On April 19, 2021 ERYTECH Pharma (Nasdaq & Euronext: ERYP), a clinical-stage biopharmaceutical company developing innovative therapies by encapsulating therapeutic drug substances inside red blood cells, reported the completion of enrollment of the first treatment cohort and the escalation to the next and potentially final dose level in a Phase 1 investigator sponsored clinical trial (IST), named rESPECT, of its lead product candidate eryaspase for the first-line treatment of pancreatic cancer (Press release, ERYtech Pharma, APR 19, 2021, https://erytech.com/erytech-announces-completion-of-first-cohort-in-a-phase-1-investigator-sponsored-trial-of-eryaspase-in-first-line-pancreatic-cancer/ [SID1234578158]).

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On April 18, 2021 Sirnaomics, Inc., a biopharmaceutical company engaged in the discovery and development of RNAi therapeutics against cancer and fibrotic diseases, reported the initiation of a Phase 2 study of the company’s lead drug candidate, STP705, for Keloid scar prevention (Press release, Sirnaomics, APR 18, 2021, View Source [SID1234578167]). The Phase 2, multi-center, randomized, double-blind, multiple-arm, controlled study is designed to evaluate the safety and efficacy of various doses of STP705 in reducing post-keloidectomy keloid recurrence.

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The primary endpoint of this trial is to measure the rate of recurrence in patients who have undergone surgery alone (receiving placebo) versus surgery and STP705 at three months, six months, and 12 months post-surgical excision.

"We anticipate that this important study should render additional data on STP705’s unique mechanism of action on skin fibrotic scarring, following the promising results in previous studies," said Patrick Lu, Ph.D., founder, President and CEO of Sirnaomics. "This approach for Keloid scar prevention is indicative of our strategy to leverage STP705’s dual-targeted inhibitory property and polypeptide nanoparticle-enhanced delivery to develop improved options for treating different cancer indications."

"Keloid scarring is a serious medical condition resulting in abnormal scar formation and pain along with having a very negative psychological impact on patients who suffer from the disease," said Michael Molyneaux M.D., Chief Medical Officer. "There is currently no treatment apart from surgical removal and this carries a very high recurrence rate. We seek to offer patients a viable alternative to prevent recurrence after surgical resection."

Sirnaomics expects to report initial clinical data from the Phase 2 trial in the second half of 2021. Additional information about this clinical trial is available at clinicaltrials.gov using the identifier: NCT04844840.

About Keloid Scar
Keloids form as a result of aberrations of physiologic wound healing and may arise following any insult to the deep dermis. By causing pain, pruritus and contractures, excessive scarring significantly affects the patient’s quality of life, both physically and psychologically. Multiple studies on keloid formation have been conducted for decades and have led to a plethora of therapeutic strategies to prevent or attenuate excessive scar formation. However, most therapeutic approaches remain clinically unsatisfactory, most likely owing to poor understanding of the complex mechanisms underlying the processes of scarring and wound contraction.

Pathophysiology of keloids entails a prolonged inflammatory and proliferative phase of wound healing after injury. Among various cytokines promoting keloids formation, TGF-β1 is known as a key regulator of the aberrant fibrogenic response, while COX-2 acts a potent proinflammatory and proliferative mediator. When STP705 was locally injected into the human hypertrophic scar tissues, which were surgically removed from patients and implanted onto nude mice, the company observed scar size reductions, accompanying with not only target gene silencing but down regulation of fibrogenic markers including α-SMA, hydroxyproline, Collagen 1, and Collagen 3. Further analysis revealed that STP705 is able to induce fibroblast apoptosis both in vitro and in vivo. Therefore, the synergistic effect of simultaneous silencing of TGF-β1 and COX-2 may reverse skin fibrotic scarring through minimizing inflammation and activating fibroblast apoptosis. This mechanism of action of STP705 can be widely applied for treatment of many fibrotic conditions.

