On July 13, 2021 Antengene Corporation Limited ("Antengene", SEHK: 6996.HK), a leading innovative biopharmaceutical company dedicated to discovering, developing and commercializing global first-in-class and/or best-in-class therapeutics in hematology and oncology, reported that it has submitted a New Drug Application (NDA) to the Taiwan Food and Drug Administration (TFDA) for selinexor, a first-in-class XPO1 inhibitor, for three indications: in combination with bortezomib and dexamethasone (XVd), or in combination with dexamethasone (Xd) for the treatment of patients with relapsed and/or refractory multiple myeloma (RRMM); and as monotherapy in adult patients with relapsed and/or refractory diffuse large B-cell lymphoma (rrDLBCL), including DLBCL arising from follicular lymphoma, who have received at least two lines of systemic therapy (Press release, Antengene, JUL 13, 2021, View Source [SID1234584826]).

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Antengene has submitted New Drug Applications (NDAs) for selinexor in multiple Asia Pacific markets including China, Australia, South Korea, and Singapore, and was granted Priority Review status by China’s National Medical Products Administration (NMPA) and Orphan Drug Designation by the Ministry of Food and Drug Safety of South Korea (MFDS). This NDA submitted in Taiwan marks another milestone in Antengene’s expansion in the APAC markets and the company’s effort to address the unmet clinical needs of patients with hematologic malignancies.

Dr. Jay Mei, Founder, Chairman and CEO of Antengene, commented: "Within just nine months, we have submitted NDAs in six APAC markets, demonstrating our commitment to addressing the unmet needs of patients in the APAC region. Our seasoned team has a wealth of experience and depth of insight in product registration and commercialization in the Asia Pacific markets. I am confident that Antengene will deliver on our promise to bring our portfolio of innovative therapies to patients in the APAC markets."

"Selinexor is an anti-tumor drug with a highly novel mechanism of action. It has been approved by the FDA for three indications in two tumor types (MM and DLBCL) in less than two years, demonstrating its broad anti-tumor effects," said Kevin Lynch, Chief Medical Officer of Antengene. "Selinexor treatment in combination with dexamethasone still has 25.3% overall response rate (ORR) in patients who have failed five established therapies (penta-refractory) myeloma; and for patients with multiple myeloma who received at least one prior line of treatment, the median progression-free survival (PFS) is 13.9 months, which is significantly higher than that of the control group receiving bortezomib-based standard of care. In the subgroup analysis, patients who are 65 years or older, with renal insufficiency or high-risk cytogenetics can still achieve significant benefits from the XVd regimen. These are patients who are otherwise very difficult to treat. Finally, selinexor monotherapy can enable patients with rrDLBCL to obtain deep and durable responses with a median duration of response of 23.0 months for patients with complete response. We are therefore very optimistic about the potential efficacy benefits of selinexor combination regimens in DLBCL."

Antengene and Karyopharm Therapeutics Inc. (NASDAQ: KPTI) have entered into an exclusive collaboration and license agreement for the development and commercialization of selinexor and other two XPO1 inhibitors, and a PAK4/NAMPT inhibitor in 17 APAC markets including Mainland China.

About Selinexor (XPOVIO)

Selinexor, a first-in-class and only-in-class oral selective inhibitor of nuclear export (SINE) compound discovered and developed by Karyopharm, is currently being developed by Antengene, which has the exclusive development and commercial rights in certain Asia-Pacific markets, including Greater China, South Korea, Australia, New Zealand and the ASEAN countries.

In July 2019, the US Food and Drug Administration (FDA) approved selinexor in combination with low-dose dexamethasone for the treatment of RRMM and in June 2020 approved selinexor as a single-agent for the treatment of rrDLBCL. In December 2020, selinexor also received FDA approval as a combination treatment (XVd) for MM after at least one prior therapy. In February 2021, selinexor was approved by the Israeli Ministry of Health for the treatment of patients with RRMM or rrDLBCL and in March 2021, the European Commission (EC) has granted conditional marketing authorization for selinexor (NEXPOVIO) for the treatment of adult patients with RRMM.

