On January 10, 2021 Emergent BioSolutions Inc. (NYSE:EBS) reported its financial forecast for 2021 and selected preliminary unaudited financial results for 2020 (Press release, Emergent BioSolutions, JAN 10, 2021, View Source [SID1234573752]).

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"In a year full of unprecedented challenges due to the pandemic, the Emergent team’s unwavering commitment produced incredible results," said Robert G. Kramer, president and chief executive officer. "Operationally, we rapidly responded to our customers’ needs, and financially, we delivered record revenue and earnings. We are proud to be a leader in the growing public health threat market, enabled by our development and manufacturing expertise, successful public-private partnerships, and broad portfolio of products and CDMO services. We look forward to continuing to execute on our strategy and building on the momentum created in 2020 across all four of our business units."

"Our 2020 financial performance clearly demonstrates the resilience and durability of our diversified portfolio of products and services," said Richard S. Lindahl, executive vice president and chief financial officer. "We enter 2021 with positive momentum and are poised to deliver robust double-digit gains in total revenues and non-GAAP earnings for the fifth consecutive year. One year into our five-year strategy, we are increasingly confident in the growth prospects for the business."

PRELIMINARY 2020 FINANCIAL RESULTS (Unaudited)

The Company is providing the following preliminary, unaudited financial results for full year 2020.

Revenue Metrics
Total revenues for 2020 are expected to be in the range of $1,545 and $1,555 million, an increase at the midpoint of $444 million or 40% as compared to 2019. This growth primarily reflects increased sales of contract development and manufacturing (CDMO) services to pharmaceutical and biotechnology innovators and government/non-government organization (NGO) customers, as well as higher product sales.

Profitability Metrics
The Company anticipates Adjusted EBITDA of $625 to $645 million, which at the midpoint represents an increase of $355 million or 127% as compared to 2019. The Company anticipates Adjusted Net Income of $415 to $430 million, which at the midpoint represents an increase of $270 million or 177% as compared to 2019. This growth primarily reflects the forecasted increase in total revenues discussed above. (See "Reconciliation of Non-GAAP Measures" for a definition of the terms and reconciliation tables.)

Note:
The preliminary 2020 financial results are unaudited, subject to revision, and anticipated to be finalized by late February 2021. The Company’s final audited financial results could differ materially from these selected preliminary results.

2021 FINANCIAL FORECAST

The Company is providing the following forecast of selected financial metrics for full year 2021.

Total Revenues
The 2021 forecast for total revenues reflects continued growth in aggregate product revenues and significant growth in services revenue from the CDMO business.

Adjusted EBITDA and Adjusted Net Income (1)
The 2021 forecast reflects an anticipated mix of product and services gross margin, continued investment in research and development, and scale efficiencies in selling, general & administration expenses.

2021 Product/Service Level Revenues – Select Assumptions

Anthrax vaccine revenues are expected to be at a more normalized annual level and continue to primarily reflect procurement of AV7909 (Anthrax Vaccine Adsorbed, adjuvanted) under the Company’s existing contract with the Biomedical Advanced Research and Development Authority (BARDA).
ACAM2000, (Smallpox (Vaccinia) Vaccine, Live) vaccine deliveries are expected to continue under the terms of the Company’s existing contract with the U.S. Department of Health and Human Services (HHS) at unit volume levels consistent with 2020 deliveries.
Narcan (naloxone HCl) Nasal Spray revenues assume no generic competition prior to the resolution of the Company’s appeal of the patent litigation regarding the 4mg form of this intranasal spray product.
CDMO Services assumes continued growth in Development Services (DVS), Drug Substance (DS) manufacturing, and Drug Product (DP) manufacturing and Packaging for both clinical- and commercial-stage projects on behalf of a growing list of pharmaceutical and biotechnology innovators and government/NGO customers.
Other 2021 Assumptions

Gross margin is expected to be approximately 65% on a GAAP basis, influenced by the mix of product and services revenues.
A follow-on procurement contract with HHS is expected for the delivery of raxibacumab, the Company’s Food and Drug Administration-approved anthrax monoclonal antibody therapeutic, to the Strategic National Stockpile (SNS).
Pipeline progress is expected across the vaccines, therapeutics, and devices portfolios, anticipating at least one Phase 3 launch and one Biologics License Application (BLA)/Emergency Use Authorization (EUA) filing.
Capital expenditures, net of reimbursement, are expected to be in a range of 8% to 9% of total revenues, reflecting ongoing investments in capacity and capability expansions related to the CDMO business and the Company’s product portfolio.
FOOTNOTES

(1) See "Reconciliation of Non-GAAP Measures" for a definition of terms and applicable reconciliation tables.