About STP705
Sirnaomics’ leading product candidate, STP705, is a siRNA (small interfering RNA) therapeutic that takes advantage of a dual-targeted inhibitory property and polypeptide nanoparticle (PNP)-enhanced delivery to directly knock down both TGF-β1 and COX-2 gene expression. The product candidate has received multiple IND approvals from both the US FDA and Chinese NMPA, including treatments of cholangiocarcinoma, nonmelanoma skin cancer and hypertrophic scar. STP705 has also received Orphan Drug Designation for treatment of cholangiocarcinoma and primary sclerosing cholangitis. A Phase 2a study of STP705 for treatment of squamous cell skin cancer (isSCC) in adult patients demonstrated positive efficacy and safety results, with 76% of all patients (19/25) achieving complete histologically clearance and the two optimal dosing ranges achieving 90% histological clearance of tumor cell in the lesion. No significant or serious adverse events, including no significant cutaneous skin reactions, were reported in the study, and the company was able to define a clear therapeutic window in advance of later stage studies.

On April 18, 2021 Transcenta Holding Limited (Transcenta), a clinical stage global biotherapeutics company with fully-integrated capabilities in discovery, development and manufacturing of antibody-based therapeutics, reported the first patient has been successfully dosed in Phase I clinical study of TST001, a humanized monoclonal antibody targeting human Claudin18.2 (CLDN18.2), in combination with CAPOX for the treatment of patients with first-line locally advanced unresectable or metastatic gastric cancer (Press release, Transcenta, APR 18, 2021, View Source [SID1234578147]).

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TST001 is a second-generation recombinant humanized monoclonal antibody targeted CLDN18.2，generated by Transcenta’s independently-developed Immune Tolerance Breaking Technology (IMTB) platform. TST001 can target and kill tumor cells expressing CLDN18.2 by leveraging mechanisms such as complement-dependent cytotoxicity (CDC) and antibody-dependent cellular cytotoxicity (ADCC) through combining CLDN18.2 with high specificity and affinity. With advanced technology, the fucose content of TST001 was significantly reduced during the production, which further enhancing the ADCC-mediated tumor killing activity of TST001. TST001 demonstrated better antitumor efficacy than IMAB362 analogues in pharmacodynamic experiments in mice (in vivo). TST001 has been launched in clinical research in China and US in July 2020 (NCT04396821, NCT04495296/CTR20201281).

"CLDN18.2 is found to be overexpressed in many tumors including gastric cancer，pancreatic cancer and esophageal cancer. At present, the first-in-class drug IMAB362 has shown promising efficacy in phase II FAST study mainly in first-line gastric cancer patients with high CLDN18.2 expression. "Professor Lin Shen from Beijing Cancer Hospital, believes that "Rapidly advancing the study of the combination of TST001 and standard chemotherapy in first-line gastric cancer patients could help us to accelerate the assessment of its therapeutic value in large number of gastric cancer patients expressing CLDN18.2."

"Gastric cancer is the most common tumor species in China and Asia. About 70% of gastric cancer patients express CLDN18.2. TST001 is a second-generation recombinant humanized monoclonal antibody targeted CLDN18.2. Preclinical studies have found that TST001 can not only efficiently kill gastric cancer cells with high expression of CLDN18.2, but also be very effective against gastric cancer cells with moderate to high expression of CLDN18.2. TST001 has been launched in clinical research in China and US in July 2020. We hope to explore the safe tolerance and anti-tumor activity of TST001 combined with CAPOX in first-line gastric cancer patients through this study, so as to provide a basis for later registrational clinical studies." said Dr. Michael Shi, EVP, Head of Global R&D and CMO of Transcenta.

On April 16, 2021 T-Cure Bioscience, Inc., a privately held company focused on developing T cell receptor (TCR) therapy products for the treatment of solid tumors, reported that management will present virtually at the following upcoming webinar and healthcare conference (Press release, T-Cure Bioscience, APR 16, 2021, View Source [SID1234580486]):

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Beacon Targeted Therapies’ Cell Therapies-Frontiers in Solid Tumor Treatment

Gang Zeng, Chief Executive Officer, will participate the fireside panel discussion on Tuesday, April 20, 2021 at 12:00 p.m. (EDT). Dr. Zeng will cover general topics of adoptive cell therapy, including opportunities to overcome current challenges in treating solid tumors.
TCR-based Therapies Summit

Erika von Euw, Senior Director of Translational Research, will lead a workshop on Tuesday, June 8, at 9:30 a.m. (EDT), titled "Development of Novel TCRs Derived from Responding Patients."
Gang Zeng, will give a presentation on Tuesday, June 8, at 3:00 p.m. (EDT), titled "Leveraging the Insights Gained from TIL Therapy to Improve Solid Tumor Immunotherapy".