Selinexor is so far the first and only oral SINE compound approved by the FDA and is the first drug approved for the treatment of both MM and DLBCL. Selinexor is also being evaluated in several other mid-and later-phase clinical trials across multiple solid tumor indications, including liposarcoma and endometrial cancer. In November 2020, at the Connective Tissue Oncology Society 2020 Annual Meeting (CTOS 2020), Antengene’s partner, Karyopharm, presented positive results from the Phase III randomized, double blind, placebo controlled, cross-over SEAL trial evaluating single agent, oral selinexor versus matching placebo in patients with liposarcoma. Karyopharm also announced that the ongoing Phase III SIENDO trial of selinexor in patients with endometrial cancer passed the planned interim futility analysis and the Data and Safety Monitoring Board (DSMB) recommended the trial should proceed as planned without any modifications. Top-line SIENDO trial results are expected in the second half of 2021.

Antengene is currently conducting five late-stage clinical trials of selinexor in China for the treatment of MM, DLBCL, non-small cell lung cancer, and peripheral T and NK/T-cell lymphoma.

On July 13, 2021 Prime Medicine, a company delivering on the promise of Prime Editing to provide lifelong cures to patients, reported its launch with $315 million in financing (Press release, Prime Medicine, JUL 13, 2021, View Source [SID1234621403]).

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The financing comprised a $115 million Series A; based on the rapid progress of the science and the company, Prime Medicine expanded its syndicate support with a $200M Series B financing approximately nine months after the company began operations. Investors in the Series A included ARCH Venture Partners, F-Prime Capital, GV, and Newpath Partners. The Series B included all Series A investors, plus Casdin Capital, Cormorant Asset Management, Moore Strategic Ventures, Public Sector Pension Investment Board (PSP Investments), Redmile Group, Samsara BioCapital, funds and accounts advised by T. Rowe Price Associates, Inc., and a number of additional, unnamed life sciences investment funds.

"Prime Editing is a wonderful example of the revolution in genetic medicine that we are living through," said Robert Nelsen, co-founder and Managing Director of ARCH Venture Partners. "When mature, gene editing technologies like this could totally change our conception of what’s possible in treating disease."

"This is an opportunity to take a giant step toward cures for a much wider range of diseases than previously possible," said Stephen Knight, MD, President and Managing Partner of F-Prime Capital.

The funds raised will be used to continue building the company, rapidly advance towards clinical indications, expand the capabilities of the platform, and to further enhance the exceptional promise of Prime Editing. By the end of 2021, Prime Medicine expects to employ more than 100 people full-time.

"Prime Editing represents an opportunity to do what no gene editing approach has yet been capable of – correcting nearly all types of pathogenic gene mutations, correcting multiple mutations at once, and bringing durable cures to patients across multiple disease areas, potentially with a single ‘once and done’ treatment approach," said David Schenkein, MD, General Partner at GV. "We are tremendously excited about the potential of this technology, and about the talented team at Prime working to bring it to patients."

Prime Editing is a next-generation gene editing technology that acts like a DNA word processor to "search and replace" disease-causing genetic sequences at their precise location in the genome, without resulting in double-strand DNA breaks that cause unwanted cellular changes. It is versatile, with the potential to address more than 90 percent of known disease-causing mutations, and works in a variety of dividing and non-dividing primary human cells, as well as in animals. Prime editing has been shown by multiple independent laboratories to make genome edits with high fidelity, making edits precisely at the desired location with minimal or no editing in other parts of the genome. Together, these features overcome several technical barriers attributed to earlier gene editing technologies.