PRESENTATION WEBCAST
The Company will provide an update on the current business and discuss preliminary 2020 financial results, the forecast and corporate goals for 2021, and long-term goals during its presentation at the 39th Annual J.P. Morgan Healthcare Conference on January 11, 2021 at 8:20 AM Eastern time.

A live webcast of the presentation can be accessed through Emergent’s website. An on-demand replay of the webcast can also be accessed in the investors section after the presentation has concluded.

On January 10, 2021 Ascendis Pharma A/S (Nasdaq: ASND), a biopharmaceutical company that utilizes its innovative TransCon technologies to create product candidates that address unmet medical needs, reported that it will provide an update on Vision 3×3 and the company’s 2021 key milestones at the 39th Annual J.P. Morgan Healthcare Conference (Press release, Ascendis Pharma, JAN 10, 2021, View Source;3-update-39th-annual-jp-morgan [SID1234573750]).

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"In 2019, we introduced Vision 3×3, the company’s strategic roadmap through 2025, to build a leading biopharma company by achieving sustainable growth through multiple approaches. 2020 was a remarkable year of progress for Ascendis reflecting the dedication and commitment of our employees worldwide as we made meaningful strides to achieve our vision, and meet or accelerate completion of our key milestones," said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer.

"We expect 2021 will mark a number of key clinical and commercial milestones. By the end of the year, we expect to have five independent TransCon product candidates in clinical development leveraging TransCon technologies through our algorithm for product innovation. We expect to further advance our late stage endocrinology pipeline with the anticipated approval and launch in the United States and the approval in Europe of TransCon hGH for pediatric growth hormone deficiency, and to obtain phase 3 results for TransCon PTH in adult hypoparathyroidism. Development of TransCon CNP is progressing as planned with the recent initiation by VISEN Pharmaceuticals of a second phase 2 trial in patients with achondroplasia, the ACcomplisH China Trial, which provides for dose expansion at an effective dose determined from the ACcomplisH Trial. Finally, we expect to have the first two product candidates from our second therapeutic area of oncology in clinical development," Mr. Mikkelsen added.

Pipeline Updates

•TransCon hGH (lonapegsomatropin): Lonapegsomatropin is an investigational long-acting prodrug of somatropin (human growth hormone or hGH) currently under review for use in pediatric growth hormone deficiency (GHD) by the United States Food and Drug Administration (FDA) and the European Medicines Agency (EMA):
οThe company submitted its Biologic License Application to the FDA which has set a Prescription Drug User Fee Act (PDUFA) date for June 25, 2021. If approved on the PDUFA date, Ascendis anticipates commercial launch in the third quarter of 2021.
οThe company submitted its Marketing Authorisation Application (MAA) to the EMA in September 2020 and anticipates receiving MAA approval for lonapegsomatropin for use in pediatric GHD in the fourth quarter of 2021.
οAscendis anticipates completion of enrollment in foresiGHt, a global phase 3 trial evaluating the safety and efficacy of lonapegsomatropin in adult patients with GHD, by late 2021 or early 2022.

•TransCon PTH: TransCon PTH is an investigational long-acting prodrug of parathyroid hormone (PTH) in development as a potential once-daily replacement therapy for adult hypoparathyroidism (HP):
οFrom the PaTH Forward phase 2 trial, 58 out of 59 randomized subjects continue receiving TransCon PTH in the open label extension (OLE) as of January 5, 2021.
οAfter 26 weeks of follow-up in the PaTH Forward Trial, bone densitometry data from subjects treated with TransCon PTH demonstrated trends towards normalization of bone mineral density. In addition, quality of life as measured by the SF-36 Health Survey showed normalization of mean scores for all summary domains and all subdomains.
οDuring the second quarter of 2021, Ascendis expects to provide a 12-month OLE update and plans to submit a Clinical Trial Notification for a clinical trial evaluating TransCon PTH for adult HP in Japan.
οTop-line results from PaTHway, a phase 3 randomized, double-blind, placebo-controlled clinical trial in North America and Europe, investigating the safety, tolerability, and efficacy of TransCon PTH in adults with HP are expected in the fourth quarter of 2021.

