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Repare Therapeutics Reports Third Quarter 2020 Financial Results and Operational Highlights

On November 11, 2020 Repare Therapeutics Inc. ("Repare" or the "Company") (Nasdaq: RPTX), a leading clinical-stage precision oncology company enabled by its proprietary synthetic lethality approach to the discovery and development of novel therapeutics, reported financial results for the third quarter ended September 30, 2020, as well as recent business highlights (Press release, Repare Therapeutics, NOV 11, 2020, View Source [SID1234570677]).

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"Since the closing of our IPO in June, we have made substantial and consistent progress to advance the development of our lead RP-3500 program, entering the clinic in July following the opening of a Phase 1/2 US IND for use as a monotherapy and in combination with talazoparib, all in patients with solid tumors," said Lloyd M. Segal, President and Chief Executive Officer of Repare. "We also expect to initiate a Phase 1 clinical trial for RP-6306 in the third quarter of 2021, ahead of our previously conveyed timeline where we anticipated an IND filing in the second half of 2021. We believe that our work in advancing a first-in-class product candidate into the clinic further validates our progress in identifying new synthetic lethal pairs and developing potent and selective inhibitors."

Third Quarter 2020 Review and Operational Updates:

Advanced CCNE-1 synthetic lethal inhibitor (now designated RP-6306) program into Good Laboratory Practice (GLP) toxicology studies ahead of original timeline. The Company anticipates initiating a Phase 1 clinical trial for RP-6306 in the third quarter of 2021, which is ahead of its original guidance of an IND filing in the second half of 2021.
Initiated a Phase 1/2 clinical trial evaluating RP-3500 as a monotherapy and in combination with Pfizer’s PARP inhibitor, talazoparib, in patients with solid tumors. In July 2020, the Company began dosing in a Phase 1/2 clinical trial of RP-3500, a potent and selective oral small molecule inhibitor of ATR (Ataxia-Telangiectasia and Rad3-related protein kinase) for the treatment of solid tumors in patients with specific genome instability-related genetic alterations, including those in the ATM gene (ataxia telangiectasia mutated kinase). RP-3500 will be evaluated as a monotherapy and in combination with Pfizer’s PARP inhibitor, talazoparib. Topline results are expected to be reported in the second half of 2021.
Inaugurated a newly expanded laboratory and office facility in Montreal, Quebec. In September 2020, the Company materially expanded its research footprint with the addition of 17,000 square feet of combined laboratory and office space in a newly built facility. The new facility more than doubled the Company’s laboratory capacity for its CRISPR-enabled genome-wide synthetic lethal target platform, SNIPRx, including dedicated space for work related to accelerating all of Repare’s preclinical assets, including those under its research collaboration with Bristol Myers Squibb.
Appointed new executive officer. In October 2020, Repare appointed Dr. Laurence F. Akiyoshi as its Executive Vice-President, Organizational and Leadership Development. Dr. Akiyoshi has joined Repare’s executive team after having served as an independent consultant to the Company for the past two years. In addition to his work with Repare, Dr. Akiyoshi has operated a private organizational development consulting practice that has advised numerous companies on scaling their organizations to support rapid growth. His clients have included leadership teams at Apple, LinkedIn, CrowdStrike and Box. Dr. Akiyoshi will be principally focused on organization design, leadership development, and attracting and retaining key team members necessary for Repare’s achievement of its corporate objectives.
Third Quarter 2020 Financial Results:

