On January 28, 2022 Blueprint Medicines Corporation (NASDAQ: BPMC) reported that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has adopted a positive opinion, recommending to expand the current indication for AYVAKYT (avapritinib) to include monotherapy for the treatment of adult patients with aggressive systemic mastocytosis (ASM), systemic mastocytosis with an associated hematological neoplasm (SM-AHN) or mast cell leukemia (MCL), after at least one systemic therapy (Press release, Blueprint Medicines, JAN 28, 2022, View Source [SID1234607485]).

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"The positive opinion from the CHMP is a result of nearly a decade of collaboration with the systemic mastocytosis community and a dedication to bring a transformative therapy to treat and manage this rare, life-threatening disease," said Becker Hewes, M.D., Chief Medical Officer at Blueprint Medicines. "Today’s announcement brings patients with advanced forms of SM in Europe one step closer to accessing AYVAKYT, the first specifically designed precision therapy to selectively target the primary driver of their disease. Blueprint Medicines remains committed to improving outcomes for patients living with systemic mastocytosis around the world."

"Patients across Europe are waiting for innovative treatment options for advanced systemic mastocytosis, which is associated with organ damage due to mast cell proliferation and poor survival outcomes," said Prof. Dr. Andreas Reiter, M.D., Center of Excellence for Myeloproliferative Neoplasms, University Medicine Mannheim. "A precision therapy associated with potent and selective targeting of the KIT D816V mutation brings the prospect of changing the course of disease and transforming treatment for patients to set a new standard of care in Europe."

The CHMP opinion will now be reviewed by the European Commission, which has the authority to grant and expand marketing authorizations for medicinal products in the European Union. A final decision on the application to expand the indication for AYVAKYT is anticipated by early April 2022.

The CHMP based its opinion on results from the Phase 1 EXPLORER trial and Phase 2 PATHFINDER trial, in which AYVAKYT showed durable clinical efficacy in advanced SM patients across disease subtypes and a generally well-tolerated safety profile. These two studies, which enrolled approximately 150 patients with up to four years of follow-up and comprise the largest ever reported dataset in advanced systemic mastocytosis, were recently published in Nature Medicine.

About AYVAKYT (avapritinib)
AYVAKYT (avapritinib) is a kinase inhibitor approved by the European Commission for the treatment of adults with unresectable or metastatic gastrointestinal stromal tumors (GIST) harboring the PDGFRA D842V mutation. Under the brand name AYVAKIT, the medicine is approved in the U.S. for the treatment of adults with unresectable or metastatic GIST harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations, and for the treatment of adults with Advanced SM, including aggressive SM (ASM), SM-AHN and mast cell leukemia (MCL).1 It is also approved under the brand name AYVAKIT in Mainland China for the treatment of adults with unresectable or metastatic GIST harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations, and in Hong Kong and Taiwan for the treatment of adults with unresectable or metastatic GIST harboring a PDGFRA D842V mutation.2-4

AYVAKYT/AYVAKIT is not approved for the treatment of any other indication in the Europe, U.S., or Greater China, or for any indication in any other jurisdiction by any other health authority.

Blueprint Medicines is developing AYVAKYT/AYVAKIT globally for the treatment of advanced and non-advanced SM. The European Commission granted orphan medicinal product designation for AYVAKYT for the treatment of GIST and mastocytosis. The U.S. Food and Drug Administration (FDA) granted breakthrough therapy designation to AYVAKIT for the treatment of moderate to severe indolent SM.

To learn about ongoing or planned clinical trials, contact Blueprint Medicines at [email protected] and +31 85 064 4001. Additional information is available at blueprintclinicaltrials.com and clinicaltrials.gov.

Please click here to see the Summary of Product Characteristics for AYVAKYT.

About Systemic Mastocytosis

Systemic mastocytosis (SM) is a rare disease driven by the KIT D816V mutation. Uncontrolled proliferation and activation of mast cells result in chronic, severe and often unpredictable symptoms for patients across the spectrum of SM. The vast majority of those affected have non-advanced (indolent or smoldering) SM, with debilitating symptoms that lead to a profound, negative impact on quality of life. A minority of patients have advanced SM, which encompasses a group of high-risk SM subtypes including ASM, SM-AHN and MCL. In addition to mast cell activation symptoms, advanced SM is associated with organ damage due to mast cell infiltration and poor survival. In Europe, there are about 40,000 patients with SM, and advanced SM represents about 5 to 10 percent of this patient population.5

