BluePrint Medicines

Part
01
of five
Part
01

BluePrint Medicines - Leaders

The ten key executives listen on BluePrint's website include Andy Boral, Kate Haviland, Jeff Albers, Tracy McCain, Christopher Murray, and Marion Dorsch among others. Our research team has retrieved information about where they worked before joining BluePrint Medicines and the titles they held. The data is presented as follows.

JEFF ALBERS

Jeff Albers was the President of Algeta ASA from January 2012 to April 2014. He has worked at BluePrint Medicines for a period of 4 years and 11 months, from July 2014 to present.

ANDY BORAL

Andy Boral worked at Novartis Institutes for Biomedical Research as the Executive Director, Oncology Translational Medicine from the year 2010 to 2015. Andy Boral was employed for 5 years at the company and was located at Cambridge, Massachusetts.

Andy was then employed at BluePrint Medicines in January 2016 and worked there to date. He has worked at BluePrint Medicines for a period of 3 years and 5 months as a Chief Medical Officer. He is based at Cambridge, Massachusetts. He is also the Senior Vice President, Clinical Development from 2015 to present.

MARION DORSCH

Marion Dorsch worked at Agios as the Vice President of Biology from April 2012 to November 2016, a period of 4 years and 8 months. She has worked at BluePrint Medicines for a period of 2 years and 7 months, from November 2016 to present, as the Chief Scientific Officer. She is based at Cambridge, Massachusetts.

KATE HAVILAND

Kate Haviland worked at Idera Pharmaceuticals as the Vice President Rare Diseases and Oncology Program Leadership for a period of 1 year and 9 months, from April 2014 to December 2015. He was based at Cambridge, Massachusetts.

Kate Haviland has worked for BluePrint Medicines for a period of 3 years and 5 months. She has the Chief Operating Officer from February 2019. From January 2016 to February 2019, she was the Chief Business Officer. She is located at Cambridge, Massachusetts.

DEBBIE DURSO-BUMPUS

Debbie Durso-Bumpus worked at Cubist Pharmaceuticals for a period of 6 years and 1 month. From February 2015 to April 2015, she was the Interim Head of Human Resources and was located in Lexington, Massachusetts. From June 2013 to February 2015, she was the Global Senior Director, Talent Management & Organizational Development.

Debbie Durso-Bumpus has worked for BluePrint Medicines for a period of 4 years and 2 months now. For 2 years and 4 months now, she has been the Senior Vice President, Human Resources, from February 2017 to present. Debbie Durso-Bumpus was the Vice President, Human Resources for a period of 1 year and 11 months, from April 2015 to February 2017. She is based at Cambridge, Massachusetts.

MICHAEL LANDSITTEL

Michael Landsittel worked for Algeta ASA as the Senior Director, Finance, from October 2012 to July 2014. That was a period of 1 year and 10 months. He was based at the Greater Boston area. He has worked at BluePrint Medicines for a period of 4 years and 9 months. He was employed as a Chief Financial Officer, from February 2019 to present, a period of 4 months. He was the Vice President, Finance from September 2014 to February 2019. He is based at Cambridge, Massachusetts.

TRACEY MCCAIN

Tracey McCain worked for Genzyme Corporation as the Senior Vice President and the Head of Legal for 19 years and 5 months. That is, from May 1997 to September 2016. He then came to BluePrint Medicines in September 2016 where he works to date as the Executive Vice President and Chief Legal Officer. He is based at Cambridge, Massachusetts.

CHRISTOPH LENGAUER

Christoph Lengauer worked at Johns Hopkins University for 25 years as an Associate Professor. He was employed there in the year 2005 and works there until now. The University is located at Baltimore, Maryland, area. He was also a Principal Investigator from the year 1994 to the year 2005. This makes 11 years. He was based in Baltimore, Maryland area. Christoph Lengauer was employed at BluePrint Medicines in the year 2016 and he still works there. He is the Executive Vice President of the company. He has been employed by BluePrint Medicines for a period of 2 years and 7 months.

CHRISTOPHER MURRAY

Christopher Murray worked at ARIAD Pharmaceuticals Inc for a period of 12 years and 7 months before moving to BluePrint Medicines. He has worked at BluePrint Medicines for a period of 1 year and 8 months now as the Senior Vice President, Technical Operations. He was hired at the company in October 2017.

