Ascentage Pharma Enters into an Agreement with University of Michigan to obtain an exclusive license for a MDM2 Degrader using PROTAC Technology

SUZHOU, China and ROCKVILLE, Md., Nov. 29, 2020 /PRNewswire/ — Ascentage Pharma (6855.HK), a globally focused, clinical-stage biotechnology company engaged in developing novel therapies for cancers, chronic hepatitis B (CHB), and age-related diseases, today announced it has entered into an agreement with the University of Michigan, through which the company may obtain the exclusive global rights to a MDM2 protein degrader developed using the Proteolysis-Targeting Chimeras (PROTACs) technology. The drug candidate is currently entering IND-enabling studies.

MDM2 is a key regulator of the tumor suppressor p53 and one of the most potent inhibitors of apoptosis discovered thus far. It has high expression in tumors and plays a key role in the occurrence and development of tumors. Binding to the MDM2 protein with high affinity, MDM2 inhibitor blocks the MDM2-p53 interactions and restore the tumor-suppressing activity of p53.1 Meanwhile, these MDM2 inhibitors present some challenges, including dose-limiting hematological toxicities, thus the urgent need to develop new generation of MDM2-targeting therapies in the treatment of cancer. The PROTAC technology has emerged as a new and promising approach that induces the degradation of targeted proteins through the ubiquitin-proteasome system (UPS), and it has received widespread interest from both the scientific community and industries since its introduction. Compared to conventional “occupancy-driven” pharmacological modality, the “event-driven” PROTAC technology has many advantages, such as high potency, high selectivity, with catalytic mode of action, and the ability to target undruggable proteins.2

Prof. Shaomeng Wang, Ph.D., C-Founder of Ascentage Pharma and Chairman of its Scientific Advisory Board, and Warner-Lambert/Parke-Davis Professor in Medicine, Professor of Internal Medicine, Pharmacology and Medicinal Chemistry, Director of Michigan Center for Therapeutic Innovation, University of Michigan, is a leading researcher in the field. Through structure-function studies of their previously discovered MDM2 inhibitors using the PROTAC technology, Dr. Wang’s research team has obtained potent and efficacious MDM2 degraders that could effectively induce rapid degradation of MDM2. The lead MDM2 degrader has achieved complete and durable tumor regression in a xenograft tumor model in mice3.

“As a new strategy to induce protein degradation, PROTAC has emerged as a novel modality in drug discovery,” said Dr. Wang. “Studies showed, PROTAC-induced MDM2 degradation can not only enhance the potency of MDM2 inhibitors, but also maintain a long-lasting suppression of MDM2 protein levels, providing a new strategy to the treatment of MDM2-driven tumors such as leukemia. We look forward to the further development of the asset by Ascentage Pharma.”

“The emergence of the PROTAC technology represents another breakthrough in the identification of small molecule drugs. The technology has received tremendous interest for its ability to target undruggable proteins,” said Dr. Dajun Yang, Chairman & CEO of Ascentage Pharma. “We are very pleased to reach this agreement with the University of Michigan, to begin the assessment of this PROTAC-based MDM2 degrader and potentially bring this important addition to our existing pipeline. Through the assessment and potential development of the MDM2 degrader, we hope soon that it will offer an effective therapy

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EU seeks fast-track bypassing of pharma patents in emergencies

By Francesco Guarascio

The EU flag is reflected in a drop on a syringe needle in this illustration

© Reuters/DADO RUVIC
The EU flag is reflected in a drop on a syringe needle in this illustration

BRUSSELS (Reuters) – The European Union wants faster procedures to produce generic versions of drugs without the consent of patent holders, an EU document says, in a move meant to bypass usual intellectual rights protections in exceptional circumstances.


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So-called compulsory licensing is allowed under World Trade Organization (WTO) rules in emergencies as a waiver of normal regulations and could be applied during the COVID-19 pandemic.

“The Commission sees the need to ensure that effective systems for issuing compulsory licences are in place, to be used as a means of last resort and a safety net, when all other efforts to make IP (intellectual property) available have failed,” the document published on Wednesday said.

The measure, if ever applied, would effectively allow EU states to produce generic drugs without the consent of the pharmaceutical companies that developed them and still own the intellectual property rights.

Ironically, the proposal is part of an EU action plan on intellectual property that is mostly aimed at strengthening the protection of EU companies’ patents against foreign actors.