"Prime Editing is a transformative technology that we believe will make a significant impact by addressing the fundamental causes of genetic disease," said Keith Gottesdiener, MD, CEO of Prime Medicine. "Since Prime began operations in the summer of 2020, we have continued to make great progress in advancing the performance of Prime Editing, which allowed us to close our Series B financing nine months later. We are operating from a position of financial strength, and look forward to further developing the technology and progressing our preclinical programs toward the clinic, with the hope that they may cure or halt the progression of genetic diseases for patients."

On July 13, 2021 Moleculin Biotech, Inc., (Nasdaq: MBRX) ("Moleculin" or the "Company"), a clinical stage pharmaceutical company with a broad portfolio of drug candidates targeting highly resistant tumors and viruses, reported that it has received approval from the Bioethics Committee of the Medical University of Karol Marcinkiewicz in Poznań (Ethics Committee) as well as an allowance from the Polish Department of Registration of Medicinal Products (URPL) for a protocol amendment for its Phase 1/2 evaluating Annamycin for the treatment of subjects with acute myeloid leukemia (AML) that is refractory to or relapsed after induction therapy (Press release, Moleculin, JUL 13, 2021, View Source [SID1234584811]).

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Annamycin is the Company’s next-generation anthracycline that has demonstrated a lack of cardiotoxicity in recently conducted human clinical trials for the treatment of AML. Additionally, Annamycin has been shown in animal models to accumulate in the lungs at up to 30-fold the level of doxorubicin. The Company believes that the use of Annamycin may not face the same usage limitations imposed on doxorubicin. Annamycin is currently in development for the treatment of AML and STS lung metastases.

"Based on the preliminary data seen demonstrating clinical benefit for patients receiving a full course of treatment in the 240 mg/m2 cohort and the recommendation from our medical advisors on the dose limiting toxicity criteria, we are pleased to have Ethics Committee approval and the allowance from the URPL to amend the protocol for future patients. This amendment will allow us to continue dose escalation in the Phase 1 portion of the trial and establish the maximum tolerated dose as we work toward the recommended dose for the Phase 2 portion of the study. Our team is committed to advancing this important clinical program forward and to potentially address the limitations with current treatment options for AML patients," commented Walter Klemp, Chairman and CEO of Moleculin.

The Phase 1/2 AML trial in Poland remains ongoing and is currently dosing patients at 240 mg/m2. Under the previous protocol transient elevated liver enzymes (AST and ALT) observed in two patients were considered a dose limiting toxicity (DLT), which investigators believe would inappropriately limit the potential for continued dose escalation. The amendment to the Annamycin clinical trial protocol allows for a change in the DLT criteria as it relates to transient grade 3 elevations and allows dosing of three additional patients in the 240 mg/m2 cohort. If no DLT (as defined by the new criteria) is experienced with these next three patients, the Company plans to escalate dosing in new cohorts by 30 mg/m2 instead of the 60 mg/m2 previously planned, and with a de-escalation of 15 mg/m2 at the DLT dose if future patients experience a DLT.

The results from the Phase 1 portion of the Company’s U.S. Phase 1/2 clinical trial of Annamycin for the treatment of AML met its primary endpoint and demonstrated a clean safety profile with no evidence of cardiotoxicity when delivered to patients at or below the lifetime maximum anthracycline dose established by the FDA. To date, an independent expert assessment of the absence of cardiotoxicity in the first 19 patients treated with Annamycin in both the Company’s U.S. and European Phase 1 clinical trials in which an independent expert concluded that he "does not see evidence of cardiotoxicity."

Moleculin Biotech expects to continue reporting cohort topline results from the ongoing Phase 1/2 study for treatment of AML and to report the study’s topline results in the second half of 2022. Annamycin has been granted Fast Track Status and Orphan Drug Designation from the U.S. Food and Drug Administration for the treatment of AML.

On July 13, 2021 Jazz Pharmaceuticals plc (Nasdaq: JAZZ) reported that the company will host a webcast on Tuesday, July 20, 2021 at 4:30 p.m. ET / 9:30 p.m. IST to provide an update on Rylaze (asparaginase erwinia chrysanthemi (recombinant)-rywn), which was approved by the U.S. Food and Drug Administration (FDA) on June 30, 2021 (Press release, Jazz Pharmaceuticals, JUL 13, 2021, View Source [SID1234584827]).

