compared to $68.1 million for the same period in 2016. The increase in SG&A expense, across both periods, is primarily due to commercialization activities for INGREZZA.

The Company’s balance sheet at December 31, 2017, reflected total assets of $817.6 million, including cash, investments and receivables of $797.6 million, compared with total asset balances at December 31, 2016 of $365.1 million.

2018 Financial Guidance

Revenue milestones under the AbbVie agreement for 2018 are expected to be $40 million contingent on an FDA approval of elagolix for endometriosis. Ongoing operating expenses for 2018 should approximate $365 to $395 million. The 2018 anticipated expenses include an estimated $50 million of share-based compensation expense. The increase in operating expenses is largely attributable to increased investment in INGREZZA sales and marketing activities coupled with increased R&D efforts for Tourette syndrome, opicapone, new early stage programs, and post-marketing clinical activities.

Pipeline Highlights and Upcoming Events in 2018

INGREZZA Update

INGREZZA received FDA approval on April 11, 2017, becoming the first medicine approved in the United States for the treatment of adults with tardive dyskinesia. Full commercial efforts for the 40 mg capsule of INGREZZA began on May 1, 2017. On October 4, 2017, the FDA approved the supplemental New Drug Application (NDA) for the 80 mg capsule strength of INGREZZA.

In March 2015, the Company announced that it had entered into an exclusive collaboration and licensing agreement for the development and commercialization of INGREZZA in Japan and other select Asian markets with Mitsubishi Tanabe. In 2017, Mitsubishi Tanabe initiated a pivotal trial of INGREZZA in Asia for the treatment of tardive dyskinesia which generated a $15 million milestone during the third quarter of 2017.

INGREZZA is being investigated in Tourette syndrome and has been granted Orphan Drug Designation by the FDA for the treatment of pediatric patients with Tourette syndrome. Orphan Drug Designation is granted by the FDA to drugs that are intended to treat rare diseases or conditions in the United States.

In addition, the Company has advanced the valbenazine Tourette syndrome program into Phase IIb by initiating the T-Force GOLD study in pediatric patients with Tourette syndrome. This study is a multicenter, randomized, double-blind, placebo-controlled, parallel group, Phase IIb study to evaluate the safety, tolerability, efficacy and optimal dose of once-daily valbenazine in up to 120 pediatric patients with moderate to severe Tourette syndrome over 12 weeks of treatment. The primary endpoint of this study is the change from baseline of the Yale Global Tic Severity Scale between placebo and active treatment groups at the end of week 12 with top-line data expected in late 2018.

Elagolix Update

On October 27, 2017, AbbVie, in collaboration with Neurocrine, announced that the FDA had granted Priority Review to elagolix for the management of endometriosis with associated pain. The FDA grants Priority Review to medicines that it determines have potential to provide significant improvements in the safety and effectiveness of the treatment of a serious disease. Priority Review shortens the FDA review timeframe from ten months from acceptance of the NDA filing to six months. The Prescription Drug User Fee Act (PDUFA) date for the FDA to complete its review is in the second quarter of 2018.

AbbVie is currently completing two replicate Phase III randomized, parallel, double-blind, placebo-controlled clinical trials evaluating elagolix alone or in combination with add-back therapy in women with heavy uterine bleeding associated with uterine fibroids. The studies enrolled approximately 400 subjects each for an initial six-month placebo-controlled dosing period. At the end of the six months of placebo-controlled evaluation, subjects are eligible to enter an additional six-month safety extension study. The primary efficacy endpoint of the study is an assessment of the change in menstrual blood loss utilizing the alkaline hematin method comparing baseline to month six. Additional secondary efficacy endpoints will be evaluated including assessing the change in fibroid volume and hemoglobin. Bone mineral density will be assessed via dual-energy x-ray absorptiometry (DEXA) scan at baseline, at the conclusion of dosing, and six months post-dosing. AbbVie expects initial top-line efficacy data from the uterine fibroid Phase III program during the first quarter of 2018. These two studies will form the basis for an anticipated 2019 supplemental NDA submission to the FDA for the approval of elagolix in the treatment of uterine fibroids.

