On December 14, 2021 Incyte (Nasdaq:INCY) reported that it will present at the following investor conferences during the month of January (Press release, Incyte, DEC 14, 2021, View Source [SID1234597058]):

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Goldman Sachs 14th Annual Healthcare CEOs Unscripted Conference: A View from the Top (Virtual) on Thursday, January 6, 2022 at 3:00 pm (EST)

40th Annual J. P. Morgan Healthcare Conference on Monday, January 10, 2022 at 7:30 am (PST)
The presentations will be webcast live and can be accessed at Investor.Incyte.com and will be available for replay.

On December 14, 2021 Ascendis Pharma A/S (Nasdaq: ASND) reported to provide an update on two of its investigational endocrinology rare disease development programs, TransCon PTH for hypoparathyroidism, and TransCon CNP for achondroplasia, and on one of its investigational oncology product candidates, TransCon TLR7/8 Agonist at the Company’s virtual R&D Program Update (Press release, Ascendis Pharma, DEC 14, 2021, View Source [SID1234597057]).

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"Today marks an important milestone for Vision 3×3, our strategic roadmap for achieving sustainable growth and building a leading global biopharma company. In addition to providing updates on our Endocrinology Rare Disease pipeline, we are also presenting the first early-stage clinical data from our second therapeutic area, Oncology," said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer. "I am very encouraged that TransCon TLR7/8 Agonist is so far performing as designed, demonstrating early signs of anti-tumor activity with a well-tolerated safety profile as monotherapy or in combination with pembrolizumab."

Ascendis’ management outlined continued clinical advancement on each of the three rare disease development programs. Highlights include:

Update from ongoing TransCon PTH development program:
Reviewed clinical data from ongoing Phase 2 PaTH Forward Trial
Topline results from the Phase 3 PaTHway Trial expected in Q1 2022
Planned NDA submission in Q3 2022 for the treatment of adults with hypoparathyroidism
Topline results from the PaTHway Japan Trial expected in Q3 2022
Planned MAA submission in Q4 2022 for the treatment of adults with hypoparathyroidism
Initiation of the pediatric hypoparathyroidism program planned for Q4 2022

Update from ongoing TransCon CNP development program:

Interim Phase 2 ACcomplisH Trial Update
ACcomplisH enrollment complete (N=57)
Preliminary PK data demonstrated that TransCon CNP provided continuous and dose-dependent exposure with a half-life of ~110 hours mirroring Phase 1 data
Well tolerated in children with achondroplasia with up to 65 weeks follow-up, with mean orthostatic vital signs unchanged
Interim blinded data from the ACcomplisH Trial informed dose selection of 50 and 100 µg/kg/week for the ACcomplisH China Trial
ACcomplisH Infants Trial (0-2 years of age) IND or equivalent submission planned for Q2 2022
ACcomplisH Trial topline unblinded data anticipated in Q4 2022
Interim update from ongoing TransCon TLR7/8 Agonist first-in-human trial (transcendIT-101):
Early signs of clinical activity in three out of three efficacy-evaluable cancer patients treated with TransCon TLR7/8 Agonist as monotherapy or in combination with pembrolizumab
PK data indicate sustained release of active drug with a half-life of ~7 days and low systemic exposure providing a wide safety margin
Consistent and robust immune activation in tumor tissue was observed for at least 7 days post dose
In the safety-evaluable population observed to date (n=8), TransCon TLR7/8 Agonist was well-tolerated with no dose-limiting toxicities or drug-related systemic side effects; the only related adverse events reported were transient, mild injection site-related reactions (Grade 1/2)
transcendIT-101 dose expansion expected to start enrollment in Q2 2022
Virtual R&D Program Update Conference Call & Webcast information

Date Tuesday, December 14, 2021
Time 9:00 a.m. to 11:30 a.m. Eastern Time
Dial In (U.S.) 877-870-9135
Dial In (International) 646-741-3167
Access Code 2169055
A live webcast of the event will be available on the Investors & News section of the Ascendis Pharma website at View Source A webcast replay will be available on the site shortly after conclusion of the event and will stay available for 30 days.

