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Neurocrine Biosciences Reports Second Quarter 2019 Financial Results

On July 30, 2019 Neurocrine Biosciences, Inc. (NASDAQ: NBIX) reported its financial results for the quarter ended June 30, 2019 and provided an update on the launch of INGREZZA (valbenazine) and its clinical development programs (Press release, Neurocrine Biosciences, JUL 30, 2019, View Source [SID1234537867]).

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"Over the past two years, our team has done an excellent job building awareness of tardive dyskinesia within the healthcare community and providing patients access to INGREZZA. We continue to focus on educating about tardive dyskinesia and the benefits of INGREZZA in providing relief for patients suffering from this debilitating movement disorder," said Kevin Gorman, Ph.D., Chief Executive Officer of Neurocrine Biosciences. "We have also made progress across our development pipeline with the FDA acceptance of our New Drug Application for opicapone and the anticipated regulatory submission of elagolix for uterine fibroids by AbbVie. In addition, we continued to advance our congenital adrenal hyperplasia program with the recent initiation of our pediatric trial and look forward to our planned discussion with the FDA on the adult program. We are pleased with this exciting progress and remain on track to have three FDA-approved treatments in four indications by 2020."

Financial Results

Total revenues for the three and six months ended June 30, 2019, was $183.6 million and $322.0 million, respectively, compared to $96.9 million and $168.0 million for the same periods in 2018.

Total revenues were comprised of the following (unaudited):

For the second quarter of 2019, the Company reported net income of $51.3 million, or $0.54 diluted earnings per share, compared to a net loss of $5.9 million, or $0.07 net loss per share, for the same period in 2018. The increase in net income is due to increased INGREZZA net product sales and a $21.0 million unrealized gain on the Company’s Voyager Therapeutics equity investment. For the six months ended June 30, 2019, the Company reported a net loss of $50.8 million, or $0.56 net loss per share, compared to a net loss of $47.7 million, or $0.53 net loss per share, for the same period in 2018. The increase in net loss for the first half of 2019 is primarily due to $118.1 million of in-process research and development (IPR&D) in connection with the strategic collaboration with Voyager. This was partially offset by increased INGREZZA net product sales and a $22.6 million unrealized gain on the Company’s equity investment in Voyager.

Research and development (R&D) expenses increased to $61.7 million for the second quarter of 2019 from $37.0 million for the same period in 2018. The increase in R&D expenses primarily reflects funding of development activities and a $10.0 million milestone expense related to the U.S. Food and Drug Administration (FDA) acceptance of the opicapone New Drug Application (NDA). For the six months ended June 30, 2019, R&D expenses increased to $99.3 million, compared to $85.9 million for the same period in 2018. The increase in R&D expenses for the first half of 2019 primarily reflects funding of development activities in connection with the Company’s collaboration with Voyager.

In connection with the Voyager collaboration, the Company recognized IPR&D of $118.1 million during the first six months of 2019, including a $5.0 million payment during the second quarter to obtain rights outside the United States to the Friedreich’s ataxia program. In addition, the Company made an equity investment in Voyager which is required to be marked to market each quarter resulting in an unrealized gain of $21.0 million and $22.6 million for the second quarter and first six months, respectively, and is reflected in Other Income.

Sales, general and administrative (SG&A) expenses increased to $80.8 million for the second quarter of 2019, from $60.9 million for the same period in 2018. For the six months ended June 30, 2019, SG&A expenses increased to $168.4 million, compared to $119.6 million for the same period in 2018. The increase in SG&A expenses for both periods is primarily due to the sales force expansion completed in the third quarter of 2018, the national launch of a patient-focused disease state awareness campaign, Talk About TD, and an increase in the Branded Pharmaceutical Drug fee expense.

As of June 30, 2019, the Company’s cash and available-for-sale investments was $766.5 million.

Updated 2019 SG&A and R&D Expense Guidance

SG&A, IPR&D, and R&D expenses for 2019 are expected to be $658 million to $688 million. Ongoing SG&A and R&D expenses for 2019, excluding IPR&D, are now expected to approximate $540 to $570 million, which compares to the prior SG&A and R&D expense guidance of $550 million to $600 million.