Opicapone Update

In February 2017, the Company entered into an exclusive licensing agreement with BIAL for the development and commercialization of opicapone in the United States and Canada. Opicapone is a once-daily, peripherally-acting, highly-selective catechol-o-methyltransferase inhibitor being developed as an adjunct therapy to preparations of levodopa/DOPA decarboxylase inhibitors for adult patients with Parkinson’s disease and motor fluctuations. The Company met with the FDA in January to discuss the necessary activities to support an NDA submission and will provide an update on the regulatory path and program timing after official meeting minutes are received later this month.

Congenital Adrenal Hyperplasia (CAH) Program (NBI-74788) Update

In the second quarter of 2017, the Company successfully completed the Phase I, IND-opening study of NBI-74788 in healthy volunteer subjects. The study was a randomized, open-label, two-period crossover study to evaluate the pharmacokinetics (PK), the effect of food on PK, and the safety of NBI-74788 in a total of 16 healthy adults.

The Company began recruitment for a Phase II, proof-of-concept study examining the PK, pharmacodynamics, and safety of NBI-74788 in adult males and females with classic, 21-hydroxylase deficiency CAH in November of 2017. The study will evaluate the relationship between NBI-74788 exposures and specific steroid hormone levels in these subjects and expect data to be available during the first half of 2018.

Conference Call and Webcast Today at 5:00 PM Eastern Time

Neurocrine will hold a live conference call and webcast today at 5:00 p.m. Eastern Time (2:00 p.m. Pacific Time). Participants can access the live conference call by dialing 888-632-3389 (US) or 785-424-1673 (International) using the conference ID: NBIX. The call can also be accessed via the webcast through the Company’s website at View Source

About INGREZZA (valbenazine) Capsules
INGREZZA, a selective VMAT2 inhibitor, is the first FDA approved product indicated for the treatment of adults with tardive dyskinesia, a condition associated with uncontrollable, abnormal and repetitive movements of the trunk, extremities and/or face.

INGREZZA is thought to work by reducing the amount of dopamine released in a region of the brain that controls movement and motor function, helping to regulate nerve signaling in adults with tardive dyskinesia. VMAT2 is a protein in the brain that packages neurotransmitters, such as dopamine, for transport and release in presynaptic neurons. INGREZZA, developed in Neurocrine’s laboratories, is novel in that it selectively inhibits VMAT2 with no appreciable binding affinity for VMAT1, dopaminergic (including D2), serotonergic, adrenergic, histaminergic, or muscarinic receptors. Additionally, INGREZZA can be taken for the treatment of tardive dyskinesia as one capsule, once-daily, together with psychiatric medications such as antipsychotics or antidepressants.

Important Safety Information
Warnings & Precautions
Somnolence
INGREZZA can cause somnolence. Patients should not perform activities requiring mental alertness such as operating a motor vehicle or operating hazardous machinery until they know how they will be affected by INGREZZA.

QT Prolongation
INGREZZA may prolong the QT interval, although the degree of QT prolongation is not clinically significant at concentrations expected with recommended dosing. INGREZZA should be avoided in patients with congenital long QT syndrome or with arrhythmias associated with a prolonged QT interval. For patients at increased risk of a prolonged QT interval, assess the QT interval before increasing the dosage.

Adverse Reactions
The most common adverse reaction (≥5% and twice the rate of placebo) is somnolence. Other adverse reactions (≥2% and >placebo) include: anticholinergic effects, balance disorders/falls, headache, akathisia, vomiting, nausea, and arthralgia.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit MedWatch at www.fda.gov/medwatch or call 1-800-FDA-1088.

Please see INGREZZA full Prescribing Information at www.INGREZZA.com/HCP

On February 13, 2018 RedHill Biopharma Ltd. (NASDAQ:RDHL) (Tel-Aviv Stock Exchange:RDHL) ("RedHill" or the "Company"), a specialty biopharmaceutical company primarily focused on late clinical-stage development and commercialization of proprietary drugs for gastrointestinal diseases and cancer, reported that it will report its fourth quarter and full-year 2017 financial results on Thursday, February 22, 2018 (Press release, RedHill Biopharma, FEB 13, 2018, View Source [SID1234523982]).

