On January 26, 2021 BridgeBio Pharma, Inc. ("BridgeBio") (Nasdaq: BBIO) reported that it has completed its acquisition of all of the outstanding shares of Eidos Therapeutics, Inc. ("Eidos") (formerly Nasdaq: EIDX) common stock that BridgeBio did not already own (Press release, BridgeBio, JAN 26, 2021, View Source [SID1234576240]). The transaction was overwhelmingly approved by BridgeBio and Eidos stockholders.

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The merger reunites the teams at BridgeBio and Eidos and allows BridgeBio to deploy its full clinical and commercial infrastructure to support the development and global commercialization plans underway for Eidos’ acoramidis, a potential best-in-class therapy for patients with transthyretin (TTR) amyloidosis (ATTR). BridgeBio’s mission is to discover, create, test and deliver transformative medicines to treat patients who suffer from genetic diseases and cancers with clear genetic drivers.

"2021 is an important year for BridgeBio and the patients we serve," said Neil Kumar, Ph.D., founder and CEO of BridgeBio. "With significant near-term pivotal and proof-of-concept data anticipated in our four core programs, including acoramidis, we are eager to accelerate our critical work for patients as a single unified company."

Acoramidis for ATTR is one of BridgeBio’s four core value driver programs, along with encaleret (CaSR inhibitor) for autosomal dominant hypocalcemia type 1 (ADH1), low-dose infigratinib (FGFR inhibitor) for achondroplasia, and BBP-631, an AAV5 gene therapy for congenital adrenal hyperplasia (CAH). 2021 is poised to be a transformational year for BridgeBio with major catalysts in all four programs anticipated in 2021 or the first quarter of 2022. This year BridgeBio also expects to launch two drugs, if approved, and is building its global commercial capabilities.

Acoramidis (AG10) – TTR stabilizer for ATTR: Topline results from Part A of the ATTRibute-CM trial are expected in late 2021 or early 2022 and from Part B in 2023. If Part A is successful, BridgeBio expects to file for regulatory approval of acoramidis in 2022. ATTR is a form of amyloidosis caused by the accumulation of misfolded TTR protein. It is estimated to affect more than 400,000 people worldwide and is largely undiagnosed today.

Encaleret – calcium-sensing receptor (CaSR) inhibitor for ADH1: Phase 2 proof-of-concept results are anticipated in the third quarter of 2021. If the development program is successful, encaleret would be the first approved therapy for ADH1, a condition caused by gain of function variants in the CASR gene estimated to be carried by 12,000-13,000 individuals in the United States alone.

Low-dose infigratinib – FGFR1-3 inhibitor for achondroplasia: Initial data from the ongoing Phase 2 dose ranging study are expected in the fourth quarter of 2021. Achondroplasia is the most common form of genetic short stature and one of the most common genetic diseases, with 55,000 cases in the United States and European Union. Low-dose infigratinib is the only known therapy in development for achondroplasia that targets the disease at its genetic source and the only orally administered product candidate in clinical stage development.

BBP-631 – AAV5 gene therapy candidate for CAH: Initiation of a first-in-human Phase 1/2 study is expected in 2021, with initial data anticipated in the fourth quarter of 2021 or the first quarter of 2022. CAH is one of the most prevalent genetic diseases potentially addressable with AAV gene therapy, with more than 75,000 cases in the United States and European Union. The disease is caused by deleterious mutations in the gene encoding an enzyme called 21-hydroxylase, leading to lack of endogenous cortisol production. BridgeBio’s AAV gene therapy candidate is designed to provide a functional copy of the 21-hydroxylase-encoding gene (CYP21A2) and potentially address many aspects of the disease course.
As a result of the merger, former Eidos stockholders are entitled to receive, for each share of Eidos common stock issued and outstanding immediately prior to the effective time of the merger that was not owned by BridgeBio or any of its subsidiaries and that was not a restricted share award, either (i) 1.85 shares of BridgeBio common stock or (ii) if an election to receive cash was properly made prior to 5:00 P.M., New York City time, on January 21, 2021, $73.26 in cash. Eidos stockholders should contact American Stock Transfer & Trust Company, LLC, the exchange agent for the transaction, by calling toll-free at (877) 248-6417 or at (718) 921-8317, if they have any questions regarding the consideration to which they are entitled.