Cash and restricted cash: Cash and restricted cash as of September 30, 2020 were $348.1 million.
Research and development expenses, net of tax credits (Net R&D): Net R&D expenses were $10.1 million and $27.7 million for the three and nine month periods ended September 30, 2020, respectively, as compared to $5.6 million and $14.2 million in the same periods in the prior year, respectively. Increases in R&D for the three and nine month periods ended September 30, 2020 were primarily due to increases in development costs related to Repare’s RP-3500 and RP-6306 programs, as well as increases in personnel-related expenses and certain other R&D expenses.
General and administrative (G&A) expenses: G&A expenses were $4.0 million and $9.6 million for the three and nine month periods ended September 30, 2020, respectively, as compared to $1.3 million and $3.4 million in the same periods in the prior year, respectively. Increases in G&A for the three and nine month periods ended September 30, 2020 were due to increases in payroll and personnel costs as well as increases in legal, professional and D&O insurance costs in connection with preparations for becoming and now operating as a public company.
Net loss: Net loss was $13.8 million, or $0.37 per share in the third quarter of 2020 and $38.2 million, or $2.63 per share, in the first nine months of 2020.
About Repare Therapeutics’ SNIPRx Platform

Repare’s SNIPRx platform is a genome-wide CRISPR-based screening approach that utilizes proprietary isogenic cell lines to identify novel and known synthetic lethal gene pairs and the corresponding patients who are most likely to benefit from the Company’s therapies based on the genetic profile of their tumors. Repare’s platform enables the development of precision therapeutics in patients whose tumors contain one or more genomic alterations identified by SNIPRx screening, in order to selectively target those patients most likely to achieve clinical benefit from resulting product candidates.

The US Oncology Network Enrolls 100,000th Patient in the Oncology Care Model, Enhancing Care While Saving Medicare Over $100 Million

On November 11, 2020 The US Oncology Network (The Network) reported that reached a major milestone recently in its journey to provide high-quality, value-based care to local communities by enrolling its 100,000th patient in the Centers for Medicare & Medicaid Innovation’s (CMMI) Oncology Care Model (OCM) (Press release, US Oncology, NOV 11, 2020, View Source [SID1234570676]). In realizing this goal, the participating Network practices delivered more than $122 million in cumulative savings to Medicare over the program’s first six performance periods (PP). The OCM is a five-year pilot program developed by CMMI to provide higher quality, more coordinated cancer care at the same or lower cost to Medicare. The program is part of Medicare’s ongoing effort to move healthcare to a system based on value rather than volume.

"We are very excited to enroll our 100,000th patient in the Oncology Care Model while also providing over $100 million in cumulative savings to Medicare," said Michael Seiden, MD, PhD, president, The US Oncology Network. "These achievements showcase the ability of practices in The Network to consistently perform well in the OCM while demonstrating that high-quality care and cost management can work in tandem to provide value to all stakeholders."

The OCM is a very large, complex program that requires enhanced patient care, effective cost management and comprehensive clinical data reporting to Medicare, to highlight only a few key elements of the program. Practices across The Network are performing well in the OCM, and most have shown improvement for each subsequent performance period.

"Celebrating our 100,000th OCM patient and seeing better performance each period remind us that success is an ongoing process," said Beatrice Mautner, vice president of Clinical & Innovent Services, The US Oncology Network. "We are continually striving to be at the forefront of value-based care by constantly developing innovative resources and solutions to challenges as they arise, empowering community oncology to lead the way forward and succeed in this new environment."

At the end of the most recent OCM performance period, PP6, significant gains occurred in critical performance areas across the participating Network practices, resulting in:

Improved quality compared to baseline performance during Performance Period 1
Hospitalizations reduced by 7 percent
Emergency department visits decreased by 3 percent
Hospice utilization increased by 5 percent
Increased focus on pain and depression management
The pain level of 94 percent of patients was captured and a plan implemented for elevated pain control
80 percent of patients who reported depression were screened and given strategies for improvement
Enhanced patient experience and services
Shared decision making with patients became a priority at the start of treatment
Patients received a comprehensive treatment plan explained by the physician
Access to care was improved with "Call Us First" campaigns, proactive high-risk outreach, enhanced triage, electronic patient-reported outcomes and same day/next morning urgent care access
Increased support and care coordination were provided through highly trained navigators and social workers
"The impressive results achieved by The Network is a testament to the commitment by the practices to deliver high-quality, value-based care," said Marcus Neubauer, MD, chief medical officer, The US Oncology Network. "Enrolling 100,000 patients into the OCM and earning high performance marks during PP6 are proof that the participating Network practices are major contributors to this program by providing quality care and saving healthcare dollars."