CHRISTINA ROSSI

Christina Rossi worked at Sanofi Genzyme for 6 years before she came to BluePrint Medicines. She came to BluePrint Medicines in October 2018 and has worked there for 8 months now as the chief commercial officer. The company is located at Cambridge, Massachusetts.
Part
02
of five
Part
02

BluePrint Medicines - History

BluePrint Medicines is headquartered Cambridge, Massachusetts, and has 217 employees. It has received over $240 million in funding since its establishment. BluePrint Medicines is focused on creating precision therapies and conducting clinical trials for cancer and rare diseases patients.

BLUEPRINT MEDICINES FUNDING HISTORY

Since its establishment, BluePrint Medicines has raised over $240 million in funding. They received $40 million from Third Rock Ventures, one of its co-founders on April 11, 2011, to help "translate new molecular data into personalized cancer treatments." Eight Roads Ventures invested an undisclosed amount on Jan 1, 2012.

In May 2013, BluePrint Medicines received $5 million in funding from Silicon Valley Bank, and $25 million from Next Invest and other investors including Fidelity Biosciences and Third Rock Ventures, among others on Jan 7, 2014. In November 2014, they borrowed another $5 million from Silicon Valley Bank. BluePrint received $50 million in Series C funding from Partner Fund Management along with Wellington Management Company, Sabby Capital, and other investors, which they planned to use in initiating clinical trials. Morgan Stanley, Goldman Sachs & Co, and Cowen & Company invested $125 million which the company planned to use in advancing clinical trials of cancers, to increase capital as well as for other corporate purposes.

FINANCIAL STATUS

BluePrint Medicines became a public company on April 30, 2015.

NUMBER OF EMPLOYEES

Blueprint Medicines had 217 full-time employees as of February 15, 2019. 76 of their employees have either M.D. or Ph.D. degrees.

BLUEPRINT MEDICINES HEADQUARTERS

Blueprint Medicines has its headquarters at 45 Sidney Street, Cambridge, Massachusetts 02139.

BLUEPRINT MEDICINES SPECIALTIES

BluePrint Medicines focuses on advancing precision therapies that will improve human health. They have multiple research programs and are also conducting clinical trials for patients who have rare diseases and cancers. The company is focused on identifying people who best respond to their therapies, increasing the efficiency of clinical trials, and delivering transformational therapies. Their research areas include genomically-defined cancers, rare diseases, and cancer immunotherapy.

BluePrint Medicines uses a scientific platform to quickly design kinase medicines which target drivers of disease and increase the chances of clinical success. They develop appropriate investigational therapies to mark the causes behind "genomically defined diseases." BluePrint Medicines conducts clinical trials in gastrointestinal stromal tumors, systemic mastocytosis, RET-altered cancers, and hepatocellular carcinoma. (Source 11)
They create special therapies by combining their knowledge in the genetic blueprint of cancer, kinase biology, and rare diseases. Some diseases that they target include medullary thyroid cancer, Fibrodysplasia ossificans progressiva, and RET-altered non-small cell lung cancer, among others.

Part
03
of five
Part
03

BluePrint Medicines - Financials

At the end of 2018, BluePrint reported $494 million in cash, cash equivalents, and investments – a drop from the $673.4 million it reported at the end of 2017. Unfortunately, based on public data, some sections of the requirements were unanswered; however, thorough research strategies explaining the search attempts deployed to try to uncover the findings are elaborated below. Information regarding most of the specifications on the subject was retrieved with the exception of information on customer perceptions to Blueprint’s products, marketing spend for 2018, and the advertising agencies used. Three distinct methodologies outlining the search avenues consulted to try to find the missing information are included below.

RESEARCH STRATEGY

We commenced our search by exploring the company website, which had most of the details in its annual reports and the website contents regarding its products. Unluckily, Blueprint’s website did not feature information regarding its marketing budget and the adverting agencies it uses. Equally, the financial statements provided the company's total expenditure without any breakdowns. Besides, the reports did not include any data on the advertising agencies BluePrint uses or customer perceptions of the various products it provides.

Next, we examined multiple clinical studies published by BluePrint Medicines about its products to check whether they featured any details on customer perceptions, marketing spend, and the agencies contracted. Fortunately, we found a report titled “2020 Blueprint global business strategy” highlighting the company’s strategic plan to 2020. The report included data on Blueprint’s future positioning in the global market but did not feature data specific to the expected spend on marketing to attain the positioning it targets. Additionally, the report lacked historical data on the company's marketing budget, agencies contracted for advertising, and customer perceptions towards its products.