•TransCon CNP: TransCon CNP, an investigational long-acting prodrug of C-type natriuretic peptide (CNP), as a potential therapeutic option for patients with achondroplasia (ACH):
οDosing of sequential ascending dose cohorts continues in the ACcomplisH Trial, a phase 2 randomized, double-blind, placebo-controlled clinical trial in North America, Europe, and Oceania.
οVISEN Pharmaceuticals (VISEN), our strategic investment in China, received approval from China’s Center for Drug Evaluation to conduct the ACcomplisH China Trial, a phase 2 randomized, double-blind, placebo-controlled clinical trial.
οAscendis expects that the ACcomplisH and ACcomplisH China Trials will enroll more than 120 subjects in total (ages 2-10), to be followed for 12 months.
οThe company plans to provide a TransCon CNP clinical program update in the fourth quarter of 2021.

•TransCon TLR7/8 Agonist: TransCon TLR7/8 Agonist is an investigational long-acting prodrug of resiquimod, a small molecule agonist of Toll-like receptors (TLR) 7 and 8 designed to provide sustained activation of intratumoral antigen-presenting cells driving tumor antigen presentation and induction of immune stimulatory cytokines for weeks or months with a single intratumoral injection:
οSubmitted IND to the FDA in December 2020 to initiate clinical program.
οDuring the second quarter of 2021, following monotherapy evaluation, the company plans to initiate TransCon TLR7/8 Agonist dose escalation in combination with a checkpoint inhibitor.
οInitial monotherapy dose escalation results are expected in the fourth quarter of 2021.

•TransCon IL-2 β/γ: TransCon IL-2 β/γ is an investigational novel long-acting prodrug of IL-2 β/γ designed to selectively bind and activate the IL-2Rβ/γ:
οAscendis reported pre-clinical data for TransCon IL-2 β/γ demonstrating:
•Independently optimized receptor bias and potency as well as pharmacokinetics to create a potentially best-in-class IL-2 product.
•An effective half-life of approximately 32 hours in non-human primates (NHP).
•Following a single dose of TransCon IL-2 β/γ in NHP, observed potential best-in-class expansion and activation of cytotoxic lymphocyte subsets with minimal effect on eosinophils, minimal IL-5 and IL-6 levels which suggests low risk of vascular leak syndrome.
οThe company expects to submit an IND or similar for TransCon IL-2 β/γ in the third quarter of 2021.

Global Endocrinology Rare Disease Commercial Strategy

In anticipation of regulatory approvals for lonapegsomatropin in the United States and Europe, Ascendis is establishing a global commercial presence through multiple approaches. This global commercial approach will be laying the groundwork for future potential endocrinology rare disease launches as TransCon PTH and TransCon CNP advance in clinical development.

The company’s US commercial organization is in place and commercial manufacturing is ongoing for the potential launch of lonapegsomatropin in pediatric GHD planned for the third quarter of 2021 after anticipated regulatory approval.

In addition, Ascendis is preparing for potential commercialization in Europe, building an integrated organization in select European countries and evaluating established distribution channels in other European countries to be ready for the anticipated MAA approval of lonapegsomatropin in pediatric GHD in the fourth quarter of 2021.

Lastly, Ascendis plans to serve patients in other regions around the world through established sales and distribution networks and following applicable regulatory approvals. The company has invested in VISEN in Greater China and plans to partner in Japan and South Korea when appropriate.

Presentation at J.P. Morgan Healthcare Conference on Monday, January 11th

Live webcast of the J.P. Morgan presentation will be available on the Events & Presentations section of the investor relations webpage at View Source The presentation will begin at 11:40 a.m. ET. A webcast replay will also be available for 30 days.

The company’s slides from the J.P. Morgan presentation will be available on the investor relations website.

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 optimize therapeutic effect, including efficacy and safety and through 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. The TransCon technology platform can be applied broadly to proteins, peptides or small molecules in multiple therapeutic areas, and can be designed for systemic or localized release.