On January 26, 2021 BriaCell Therapeutics Corp. ("BriaCell" or the "Company") (TSX-V:BCT) (OTCQB:BCTXF), a clinical-stage biotechnology company specializing in targeted immunotherapies for advanced breast cancer, reported the presentation of results from clinical studies with its lead product candidate, Bria-IMT, summarized in a poster session held January 25-27 during the 2021 Keystone Symposium, "Emerging Cell Therapies: Realizing the Vision of NextGen Cell Therapeutics," a virtual scientific conference (Press release, BriaCell Therapeutics, JAN 26, 2021, View Source [SID1234574784]).

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Overall, the data suggests a potentially significant survival benefit for the patients treated with the Bria-IMT regimen alone or in combination with checkpoint inhibitors, especially in those with moderately or well differentiated tumors or those who match Bria-IMT at 2 HLA alleles. We hypothesize that Bria-IMT, with a molecular signature most closely related to moderately or well differentiated tumors, may result in disease control especially in patients with moderately-well differentiated tumors. The patient data summarized and discussed belong to previously-disclosed patients (i.e., no incremental numbers enrolled).

Dr. Bill Williams, BriaCell’s President & CEO, presented the clinical and pathological data of Bria-IMT monotherapy (i.e., the Bria-IMT regimen alone) and Bria-IMT in combination with immune checkpoint inhibitors including pembrolizumab (KEYTRUDA; manufactured by Merck & Co., Inc.), and more recently, Incyte’s INCMGA00012 (under a corporate collaboration with Incyte Corporation), in advanced breast cancer.

Details and results on the poster presentation are summarized below:

Poster Title: Personalized off-the-shelf whole cell immunotherapy for cancer

Monotherapy Study – 27 patients: The patients were heavily pre-treated (median of 5 prior therapy regimens). Disease control including stable disease (SD), partial responses (PR) or complete responses (CR) was seen in 8 patients (30%) suggesting clinical benefit. Importantly, disease control was more frequent in patients who had 2 or more HLA matches with Bria-IMT (67%) or had moderately or well differentiated tumors (63%) suggesting the potential importance of these factors in designing the next generation of personalized immunotherapies.

Combination Study – 12 patients (Bria-IMT regimen with checkpoint inhibitors): The patients were heavily pre-treated (median of 6 prior therapy regimens). Disease control was seen in 4 patients (33%). Importantly, patients with moderately or well differentiated tumors were more likely to achieve disease control (3/4, 75%) suggesting clinical benefit in this subset of patients. Furthermore, the data suggests the potential additive or synergistic effects of check point inhibitors when combined with the Bria-IMT regimen.

Patients with moderately or well differentiated tumors: The patients with moderately or well differentiated tumors (monotherapy and combination therapy studies combined) were heavily pre-treated (median of 8 prior therapy regimens). Disease control was seen in 7 of 11 patients (64%) suggesting clinical benefit in this subset of patients. This included 2 of 5 patients with objective responses, both of whom had 2 or more HLA matches with Bria-IMT. Overall survival (OS), collected in 6 patients, was 12.5 months suggesting clinical benefit. In comparison, an OS of 7.2-9.8 months was reported in similar patients in the third line setting (Kazmi S, et al., Breast Cancer Res Treat. 2020).

Capitalizing on these findings, BriaCell has engineered cell lines to express specific HLA alleles allowing a single HLA match with ~99% and a double HLA match with ~90% of the population. These cell lines will provide a personalized approach to cancer immunotherapy that is pre-manufactured rather than prepared individually per patient, eliminating the time and complex manufacturing logistics of other personalized immunotherapies.

A copy of the poster will be posted at the following: View Source

On January 26, 2021 Translational Genomics Research Institute (TGen), an affiliate of City of Hope reported that Breast cancer, even at its initial stages, could be detected earlier and more accurately than current techniques using blood samples and a unique proteomics-based technology (Press release, TGen, JAN 26, 2021, View Source [SID1234574442])

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Patrick Pirrotte, Ph.D., an Assistant Professor and Director of TGen’s Collaborative Center for Translational Mass Spectrometry, and an international team of researchers developed a test that can detect infinitesimally small breast cancer biomarkers that are shed into the bloodstream from cells surrounding cancer known as extracellular matrix (ECM), according to the findings of their study recently published in the scientific journal Breast Cancer Research.

For decades, physicians have relied on mammography breast imaging to look for cancer in a quest to provide prevention, early detection and reduce deaths. But the unintended consequences of both false positives and false negatives have off-set the hoped-for gains of this inexact type of screening, including complications from surgery and cardiovascular disease, and unnecessary biopsies of what turn out to be benign lesions.

ECM is the network of molecules — including collagen, enzymes and glycoproteins — that provide structural and biochemical support to surrounding cells, including cancer cells. During the early stages of cancer, these proteins and protein fragments — which form the tumor microenvironment — leak into circulating blood.