Third, we attempted to search for the marketing spend for each of the products the company sells to try to triangulate the overall marketing figure. Unfortunately, all the documents uncovered did not include any details about the company’s spend on marketing and promotion activities of its products. We further searched for information and mentions across news and marketing reports about the agencies involved in the marketing function of Blueprint, but only got partnerships, like CStone in China, which represent the company’s proxies in different countries. Again, there was no mention of any advertising agencies contracted. In this regard, we concluded that information regarding the company’s marketing spending and advertising agencies are kept private and confidential to the company and that surveys on consumer perceptions regarding the company’s products are yet to be conducted.

BLUEPRINT'S FINANCIAL PERFORMANCE

At the end of 2018, BluePrint reported $494 million in cash, cash equivalents, and investments indicating a drop from 2017 figure of $673.4 million. According to the company, the decline was attributed to increases in operating activities spend. The company reported a revenue of $44.5 million for the year ended December 2018, a significant improvement from the $21.4 million reported in December 2017.

Department wise, research and development expenses increased to $243.6 million in 2018, compared to $144.7 million in 2017. The increase in R&D stemmed from a rise in clinical and manufacturing expenses, which were facilitated by Blueprint Medicines' “lead development candidates and increased personnel-related expenses.” General and administrative expenses stood at $47.9 million in 2018 compared to $28.0 million in 2017. Overall, BluePrint closed 2018 with a net loss of $236.6 million and a net loss of $5.39 per share compared to 2017’s net loss of $148.1 million or $3.92 per share.

BLUEPRINT PRODUCTS

  • BLU-667
BLU-667 is an oral precision therapy for oncogenic RET alterations and resistance mutants. It is currently part of Blueprint's global Phase 1 ARROW clinical trial to be used for treating patients with NSCLC (RET-altered non-small cell lung cancer), medullary thyroid cancer (MTC)” and other complex tumors. In China, CStone has already initiated Phase 1 clinical trials for RET inhibitor BLU-667 following regulatory approval.

  • BLU-554
BLU-554 is a potent, irreversible inhibitor of fibroblast growth factor receptor 4 (FGFR4) administered orally. The drug selectively inhibits FGFR4 and spares other kinases. The FDA granted the drug orphan drug designation because of its contribution to effective and safe treatment, diagnosis, and prevention of rare diseases and disorders.

  • BLU-782
BLU-782 is also an orally administered drug that targets the underlying genetic driver of fibrodysplasia ossificans progressiva (FOP), which is a rare, severely disabling, and life-shortening genetic disease. The product is crucial for people suffering from FOP and is anticipated to revolutionize the treatment of FOP.

  • AVAPRITINIB
AVAPRITINIB is also a potent and selective inhibitor of activated KIT and PDGFRA mutant kinases. It works by binding and inhibiting the active conformation of these proteins.

BLUEPRINT MEDICINES MARKETING SPEND AND ADVERTISING AGENCY

Based on the company’s strategic report for 2020, it intends to have two marketed products and at least four additional marketing applications pending in the United States and Europe. Also, under this strategy, Blueprint seeks to have 6 therapeutic candidates under development globally and 8 research programs by 2020.
Part
04
of five
Part
04

BluePrint Medicines - BLU-667

The molecule of BLU-667 is an RET (Rearranged during Transfection) inhibiting molecule that has shown promise in the ongoing clinical trials for reducing the size of hard tumors in cancers where RET is altered. The only competitor in the market for BLU-667 is LOXO Oncology's molecule LOXO-292.

METHODOLOGY

In order to obtain the required information on the molecule of BLU-667, we began our search by looking through the official website of BluePrint Medicines, medical databases for clinical trials such as the United States Library of Medicine, academic journals from the American Association for Cancer Research, industry-specific websites such as MD Anderson Center and The ASCO Post. An extensive search through these channels provided us with the description of the molecule and its activity, its performance in one clinical trial, and one competitor in the market for BLU-667.
To find the second competitor to BLU-667, we consulted the National Cancer Institute and the American Association for Cancer Research to look for news about presentations concerning RET inhibitor treatments, technology, and companies that provide these. We found that BluePrint Medicines, the developer of BLU-667, and LOXO Oncology, the developer of LOXO-292, are the only two companies that have been reported to be developing RET inhibitors. We then attempted to gauge the size of the market to hopefully be able to obtain a figure for the number of companies that are developing RET inhibiting therapies. However, we found no account of credible information that provides data on this particular space.
In search for how BLU-667 has performed in a second clinical trial, we searched through numerous oncological resources such as Cancer Discovery, AACR, and the University of Texas to glean what new advancements, studies, and treatments are currently available. However, apart from the Phase 1 ARROW trials, no other clinical trial for BLU-667 is available. We then expanded our search to look for information from past RET inhibitor studies but found that BLU-667 is the first RET inhibiting molecule to go to clinical trials. It can be assumed that because BLU-667 was so recently discovered and applied to RET altered cancer treatments, there are no other clinical trials that are currently in place.