In the first months of the COVID-19 pandemic, lawmakers and activists had urged the EU to use the WTO waiver to gain access to potential vaccines and drugs against the new coronavirus, which have mostly been developed outside the 27-nation bloc.

The EU executive has so far refused to invoke the waiver and has struck multibillion-euro deals with drugmakers to secure COVID-19 shots and therapeutics.

However, the COVID-19 emergency has led to a reconsideration of existing procedures, which largely fall under the remit of national governments in the EU.

“The Commission calls on member states to ensure that the tools they have are as effective as possible; for instance, by putting in place fast-track procedures for issuing compulsory licences in emergency situations,” the document says.

To avoid distortions to trade and innovation, the Commission is also considering creating an emergency co-ordination mechanism that would be triggered at short notice when an EU government wants to issue a compulsory licence.

(Reporting by Francesco Guarascio @fraguarascio; Editing by David Goodman)

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Ascendis Pharma A/S Announces Third Quarter 2020 Financial Results and Business Update Conference Call on November 11

COPENHAGEN, Denmark, Nov. 02, 2020 (GLOBE NEWSWIRE) — Ascendis Pharma A/S (Nasdaq: ASND), a biopharmaceutical company that utilizes its innovative TransCon™ technologies to address unmet medical needs, today announced that the company will hold a conference call and live webcast on Wednesday, November 11, 2020 at 4:30 p.m. Eastern Time (ET) to review its third quarter 2020 financial results and provide a business update.

Conference Call Details


Wednesday, November 11, 2020


4:30 p.m. ET/1:30 p.m. Pacific Time

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A live webcast of the conference call will be available on the Investors and News section of the Ascendis Pharma website at A webcast replay will be available on this website shortly after conclusion of the event for 30 days.

About Ascendis Pharma A/S

Ascendis Pharma is applying its innovative platform technology to build a leading, fully integrated biopharma company focused on making a meaningful difference in patients’ lives. Guided by its core values of patients, science and passion, the company utilizes its TransCon™ technologies to create new and potentially best-in-class therapies.

Ascendis Pharma currently has a pipeline of three independent endocrinology rare disease product candidates in clinical development and is advancing oncology as its second therapeutic area of focus. The company continues to expand into additional therapeutic areas to address unmet patient needs. Ascendis is headquartered in Copenhagen, Denmark, with additional offices in Heidelberg and Berlin, Germany, and in Palo Alto and Redwood City, California.

For more information, please visit

Forward-Looking Statements

This press release contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical facts, included in this press release regarding Ascendis’ future operations, plans and objectives of management are forward-looking statements. Examples of such statements include, but are not limited to, statements relating to (i) Ascendis’ ability to apply its platform technology to build a leading, fully integrated biopharma company, (ii) Ascendis’ product pipeline and expansion into additional therapeutic areas and (iii) Ascendis’ expectations regarding its ability to utilize its TransCon technologies to create new and potentially best-in-class therapies. Ascendis may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions, expectations and projections disclosed in the forward-looking statements. Various important factors could cause actual results or events to differ materially from the forward-looking statements that Ascendis makes, including the following: unforeseen safety or efficacy results in its oncology programs, TransCon hGH, TransCon PTH and TransCon CNP or other development programs; unforeseen expenses related to the development and potential commercialization of its oncology programs, TransCon hGH, TransCon PTH and TransCon CNP or other development programs, selling, general and administrative expenses, other research and development expenses and Ascendis’ business generally; delays in the development of its oncology programs, TransCon hGH, TransCon PTH and TransCon CNP

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Ascendis Pharma A/S Announces Presentations for its Endocrinology Rare Disease Clinical Programs at Upcoming Medical Conferences

COPENHAGEN, Denmark, Oct. 21, 2020 (GLOBE NEWSWIRE) — Ascendis Pharma A/S (Nasdaq: ASND), a biopharmaceutical company that utilizes its innovative TransCon™ technologies to address unmet medical needs, today announced five presentations featuring the company’s endocrinology rare disease programs at two upcoming medical conferences: European Calcified Tissue Society (ECTS) 2020, taking place online October 22–24, 2020, and the Pediatric Endocrinology Nursing Society (PENS) national conference, taking place online November 2–5, 2020.