On April 16, 2021 AstraZeneca reported that proposed acquisition of Alexion Pharmaceuticals, Inc (Alexion) has achieved an important step toward completion, having cleared US Federal Trade Commission review (Press release, AstraZeneca, APR 16, 2021, View Source [SID1234578146]). This follows the conclusion of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act.

The announcement follows competition clearances in Canada, Brazil, Russia and other countries globally, with a full list available on astrazeneca.com. Additional global regulatory clearances are pending, including but not limited to the UK, EU and Japan.

Marc Dunoyer, Executive Director and Chief Financial Officer, said: "These clearances further advance us towards closing our acquisition of Alexion. We remain focused on the next chapter for AstraZeneca and Alexion, building on our combined expertise in immunology and precision medicines and our shared ambition to bring more innovative medicines to patients worldwide. We look forward to working closely with other global authorities as we progress toward this goal."

The proposed acquisition, first announced in December 2020, would enhance the Company’s scientific presence in immunology by adding Alexion’s innovative complement-technology platforms and strong pipeline. Rare diseases represent a high-growth disease area with rapid innovation and significant unmet medical need. The acquisition remains expected to close in Q3 2021, subject to receipt of additional global regulatory clearances and approval by shareholders of both companies with shareholder voting anticipated on 11 May 2021.

Subject to a successful completion of the acquisition, a dedicated business unit will be created, known as ‘Alexion, The AstraZeneca Rare Disease Unit’, headquartered in Boston, US. AstraZeneca will have an enhanced global footprint and broad coverage across primary, speciality and highly specialised care, and is expected to deliver double-digit revenue growth through 2025, double-digit core EPS accretion for the first three years as well as strong cash flow with an ambition to increase the dividend.

Rare diseases
Over 7,000 rare diseases are known today, and only c.5% have US Food and Drug Administration-approved treatments.1 Demand in the global rare disease space is forecasted to grow by a low double-digit percentage in the future.2

Important additional information
In connection with AstraZeneca’s proposed acquisition of Alexion (the Acquisition), AstraZeneca filed a registration statement on Form F-4 (the Registration Statement), which has been declared effective by the United States Securities and Exchange Commission, and which includes a document that serves as a prospectus of AstraZeneca and a proxy statement of Alexion (the proxy statement/prospectus). Alexion filed the proxy statement/prospectus as a proxy statement and AstraZeneca filed the proxy statement/prospectus as a prospectus with the SEC on 12 April 2021, and each party will file other documents regarding the Acquisition with the SEC. Investors and security holders of Alexion are urged to carefully read the entire Registration Statement and proxy statement/prospectus and other relevant documents filed with the SEC when they become available, because they will contain important information. Investors and security holders may obtain the Registration Statement and the proxy statement/prospectus free of charge from the SEC’s website or from AstraZeneca or Alexion as described in the paragraphs below.

The documents filed by AstraZeneca with the SEC may be obtained free of charge at the SEC’s website at www.sec.gov. These documents may also be obtained free of charge on AstraZeneca’s website at View Source under the tab "Investors". The documents filed by Alexion with the SEC may be obtained free of charge at the SEC’s website at www.sec.gov. These documents may also be obtained free of charge on Alexion’s website at View Source under the tab, "Investors" and under the heading "SEC Filings" or by contacting Alexion’s Investor Relations Department at [email protected].