Pipeline Highlights

Opicapone Update

In February 2017, the Company entered 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. Opicapone is a once-daily, oral, selective catechol-O-methyltransferase inhibitor, being developed as an adjunctive therapy to levodopa/carbidopa in patients with Parkinson’s disease experiencing OFF episodes. The Company met with the FDA in January 2018 and based upon the BIPARK-1 and BIPARK-2 pivotal Phase III studies conducted by BIAL, the FDA did not require additional Phase III trials to form an NDA submission. The NDA for opicapone was submitted to the FDA during the second quarter of 2019. The NDA was accepted by the FDA with a Prescription Drug User Fee Act (PDUFA) target action date of April 26, 2020. As a result of the FDA’s acceptance of the NDA submission, the Company will pay a $10.0 million milestone payment to BIAL.

Elagolix Update

On July 24, 2018, AbbVie, in collaboration with Neurocrine Biosciences, announced FDA approval and in October 2018 Health Canada approval for ORILISSA (elagolix) for the management of endometriosis with associated moderate to severe pain. The FDA granted priority review to ORILISSA. The FDA grants priority review designation to medicines that, if approved, would provide a significant improvement in the safety or effectiveness of treatment of a serious condition. AbbVie began commercialization of ORILISSA in the United States in August 2018.

AbbVie provided positive top-line efficacy data from two Phase III studies in women with uterine fibroids in the first quarter of 2018 and from the associated six-month safety extension study during the third quarter of 2018. The ELARIS UF-I and UF-II studies of elagolix met all primary and ranked secondary endpoints at month six. Results from these studies will form the basis for an anticipated NDA submission to the FDA for the approval of elagolix in the treatment of uterine fibroids in 2019.

AbbVie intends to initiate a study of elagolix in women with polycystic ovary syndrome (PCOS).

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

The Company began an adaptive, Phase II proof-of-concept study examining the pharmacokinetics, pharmacodynamics, and safety of NBI-74788 in adults with classic 21-hydroxylase deficiency congenital adrenal hyperplasia (CAH) in November 2017. This study evaluates the safety and tolerability of NBI-74788 in patients with CAH together with the relationship between exposure and specific steroid hormone levels in these patients. In March 2019, positive interim results from this ongoing study demonstrated a clinically meaningful reduction in key biomarkers associated with the management of CAH. NBI-74788 was shown to be well tolerated with no serious adverse events reported to date. The Company plans to discuss the NBI-74788 program in adult CAH patients with the FDA during the third quarter of 2019.

In July 2019, the Company initiated an adaptive, Phase II proof-of-concept study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of NBI-74788 in pediatric patients with classic CAH.

Voyager Collaboration and VY-AADC Program

During the first quarter of 2019, Neurocrine Biosciences formed a strategic collaboration with Voyager Therapeutics focused on the development and commercialization of Voyager’s gene therapy programs, VY-AADC for Parkinson’s disease and VY-FXN01 for Friedreich’s ataxia, as well as rights to two programs to be determined. This collaboration combines Neurocrine Biosciences’ expertise in neuroscience, drug development and commercialization with Voyager’s innovative gene therapy programs targeting severe neurological diseases.

Based on the results from the VY-AADC Phase I programs in Parkinson’s disease, RESTORE-1, a Phase II, randomized, placebo-surgery controlled, double-blinded, multi-center, clinical trial was initiated to evaluate the safety and efficacy of VY-AADC in patients who have been diagnosed with Parkinson’s disease for at least four years, are not responding adequately to oral medications, and have at least three hours of OFF time during the day as measured by a validated self-reported patient diary.

Conference Call and Webcast Today at 4:30 PM Eastern Time

Neurocrine Biosciences will hold a live conference call and webcast today at 4:30 p.m. Eastern Time (1:30 p.m. Pacific Time). Participants can access the live conference call by dialing 866-342-8591 (US) or 203-518-9713 (International) using the conference ID: NBIX. The webcast can also be accessed on Neurocrine Biosciences’ website under Investors at www.neurocrine.com. A replay of the webcast will be available on the website approximately one hour after the conclusion of the event and will be archived for approximately one month.

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 face, torso, and/or other body parts.