Although many practices participating in the OCM are still looking for a path to success, those in The Network have succeeded in part because they have access to comprehensive, proven resources that have helped them successfully transition to value-based care. These resources include industry-leading technologies that drive evidence-based decision-making at the point of care, advanced analytics for optimal data management and reporting, and innovative pharmacy solutions for efficient drug management. The Network, supported by McKesson, also provides practices access to thought leaders and key staff who have deep expertise in the OCM and value-based care.

"At least 50 percent of The Network practices participating in the OCM have chosen two-sided risk for 2020," noted Stuart Staggs, senior director of Strategic Programs, McKesson. "This demonstrates a very high level of confidence in the support they are receiving from The Network and McKesson that enables them to successfully meet the complex challenges the OCM presents."

The US Oncology Network is committed to ensuring community practices have access to all the resources necessary to successfully accomplish the massive practice transformation required by value-based and alternative payment models. To learn more, visit usoncology.com.

Centers for Medicare & Medicaid Services (CMS) Issues Permanent J-Code for UroGen’s Jelmyto® Effective January 1, 2021

On November 11, 2020 UroGen Pharma Ltd. (Nasdaq: URGN), a biopharmaceutical company dedicated to building and commercializing novel solutions that treat specialty cancers and urologic diseases, reported that the Centers for Medicare & Medicaid Services (CMS) has established a permanent and product-specific J-code for Jelmyto (mitomycin) for pyelocalyceal solution that will take effect on January 1, 2021 (Press release, UroGen Pharma, NOV 11, 2020, View Source [SID1234570673]). The J-code is expected to replace the previously issued temporary C-code and will standardize and facilitate reimbursement in the hospital outpatient, ambulatory surgery center and physician office settings of care beginning on January 1, 2021.

"Having a permanent J-code will simplify billing and streamline reimbursement, making it even more timely and reliable for physicians treating patients with Jelmyto," said Jeffrey Bova, Chief Commercial Officer of UroGen Pharma. "This should translate into improved access to Jelmyto for adult patients seeking a minimally invasive, potentially kidney-sparing treatment option for low-grade upper tract urothelial cancer."

J-codes are reimbursement codes used by commercial insurance plans, Medicare, Medicare Advantage, and other government payers for Medicare Part B drugs like Jelmyto that are administered by a physician. Claims submission and payment are standardized with a J-code, facilitating and streamlining billing and reimbursement.

The J-code for Jelmyto (J9281) has been published online in the CMS Healthcare Common Procedure Coding System (HCPCS) Application Summaries and Coding Decisions (page 36).

About Jelmyto

Jelmyto (mitomycin) for pyelocalyceal solution, is a drug formulation of mitomycin indicated for the treatment of adult patients with low-grade upper tract urothelial cancer (LG-UTUC). Utilizing the RTGel technology platform, UroGen’s proprietary sustained release, hydrogel-based formulation, Jelmyto is designed to enable longer exposure of urinary tract tissue to mitomycin, thereby enabling the treatment of tumors by non-surgical means. Jelmyto is delivered to patients using standard ureteral catheters or nephrostomy tube. The U.S. FDA previously granted Orphan Drug, Fast Track, and Breakthrough Therapy Designations to Jelmyto for the treatment of LG-UTUC. On April 15, 2020, the FDA approved Jelmyto, making it the first drug approved for the treatment of LG-UTUC in adult patients.

APPROVED USE FOR JELMYTO

JELMYTO is a prescription medicine used to treat adults with a type of cancer of the lining of the upper urinary tract including the kidney called low-grade Upper Tract Urothelial Cancer (LG-UTUC).

IMPORTANT SAFETY INFORMATION

You should not receive JELMYTO if you have a hole or tear (perforation) of your bladder or upper urinary tract.