Participants in the solicitation
AstraZeneca, Alexion and certain of their directors, executive officers and employees may be deemed participants in the solicitation of proxies from Alexion shareholders in connection with the Acquisition. Information regarding the persons who may, under the rules of the SEC, be deemed participants in the solicitation of the shareholders of Alexion in connection with the Acquisition, including a description of their direct or indirect interests, by security holdings or otherwise, is set forth in the proxy statement/prospectus or proxy statement filed with the SEC on 12 April 2021. Information about the directors and executive officers of Alexion and their ownership of Alexion shares is set forth in Alexion’s Annual Report on Form 10-K/A, as previously filed with the SEC on 16 February 2021. Free copies of these documents may be obtained as described in the paragraphs above.

Important notices relating to financial advisors
Evercore Partners International LLP (Evercore), which is authorised and regulated by the FCA in the United Kingdom, is acting exclusively for AstraZeneca and no one else in connection with the Acquisition and the matters referred to in this announcement and will not regard any other person as a client in relation to the matters set out in this announcement (whether or not a recipient of this announcement) and will not be responsible to anyone other than AstraZeneca for providing the protections afforded to its clients, nor for providing advice in relation to the Acquisition or any other matter referred to in this announcement. Neither Evercore nor any of its subsidiaries, holding companies, branches or affiliates owes or accepts any duty, liability or responsibility whatsoever (whether direct or indirect, whether in contract, in tort, under statute or otherwise) to any person who is not a client in connection with the Acquisition or any statement contained in this announcement or otherwise. Apart from the responsibilities and liabilities, if any, which may be imposed on Evercore by the Financial Services and Markets Act 2000 (FSMA), or the regulatory regime established thereunder, or under the regulatory regime of any jurisdiction where exclusion of liability under the relevant regulatory regime would be illegal, void or unenforceable, neither Evercore nor any of its affiliates accepts any responsibility or liability whatsoever for the contents of this announcement, and no representation, express or implied, is made by it, or purported to be made on its behalf, in relation to the contents of this announcement, including their accuracy, fairness, sufficiency, completeness or verification of any statement contained in this announcement or any other statement made or purported to be made by it, or on its behalf, in connection with AstraZeneca or the matters described in announcement, and nothing in this announcement is, or shall be relied upon as, a promise or representation in this respect, whether as to the past or the future. To the fullest extent permitted by applicable law, each of Evercore and its affiliates accordingly disclaim all and any responsibility or liability whether arising in tort, contract or otherwise (save as referred to above) which they might otherwise have in respect of this announcement or any statement contained in this announcement.

Centerview Partners UK LLP (Centerview Partners), which is authorised and regulated by the FCA in the United Kingdom, is acting exclusively for AstraZeneca and no one else in connection with the Acquisition and the matters referred to in this announcement and will not regard any other person as a client in relation to the matters set out in this announcement (whether or not a recipient of this announcement) and will not be responsible to anyone other than AstraZeneca for providing the protections afforded to its clients, nor for providing advice in relation to the Acquisition or any other matter referred to in this announcement. Neither Centerview Partners nor any of its subsidiaries, holding companies, branches or affiliates owes or accepts any duty, liability or responsibility whatsoever (whether direct or indirect, whether in contract, in tort, under statute or otherwise) to any person who is not a client in connection with the Acquisition or any statement contained in this announcement or otherwise. Apart from the responsibilities and liabilities, if any, which may be imposed on Centerview Partners by the FSMA, or the regulatory regime established thereunder, or under the regulatory regime of any jurisdiction where exclusion of liability under the relevant regulatory regime would be illegal, void or unenforceable, neither Centerview Partners nor any of its affiliates accepts any responsibility or liability whatsoever for the contents of this announcement, and no representation, express or implied, is made by it, or purported to be made on its behalf, in relation to the contents of this announcement, including their accuracy, fairness, sufficiency, completeness or verification of any statement contained in this announcement or any other statement made or purported to be made by it, or on its behalf, in connection with AstraZeneca or the matters described in this announcement, and nothing in this announcement is, or shall be relied upon as, a promise or representation in this respect, whether as to the past or the future. To the fullest extent permitted by applicable law, each of Centerview Partners and its affiliates accordingly disclaim all and any responsibility or liability whether arising in tort, contract or otherwise (save as referred to above) which they might otherwise have in respect of this announcement or any statement contained in this announcement.

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