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The Company will host a conference call on Thursday, February 22, 2018 at 9:00 am EST to review the financial results and business highlights.

To participate in the conference call, please dial one of the following numbers 15 minutes prior to the start of the call: United States: +1-800-281-7829; International: +1-646-828-8143; and Israel: +972-3-721-9463. The access code for the call is: 2134987.

The conference call will be broadcasted live and will be available for replay on the Company’s website, View Source, for 30 days. Please access the Company’s website at least 15 minutes ahead of the conference call to register, download and install any necessary audio software.

On February 13, 2018 ERYTECH Pharma (Euronext: ERYP- Nasdaq: ERYP), a clinical-stage biopharmaceutical company developing innovative therapies by encapsulating therapeutic drug substances inside red blood cells, reported the selection of Triple Negative Breast Cancer as the next target indication for broadening the scope of eryaspase (GRASPA) development in solid tumors (Press release, ERYtech Pharma, FEB 13, 2018, View Source [SID1234523942]).

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L-asparaginase is a cornerstone treatment in acute lymphoblastic leukemia (ALL), especially for pediatric patients, but the excessive toxicity of conventional L-asparaginase formulations has limited its use in other indications.

The positive Phase 2b result of eryaspase (L-asparaginase encapsulated in red blood cells) in metastatic pancreatic cancer, reported in 2017, represents, to our knowledge, the first-ever evidence of clinical benefit of an asparaginase-based product in a solid tumor indication. In this 141-patient randomized Phase 2b study, eryaspase in combination with chemotherapy demonstrated a 40% reduction in risk of death rate (HR=0.60; p=0.009) compared to chemotherapy alone.

Following these very encouraging results, ERYTECH conducted a comprehensive evaluation to determine other potential solid-tumor indications for developing eryaspase. Metastatic Triple-Negative Breast Cancer (TNBC) has now been selected as the next indication to expand the potential use of eryaspase in solid tumors. TNBC is an aggressive and metabolically active form of breast cancer with high rates of symptomatic metastases. A recent publication in Nature1 supports the hypothesis that restricting availability of asparagine can reduce metastatic progression of cancer cells in breast cancer. One of the most striking observations from our Phase 2b trial in pancreatic cancer was the 40% reduction of new lesions in the eryaspase arm compared to the control arm.

"TNBC is a heterogenous subgroup of breast cancer associated with poor patient outcome and high risk of recurrence compared to other breast cancer subtypes. Aside from BRCA1/2 mutation status, treatment options for TNBC remain limited," commented Iman El-Hariry, MD, PhD, Chief Medical Officer of ERYTECH. "There is growing evidence of altered metabolism in TNBC. The evaluation of eryaspase in metastatic TNBC provides a promising new therapeutic approach, which capitalizes on reprogramming of the metabolic pathways in this disease."

Gil Beyen, Chairman and CEO of ERYTECH, added, "The selection of TNBC as the second solid tumor indication for evaluating eryaspase anti-tumor activity brings hope for improving the health of these women. The safety profile of eryaspase provides additional rationale for combination with currently existing therapies to increase treatment options in TNBC."

The development in TNBC complements ERYTECH’s pipeline of programs, which focus on the development of therapies that target amino acid metabolism of tumor cells. Set-up activities of a Phase 2 proof-of-concept clinical study have started and ERYTECH expects to enroll the first patient in Q3 2018.

About Triple-Negative Breast Cancer (TNBC)

Breast cancer is the most commonly diagnosed cancer in women globally with nearly 1.8 million new cases diagnosed annually2. It is estimated that over 600,000 women each year are diagnosed with breast cancer in the United States and Europe in aggregate3. Approximately 10-20% of breast cancers are TNBC, a form of breast cancer that lacks expression of estrogen receptor (ER), progesterone receptor (PR) and does not overexpress HER24. TNBC is associated with a poorer prognosis when compared to other breast cancer subtypes. As commonly utilized hormone therapy and HER2 targeting agents are not treatment options for women with TNBC, there is significant unmet need for novel therapeutic approaches in this subtype of breast cancer.