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 from 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

Contraindications
INGREZZA is contraindicated in patients with a history of hypersensitivity to valbenazine or any components of INGREZZA. Rash, urticaria, and reactions consistent with angioedema (e.g., swelling of the face, lips, and mouth) have been reported.

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.

Parkinsonism
INGREZZA may cause Parkinsonism in patients with tardive dyskinesia. Parkinsonism has also been observed with other VMAT2 inhibitors. Reduce the dose or discontinue INGREZZA treatment in patients who develop clinically significant parkinson-like signs or symptoms.

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.

City of Hope Named 11th Best Cancer Hospital in the Nation by U.S. News & World Report

On July 30, 2019 City of Hope reported that it has been named the 11th best cancer hospital in the nation by U.S. News & World Report’s 2019-20 Best Hospitals: Specialty Ranking (Press release, City of Hope, JUL 30, 2019, View Source [SID1234537861]). This makes City of Hope the highest ranked cancer hospital in the West and marks the 13th consecutive year it has been distinguished as one of the nation’s elite cancer hospitals.

"City of Hope is extremely honored to be recognized by U.S. News & World Report for our long-standing commitment to providing the best treatment and care to our patients," said Robert Stone, City of Hope president and CEO. "It’s a reflection of our exceptional, compassionate care and our dedication to turning powerful science into lifesaving new treatments for our patients."

"A steadfast collaboration between City of Hope scientists and physicians, our strong translational research model and our top-notch biomedical research and manufacturing facilities enable us to deliver excellent cancer care," Stone added.

This year, U.S. News & World Report added data from Hospital Consumer Assessment of Healthcare Providers and Systems, which tracks exemplary patient experience. City of Hope was fourth in the country out of the 885 hospitals surveyed.

City of Hope was also ranked "high-performing" in lung cancer surgery and colon cancer surgery.

"Although City of Hope is not driven by the accolades we receive, we know that patients and their families often turn to rankings and ratings to decide where to get their care," said Michael Caligiuri, M.D., president of City of Hope National Medical Center and the Deana and Steve Campbell Physician-in-Chief Distinguished Chair. "We are gratified to be recognized as a leading-edge cancer center where a patient’s hope can turn into reality through our innovative treatments and exemplary care."

The cancer hospital ranking is compiled based on data from 885 eligible cancer hospitals nationwide; U.S. News & World Report’s rankings cover 16 specialty areas in total. Only 12.5% of the more than 4,500 hospitals analyzed in all the specialty areas received the "Best" status. A cancer hospital’s overall score reflects performance in such areas as advanced technologies, designation as a National Cancer Institute comprehensive cancer center and survival score data.

City of Hope is one of 50 comprehensive cancer centers in the nation, the highest designation possible from the National Cancer Institute and a metric used by U.S. News & World Report. To earn this designation, a center must undergo a rigorous peer-review process, maintain a high number of cancer research projects and be heavily involved in community outreach, as well as meet other academic and public service requirements. City of Hope earned its comprehensive cancer center designation in 1998 and has maintained it since.

City of Hope is also a founding member of the National Comprehensive Cancer Network, making it a leader in advancing research and treatment protocols throughout the nation. City of Hope is currently conducting more than 500 clinical trials, enrolling more than 6,200 patients.

The American College of Surgeons Commission on Cancer recently awarded City of Hope a "Three-Year with Commendation" designation, its highest level of accreditation recognizing exceptional cancer care.

City of Hope was a pioneer in bone marrow and stem cell transplants, and continues to have one of the largest, most successful such programs of its kind in the U.S. The Center for International Blood and Marrow Transplant Research Center has ranked City of Hope as an "overperforming" transplant center, making it the only U.S. transplant center with this recognition for 14 consecutive years. Interlink COE Networks and Programs also recently designated City of Hope’s bone marrow and stem cell transplant program as No. 1 in the nation.

A leader in the CAR T field for being the first to offer CAR T trials for specific targets on glioblastoma and acute myeloid leukemia cells, City of Hope has treated more than 350 patients in CAR T trials and currently has 20 ongoing CAR T and T cell trials.

Complete lists of U.S. News’ "Best Hospitals" by specialties and their ranking methodology are available online beginning July 30 at 12:01 a.m. EDT.