BLU-667 MOLECULE

BLU-667 is a single molecule that was discovered and developed by BluePrint Medicines to essentially target RET which is a kinase driver of disease for patients with medullary thyroid, non-small cell lung cancer, and other types of tumor. BLU-667 is currently one of the first molecules the company has discovered and developed from a starting point in its library. The company’s kinase inhibitor library has more than 10,000 molecules that act against a wide range of kinases.
BLU-667 is developed as an oral precision therapy for highly selective targeting of resistance mutants and oncogenic RET alterations. Kinase activation drives the formation and growth of tumors due to the oncogenic alterations in RET. Mutations and fusions are two key mechanisms of oncogenic activation in RET. It was found that against some of the most common RET fusions, resistance mutations, and activating mutations, BLU-667 reached sub-nanomolar potency in preclinical studies. BLU-667 has the ability to prevent and overcome the emergence of clinical resistance by suppressing RET’s primary and secondary mutants. BLU-667 was found to achieve better selectivity for RET in comparison to other approved multi-kinase inhibitors such as vandetanib and cabozantinib.
According to a study published in the American Association for Cancer Research Journals on Small-Molecule Kinase Inhibitors, BLU-667 is predicted to inhibit RET at doses that are clinically achievable. The study also suggests that this investigational agent shows promise to offer patients, who have RET-driven malignancies, a chance for more effective and durable treatment.

BLU-667 in Clinical Trials — Phase 1 Arrow Study

Phase 1 of the ARROW Study (NCT03037385) is a first-in-human and open-label study designed to determine the tolerability, safety, preliminary antineoplastic activity, pharmacodynamics (PD), and pharmacokinetics (PK) of BLU-667 administered orally in those patients with RET-altered non-small cell lung cancer (NSCLC), medullary thyroid cancer, and other types of RET-altered solid tumors. The study began on March 17, 2017, and the primary part of the study is expected to be completed in March 2021. The entire study is estimated to be performed with 360 participants and will be completed in March 2023.
According to the lead author of the study, assistant professor, Vivek Subbiah, MD, of Investigational Cancer Therapeutics in MD Anderson Cancer Center, Houston, durable responses and tumor reductions were noticed in most patients, significantly more for those whose cancers advanced with multi-kinase inhibitors and chemotherapy. The study revealed a 45% overall response rate for RET-driven cancers, a 40% response rate for RET-mutant medullary thyroid cancer (MTC), and a 50% response rate for RET-fusion NSCLC. At the preliminary stage, 53 patients with various RET-driven cancer types enrolled and currently, 41 patients are still on study. Majority of the adverse effects were found to be grade 1 which include raised alanine aminotransferase, fatigue, diarrhea, and constipation. Of the 53 patients, only three patients experienced grade 3 adverse effects that include tumor lysis syndrome, hypertension, and elevated alanine aminotransferase. Overall, 83% of the patients were found with reduced tumors when treated with doses of 60mg/day of BLU-667.
The data obtained from the study indicates that BLU-667, precision-targeted therapy, can have a significant impact on patients having RET-driven cancers. By providing a selectively tailored medicine to this oncogenic driver, this new oral therapy will help patients benefit from the advancements made in genomic profiling. Currently, more patients are being enrolled for the clinical trial.

BLU-667 Competitor — LOXO-292

Similar to the BLU-667, LOXO-292 is a selective oral investigational drug designed to treat patients with RET-driven cancers. LOXO-292 was designed and developed by LOXO Oncology to inhibit RET signaling and acquired resistance mechanisms which could otherwise restrict the activity of LOXO-292 approach. This investigational drug is being studied at present in the LIBRETTO-001 Phase 1/2 trial. The primary phase of the clinical trial is estimated to be completed in November 2021 and the entire study is expected to be completed by October 2022.
In LIBRETTO-001, LOXO-292 is orally administered to those patients with solid tumors that include RET fusion-positive tumors, medullary thyroid cancer (MTC), and other RET-activated tumors. It is also being used to treat pediatric patients with advanced primary nervous system tumors. Currently, there are no results published for public consumption.