During ECTS, results from the phase 2 PaTH Forward Trial of TransCon PTH in adult hypoparathyroidism (HP) will be presented. During PENS, data from the phase 3 fliGHt Trial of TransCon hGH (lonapegsomatropin) in pediatric growth hormone deficiency (GHD) and from two clinical trials of the auto-injector for lonapegsomatropin. Additionally, the company will present posters highlighting the impact of achondroplasia (ACH) on the quality of life in children and their parents, which will help inform the TransCon CNP program.

“Ascendis Pharma is committed to supporting the endocrinology community, and we are excited to present data across all three of our endocrinology rare disease programs,” said Aimee Shu, M.D., Senior Medical Director, Clinical Development at Ascendis Pharma. “The data being presented at this year’s ECTS and PENS meetings highlight our portfolio of investigational product candidates and demonstrate important clinical and quality of life outcomes for patients and caregivers, including our first presentation of clinical data on our proprietary auto-injector for lonapegsomatropin.”

Presentation Details


Poster Presentation





Design and Topline Results of TransCon PTH, a Long-acting Parathyroid Hormone (PTH), Phase 2 Trial in Patients with Hypoparathyroidism


Presented on Saturday, October 24, 2020 at 4:55–5:00 p.m. (CST) with live Q&A to follow.


PENS 2020

Poster Presentations





Introduction of a Novel GH Auto-Injector for Once-weekly Administration of TransCon hGH (lonapegsomatropin)


Posters are available during PENS with live Q&A on Thursday, November 5, 2020 from 2:15–2:45 p.m. (ET).


Treatment Experience of Children with Growth Hormone Deficiency in the Phase 3 fliGHt Trial: Switching from Daily Growth Hormone to Once-weekly TransCon hGH (lonapegsomatropin)



Pediatric Achondroplasia: Impacts on Children’s Functioning and Well-being



Experiences of Parents of Children with Achondroplasia: Impacts on Quality of Life



The posters will be available on the Ascendis website under Selected Publications in the Pipeline section:   If you are a healthcare provider who would like more information, please contact: [email protected]

About TransCon™ Technology Platform

TransCon refers to “transient conjugation.” The proprietary TransCon platform is an innovative technology to create new therapies that are designed to potentially optimize therapeutic effect, including efficacy, safety and dosing frequency. TransCon molecules have three components: an unmodified parent drug, an inert carrier that protects it, and a linker that temporarily binds the two. When bound, the carrier inactivates and shields the parent drug from clearance. When injected into the body, physiologic conditions (e.g., pH and temperature) initiate the release of the active, unmodified parent drug in a predictable manner. Because the parent drug is unmodified, its original mode of action is expected to be maintained. TransCon technologies can be

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Vertex Pharma Off After Analysts Comment on Drug-Trial Halt

Vertex Pharmaceuticals  (VRTX) – Get Report shares dropped Thursday after the company said it’s halting development of its VX-814 drug for a lung and liver malady due to liver toxicity, and some analysts reacted negatively.

Vertex shares recently traded at $229, down 16%. They had gained 24% year to date through Wednesday amid enthusiasm for VX-814 and the Boston company’s other drugs.

As for the analysts, SVB Leerink’s Geoffrey Porges lowered his price target to $267 from $283, though he affirmed his rating at outperform.

The VX-814 halt “shows how bare” Vertex’s pipeline is, he wrote in a commentary, according to Bloomberg. 

Vertex doesn’t have nearly the pipeline it needs to sustain a company of its size, Porges said. So he expects acquisitions in the next year.

But Vertex management doesn’t have a good history with acquisitions, and investors won’t tolerate bad deals, Porges said.

Credit Suisse analyst Evan Seigerman wrote in a commentary that the liver-toxicity issue throws into question Vertex’s non-cystic-fibrosis pipeline, Bloomberg reports. But he still rates the stock outperform with a share-price target of $328.

After the VX-814 data, investors are “now concerned about the expansion beyond” cystic fibrosis, the company’s linchpin, Seigerman said. 

As for deal-making, Vertex is unlikely to purchase commercial-stage assets in light of management’s comments, he said.

After Vertex released its earnings in July, Morningstar analyst Anna Baran offered some positive comments. 

“Vertex reported second-quarter results slightly ahead of our expectations, with Trikafta, the triple combination therapy for cystic fibrosis, continuing to perform well and add new patients,” she wrote. Baran put fair value for the stock at $259.

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