Before receiving JELMYTO, tell your healthcare provider about all your medical conditions, including if you:

are pregnant or plan to become pregnant. JELMYTO can harm your unborn baby. You should not become pregnant during treatment with JELMYTO. Tell your healthcare provider right away if you become pregnant or think you may be pregnant during treatment with JELMYTO.
Females who are able to become pregnant: You should use effective birth control (contraception) during treatment with JELMYTO and for 6 months after the last dose.
Males being treated with JELMYTO: If you have a female partner who is able to become pregnant, you should use effective birth control (contraception) during treatment with JELMYTO and for 3 months after the last dose.
are breastfeeding or plan to breastfeed. It is not known if JELMYTO passes into your breast milk. Do not breastfeed during treatment with JELMYTO and for 1 week after the last dose.
Tell your healthcare provider if you take water pills (diuretic).
How will I receive JELMYTO?

Your healthcare provider will tell you to take a medicine called sodium bicarbonate before each JELMYTO treatment.
You will receive your JELMYTO dose from your healthcare provider 1 time a week for 6 weeks. It is important that you receive all 6 doses of JELMYTO according to your healthcare provider’s instructions. If you miss any appointments, call your healthcare provider as soon as possible to reschedule your appointment. Your healthcare provider may recommend up to an additional 11 monthly doses.
JELMYTO is given to your kidney through a tube called a catheter.
During treatment with JELMYTO, your healthcare provider may tell you to take additional medicines or change how you take your current medicines.
After receiving JELMYTO:

JELMYTO may cause your urine color to change to a violet to blue color. Avoid contact between your skin and urine for at least 6 hours.
To urinate, males and females should sit on a toilet and flush the toilet several times after you use it. After going to the bathroom, wash your hands, your inner thighs, and genital area well with soap and water.
Clothing that comes in contact with urine should be washed right away and washed separately from other clothing.
JELMYTO may cause serious side effects, including:

Swelling and narrowing of the tube that carries urine from the kidney to the bladder (ureteric obstruction). If you develop swelling and narrowing, and to protect your kidney from damage, your healthcare provider may recommend the placement of a small plastic tube (stent) in the ureter to help the kidney drain. Tell your healthcare provider right away if you develop side pain or fever during treatment with JELMYTO.
Bone marrow problems. JELMYTO can affect your bone marrow and can cause a decrease in your white blood cell, red blood cell, and platelet counts. Your healthcare provider will do blood tests prior to each treatment to check your blood cell counts during treatment with JELMYTO. Your healthcare provider may need to temporarily or permanently stop JELMYTO if you develop bone marrow problems during treatment with JELMYTO.
The most common side effects of JELMYTO include: side pain, urinary tract infection, blood in your urine, kidney problems, tiredness, nausea, stomach pain, trouble with urination, vomiting, low red blood cell count, frequent urination, itching, chills, and fever.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda/gov/medwatch or call 1‑800‑FDA‑1088. You may also report side effects to UroGen Pharma at 1-855-987-6436.

Please see JELMYTO Full Prescribing Information, including the Patient Information at www.jelmyto.com.

About Upper Tract Urothelial Cancer (UTUC)

Urothelial cancer is the ninth most common cancer globally and the eighth most lethal neoplasm in men in the U.S. Between five percent and ten percent of primary urothelial cancers originate in the ureter or renal pelvis and are collectively referred to as upper tract urothelial cancers (UTUC). In the U.S., there are approximately 6,000 – 7,000 new or recurrent low-grade UTUC patients annually. Most cases are diagnosed in patients over 70 years old, and these older patients often face comorbidities. There are limited treatment options for UTUC, with the most common being endoscopic surgery or nephroureterectomy (removal of the entire kidney and ureter). These treatments can lead to a high rate of recurrence and relapse.