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Jazz senior management will be joined by Dr. Luke Maese, associate professor of pediatrics, University of Utah – Huntsman Cancer Institute, Primary Children’s Hospital, to discuss the Rylaze FDA approval, commercial launch and an overview of acute lymphoblastic leukemia (ALL) and lymphoblastic lymphoma (LBL) and the need for recombinant, non-E. coli derived asparaginase treatments.

About Rylaze (asparaginase erwinia chrysanthemi (recombinant)-rywn)
Rylaze, also known as JZP458, is approved in the U.S. for use as a component of a multi-agent chemotherapeutic regimen for the treatment of acute lymphoblastic leukemia (ALL) or lymphoblastic lymphoma (LBL) in pediatric and adult patients one month and older who have developed hypersensitivity to E. coli-derived asparaginase. Rylaze has orphan drug designation for the treatment of ALL/LBL in the United States. Rylaze is a recombinant erwinia asparaginase that uses a novel Pseudomonas fluorescens expression platform. JZP458 was granted Fast Track designation by the U.S. Food and Drug Administration (FDA) in October 2019 for the treatment of this patient population. Rylaze was approved as part of the Real-Time Oncology Review program, an initiative of the FDA’s Oncology Center of Excellence designed for efficient delivery of safe and effective cancer treatments to patients.

The full U.S. Prescribing Information for Rylaze is available at: <View Source>

Important Safety Information

RYLAZE should not be given to people who have had:

Serious allergic reactions to RYLAZE
Serious swelling of the pancreas (stomach pain), serious blood clots, or serious bleeding during previous asparaginase treatment
RYLAZE may cause serious side effects, including:

Allergic reactions (a feeling of tightness in your throat, unusual swelling/redness in your throat and/or tongue, or trouble breathing), some of which may be life-threatening
Swelling of the pancreas (stomach pain)
Blood clots (may have a headache or pain in leg, arm, or chest)
Bleeding
Liver problems
Contact your doctor immediately if any of these side effects occur.

Some of the most common side effects with RYLAZE include: liver problems, nausea, bone and muscle pain, tiredness, infection, headache, fever, allergic reactions, fever with low white blood cell count, decreased appetite, mouth swelling (sometimes with sores), bleeding, and too much sugar in the blood.

RYLAZE can harm your unborn baby. Inform your doctor if you are pregnant, planning to become pregnant, or nursing. Females of reproductive potential should use effective contraception (other than oral contraceptives) during treatment and for 3 months following the final dose. Do not breastfeed while receiving RYLAZE and for 1 week after the final dose.

On July 12, 2021 Vaccibody AS (Euronext Growth (Oslo): VACC), a clinical-stage biopharmaceutical company dedicated to the discovery and development of vaccines and novel immunotherapies, reported that it has entered into an exclusive license agreement with Adaptive Biotechnologies Corporation (Nasdaq: ADPT), a commercial stage biotechnology company that aims to translate the genetics of the adaptive immune system into clinical products to diagnose and treat disease, to use a broad selection of virus-specific T cell epitopes identified by Adaptive for Vaccibody to design and develop novel SARS-CoV-2 vaccines (Press release, Vaccibody, JUL 12, 2021, View Source [SID1234584775]).

