On September 28, 2020 Junshi Biosciences (HKEX: 1877; SSE: 688180), a leading innovation-driven biopharmaceutical company dedicated to the discovery, development and commercialization of novel therapies, announced today that an Independent Monitoring Committee (IDMC) determined that the randomized, double-blind, placebo-controlled, international multi-center Phase III study JUPITER-02 at the interim analysis had met its pre-specified primary endpoint (Press release, Shanghai Junshi Bioscience, SEP 29, 2020, View Source [SID1234567842]). The results of the study showed that Toripalimab in combination with Gemcitabine/Cisplatin as a first-line treatment for patients with recurrent or metastatic nasopharyngeal carcinoma (NPC) significantly extended the progression-free survival than the current standard treatment of Gemcitabine/Cisplatin. The Company will submit biologic license application (BLA) to the National Medical Products Administration (the "NMPA") and regulatory authorities in other relevant countries in the near future.

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About NPC
Nasopharyngeal carcinoma (NPC), a subtype of head and neck cancer, is a malignant tumor that occurs in the nasopharynx. According to statistics from the World Health Organization, NPC is widely distributed in Southeast Asia and the incidence in the region accounts for approximately 84.6% of the new cases worldwide. NPC is prone to distant metastasis at diagnosis, while early stage patients tend to relapse after radiation or radio-chemotherapy. Platinum-based therapy is currently standard care for recurrent or metastatic NPC. In addition, there is no standard treatment after failure of the first-line systemic chemotherapy. The 5-year overall survival rate of patients with advanced disease is less than 10%.

ABOUT JUPITER-02 STUDY
The JUPITER-02 Study (NCT03581786) is a randomized, double-blind, placebo-controlled, international multi-center Phase III clinical study to compare the efficacy and safety of Toripalimab versus placebo in combination with Gemcitabine/Cisplatin, as a first-line treatment for patients with recurrent or metastatic nasopharyngeal carcinoma. Professor Xu Ruihua from Sun Yat-sen University Cancer Centre is the leading principal investigator. The JUPITER-02 Study enrolled a total of 289 patients. The primary endpoint of the study is progression-free survival (PFS), and the secondary endpoints include overall survival (OS), objective response rate (ORR), duration of response (DOR), disease control rate (DCR) and safety.

At the interim analysis, the Independent Data Monitoring Committee (IDMC) determined that the global study met its primary endpoint of progression free survival (PFS). Compared with the placebo control, Toripalimab in combination with Gemcitabine/Cisplatin significantly prolonged the PFS as a first-line treatment for patients with recurrent or metastatic nasopharyngeal carcinoma.

About Toripalimab
Toripalimab is a PD-1 monoclonal antibody developed by Junshi Biosciences. Toripalimab received its first approval for 2nd line treatment of metastatic melanoma on December 17, 2018 in China and was commercially launched in February 2019. More than 30 clinical studies covering more than ten cancer indications have been conducted in China, the United States and other countries.

In April 2020, the supplemental New Drug Application ("NDA") of Toripalimab for the treatment of recurrent/metastatic nasopharyngeal carcinoma after failure of at least two prior systemic treatments has been accepted by the NMPA. This supplemental NDA is the world’s first NDA of PD-1 blockade therapy for the treatment of nasopharyngeal carcinoma. In May 2020, the supplemental NDA of Toripalimab for the treatment of locally advanced or metastatic urothelial carcinoma after systemic treatment has been accepted by the NMPA. Both supplemental NDAs received priority review status by the NMPA in July 2020.

In March 2020, Toripalimab in combination with axitinib for the treatment of mucosal melanoma was granted the orphan-drug designation by the US FDA. In May and September 2020, Toripalimab also received the orphan-drug designation by the FDA for the treatment of nasopharyngeal carcinoma and soft tissue sarcoma. In September 2020, Toripalimab for the treatment of nasopharyngeal carcinoma was granted the Breakthrough Therapy designation by the FDA.

On September 28, 2020 Carmine Therapeutics reported that it has been named by FierceBiotech as one of 2020’s Fierce 15 biotechnology companies, designating it as one of the most promising private biotechnology companies in the industry (Press release, Carmine Therapeutics, SEP 28, 2020, View Source [SID1234567822]).

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The 2020 Fierce 15 comes at a time when the world is focused on a pandemic, but even as coronavirus remains a threat, patients with cancer, rare diseases and other disorders still need treatment, FierceBiotech Senior Editor Ben Adams said. "This year, we’ve chosen from a diverse range of those fighting COVID, as well as those fighting longer term plagues against our biology," Adams said.