Debilitating symptoms, including anaphylaxis, maculopapular rash, pruritis, diarrhea, brain fog, fatigue and bone pain, often persist across all forms of SM despite treatment with a number of symptomatic therapies. Patients often live in fear of severe, unexpected symptoms, have limited ability to work or perform daily activities, and isolate themselves to protect against unpredictable triggers. Historically, there had been no approved therapies for the treatment of SM that selectively inhibit D816V mutant KIT.6,7
(Press release, Blueprint Medicines, JAN 28, 2022, View Source [SID1234607485])

On January 28 2022 Sareum Holdings plc (AIM: SAR), the specialist drug development company, reported that it has issued warrants over 29,000,000 new ordinary shares of 0.025p each in the capital of the Company to individuals who provided introductory services leading to the share subscriptions announced by the Company during 2021 (Press release, Sareum, JAN 28, 2022, View Source [SID1234607483]). The warrants have an exercise price of 5p per share, with exercise being conditional on the shares having traded at or above 7p for five consecutive days, and a subscription period that ends on 31 May 2026.

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On January 28, 2022 Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) and Sanofi reported the voluntary withdrawal of the supplemental Biologics License Application (sBLA) for Libtayo (cemiplimab-rwlc) as a second-line treatment for patients with advanced cervical cancer (Press release, Regeneron, JAN 28, 2022, View Source [SID1234607482]). The decision was made after the companies and the U.S. Food and Drug Administration (FDA) were not able to align on certain post-marketing studies. Discussions with regulatory authorities outside of the U.S. are ongoing.

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About Cervical Cancer
It is estimated that approximately 570,000 people are diagnosed with cervical cancer worldwide each year, with deaths exceeding 250,000. In the U.S. there are 14,500 new cases diagnosed annually and approximately 4,000 die each year. Since we filed our sBLA, another PD-1 inhibitor was approved as first-line treatment for patients with persistent, recurrent or metastatic cervical tumors that express PD-L1. The use of Libtayo in advanced cervical cancer is not approved by the FDA.

About Libtayo
Libtayo is a fully human monoclonal antibody targeting the immune checkpoint receptor PD-1 on T-cells. By binding to PD-1, Libtayo has been shown to block cancer cells from using the PD-1 pathway to suppress T-cell activation. Libtayo is indicated in certain patients with advanced basal cell carcinoma (BCC), advanced cutaneous squamous cell carcinoma (CSCC), and advanced non-small cell lung cancer (NSCLC).

The extensive clinical program for Libtayo is focused on difficult-to-treat cancers. Libtayo is currently being investigated in trials as a monotherapy, as well as in combination with either conventional or novel therapeutic approaches for other solid tumors and blood cancers. These potential uses are investigational, and their safety and efficacy have not been evaluated by any regulatory authority.

Libtayo, which was invented using Regeneron’s proprietary VelocImmune technology, is being jointly developed by Regeneron and Sanofi under a global collaboration agreement.

U.S. FDA-approved Indications
Libtayo is a prescription medicine used to treat people with:

A type of skin cancer called advanced CSCC that has spread or cannot be cured by surgery or radiation.
A type of skin cancer called BCC:
That cannot be removed by surgery (locally advanced BCC) and have received treatment with a hedgehog inhibitor (HHI), or cannot receive treatment with an HHI.
That has spread (metastatic BCC) and have received treatment with an HHI, or cannot receive treatment with an HHI. This use is approved based on how many patients responded to treatment and how long they responded. Studies are ongoing to provide additional information about clinical benefit.
A type of lung cancer called NSCLC. Libtayo may be used as your first treatment when your lung cancer has not spread outside your chest (locally advanced lung cancer) and you cannot have surgery or chemotherapy with radiation, or your lung cancer has spread to other areas of your body (metastatic lung cancer), and your tumor tests positive for high "PD-L1" and your tumor does not have an abnormal "EGFR", "ALK" or "ROS1" gene.
It is not known if Libtayo is safe and effective in children.

About Regeneron’s VelocImmune Technology
Regeneron’s VelocImmune technology utilizes a proprietary genetically engineered mouse platform endowed with a genetically humanized immune system to produce optimized fully human antibodies. When Regeneron’s President and Chief Scientific Officer George D. Yancopoulos was a graduate student with his mentor Frederick W. Alt in 1985, they were the first to envision making such a genetically humanized mouse, and Regeneron has spent decades inventing and developing VelocImmune and related VelociSuite technologies. Dr. Yancopoulos and his team have used VelocImmune technology to create approximately a quarter of all original, FDA-approved or authorized fully human monoclonal antibodies currently available. This includes REGEN-COV(casirivimab and imdevimab), Dupixent (dupilumab), Libtayo (cemiplimab-rwlc), Praluent(alirocumab), Kevzara (sarilumab), Evkeeza (evinacumab-dgnb) and Inmazeb (atoltivimab, maftivimab, and odesivimab-ebgn).

IMPORTANT SAFETY INFORMATION AND INDICATION FOR U.S. PATIENTS

What is the most important information I should know about Libtayo?
Libtayo is a medicine that may treat certain cancers by working with your immune system. Libtayo can cause your immune system to attack normal organs and tissues in any area of your body and can affect the way they work. These problems can sometimes become severe or life-threatening and can lead to death. You can have more than one of these problems at the same time. These problems may happen anytime during treatment or even after your treatment has ended.

Call or see your healthcare provider right away if you develop any new or worsening signs or symptoms, including:

Lung problems: cough, shortness of breath, or chest pain
Intestinal problems: diarrhea (loose stools) or more frequent bowel movements than usual, stools that are black, tarry, sticky or have blood or mucus, or severe stomach area (abdomen) pain or tenderness
Liver problems: yellowing of your skin or the whites of your eyes, severe nausea or vomiting, pain on the right side of your stomach area (abdomen), dark urine (tea colored), or bleeding or bruising more easily than normal
Hormone gland problems: headache that will not go away or unusual headaches, eye sensitivity to light, eye problems, rapid heartbeat, increased sweating, extreme tiredness, weight gain or weight loss, feeling more hungry or thirsty than usual, urinating more often than usual, hair loss, feeling cold, constipation, your voice gets deeper, dizziness or fainting, or changes in mood or behavior, such as decreased sex drive, irritability, or forgetfulness
Kidney problems: decrease in your amount of urine, blood in your urine, swelling of your ankles, or loss of appetite
Skin problems: rash, itching, skin blistering or peeling, painful sores or ulcers in mouth or nose, throat, or genital area, fever or flu-like symptoms, or swollen lymph nodes
Problems can also happen in other organs and tissues. These are not all of the signs and symptoms of immune system problems that can happen with Libtayo. Call or see your healthcare provider right away for any new or worsening signs or symptoms, which may include: chest pain, irregular heartbeat, shortness of breath or swelling of ankles, confusion, sleepiness, memory problems, changes in mood or behavior, stiff neck, balance problems, tingling or numbness of the arms or legs, double vision, blurry vision, sensitivity to light, eye pain, changes in eyesight, persistent or severe muscle pain or weakness, muscle cramps, low red blood cells, or bruising
Infusion reactions that can sometimes be severe. Signs and symptoms of infusion reactions may include: nausea, chills or shaking, itching or rash, flushing, shortness of breath or wheezing, dizziness, feel like passing out, fever, back or neck pain, or facial swelling.
Rejection of a transplanted organ. Your healthcare provider should tell you what signs and symptoms you should report and monitor you, depending on the type of organ transplant that you have had.
Complications, including graft-versus-host disease (GVHD), in people who have received a bone marrow (stem cell) transplant that uses donor stem cells (allogeneic). These complications can be serious and can lead to death. These complications may happen if you underwent transplantation either before or after being treated with Libtayo. Your healthcare provider will monitor you for these complications.
Getting medical treatment right away may help keep these problems from becoming more serious.
Your healthcare provider will check you for these problems during your treatment with Libtayo. Your healthcare provider may treat you with corticosteroid or hormone replacement medicines. Your healthcare provider may also need to delay or completely stop treatment with Libtayo if you have severe side effects.

Before you receive Libtayo, tell your healthcare provider about all your medical conditions, including if you:

have immune system problems such as Crohn’s disease, ulcerative colitis, or lupus
have received an organ transplant
have received or plan to receive a stem cell transplant that uses donor stem cells (allogeneic)
have a condition that affects your nervous system, such as myasthenia gravis or Guillain-Barré syndrome are pregnant or plan to become pregnant. Libtayo can harm your unborn baby
Females who are able to become pregnant:
Your healthcare provider will give you a pregnancy test before you start treatment.
You should use an effective method of birth control during your treatment and for at least 4 months after your last dose of Libtayo. Talk with your healthcare provider about birth control methods that you can use during this time.
Tell your healthcare provider right away if you become pregnant or think you may be pregnant during treatment with Libtayo.
are breastfeeding or plan to breastfeed. It is not known if Libtayo passes into your breast milk. Do not breastfeed during treatment and for at least 4 months after the last dose of Libtayo.
Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

The most common side effects of Libtayo include muscle or bone pain, tiredness, rash, and diarrhea. These are not all the possible side effects of Libtayo. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088. You may also report side effects to Regeneron Pharmaceuticals and Sanofi at
1-877-542-8296.

On Janaury 28, 2022 Quest Diagnostics (NYSE: DGX), the world’s leading provider of diagnostic information services, reported that it has received a score of 100% on the Human Rights Campaign Foundation’s 2022 Corporate Equality Index (CEI), the nation’s foremost benchmarking survey and report measuring corporate policies and practices related to LGBTQ+ workplace equality (Press release, Quest Diagnostics, JAN 28, 2022, View Source [SID1234607481]).

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"We are honored to earn a 100% score on the Corporate Equality Index for the sixth consecutive year," said Cecilia McKenney, Quest’s Chief Human Resources Officer and Pride Employee Business Network (EBN) co-sponsor. "Quest is committed to providing our employees with a diverse, equitable and inclusive workplace where employees feel safe to bring their authentic selves to work. Our diverse workforce empowers the valuable insights and inclusive care we are able to deliver to our patients and communities."

Quest has 10 EBNs, including the Pride EBN, an all-inclusive resource group for LGBTQ employees, straight/cis allies, parents and family members of LGBTQ individuals. For over 20 years, the Pride EBN has engaged colleagues and senior leaders to promote awareness, education, and advocacy of social justice for the LGBTQ+ community.

Inclusion and Diversity is an integral part of Quest. By consistently inviting new perspectives and exploring new experiences, the company strives to create an inclusive, respectful and empathetic workplace where everyone feels they belong. An example of the company’s commitment to equity is the Quest Inclusion and Diversity (I&D) Council, which advises senior leaders as I&D goals and priorities are established, acts as a visible I&D leader and represents the needs and perspectives of diverse demographics across the company and communities.

The CEI rates companies on detailed criteria under four central pillars: non-discrimination policies across business entities; equitable benefits for LGBTQ+ workers and their families; supporting an inclusive culture; and corporate social responsibility.

Read more about Quest’s award-winning culture on the company’s ESG Resources page. Find the full Human Rights Campaign Foundation’s 2022 Corporate Equality Index (CEI) here.

On January I-Mab (the "Company") (Nasdaq: IMAB), a clinical-stage biopharmaceutical company committed to the discovery, development, and commercialization of novel biologics (Press release, I-Mab Biopharma, JAN 28, 2022, View Source [SID1234607480]). The Company has noted the recent unusual fluctuations in the price and trading volume of the American depositary shares (the "ADSs") of the Company and emphasizes that its business and pipeline developments are all well on track and have no adverse changes . The Company has strong confidence in its global competitive pipeline and is accelerating ongoing transformation towards an integrated global biopharma as planned.

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Latest Development of Lemzoparlimab and Uliledlimab:

Lemzoparlimab: multiple clinical studies are ongoing in both the U.S. and China, including lemzoparlimab in combination with azacytidine in myelodysplastic syndrome (MDS) and acute myelocytic leukemia (AML), in combination with rituximab in non-Hodgekin’s lymphoma (NHL), and in combination with pembrolizumab (Keytruda ) or toripalimab (TUOYI ) in advanced solid tumors. Based on data from multiple on-going clinical trials so far, approximately 180 patients, including 120 patients with hemetological maglinancies, have been treated with lemzoparlimab without the need for priming dosing, and showed anti-tumor activity and a favorable safety profile and lack of a unwanted "sink effect". Data readout from MDS, NHL and solid tumor clinical trials is expected in 2022.

Uliledlimab in combination with toripalimab (TUOYI) : The China phase 2 cohort expansion study in patients with advanced or metastatic cancers who are refractory to or intolerant of all available therapies is ongoing. The company expects to submit the preliminary data to ASCO (Free ASCO Whitepaper) 2022.

In 2022, the Company expects to deliver a series of significant clinical milestones. As a result, the pipeline will be advanced to include 3—4 registrational clinical trials (lemzoparlimab, estansomatropin, felzartamab), 11 Phase 2 clinical trials (including 8 new trials) and 3 Phase 1 clinical trials by end of 2022.

Financial Position:

The company has strong cash position with approximately $700 million, as of 31 December, 2021.

Between 2022 to 2023, the company expects to receive over $100 million cash from the existing out-licensing deals. The current cash on hand is sufficient to fund the Company’s operations for three years.

The Company’s fundamentals remain very strong. In 2021, I-Mab has successfully delivered planned important clinical and corporate milestones and will continue building on the strong momentum to deliver critical new milestones, including reporting multiple key clinical data readouts, initiating new registrational trials and accelerating dual listing on The Main Board of The Stock Exchange of Hong Kong Limited, continually creating value for patients and shareholders.