Athenex, Inc. to Report Second Quarter 2019 Earnings Results on August 7, 2019

On July 29, 2019 Athenex, Inc. (Nasdaq: ATNX), a global biopharmaceutical company dedicated to the discovery, development and commercialization of novel therapies for the treatment of cancer, reported that it will release second quarter and six-months ended June 30, 2019 financial results on Wednesday, August 7, 2019, before the market opens, and host a conference call and live audio webcast 8:00am Eastern Time to discuss the financial results and provide a business update (Press release, Athenex, JUL 29, 2019, View Source [SID1234573889]).

To participate in the call, dial 877-407-0784 (domestic) or 201-689-8560 (international) fifteen minutes before the conference call begins and reference the conference passcode 13691069. The live conference call and replay can also be accessed via audio webcast at the Investor Relations section of the Company’s website, located at View Source

NEC and OncoImmunity AS combine forces to use AI to empower personalized cancer immunotherapy

On July 29, 2019 OncoImmunity AS reported that it has been acquired by NEC Corporation, the renowned Japanese IT and network company, that recently launched its Artificial Intelligence (AI) driven drug discovery business (Press release, NEC OncoImmunity, JUL 29, 2019, View Source [SID1234555210]). OncoImmunity AS will now become a subsidiary of NEC and operate under the name of NEC OncoImmunity AS.

OncoImmunity AS, founded in 2014, develops software that identifies neoantigen targets for truly personalized cancer vaccines, cell therapies and optimal patient selection for cancer immunotherapy clinical trials. Prior to the acquisition, OncoImmunity AS was funded by a consortium of Norwegian life science and tech investors led by Radforsk, who played a proactive role in developing the company under the guidance of the company’s Chairman Anders Tuv, who was instrumental for and also managed the sales process on behalf of all shareholders. Under the acquisition, NEC will maintain its focus on drug discovery, while NEC OncoImmunity AS continues its neoantigen prediction services.

Both NEC and OncoImmunity AS share a common vision that individualized cancer treatments can be significantly improved by harnessing the power of AI. AI holds tremendous promise in enabling each and every patient to receive the optimal personalized therapy. OncoImmunity AS will benefit from NEC’s proprietary neoantigen prediction modules and its wide array of IT expertise such as data management and security. The OncoImmunity AS platform has unique advantages in accurately predicting antigen presentation to the tumor cell surface, a key determinant for identifying clinically relevant immunogenic neoantigens. By integrating complementary knowledge and resources, this acquisition makes NEC OncoImmunity AS a forerunner in the field of neoantigen prediction. The acquisition signals positive momentum in the field of personalized cancer immunotherapy, where the prediction of immunogenic neoantigens remains a central challenge for clinical success, with two of the predominant players in the neoantigen prediction field now joining forces.

"This acquisition creates a scientific synergy that will enable OncoImmunity AS’s continued dedication to the development of bioinformatics solutions for truly personalized cancer immunotherapy. The combined technology will be of tremendous benefit to our current and future clinical partners," said Dr. Trevor Clancy, Chief Scientific Officer and Co-founder of OncoImmunity AS.

"The OncoImmunity AS team are delighted to be joining the NEC Group and firmly believe that the union will realize great synergies, marrying the mutual strengths of OncoImmunity AS’s and NEC’s neoantigen prediction pipelines with NEC’s expertise in AI, data management and security. These synergies will help strengthen NEC’s individualized immunotherapy programs, whilst simultaneously positioning NEC OncoImmunity AS as the leading neoantigen prediction service provider in the field," said Dr. Richard Stratford, Chief Executive Officer and Co-founder of OncoImmunity AS.

"NEC strongly believes that healthcare based on genomics paves the way for individualized medicine. OncoImmunity AS is a recognized player in the neoantigen prediction field, and their compelling systems are expected to form valuable synergies with NEC. We are confident that this acceleration of development will provide promising new avenues that lead to better cancer treatments," said Osamu Fujikawa, Senior Vice President, NEC Corporation.

CRISPR Therapeutics Provides Business Update and Reports Second Quarter 2019 Financial Results

On July 29, 2019 CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, reported financial results for the second quarter ended June 30, 2019 (Press release, CRISPR Therapeutics, JUL 29, 2019, View Source [SID1234537901]).