Carmine Therapeutics is pioneering a novel class of therapeutics based on its REGENT technology – an innovative approach which leverages red blood cell extracellular vesicles (RBCEVs). First published in Nature Communications in 2018, an initial focus is non-viral gene therapies.

In June 2020, Carmine Therapeutics signed a research collaboration agreement with Takeda Pharmaceutical Company Limited to discover, develop and commercialize transformative non-viral gene therapies. Carmine Therapeutics will be eligible for over $900M USD in total milestone payments plus tiered royalties.

XQ Lin, Founding CEO of Carmine Therapeutics and Managing Partner of Esco Ventures X, commented, "We are thrilled to be recognized as a Fierce 15 company, an honor that reflects the innovative nature of our RBCEV platform and the dedication of our team. This comes following a year of successes and we are excited to build on this momentum. We believe that our novel approach will have a tremendous impact on transforming patient lives."

The Fierce 15 celebrates the spirit of being "fierce" – championing innovation and creativity, even in the face of intense competition. This is FierceBiotech’s 18th annual Fierce 15 selection.

An internationally recognized daily report reaching a network of over 285,000 biotech and pharma industry professionals, FierceBiotech provides subscribers with an authoritative analysis of the day’s top stories. Every year FierceBiotech evaluates hundreds of private companies from around the world for its annual Fierce 15 list, which is based on a variety of factors such as the strength of its technology, partnerships, venture backers and a competitive market position.

On September 28, 2020 CureVac reported that Its focus is squarely on getting an mRNA vaccine against COVID-19 onto the market, but has bigger ambitions from its deep pipeline (Press release, CureVac, SEP 28, 2020, View Source [SID1234567807])

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CEO: Franz-Werner Haas
Based: Tübingen, Germany
Founded: 2000
Clinical focus: Prophylactic vaccines, cancer immunotherapies and protein therapies

The scoop: CureVac, alongside BioNTech and Moderna, is one of the biotechs that put mRNA on the map. It suffered the field’s first big clinical setback when prostate cancer vaccine CV9104 flunked a phase 2b in 2017, only to bounce back through the progression of other programs and a significant infectious disease pact with Eli Lilly.

Then COVID-19 happened. As the coronavirus spread, CureVac received €300 million ($356 million) from the German government, struck a broad, big-ticket deal with GlaxoSmithKline, completed a Nasdaq IPO and briefly became a mainstream news story when President Donald Trump reportedly tried to buy it.

What makes CureVac Fierce: The biotech that emerged from the early months of the pandemic has a new CEO, Franz-Werner Haas, a new near-term focus, COVID-19, and, by extension, a radically revised outlook and strategy for the next year or two.

"[COVID-19] is really transforming the company," Haas said.

CureVac went into 2020 thinking it was still years away from bringing a product to market, meaning its focus was on running clinical trials and manufacturing materials for use in them. Now, CureVac is planning to bring a COVID-19 vaccine to market next year and lining up a contract to sell up to 405 million doses to the European Union.

The rapid acceleration of CureVac’s time to market has changed the focus of the leadership team. Today, Haas is occupied with questions about how to establish the expertise and scale CureVac will need to succeed as a commercial company. In January, those questions were distant concerns.

CureVac has a little more time to address the questions than some other developers of COVID-19 vaccine. While its peers were racing into the clinic, CureVac spent more time in preclinical doing the optimization work that it thinks will enable its vaccine to trigger balanced immune responses and be readily manufactured at scale. CureVac took a different approach to other mRNA vaccine developers.

"These other candidates, they’re building upon chemical modification of the RNA, which we don’t do. We are optimizing the RNA on the RNA level, so using the potency of the untranslated regions. That’s a differentiating factor," Haas said. One consequence of CureVac’s approach is the maximum dose in its first clinical trial is 8 µg. Moderna is testing a 100-µg dose in its phase 3.

The relatively small amount of mRNA used in its vaccine should enable CureVac to get more doses out of the manufacturing capacity it is adding. Infrastructure set up to support the COVID-19 vaccine could prove valuable as other assets get to market. CureVac’s rabies vaccine could come to market within the next five years. Perhaps more significantly, CureVac is retaining the commercialization rights in three European countries to the five candidates covered by its deal with GSK.

"These are big products, which need huge capacity in terms of manufacturing. And so there will be a kind of decision point: Where are these five projects? How can we bring these into the market? This is a transforming platform deal for us," Haas said.