"Our data reinforces the idea that this release of ECM components into circulation, even at the earliest stages of malignancy, can be used to design a specific and sensitive biomarker panel to improve detection of breast cancer," said Dr. Pirrotte, the study’s senior author. "Using a highly specific and sensitive protein signature, we devised and verified a panel of blood-based biomarkers that could identify the earliest stages of breast cancer, and with no false positives."

To establish this protein signature, researchers employed blood samples from 20 patients with IDC breast cancer, and from 20 women without cancer who nonetheless had positive mammograms but benign pathology at biopsy. These results were compared to five groups of individuals diagnosed with other cancers: ovarian, lung, prostate, colon and melanoma.

Because the number of ECM molecules in blood is relatively low, researchers relied on proteomics and new sample preparation enrichment techniques, including the use of hydrogel nanoparticles, to accurately detect cancer-associated biomarkers. This technique binds proteins from ECM associated with cancer proliferation, migration, adhesion and metastasis, or the spread of cancer from one part of the body to another. Many of these proteins had never before been observed in blood samples.

"Our study results show a high degree of specificity of those markers as circulating proteins in breast cancer patients," said Khyatiben Pathak, Ph.D., a staff scientist in TGen’s Collaborative Center for Translational Mass Spectrometry and a study author. "Our results justify further studies with larger groups to evaluate whether this biomarker panel improves the positive predictive value of mammography for breast cancer detection."

Contributing to this research were George Mason University, Centro di Riferimento Oncologico di Aviano, Istituto Superiore di Sanità, Università Cattolica del Sacro Cuore, Fondazione Policlinico Universitario, University San Raffaele, and Istituto di Ricovero e Cura a Carattere Scientifico.

Funding for this study was provided by TGen and George Mason University. Additional support was provided by: National Cancer Institute, U.S. Department of Health and Human Services, Italian Istituto Superiore di Sanità, and Italian Ministry of Public Health.

The study — Shotgun proteomics coupled to nanoparticle-based biomarker enrichment reveals a novel panel of extracellular matrix proteins as candidate serum protein biomarkers for early-stage breast cancer detection — was published Dec. 2 in the journal Breast Cancer Research.

On January 26, 2021 Celsion Corporation (NASDAQ: CLSN), an oncology drug development company, reported the closing of its previously announced registered direct offering of 25,925,925 shares of common stock at a purchase price of $1.35 per share, priced at-the-market under Nasdaq rules, resulting in net proceeds of $32.6 million, after deducting placement agents’ fees but before expenses payable by the Company (Press release, Celsion, JAN 26, 2021, View Source [SID1234574346]).

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A.G.P./Alliance Global Partners acted as the lead placement agent for the offering.

Brookline Capital Markets, a division of Arcadia Securities, LLC, acted as co-placement agent for the offering.

Celsion intends to use the net proceeds for general corporate purposes, including research and development activities, capital expenditures and working capital.

This offering was made pursuant to an effective shelf registration statement on Form S-3 (File No. 333-227236), previously filed with the Securities and Exchange Commission (the "SEC") on September 7, 2018 and declared effective on October 12, 2018, and an additional registration statement pursuant to Rule 462(b) (File No. 333-252320) under the Securities Act of 1933, as amended. The offering of the shares of common stock were made by means of a prospectus, including a prospectus supplement, forming a part of the effective registration statement. A prospectus supplement and the accompanying prospectus relating to and describing the terms of the offering are filed with the SEC and are available on the SEC’s website at View Source or by contacting A.G.P./Alliance Global Partners, 590 Madison Avenue, 28th Floor, New York, NY 10022, or by telephone at (212) 624-2060, or by email at [email protected].

This press release shall not constitute an offer to sell or a solicitation of an offer to buy nor shall there be any sale of these securities in any state or other jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or other jurisdiction.

On January 26, 2021 Epigenomics AG (Frankfurt Prime Standard: ECX, OTCQX: EPGNY; the "Company")reported that successfully completed the placement of the subordinated mandatory convertible notes, which the Company’s Executive Board resolved to issue with the approval of the Supervisory Board on January 7, 2021 (Press release, Epigenomics, JAN 26, 2021, View Source [SID1234574338]). In total, the mandatory convertible bond in the nominal amount of EUR 5,500,000.00 was fully placed. The offering was significantly oversubscribed. The mandatory convertible bond consists of 500,000 bonds, which are convertible into 5,000,000 registered shares of the Company. The gross proceeds from the issuance of the mandatory convertible bond amount to EUR 5.5 million.

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