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Vaccibody’s development strategy for its second-generation SARS-CoV-2 vaccine is designed to respond to the emerging threats of evolving variants with reduced sensitivity to first generation vaccines that were developed using the 2020 prototype spike protein. The 2-armed strategy aims to develop two candidates for the broad population. First, a vaccine candidate encoding the receptor binding domain (RBD) derived from the South African beta variant of concern. And second, a T cell based vaccine candidate, encoding multiple validated immunodominant, conserved Adaptive-identified T-cell epitopes spanning multiple antigens across the SARS-CoV-2 genome. 2

"To date, SARS-CoV-2 has resulted in the death of over 4 million people globally and we are facing a tremendous threat from emerging variants of concern. We are very excited to have access to Adaptive’s T cell epitopes for our use in developing second-generation SARS-CoV-2 vaccines to specifically address current and future variants of concern. Vaccibody has exclusively licensed validated SARS-CoV-2 T-cell epitopes for use in the design and development of our T-cell vaccine candidates, including the candidate in our previously announced clinical trial" said Agnete B. Fredriksen, Chief Innovation and Strategy Officer of Vaccibody.

Adaptive has mapped the T cell immune response using more than 6,500 samples from patients impacted by COVID-19. Adaptive used its immune medicine platform, leveraging its proprietary antigen mapping and deep sequencing capabilities, to identify naturally processed and presented T-cell epitopes to SARS-CoV-2 antigens. Adaptive’s T-cell epitopes will be used by Vaccibody in its modular vaccine technology platform to target specific SARS-CoV-2 antigens to antigen presenting cells.

Michael Engsig, Chief Executive Officer of Vaccibody continued, "Our aim is to design and develop novel second-generation COVID-19 vaccines using Vaccibody’s unique modular vaccine technology platform and Adaptive’s functionally validated, immunodominant T-cell epitopes."

"The SARS-COV-2 virus’ ability to rapidly mutate can impact the efficacy of many firstgeneration vaccines. Adaptive’s unique ability to read and access the immune system enables us to identify and validate SARS-COV-2 T-cell epitopes from convalescent COVID-19 individuals. We are excited to combine the strength of validated T-cell epitopes, identified using our immune medicine platform, with Vaccibody’s innovative vaccine technology in fighting the pandemic," added Harlan Robins, Chief Scientific Officer and co-founder of Adaptive Biotechnologies.

Mikkel W. Pedersen, Ph.D., Chief Scientific Officer of Vaccibody continued, "We are thrilled to work with Adaptive Biotechnologies to accurately identify immunogenic and conserved T-cell epitopes. Adaptive’s epitopes have enabled us to create a multivalent SARS-CoV-2 T-cell vaccine that may provide more complete viral protection, long-term immunity and viral clearance compared to first generation vaccines. Our T-cell candidate may have both prophylactic and therapeutic potential and may also fit the profile of a universal SARS-CoV-2 vaccine booster for individuals previously vaccinated with Spike based vaccines."

Vaccibody has demonstrated that the SARS-CoV-2 vaccine candidate that incorporates Adaptive’s T-cell epitopes induces a rapid, strong and broad T-cell response after administration of a single dose in a humanized preclinical model.

Under the terms of the license agreement, Adaptive has provided certain selected T-cell epitopesfor exclusive use in Vaccibody’s next-generation SARS-CoV-2 vaccines. Vaccibody will 3 be responsible for further development of the potential T-cell SARS-CoV-2 vaccine candidates. Financial terms of this agreement will not be disclosed.

The phase 1/2 trial is currently in the planning phase. The CTA (Clinical Trial Application) is expected to be submitted in Q3 2021 and initiation is planned for Q4 2021. The clinical trial will be conducted in Norway. Please also referto Vaccibody’s announcement on June 29, 2021 about the clinical trial.

Webcast
Michael Engsig, Chief Executive Officer of Vaccibody, and other members of Vaccibody’s management team will host a webcast on July 12, 2021 at 4 p.m. CET / 10 a.m. EDT. Harlan Robins, Chief Scientific Officer of Adaptive Biotechnologies, will also join them to discuss how Adaptive identified the T cell epitopes that Vaccibody will use in its second generation COVID19 vaccine using its proprietary immune medicine platform. A presentation will be available on Vaccibody’s website, www.vaccibody.com/financial-reports-and-presentations, before the webcast.