Haas sees vaccines as the "sweet spot" for CureVac’s technology today as they are given at low doses in two-shot prime-boost regimens, rather than the repeated administration needed to treat disease. That is reflected in CureVac’s pipeline, which is led by vaccines against COVID-19 and rabies, but the biotech’s ambitions, like those of its mRNA rivals, extend well beyond prophylactics.

CureVac is testing its tumor microenvironment modulator CV8102 in cancer patients in combination with anti-PD-1 antibodies, and the Boehringer Ingelheim-partnered self-adjuvanting mRNA vaccine CV9202 is in early-phase development in non-small cell lung cancer. A third wing of the biotech is working on protein-based therapies, including in programs that pair its technology with the potentially complementary capabilities of CRISPR Therapeutics and Genmab.

Investors: Dievini Hopp BioTech holding, Kreditanstalt für Wiederaufbau and GlaxoSmithKline

On September 28, 2020 Medigene AG (FSE: MDG1, Prime Standard) reported its participation at the following upcoming scientific and investor conferences (Press release, MediGene, SEP 28, 2020, View Source [SID1234567736]). Please note that all mentioned conferences will be conducted virtually due to COVID-19.

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Cell Therapy Manufacturing & Gene Therapy Congress
Date: 19 – 22 October
Website: https://informaconnect.com/celltherapy/

TCR Therapies Summit
Date: 26 – 29 October
Website: https://tcr-therapies-summit.com/

BIO-Europe Digital
Date: 26 – 29 October
Website: https://informaconnect.com/bioeurope/?gclid=EAIaIQobChMIn-75yv7b6wIVFed3Ch27uAezEAAYASAAEgLuufD_BwE

Neoantigen Based Therapies Summit
Date: 3 – 5 November
Website: View Source

35th Anniversary Annual Meeting & Pre-Conference Programs (SITC 2020)
Date: 9 – 14 November
Website: View Source;Home=%2Fevents%2Fevent-description

62nd ASH (Free ASH Whitepaper) Annual Meeting and Exposition
Date: 5 – 8 December
Website: View Source

On September 28, 2020 xCures, a clinical study platform provider, reported the US Food and Drug Administration (FDA) granted their IND for an intermediate Expanded Access Program (EAP) for the ERK inhibitor ulixertinib (BVD-523) (Press release, xCures, SEP 28, 2020, View Source [SID1234567705]).

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Ulixertinib is being developed by BioMed Valley Discoveries (BVD), a clinical stage biotechnology company, as a treatment for patients with MAPK pathway aberrant cancer, including but not limited to KRAS, NRAS, HRAS, BRAF, MEK, and ERK, mutations.

The EAP is open across the United States to adolescent and adult cancer patients who cannot access an open clinical trial for the investigation of ulixertinib (BVD-523).

"xCures prospective real-world evidence generation capability transforms managed access programs such as the ulixertinib expanded access program by making them an efficient way for physicians and patients to gain access to promising therapies when clinical trials are not an option," says Mika Newton, CEO of xCures, Inc. "xCures’ programs uniquely capture high-value evidence related to the safety and efficacy from this expanded set of patients."

This intermediate-sized expanded access program (NCT04566393) is currently open and available for physicians interested in treating their patients. Physicians can reach out to [email protected] for more information. Patients can register and find more information at enroll.xcures.com/uli-eap or receive additional information via xCures patient advocacy partner Cancer Commons (www.cancercommons.org).

About ulixertinib (BVD-523):
Ulixertinib is a first-in-class and best-in-class small-molecule inhibitor of extracellular signal-regulated kinase (ERK) family kinases (ERK1 and ERK2) that is being developed as a novel anti-cancer drug. ERK kinases are downstream components of the mitogen-activated protein kinase (MAPK) signaling cascade (RAS-RAF-MEK-ERK). Ulixertinib has demonstrated promising early efficacy for patients with tumors harboring alterations in the MAPK pathway, including atypical (non-V600) BRAF alterations, for which there are currently no approved targeted agents.

About Expanded Access:
Expanded access, which is often called "compassionate use," is the use of an unapproved drug for treatment of patients with serious or life-threatening illnesses outside of a clinical trial. Expanded access is subject to oversight from the US FDA in accordance with the regulations outlined in 21 CFR 312.305.

About the Program:
This Expanded Access program provides ulixertinib for compassionate use in advanced cancer patients with a MAPK pathway-altered solid tumor(s) who have exhausted available therapies. The protocol aims to collect sufficient information about the patient’s treatment to provide a complete and accurate case report to health authorities using real-world data collection to assess response to treatment, safety, tolerability, and quality-of-life.