On January 10, 2021 Ascendis Pharma A/S (Nasdaq: ASND), a biopharmaceutical company that utilizes its innovative TransCon technologies to create product candidates that address unmet medical needs, reported that it will provide an update on Vision 3×3 and the company’s 2021 key milestones at the 39th Annual J.P. Morgan Healthcare Conference (Press release, Ascendis Pharma, JAN 10, 2021, View Source;3-update-39th-annual-jp-morgan [SID1234573750]).

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Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"In 2019, we introduced Vision 3×3, the company’s strategic roadmap through 2025, to build a leading biopharma company by achieving sustainable growth through multiple approaches. 2020 was a remarkable year of progress for Ascendis reflecting the dedication and commitment of our employees worldwide as we made meaningful strides to achieve our vision, and meet or accelerate completion of our key milestones," said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer.

"We expect 2021 will mark a number of key clinical and commercial milestones. By the end of the year, we expect to have five independent TransCon product candidates in clinical development leveraging TransCon technologies through our algorithm for product innovation. We expect to further advance our late stage endocrinology pipeline with the anticipated approval and launch in the United States and the approval in Europe of TransCon hGH for pediatric growth hormone deficiency, and to obtain phase 3 results for TransCon PTH in adult hypoparathyroidism. Development of TransCon CNP is progressing as planned with the recent initiation by VISEN Pharmaceuticals of a second phase 2 trial in patients with achondroplasia, the ACcomplisH China Trial, which provides for dose expansion at an effective dose determined from the ACcomplisH Trial. Finally, we expect to have the first two product candidates from our second therapeutic area of oncology in clinical development," Mr. Mikkelsen added.

Pipeline Updates

•TransCon hGH (lonapegsomatropin): Lonapegsomatropin is an investigational long-acting prodrug of somatropin (human growth hormone or hGH) currently under review for use in pediatric growth hormone deficiency (GHD) by the United States Food and Drug Administration (FDA) and the European Medicines Agency (EMA):
οThe company submitted its Biologic License Application to the FDA which has set a Prescription Drug User Fee Act (PDUFA) date for June 25, 2021. If approved on the PDUFA date, Ascendis anticipates commercial launch in the third quarter of 2021.
οThe company submitted its Marketing Authorisation Application (MAA) to the EMA in September 2020 and anticipates receiving MAA approval for lonapegsomatropin for use in pediatric GHD in the fourth quarter of 2021.
οAscendis anticipates completion of enrollment in foresiGHt, a global phase 3 trial evaluating the safety and efficacy of lonapegsomatropin in adult patients with GHD, by late 2021 or early 2022.

•TransCon PTH: TransCon PTH is an investigational long-acting prodrug of parathyroid hormone (PTH) in development as a potential once-daily replacement therapy for adult hypoparathyroidism (HP):
οFrom the PaTH Forward phase 2 trial, 58 out of 59 randomized subjects continue receiving TransCon PTH in the open label extension (OLE) as of January 5, 2021.
οAfter 26 weeks of follow-up in the PaTH Forward Trial, bone densitometry data from subjects treated with TransCon PTH demonstrated trends towards normalization of bone mineral density. In addition, quality of life as measured by the SF-36 Health Survey showed normalization of mean scores for all summary domains and all subdomains.
οDuring the second quarter of 2021, Ascendis expects to provide a 12-month OLE update and plans to submit a Clinical Trial Notification for a clinical trial evaluating TransCon PTH for adult HP in Japan.
οTop-line results from PaTHway, a phase 3 randomized, double-blind, placebo-controlled clinical trial in North America and Europe, investigating the safety, tolerability, and efficacy of TransCon PTH in adults with HP are expected in the fourth quarter of 2021.

•TransCon CNP: TransCon CNP, an investigational long-acting prodrug of C-type natriuretic peptide (CNP), as a potential therapeutic option for patients with achondroplasia (ACH):
οDosing of sequential ascending dose cohorts continues in the ACcomplisH Trial, a phase 2 randomized, double-blind, placebo-controlled clinical trial in North America, Europe, and Oceania.
οVISEN Pharmaceuticals (VISEN), our strategic investment in China, received approval from China’s Center for Drug Evaluation to conduct the ACcomplisH China Trial, a phase 2 randomized, double-blind, placebo-controlled clinical trial.
οAscendis expects that the ACcomplisH and ACcomplisH China Trials will enroll more than 120 subjects in total (ages 2-10), to be followed for 12 months.
οThe company plans to provide a TransCon CNP clinical program update in the fourth quarter of 2021.