"We have made significant progress across several of our development programs, including the ongoing enrollment of CTX001 studies in both beta thalassemia and sickle cell disease, initiation of a clinical trial for our allogeneic CAR-T therapy for CD19+ malignancies, and the expansion of our collaboration with Vertex into Duchenne Muscular Dystrophy and Myotonic Dystrophy Type 1," said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. "We look forward to continued execution against our clinical and regulatory milestones and expect to have data from several of our programs in 3-4 indications over the next 18 months."

Recent Highlights and Outlook

Beta thalassemia and sickle cell disease

CRISPR Therapeutics provided an update from the ongoing Phase 1/2 study of CTX001, an investigational, autologous, CRISPR/Cas9 gene-edited hematopoietic stem cell therapy being evaluated for patients suffering from transfusion-dependent beta thalassemia (TDT) and severe sickle cell disease (SCD).

Enrollment in both Phase 1/2 studies of CTX001 in patients with TDT and in patients with severe SCD is ongoing. Based on the progression of the programs, CRISPR Therapeutics expects to obtain preliminary safety and efficacy data in late 2019.

The first patient treated with CTX001 in a Phase 1/2 clinical study of patients with TDT remains transfusion independent, greater than four months following engraftment.

The first patient has been treated in a Phase 1/2 clinical study of CTX001 in severe SCD in the U.S.

Immuno-Oncology

CRISPR Therapeutics announced today that it is currently enrolling patients in its Phase 1/2 trial to assess the safety and efficacy of CTX110, its wholly-owned allogeneic CAR-T cell therapy targeting CD19+ malignancies. The Company’s proprietary CRISPR-based allogeneic CAR-Ts have the potential to create the next-generation of cell therapies that may have a superior product profile compared to current autologous therapies and allow accessibility to broader patient populations.

CRISPR Therapeutics continues to advance additional allogeneic CAR-T candidates toward clinical development including CTX120, targeting B-cell maturation antigen (BCMA) for the treatment of multiple myeloma and CTX130, targeting CD70 for the treatment of solid tumors and hematologic malignancies. The Company continues to scale its capabilities to rapidly advance these programs into and through the clinic.

Other Programs

In June, CRISPR Therapeutics and Vertex expanded their collaboration and entered into an exclusive licensing agreement to discover and develop gene editing therapies for the treatment of Duchenne Muscular Dystrophy (DMD) and Myotonic Dystrophy Type 1 (DM1). CRISPR Therapeutics continues to make advancements with programs utilizing an in vivo approach, which remains a key area of focus.

Earlier this month, CRISPR Therapeutics announced that it will present an oral presentation at the 55th Annual Meeting of the European Association for the Study of Diabetes (EASD), taking place September 16 to 20, 2019, in Barcelona, Spain (abstract #9). The presentation will demonstrate the progress made by CRISPR Therapeutics and its partner, ViaCyte, in generating an allogeneic immune-evasive clonal pluripotent stem cell line capable of differentiating into pancreatic precursor cells as a potential therapy for type 1 diabetes.

Other Corporate Matters

As the Company previously disclosed in June 2019, CRISPR Therapeutics received notification that the United States Patent and Trademark Office (USPTO) has initiated an interference proceeding at the Patent Trial and Appeal Board between certain pending U.S. patent applications co-owned by the University of California, the University of Vienna and Dr. Emmanuelle Charpentier (collectively, the "CVC Group") and certain patents and a patent application currently owned by the Broad Institute, Harvard University and the Massachusetts Institute of Technology, all of which are related to the single guide format of CRISPR/Cas9 genome editing technology in eukaryotic cells. As of July 2019, the USPTO has granted ten patents to the CVC group. None of these issued patents are involved in the interference.
Second Quarter 2019 Financial Results

Cash Position: Cash as of June 30, 2019, was $427.9 million, compared to $437.5 million as of March 31, 2019, a decrease of $9.6 million as cash operating expenses were offset by $29.7 million net proceeds from financing activities. Considering the $175 million cash received from Vertex in July under the expanded collaboration agreement for DMD and DM1, proforma cash for the company exceeds $600 million.