Vor Biopharma Announces Exclusive License of Clinical-Stage CD33 CAR-T from National Cancer Institute

On November 11, 2020 Vor Biopharma, an oncology company pioneering engineered hematopoietic stem cells (eHSCs) for the treatment of cancer, reported an exclusive licensing agreement with the National Cancer Institute (NCI), part of the National Institutes of Health (NIH), for intellectual property related to a clinical-stage anti-CD33 chimeric antigen receptor T-cell (CAR-T) therapy candidate (Press release, Vor BioPharma, NOV 11, 2020, View Source [SID1234570672]). This CAR-T construct was devised by T-cell expert Dr. Terry Fry during his tenure at the Pediatric Oncology Branch of the NCI, where he oversaw development of this therapeutic candidate from bench to bedside; it is currently being evaluated in a multi-site Phase 1/2 clinical trial in children and young adults with relapsed or refractory acute myeloid leukemia (AML).

"As a class, CAR-T cell therapies have had a major positive impact on the lives of certain patients with hematological malignancies. But because normal cells often express the same surface proteins as cancer cells, the utility and applicability of targeted therapies has been limited, in part, by on-target toxicity," said Christopher Slapak, MD, Vor’s Chief Medical Officer. "This agreement provides Vor with access to a promising CD33 CAR-T that could potentially be administered either as bridge-to-transplant therapy for relapsed or refractory patients with AML, or following transplant with our lead developmental candidate VOR33, whereby the CAR-T may selectively target leukemia cells while sparing normal myeloid cells."

Vor’s lead program VOR33, which is currently in pre-clinical development, consists of eHSCs that are engineered to provide AML patients with a donor-derived hematopoietic stem cell transplant that lacks the cell surface protein CD33, a clinically validated target for AML. The goal of removing this target is to make these eHSCs and their progeny treatment-resistant to anti-CD33 therapies. As such, Vor believes this CAR-T could be highly complementary to VOR33.

"This licensing agreement with the NCI is an important milestone for Vor, as it brings elements of a more complete AML treatment system under the same roof," said Hilary Eaton, PhD, Vor’s Senior Director of Business Development. "The combination of our next-generation, treatment-resistant eHSCs with companion therapeutics such as this CD33 CAR-T is designed to provide a single company solution for some patients suffering from hematological malignancies, potentially transforming outcomes and shifting the standard of care."

More information about the Phase 1/2 study of this CD33 CAR-T can be found at clinicaltrials.gov.

Other terms of the agreement have not been disclosed.

About VOR33

Vor’s lead product candidate, VOR33, consists of engineered hematopoietic stem cells (eHSCs) that lack the protein CD33. Once these cells are transplanted into a cancer patient, we believe that CD33 will become a far more cancer-specific target, potentially avoiding toxicity to the normal blood and bone marrow associated with CD33-targeted therapies. Vor aims to improve the therapeutic window and effectiveness of CD33-targeted therapies, thereby potentially broadening the clinical benefit to patients suffering from acute myeloid leukemia.

Novocure Receives CE Mark for NovoTTF-100L™ System

On November 11, 2020 Novocure (NASDAQ: NVCR), a global oncology company working to extend survival in some of the most aggressive forms of cancer, reported that it has received the CE Mark for the NovoTTF-100L system from the Notified Body (TÜV) (Press release, NovoCure, NOV 11, 2020, View Source [SID1234570671]). The application of the CE mark enables Novocure to commercialize the device as a first-line treatment in combination with pemetrexed and platinum-based chemotherapy for unresectable, locally advanced or metastatic, malignant pleural mesothelioma (MPM) in the European Union and Switzerland.

MPM is a rare cancer that has been strongly linked to asbestos exposure. More than 13,000 people are diagnosed with mesothelioma in Europe annually. The U.S. FDA approved the NovoTTF-100L System (known as Optune Lua in the U.S.) as a treatment for MPM in May 2019 under the Humanitarian Device Exemption (HDE) pathway. Optune Lua was the first treatment for MPM approved by the FDA in more than 15 years. Prior to the FDA approval of NovoTTF-100L, pemetrexed plus cisplatin was the only FDA-approved therapy for patients with unresectable MPM. Now having the CE mark for its MPM therapy in Europe, Novocure will begin commercialization and to identify and pursue pathways for reimbursement in selected markets.