•TransCon TLR7/8 Agonist: TransCon TLR7/8 Agonist is an investigational long-acting prodrug of resiquimod, a small molecule agonist of Toll-like receptors (TLR) 7 and 8 designed to provide sustained activation of intratumoral antigen-presenting cells driving tumor antigen presentation and induction of immune stimulatory cytokines for weeks or months with a single intratumoral injection:
οSubmitted IND to the FDA in December 2020 to initiate clinical program.
οDuring the second quarter of 2021, following monotherapy evaluation, the company plans to initiate TransCon TLR7/8 Agonist dose escalation in combination with a checkpoint inhibitor.
οInitial monotherapy dose escalation results are expected in the fourth quarter of 2021.

•TransCon IL-2 β/γ: TransCon IL-2 β/γ is an investigational novel long-acting prodrug of IL-2 β/γ designed to selectively bind and activate the IL-2Rβ/γ:
οAscendis reported pre-clinical data for TransCon IL-2 β/γ demonstrating:
•Independently optimized receptor bias and potency as well as pharmacokinetics to create a potentially best-in-class IL-2 product.
•An effective half-life of approximately 32 hours in non-human primates (NHP).
•Following a single dose of TransCon IL-2 β/γ in NHP, observed potential best-in-class expansion and activation of cytotoxic lymphocyte subsets with minimal effect on eosinophils, minimal IL-5 and IL-6 levels which suggests low risk of vascular leak syndrome.
οThe company expects to submit an IND or similar for TransCon IL-2 β/γ in the third quarter of 2021.

Global Endocrinology Rare Disease Commercial Strategy

In anticipation of regulatory approvals for lonapegsomatropin in the United States and Europe, Ascendis is establishing a global commercial presence through multiple approaches. This global commercial approach will be laying the groundwork for future potential endocrinology rare disease launches as TransCon PTH and TransCon CNP advance in clinical development.

The company’s US commercial organization is in place and commercial manufacturing is ongoing for the potential launch of lonapegsomatropin in pediatric GHD planned for the third quarter of 2021 after anticipated regulatory approval.

In addition, Ascendis is preparing for potential commercialization in Europe, building an integrated organization in select European countries and evaluating established distribution channels in other European countries to be ready for the anticipated MAA approval of lonapegsomatropin in pediatric GHD in the fourth quarter of 2021.

Lastly, Ascendis plans to serve patients in other regions around the world through established sales and distribution networks and following applicable regulatory approvals. The company has invested in VISEN in Greater China and plans to partner in Japan and South Korea when appropriate.

Presentation at J.P. Morgan Healthcare Conference on Monday, January 11th

Live webcast of the J.P. Morgan presentation will be available on the Events & Presentations section of the investor relations webpage at View Source The presentation will begin at 11:40 a.m. ET. A webcast replay will also be available for 30 days.

The company’s slides from the J.P. Morgan presentation will be available on the investor relations website.

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 optimize therapeutic effect, including efficacy and safety and through 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. The TransCon technology platform can be applied broadly to proteins, peptides or small molecules in multiple therapeutic areas, and can be designed for systemic or localized release.

On January 9, 2021 Ascendis Pharma A/S (Nasdaq: ASND), a biopharmaceutical company that utilizes its innovative TransCon technologies to create product candidates that address unmet medical needs, reported an equity investment of $12.5 million in VISEN Pharmaceuticals (VISEN) as part of VISEN’s $150 million Series B financing (Press release, Ascendis Pharma, JAN 9, 2021, View Source [SID1234573749]).

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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"Our relationship with VISEN is a key element of our Vision 3×3 strategy to extend the global reach of our TransCon endocrinology rare disease product candidates and improve patients’ lives around the world," said Jan Mikkelsen, President and CEO at Ascendis Pharma. "Since VISEN’s inception in 2018, the company has made significant progress on its clinical activities in Greater China, which we believe will enable us to get our products to patients worldwide as soon as possible."

In 2018, