On July 21, 2021 ERYTECH Pharma (Nasdaq & Euronext: ERYP), a clinical-stage biopharmaceutical company developing innovative therapies by encapsulating therapeutic drug substances inside red blood cells, reported its intention to move forward towards the submission of a BLA to the US Food and Drug Administration (FDA) for eryaspase in hypersensitive acute lymphoblastic leukemia (ALL) patients following feedback from the agency in a pre-BLA meeting (Press release, ERYtech Pharma, JUL 21, 2021, View Source [SID1234585095]).

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The purpose for the meeting was to discuss clinical, non-clinical and quality content and expectations for the Company’s potential BLA submission. Based on the discussion and the totality of the available information to the Company to date, ERYTECH believes its regulatory package can potentially support an approval of eryaspase in hypersensitive ALL patients. Pending successful completion of remaining steps, the Company anticipates submitting a BLA in the fourth quarter of 2021.

"We are encouraged by the interactive dialogue with the agency, and grateful for the feedback and guidance we received on a potential path to approval," said Gil Beyen, CEO of ERYTECH. "This is an important milestone for ERYTECH as it advances our lead product candidate eryaspase as an alternative treatment for ALL patients with hypersensitivity to PEG-asparaginase, while our Phase 3 trial in second line pancreatic cancer is nearing completion. If all goes well, we could be in a position to make this product candidate available to ALL and pancreatic cancer patients in the course of next year."

In December 2020, positive Phase 2 clinical results were presented by the Nordic Society of Pediatric Hematology and Oncology, at the 2020 American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting. Eryaspase, in combination with chemotherapy, administered every two weeks, provided a sustained asparaginase enzyme activity level, and was generally well tolerated with few hypersensitivity reactions.

About Acute Lymphoblastic Leukemia

Acute lymphoblastic leukemia (ALL) is a cancer of the blood and bone marrow that is the most common type of cancer in children in the US and Europe. More than 13,000 cases are diagnosed in the US and Europe each year with the majority of patients diagnosed before age 20. Asparaginase has been an integral component of ALL treatment for several years but is associated with treatment-limiting hypersensitivity in up to 30% of patients. Discontinuation of asparaginase therapy in ALL patients has been associated with inferior event free survival highlighting the need for additional asparaginase based treatment options.

On July 21, 2021 Samsung Biologics, one of the world’s leading contract development and manufacturing organization (CDMO), reported that signed a strategic partnership agreement with Kineta, Inc., a clinical-stage biotech company developing novel immunotherapies in oncology (Press release, Kineta, JUL 21, 2021, View Source;schBoardCtgryCcd=&schString=&schBoardYear=&boardDtm=1626706800000&page=1#new_tab&utm_source=rss&utm_medium=rss&utm_campaign=kineta-and-samsung-biologics-announce-development-and-manufacturing-agreement-for-anti-vista-antibody-immunotherapy [SID1234586786]). Samsung Biologics will provide end-to-end CDMO service from cell line development, clinical drug substance, and drug product manufacturing services to support IND filing for KVA12.1, Kineta’s novel anti-VISTA antibody in development for the treatment of solid tumors.

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VISTA is a key driver of the immunosuppressive tumor microenvironment (TME) and is overexpressed on myeloid-derived suppressor cells (MDSC) and regulatory T cells (Tregs). It is a critical myeloid cell immune-checkpoint, and VISTA blockade can reprogram suppressive myeloid cells and reactivate anti-tumor immune function. Preclinical studies demonstrate single agent anti-tumor activity with KVA12.1, and also demonstrate that targeting VISTA in combination with PD-1, PD-L1 or CTLA-4 can significantly improve the anti-tumor efficacy of those checkpoint inhibitors. Kineta’s KVA12.1 aims to reprogram the TME in hard-to-treat solid tumors.

The manufacturing cell line will be developed with support from Samsung Biologics’ R&D Center in San Francisco, and its corresponding clinical trial materials will be manufactured at Samsung Biologics headquarters in Incheon, South Korea.

"We are very glad to be partnering with Kineta, to provide support in bringing this cancer immunotherapy to market," said John Rim, CEO of Samsung Biologics. "We will fully utilize our capabilities and streamlined end-to-end processes from both our headquarters and US R&D Center, to enable high-quality development of KVA12.1 with faster speed to accelerate Kineta’s success."

"Establishing this strategic partnership with Samsung is a critical step for Kineta as we advance KVA12.1 to IND and into first-in-human clinical trials next year", said Shawn Iadonato, PhD, Chief Executive Officer at Kineta. "We are excited to collaborate with Samsung to initiate and scale up drug product manufacturing of our VISTA immunotherapy".

Samsung Biologics offers seamless one-stop CDMO research and development services both at its headquarters in Incheon, South Korea and its newly built R&D Center in San Francisco. Bringing quality-driven development services at a greater speed, the company delivers cell line development to drug substance manufacturing in six months, and to drug product manufacturing in seven months, the fastest pace in the industry. Currently building its fourth and largest facility in Incheon, Korea, Samsung Biologics will have 620,000 liters of biomanufacturing capacity upon completion of the plant in 2023.

On July 21, 2021 AbbVie (NYSE: ABBV) reported the U.S. Food and Drug Administration (FDA) granted a Breakthrough Therapy Designation (BTD) to venetoclax (VENCLEXTA) in combination with azacitidine for the potential treatment of adult patients with previously untreated intermediate-, high- and very high-risk myelodysplastic syndromes (MDS) based on revised International Prognostic Scoring System (IPSS-R) (Press release, AbbVie, JUL 21, 2021, View Source [SID1234584996]). A BTD is intended to expedite the development and review of medications to treat a serious medical condition and is granted when preliminary clinical evidence indicates the investigational therapy may demonstrate substantial improvement over existing therapies.1 This marks the sixth BTD granted to venetoclax.

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MDS are a group of diverse bone marrow disorders in which the bone marrow does not produce enough healthy blood cells.2 Patients living with MDS may experience symptoms such as infection, anemia, spontaneous bleeding, and easy bruising. Roughly 10,000 patients in the US are diagnosed with MDS each year3 and around 30 percent of those patients will progress to acute myeloid leukemia (AML). Although MDS can occur at any age, it is most commonly found in patients aged 60 and older.4

"MDS is a devastating diagnosis – not only does it have the potential to greatly impact patients’ quality of life, but 30 percent of patients will also progress to AML," said Jalaja Potluri, executive medical director, oncology, AbbVie. "This Breakthrough Therapy Designation underscores the need for more treatment options for these patients and the utility of venetoclax to potentially treat different forms of blood cancer."

This designation is supported by data from the Phase 1b M15-531 study. In addition to the Phase Ib M15-531 study, venetoclax is being investigated in combination with azacitidine for the treatment of MDS in the Phase Ib M15-522 study in patients with relapsed or refractory disease, and the Phase 3 randomized VERONA study in patients with newly diagnosed higher-risk MDS.

VENCLEXTA is being developed by AbbVie and Roche. It is jointly commercialized by AbbVie and Genentech, a member of the Roche Group, in the U.S. and by AbbVie outside of the U.S.

About VENCLEXTA (venetoclax)
VENCLEXTA/VENCLYXTO (venetoclax) is a first-in-class medicine that selectively binds and inhibits the B-cell lymphoma-2 (BCL-2) protein. In some blood cancers, BCL-2 prevents cancer cells from undergoing their natural death or self-destruction process, called apoptosis. VENCLXEXTA/VENCLYXTO targets the BCL-2 protein and works to help restore the process of apoptosis.

VENCLEXTA/VENCLYXTO is being developed by AbbVie and Roche. It is jointly commercialized by AbbVie and Genentech, a member of the Roche Group, in the U.S. and by AbbVie outside of the U.S. Together, the companies are committed to BCL-2 research and to studying venetoclax in clinical trials across several blood cancers. Venetoclax is approved in more than 80 countries, including the U.S.

Uses of VENCLEXTA (venetoclax) in US

VENCLEXTA is a prescription medicine used:

to treat adults with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL).
in combination with azacitidine, or decitabine, or low-dose cytarabine to treat adults with newly diagnosed acute myeloid leukemia (AML) who:
are 75 years of age or older, or
have other medical conditions that prevent the use of standard chemotherapy.
It is not known if VENCLEXTA is safe and effective in children.

Important VENCLEXTA (venetoclax) US Safety Information

US VENCLEXTA Important Safety Information

What is the most important information I should know about VENCLEXTA?
VENCLEXTA can cause serious side effects, including:
Tumor lysis syndrome (TLS). TLS is caused by the fast breakdown of cancer cells. TLS can cause kidney failure, the need for dialysis treatment, and may lead to death. Your healthcare provider will do tests to check your risk of getting TLS before you start taking VENCLEXTA. You will receive other medicines before starting and during treatment with VENCLEXTA to help reduce your risk of TLS. You may also need to receive intravenous (IV) fluids into your vein. Your healthcare provider will do blood tests to check for TLS when you first start treatment and during treatment with VENCLEXTA. It is important to keep your appointments for blood tests. Tell your healthcare provider right away if you have any symptoms of TLS during treatment with VENCLEXTA, including fever, chills, nausea, vomiting, confusion, shortness of breath, seizures, irregular heartbeat, dark or cloudy urine, unusual tiredness, or muscle or joint pain.

Drink plenty of water during treatment with VENCLEXTA to help reduce your risk of getting TLS.
Drink 6 to 8 glasses (about 56 ounces total) of water each day, starting 2 days before your first dose, on the day of your first dose of VENCLEXTA, and each time your dose is increased.

Your healthcare provider may delay, decrease your dose, or stop treatment with VENCLEXTA if you have side effects. When restarting VENCLEXTA after stopping for 1 week or longer, your healthcare provider may again check for your risk of TLS and change your dose.

Who should not take VENCLEXTA?
Certain medicines must not be taken when you first start taking VENCLEXTA and while your dose is being slowly increased because of the risk of increased TLS.

Tell your healthcare provider about all the medicines you take, including prescription and over-the counter medicines, vitamins, and herbal supplements. VENCLEXTA and other medicines may affect each other causing serious side effects.
Do not start new medicines during treatment with VENCLEXTA without first talking with your healthcare provider.
Before taking VENCLEXTA, tell your healthcare provider about all of your medical conditions, including if you:

have kidney or liver problems.
have problems with your body salts or electrolytes, such as potassium, phosphorus, or calcium.
have a history of high uric acid levels in your blood or gout.
are scheduled to receive a vaccine. You should not receive a "live vaccine" before, during, or after treatment with VENCLEXTA, until your healthcare provider tells you it is okay. If you are not sure about the type of immunization or vaccine, ask your healthcare provider. These vaccines may not be safe or may not work as well during treatment with VENCLEXTA.
are pregnant or plan to become pregnant. VENCLEXTA may harm your unborn baby. If you are able to become pregnant, your healthcare provider should do a pregnancy test before you start treatment with VENCLEXTA, and you should use effective birth control during treatment and for at least 30 days after the last dose of VENCLEXTA. If you become pregnant or think you are pregnant, tell your healthcare provider right away.
are breastfeeding or plan to breastfeed. It is not known if VENCLEXTA passes into your breast milk. Do not breastfeed during treatment with VENCLEXTA and for 1 week after the last dose.
What should I avoid while taking VENCLEXTA?
You should not drink grapefruit juice or eat grapefruit, Seville oranges (often used in marmalades), or starfruit while you are taking VENCLEXTA. These products may increase the amount of VENCLEXTA in your blood.

What are the possible side effects of VENCLEXTA?
VENCLEXTA can cause serious side effects, including:

Low white blood cell counts (neutropenia). Low white blood cell counts are common with VENCLEXTA, but can also be severe. Your healthcare provider will do blood tests to check your blood counts during treatment with VENCLEXTA and may pause dosing.
Infections. Death and serious infections such as pneumonia and blood infection (sepsis) have happened during treatment with VENCLEXTA. Your healthcare provider will closely monitor and treat you right away if you have a fever or any signs of infection during treatment with VENCLEXTA.
Tell your healthcare provider right away if you have a fever or any signs of an infection during treatment with VENCLEXTA.

The most common side effects of VENCLEXTA when used in combination with obinutuzumab or rituximab or alone in people with CLL or SLL include low white blood cell counts; low platelet counts; low red blood cell counts; diarrhea; nausea; upper respiratory tract infection; cough; muscle and joint pain; tiredness; and swelling of your arms, legs, hands, and feet.

The most common side effects of VENCLEXTA in combination with azacitidine or decitabine or low-dose cytarabine in people with AML include nausea; diarrhea; low platelet count; constipation; low white blood cell count; fever with low white blood cell count; tiredness; vomiting; swelling of arms, legs, hands, or feet; fever; infection in lungs; shortness of breath; bleeding; low red blood cell count; rash; stomach (abdominal) pain; infection in your blood; muscle and joint pain; dizziness; cough; sore throat; and low blood pressure.

VENCLEXTA may cause fertility problems in males. This may affect your ability to father a child. Talk to your healthcare provider if you have concerns about fertility.

These are not all the possible side effects of VENCLEXTA. Call your doctor for medical advice about side effects.

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

If you cannot afford your medication, contact genentech-access.com/patient/brands/venclexta for assistance.

On July 21, 2021 Personalis, Inc. (Nasdaq: PSNL), a leader in advanced genomics for cancer, reported it will release its second quarter 2021 financial results after the market closes on Wednesday, August 4, 2021 (Press release, Personalis, JUL 21, 2021, View Source [SID1234585028]). In conjunction with the release, the Company will host a conference call and webcast that day at 2:00 p.m. Pacific Time / 5:00 p.m. Eastern Time to discuss its financial results and recent highlights.

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Interested parties may access the live call via telephone by dialing (866) 220-8061 for domestic callers or (470) 495-9168 for international callers, using conference ID: 8564509. The live webinar of the call may be accessed by visiting the Events section of the company’s website at investors.personalis.com. A replay of the webinar will be available shortly after the conclusion of the call and will be archived on the company’s website.

On July 21, 2021 Calliditas Therapeutics AB (Nasdaq: CALT, Nasdaq Stockholm: CALTX) ("Calliditas") and STADA Arzneimittel AG ("STADA") reported they have entered into a license agreement to register and commercialize a novel specialty drug candidate for the treatment of the chronic autoimmune kidney disease Immunoglobulin A Nephropathy (IgAN) in the European Economic Area (EEA) member states, Switzerland and the UK (Press release, Calliditas Therapeutics, JUL 21, 2021, View Source [SID1234584997]).

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Under the terms of the agreement, Calliditas is entitled receive an initial upfront payment of 20M EUR ($24m) upon signing and up to an additional 77.5M EUR ($91m) in future payments linked to pre-defined regulatory and commercialization milestones. STADA is also obligated pay tiered royalties on net sales expressed as a percentage between the low twenties and the low thirties.

The partnership relates to a novel oral formulation, developed under the project name ‘Nefecon’, of a potent and well-known active substance – budesonide – designed to target down regulation of IgA1 with a view to be disease modifying. If approved, this value-added specialty medicine, which received an EU orphan-drug designation in 2016, would be the first treatment authorized in the European Union for IgAN, a rare autoimmune disease. IgAN, also known as Berger´s disease, is a serious progressive autoimmune disease in which up to 50% of patients end up at risk of developing end stage renal disease and thus requiring dialysis or a kidney transplant. Prevalence in Europe is estimated at 4 in 10,000, translating into approximately 200,000 patients.

"We are excited to be entering into this partnership with STADA to bring this IgAN therapy to market in Europe, where there is a significant unmet medical need for this patient population. We look forward to working in close collaboration with STADA to pursue marketing authorization with the goal of bringing the first ever EU-approved medication in IgAN to patients as soon as possible, utilizing STADA’s extensive marketing and sales platform throughout Europe," said Renée Aguiar-Lucander, CEO of Calliditas.

"This partnership, which leverages Calliditas’ drug-delivery expertise and clinical data in this under-served patient population, further validates STADA’s position as a go-to-partner for specialty pharmaceuticals, as well as for generics and consumer health products," commented STADA CEO Peter Goldschmidt.

"This value-added novel formulation for a large orphan indication will complement STADA’s offerings in nephrology, where we have built strong expertise over more than a decade through our epoetin zeta biosimilar and where we continue to place a clear strategic focus on seeking further opportunities to bring new options to patients."

The novel formulation is designed to deliver the drug to the Peyer’s patch region of the lower small intestine, where the disease originates as per the predominant pathogenesis models. The formulation uses a unique two-step technology, which allows for the substance to pass through the stomach and intestine without being absorbed, and to be released in a pulse like fashion only when it reaches the ileum in the lower small intestine. In addition to its potent local effect, another advantage of using this active substance is that it has very low bioavailability, with around 90% being inactivated in the liver before it reaches the systemic circulation. This means that a high concentration can be applied locally where needed, whilst limiting systemic exposure.

On May 28, 2021, Calliditas announced that the company had, under the drug-development candidate name Nefecon, submitted a Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) for a novel oral formulation of budesonide targeting down regulation of IgA1 for the treatment of primary IgAN. The company also filed an application for accelerated approval in the US on March 15, 2021 and was granted priority review in April 2021. The commercial brand name for this therapy in Europe will be determined and disclosed at a later date.

Calliditas´ oral formulation has been granted Accelerated Assessment procedure by the Committee for Human Medicinal Products (CHMP) within the European Medicines Agency, which is intended to expedite access to drugs that the CHMP considers to be of major therapeutic interest from the point of view of public health and in particular from the viewpoint of therapeutic innovation. Accelerated assessment reduces the maximum timeframe for review of the MAA to 150 days (excluding clock-stops).

IgAN is designated as an orphan disease in both the US and Europe. In Europe, an orphan disease is defined as a disease or condition affecting no more than five in 10,000 European citizens with no satisfactory method of diagnosis, prevention or treatment. Orphan incentives consist of ten years of market exclusivity from the grant date of marketing approval in the EU, protocol assistance and scientific advice, fee reductions on EMA procedural activities and eligibility for EU grants.

If approved, the product could be available to patients in Europe in the first half of 2022 and would become the first therapy specifically designed and approved for the treatment of IgAN, and which has the potential to be disease modifying.

Torreya acted as exclusive financial advisor to Calliditas on the transaction.

The information in the press release is information that Calliditas is obliged to make public pursuant to the EU Market Abuse Regulation. The information was sent for publication, through the agency of the Calliditas contact person set out above, on July 21, 2021 at 8:45 a.m. CET.