On December 14, 2021 Inceptua Group – pharmaceutical company and service partner – reported that the European Commission (EC) and the UK Medicines and Healthcare products Regulatory Agency (MHRA) has approved the transfer of the marketing authorization for Apealea (paclitaxel micellar) from Oasmia Pharmaceutical AB to Inceptua AB (Press release, Lifescience Newswire, DEC 14, 2021, View Source [SID1234597056]). Apealea is approved in combination with carboplatin for the treatment of adult patients with first relapse of platinum-sensitive epithelial ovarian cancer, primary peritoneal cancer and fallopian tube cancer.

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Inceptua have the exclusive right to distribute and commercialize Apealea in the EU, Norway, Iceland, Liechtenstein, Switzerland and the UK.

Stefan Fraenkel, Chief Executive Officer, Inceptua Group, says:

"We are pleased that the European Commission and MHRA has granted approval for Inceptua to market Apealea, Europe’s first non-Cremophor EL formulation of paclitaxel. With our deep commercialization capabilities and expertise, Inceptua is uniquely positioned to maximize the availability of Apealea for patients with ovarian cancer in this region."

Clive Whitcher, Head of Inceptua Pharma, says:

"With Apealea, ovarian cancer patients now have a treatment option that provides a shorter infusion time without mandatory premedication. We believe there is great potential for Apealea to help patients with ovarian cancer and we look forward to bringing this important treatment to patients in need throughout Europe."

On 25 March 2020, Oasmia Pharmaceutical AB and US based Elevar Therapeutics Inc. signed a global strategic partnership deal to commercialise Apealea. On 28 December 2020, Inceptua signed a licence agreement with Elevar Therapeutics Inc. to commercialise Apealea in Europe.

About Ovarian Cancer

Ovarian cancer is one of the most common female cancers affecting the primary reproductive organs.¹Globally, it is the third most common cancer among women and has the highest mortality rate²,³. Although ovarian cancer has a lower prevalence in comparison with breast cancer, it is three times more lethal, and it is predicted that, by the year 2040, the mortality rate of this cancer will rise significantly⁴,⁵. About half of the women who are diagnosed with ovarian cancer are 63 years or older and many of these patients are predisposed to age-related comorbidities, such as diabetes, which can influence treatment response and prognosis⁶.

About Apealea (paclitaxel micellar)

Apealea is a patented, water-soluble, intravenously injectable, non-Cremophor based formulation of paclitaxel that can be given without premedication such as steroids and with a shorter infusion time. Paclitaxel is a well-known chemotherapy agent used to treat breast, ovarian, lung, bladder, prostate, melanoma, and oesophageal cancer, as well as other types of solid tumour cancers. Cremophor-EL, is a formulation vehicle used for various poorly-water soluble drugs, including the anticancer agent paclitaxel and is associated with allergic reactions. Apealea received marketing authorization by the European Commission in November 2018, making it Europe’s first non-Cremophor-EL formulation of paclitaxel approved for use in ovarian cancer.

On December 14, 2021 Aprea reported that Lead investigators from clinical trials evaluating eprenetapopt in patients with TP53 mutant MDS or AML presented positive, updated data at the 2021 ASH (Free ASH Whitepaper) Annual Meeting (Press release, Aprea, DEC 14, 2021, View Source [SID1234597055]). All studies evaluated the tolerability and efficacy of Aprea Therapeutics, Inc. (Nasdaq: APRE) lead product candidate, eprenetapopt.

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Data summaries of the presentations are as follows:

Title: Phase II Trial of Eprenetapopt (APR-246) in Combination with Azacitidine (AZA) As Maintenance Therapy for TP53 Mutated AML or MDS Following Allogeneic Stem Cell Transplantation (SCT)
Presenter: Asmita Mishra, M.D., H. Lee Moffitt Cancer Center and Research Institute Tampa, Florida
Oral Abstract Session: 723. Allogeneic Transplantation: Long-term Follow-up and Disease Recurrence
Data Summary: In 33 patients enrolled in the trial, the relapse free survival (RFS) at 1 year post-transplant was 60% and the median RFS was 12.5 months. The overall survival (OS) at 1 year post-transplant was 79%, with a median OS of 20.6 months. The post- transplant regimen of eprenetapopt and azacitidine was well tolerated among patients in the clinical trial.