Part
05
of five
Part
05

Oncogenic RET alterations

The rearranged during transfer (RET) receptor is a proto-oncogene, or a type of gene that normally helps cells grow. However, when altered, the RET gene can become an oncogene, resulting in uncontrolled division of cells (i.e., cancer); thus, this manner of RET alteration is an oncogenic RET alteration. To date, there are no FDA-approved drugs that specifically inhibit the RET gene to treat RET-caused cancers, though there are two drugs in the pipeline (BLU-667 and LOXO-292) which, provided clinical trial success, will soon be on the market. Below is a complete description of oncogenic RET alterations, followed by a description of the two drugs, along with other treatment options for RET-caused cancers.

Oncogenic Ret Alterations: An Overview

The RET gene is an important one, as it "provides instructions for producing a protein that is involved in signaling within cells," which in turn promotes normal development of a variety of nerve cells. The protein encoded by the RET gene is partly responsible for the development of nerves in the intestine and kidneys, as well as parts of the autonomic nervous system (the system that controls involuntary functions such as heart rate). Because it promotes normal growth while working correctly, the RET gene is a proto-oncogene. These types of genes are important for the healthy functioning of human bodies, but alterations can turn them into oncogenes, which cause uncontrolled cell growth — in other words, cancer. Thus, these alterations can be called oncogenic alterations.

There are two types of oncogenic RET alterations: "activating fusions and point mutations," both of which "lead to overactive RET signaling and uncontrolled cell growth." RET fusions are responsible for about 2% of non-small cell lung cancer cases and 10-20% of cases of papillary thyroid cancer, as well as "subsets of other cancers, including cancers of the colon and pancreas." RET point mutations, on the other hand, are responsible for a significant amount (about 60%) of medullary thyroid cancers. Neither form of oncogenic RET alteration is inherited; rather, they are somatic mutations, which are "acquired during a person's lifetime and are present only in certain cells."

Treatments for RET-caused cancers

As described above, cancers caused by oncogenic RET alterations are the result of uncontrolled growth and division of cells caused by an uninhibited RET gene. Thus, the natural way to combat these kinds of cancers would be to inhibit the RET gene specifically. At the moment, there are "no highly selective RET inhibitors currently approved specifically for these RET-driven cancers," but two that are currently in the development pipeline are BLU-667 and LOXO-292.

BLU-667 is a RET mutation/fusion inhibitor, meaning that it can treat both types of oncogenic RET alterations and a wide range of cancer types. Currently, it has completed phase I clinical testing and been shown to be successful in treating several RET-caused cancers. LOXO-292 is an investigational RET inhibitor that has been granted breakthrough therapy status by the FDA, allowing it to move through the development process at an expedited rate. It is currently undergoing phase 1/2 clinical trials.

Currently, RET-caused cancers are treated simply via "traditional chemotherapy and earlier generations of multikinase inhibitors...[which] have had limited success, with often considerable side effects." Multikinase inhibitors (MKIs) like Cabozantinib, which are non-specific in their inhibitory actions, have been used to treat RET-caused cancers, but the results have "been disappointing," showing little effect on either type of oncogenic RET alteration. As such, the future of RET-caused cancers primarily hinges on the clinical success of RET-specific inhibitors like BLU-667 and LOXO-292.

Conclusion

The rearranged during transfer (RET) gene is an important proto-oncogene, facilitating the growth of nerves in many parts of the body. However, certain alterations to this gene, including fusion and mutation, can turn it into an oncogene, resulting in uncontrolled cell growth and division. There are no drugs currently on the market that target and inhibit RET specifically, though two such drugs, BLU-667 and LOXO-292, are in the pipeline. Aside from them, the only options for those with RET-caused cancers are traditional chemotherapy treatments and non-specific multikinase inhibitors (MKIs) like Cabozantinib, which have been largely unsuccessful in treating cancers caused by oncogenic RET alterations.
Sources
Sources