Revenues: Total collaboration revenues were $0.3 million for the second quarter of 2019 compared to $1.1 million for second quarter of 2018.

R&D Expenses: R&D expenses were $39.5 million for the second quarter of 2019 compared to $25.6 million for the second quarter of 2018. The increase was driven by headcount and services expense supporting the advancement of the hemoglobinopathies program, the broadening of the wholly-owned immuno-oncology portfolio, as well as increased investment in the Company’s CRISPR/Cas9 platform research.

G&A Expenses: General and administrative expenses were $15.8 million for the second quarter of 2019 compared to $12.7 million for the second quarter of 2018. The increase was driven by headcount-related expense and external professional and consulting service expense.

Net Loss: Net loss was $53.7 million for the second quarter of 2019 compared to a loss of $38.4 million for the second quarter of 2018, driven predominantly by increased R&D expense in the quarter.
About CTX001
CTX001 is an investigational ex vivo CRISPR gene-edited therapy that is being evaluated for patients suffering from TDT or severe SCD in which a patient’s hematopoietic stem cells are engineered to produce high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is a form of the oxygen carrying hemoglobin that is naturally present at birth and is then replaced by the adult form of hemoglobin. The elevation of HbF by CTX001 has the potential to alleviate transfusion requirements for TDT patients and painful and debilitating sickle crises for SCD patients.

CTX001 is being developed under a co-development and co-commercialization agreement between CRISPR Therapeutics and Vertex.

About the Gene-Editing Process in These Trials
Patients who enroll in these studies will have hematopoietic stem cells collected from peripheral blood. The patient’s cells will be edited using the CRISPR/Cas9 technology. The edited cells, CTX001, will then be infused back into the patient as part of a stem cell transplant, a process which involves, among other things, a patient being treated with high dose chemotherapy and/or radiation therapy. Patients undergoing stem cell transplants may encounter side effects (ranging from mild to severe) that are unrelated to the administration of CTX001. Patients will initially be monitored to determine when the edited cells begin to produce mature blood cells, a process known as engraftment. After engraftment, patients will continue to be monitored to track the impact of CTX001 on multiple measures of disease.

About the Phase 1/2 Study in Beta Thalassemia
The Phase 1/2 open-label trial is designed to assess the safety and efficacy of a single dose of CTX001 in patients ages 18 to 35 with TDT, non-beta zero/beta zero subtypes. The first two patients in the trial will be treated sequentially and, pending data from these initial two patients, the trial will open for broader concurrent enrollment. The study is currently being conducted at multiple clinical trial sites in North America and Europe.

About the Phase 1/2 Study in Sickle Cell Disease
The Phase 1/2 open-label trial is designed to assess the safety and efficacy of a single dose of CTX001 in patients ages 18 to 35 with severe SCD. Similar to the trial in beta thalassemia, the first two patients in the trial will be treated sequentially prior to broader concurrent enrollment. The study is currently being conducted at multiple clinical trial sites in North America and Europe.

About Beta Thalassemia and Sickle Cell Disease
Beta thalassemia is an inherited blood disorder caused by mutations in the beta-globin gene that results in low or no beta-globin production, which is an important building block of hemoglobin. Patients with TDT, a severe form of beta thalassemia, suffer from anemia and are dependent on blood transfusions, which can lead to iron accumulation and complications that damage organs and shorten life span.

SCD is an inherited blood disorder caused by mutations in the beta-globin gene that lead to an abnormal hemoglobin, called sickle hemoglobin (HbS). Because of this abnormal hemoglobin, red blood cells can become rigid and block small blood vessels. Patients with severe SCD can suffer from acute pain, acute chest syndrome, organ damage, as well as other potential complications, including shortened life span.

About the CRISPR-Vertex Collaboration
CRISPR Therapeutics and Vertex entered into a strategic research collaboration in 2015 focused on the use of CRISPR/Cas9 to discover and develop potential new treatments aimed at the underlying genetic causes of human disease. CTX001 represents the first treatment to emerge from the joint research program. CRISPR Therapeutics and Vertex will jointly develop and commercialize CTX001 and equally share all research and development costs and profits worldwide.