Title: Long-Term Follow-up and Combined Phase 2 Results of Eprenetapopt (APR-246) and Azacitidine (AZA) in Patients with TP53 Mutant Myelodysplastic Syndromes (MDS) and Oligoblastic Acute Myeloid Leukemia (AML)
Presenter: David Sallman, M.D., H. Lee Moffitt Cancer Center and Research Institute, Tampa, Florida
Oral Abstract Session: 637. Myelodysplastic Syndromes—Clinical and Epidemiological: Treatment of High Risk Myelodysplastic Syndrome
Data Summary: By ITT analysis (n=100), ORR was 69% and CR was 43% by International Working Group criteria; Biallelic TP53 mutation or complex karyotype was significantly associated with higher CR rate (49% versus 8%; P=0.01); combination therapy was well tolerated in treated patients.

Title: Phase I and Expansion Study of Eprenetapopt (APR-246) in Combination with Venetoclax (VEN) and Azacitidine (AZA) in TP53-Mutant Acute Myeloid Leukemia (AML)
Presenter: Guillermo Garcia-Manero, M.D., The University of Texas MD Anderson Cancer Center, Houston, Texas
Poster Abstract Session: 616. Acute Myeloid Leukemias: Investigational Therapies, Excluding Transplantation and Cellular Immunotherapies: Poster III
Data Summary: In 39 efficacy evaluable patients ORR was 64%, CR was 39%, and CR + CRi and the CR + CRh rates were each 56%. The triplet combination of eprenetapopt, venetoclax and azacitidine, was tolerable as an outpatient regimen.
Presentations of these data can be accessed from "Presentations" in the News and Events section of the Company’s website at Link.

On December 14, 2021 Ichnos Sciences Inc., a global biotechnology company developing innovative biologics in oncology and autoimmune diseases, and Almirall S.A. (BME: ALM), a global biopharmaceutical company focused on skin health, reported that they have entered into an exclusive licensing agreement for the IL-1RAP antagonist ISB 880 (Press release, Ichnos Sciences, DEC 14, 2021, View Source [SID1234597053]). Under the agreement, Almirall is granted global rights to develop and commercialize this monoclonal antibody for autoimmune diseases. Ichnos will retain rights for antibodies acting on the
IL-1RAP pathway for oncology indications.

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Within the terms of the agreement, Almirall will assume full cost and responsibility for the global development and commercialization of the compound for all autoimmune disease indications. Ichnos will receive an upfront payment of €20.8 million as well as additional development and commercial milestone payments and tiered royalties based upon future global sales.

ISB 880 is a first-in-class fully human, high affinity monoclonal antibody that targets human IL-1RAP. Blockade of IL-1RAP simultaneously abrogates multiple disease drivers among the IL-1 family of proinflammatory cytokine receptors, including IL-1R, IL-33R, and IL-36R, differentiating ISB 880 from single cytokine blockade therapies. These cytokines have been implicated in numerous autoimmune conditions, opening opportunities for ISB 880 to be positioned across broad disease indications. IND-enabling work has been conducted and filings with regulatory authorities are under development.

"This is an exciting step forward for Ichnos as we enhance our focus on oncology and accelerate the pipeline of multispecific antibodies based on our proprietary, BEAT platform," said Cyril Konto, M.D., President and CEO of Ichnos. "ISB 880 anti-IL-1RAP antagonist illustrates Ichnos’ core capabilities in biologics discovery and manufacturing, as well as its strategy to advance a pipeline with potential first- or best-in-class assets. Almirall’s excellent team is well positioned to take ISB 880 through testing and development with the goal of delivering this potential new treatment to patients."

"Given its novel mechanism of action, we believe that ISB 880 has great potential to treat underserved patients across a range of autoimmune dermatological diseases," said Karl Ziegelbauer, Ph.D., CSO of Almirall. "We look forward to beginning to work on ISB 880 and based on the significant data generated by Ichnos we plan to initiate the first-in-human study in the first half of 2022."