"We are extremely pleased to have CE marking for our NovoTTF-100L System, making our therapy commercially available for patients with MPM in Europe," said Pritesh Shah, Novocure’s Chief Commercial Officer. "Obtaining the CE mark for our NovoTTF-100L System represents another step forward on our patient-forward mission of striving to extend survival in some of the most aggressive forms of cancer. Our commercial team in EMEA is now focused on establishing pathways for reimbursement to expand access to our therapy for MPM patients."

About NovoTTF-100L

NovoTTF-100L is a noninvasive, antimitotic cancer treatment for MPM. NovoTTF-100L delivers Tumor Treating Fields, which is a cancer therapy using electric fields to disrupt cell division. Tumor Treating Fields does not stimulate or heat tissue and targets dividing cancer cells of a specific size. Tumor Treating Fields causes minimal damage to healthy cells. Mild to moderate skin irritation is the most common side effect reported. Tumor Treating Fields is approved in certain countries for the treatment of adults with glioblastoma and for mesothelioma, two of the most difficult cancer types to treat. The therapy shows promise in multiple solid tumor types – including some of the most aggressive forms of cancer.

Caution: Federal law restricts this device to sale by or on the order of a physician. Humanitarian Device. Authorized by Federal Law for use in the treatment of adult patients with unresectable, locally advanced or metastatic, malignant pleural mesothelioma concurrently with pemetrexed and platinum-based chemotherapy. The effectiveness of this device for this use has not been demonstrated.

Approved Indications

NovoTTF-100L/Optune Lua is indicated for the treatment of adult patients with unresectable, locally advanced or metastatic, malignant pleural mesothelioma (MPM) to be used concurrently with pemetrexed and platinum-based chemotherapy.

Important Safety Information

Contraindications

Do not use NovoTTF-100L/Optune Lua in patients with implantable electronic medical devices such as pacemakers or implantable automatic defibrillators, etc. Use of NovoTTF-100L/Optune Lua together with implanted electronic devices has not been tested and may lead to malfunctioning of the implanted device.

Do not use NovoTTF-100L/Optune Lua in patients known to be sensitive to conductive hydrogels. Skin contact with the gel used with NovoTTF-100L/Optune Lua may commonly cause increased redness and itching, and may rarely lead to severe allergic reactions such as shock and respiratory failure.

Warnings and Precautions

NovoTTF-100L/Optune Lua can only be prescribed by a healthcare provider that has completed the required certification training provided by Novocure.

The most common (≥10%) adverse events involving NovoTTF-100L/Optune Lua in combination with chemotherapy were anemia, constipation, nausea, asthenia, chest pain, fatigue, medical device site reaction, pruritus, and cough.

Other potential adverse effects associated with the use of NovoTTF-100L/Optune Lua include: treatment related skin toxicity, allergic reaction to the plaster or to the gel, electrode overheating leading to pain and/or local skin burns, infections at sites of electrode contact with the skin, local warmth and tingling sensation beneath the electrodes, muscle twitching, medical device site reaction and skin breakdown/skin ulcer.

If the patient has an underlying serious skin condition on the treated area, evaluate whether this may prevent or temporarily interfere with NovoTTF-100L/Optune Lua treatment.

Do not prescribe NovoTTF-100L/Optune Lua for patients that are pregnant, you think might be pregnant or are trying to get pregnant, as the safety and effectiveness of NovoTTF-100L/Optune Lua in these populations have not been established.

Please visit www.optunelua.com to see NovoTTF-100L/Optune Lua Instructions For Use (IFU) for complete information regarding the device’s indications, contraindications, warnings, and precautions. This website is primarily intended for a U.S. audience.