From Part 03
Quotes
  • "As of March 31, 2019, cash, cash equivalents and investments were $415.9 million, as compared to $494.0 million as of December 31, 2018. This decrease was primarily related to cash used in operating activities. Cash, cash equivalents and investments as of March 31, 2019 do not include the estimated net proceeds of approximately $327.2 million from the company's follow-on underwritten public offering of common stock, which closed in April 2019."
Quotes
  • "BLU-667 is an oral precision therapy designed for highly potent and selective targeting of oncogenic RET alterations and resistance mutants. Across a wide range of cancers, oncogenic alterations in RET result in kinase activation, driving tumor formation and growth. The two primary mechanisms of oncogenic RET activation are fusions and mutations."
Quotes
  • "CStone Pharmaceuticals ("CStone"; HKEX: 2616) today announced that China's National Medical Products Administration (NMPA) has approved the clinical trial application to begin a Phase 1 trial in China for BLU-667 (CS3009), a highly selective and potent RET inhibitor discovered by CStone's partner Blueprint Medicines. The study is part of Blueprint Medicines' ongoing, global Phase 1 ARROW clinical trial for patients with RET-altered non-small cell lung cancer (NSCLC), medullary thyroid cancer (MTC) and other advanced solid tumors. Trial objectives include overall clinical response, duration of response, pharmacokinetics, pharmacodynamics and safety."
Quotes
  • "Given the disabling and destructive nature of this disease, people living with FOP have a desperate need for a safe and effective treatment," said Adam Sherman, Research Development and Partnerships Director at the International Fibrodysplasia Ossificans Progressiva Association. "We are immensely grateful to have companies like Blueprint Medicines developing new treatments for those living with FOP. We look forward to seeing BLU-782 advance through the development pathway and hope a therapeutic option will one day change the course of this disease"
From Part 04
From Part 05
Quotes
  • "The rearranged during transfection (RET) receptor is a tyrosine kinase receptor that binds the glial cell line-derived neurotrophic factor (GDNF) ligand family and contributes to the development of the nervous system and kidneys. Genomic alterations in the RET kinase, which include activating fusions and point mutations, lead to overactive RET signaling and uncontrolled cell growth."
  • "RET fusions have been identified in approximately 2% of non-small cell lung cancer, 10-20% of papillary thyroid cancers, and subsets of other cancers, including cancers of the colon and pancreas. Activating RET point mutations account for approximately 60% of medullary thyroid cancer (MTC). Both RET fusion cancers and RET-mutant MTC are primarily dependent on this single activated kinase for their proliferation and survival."
Quotes
  • "The RET gene provides instructions for producing a protein that is involved in signaling within cells. This protein appears to be essential for the normal development of several kinds of nerve cells, including nerves in the intestine (enteric neurons) and the portion of the nervous system that controls involuntary body functions such as heart rate (the autonomic nervous system). The RET protein is also necessary for normal kidney development and the production of sperm (spermatogenesis)."
  • "The RET protein spans the cell membrane, so that one end of the protein remains inside the cell and the other end projects from the outer surface of the cell. This positioning of the protein allows it to interact with specific factors outside the cell and to receive signals that help the cell respond to its environment. When molecules that stimulate growth and development (growth factors) attach to the RET protein, a complex cascade of chemical reactions inside the cell is triggered."
  • "Some gene mutations are acquired during a person's lifetime and are present only in certain cells. These changes, which are called somatic mutations, are not inherited. Somatic changes in the RET gene have been identified in several nonhereditary (sporadic) cancers. "
  • "Chromosomal rearrangements involving the RET gene are one of the most common causes of a sporadic form of thyroid cancer called papillary thyroid carcinoma (also known as RET/PTC). Additionally, a nonfamilial form of medullary thyroid carcinoma (a type of thyroid cancer that can also occur as part of multiple endocrine neoplasia) can be caused by somatic mutations in the RET gene."
Quotes
  • "A first-in-human study provides proof of concept that a novel oral agent targeted to RET genetic alterations is safe and active in RET-driven cancers. The agent, called BLU-667, achieved durable disease control in patients with lung and thyroid cancers harboring the RET oncogene, according to the results of the phase I ARROW study reported at the 2018 Annual Meeting of the American Association for Cancer Research (AACR)."
  • "To date, no drug has been approved by the U.S. Food and Drug Administration to treat RET-driven cancers. “There is a critical unmet need for effective drugs against cancers that have the RET alteration...The current treatments for these cancers are limited to traditional chemotherapy and earlier generations of multikinase inhibitors."
Quotes
  • "Proto-oncogenes are genes that normally help cells grow. When a proto-oncogene mutates (changes) or there are too many copies of it, it becomes a "bad" gene that can become permanently turned on or activated when it is not supposed to be. When this happens, the cell grows out of control, which can lead to cancer. This bad gene is called an oncogene."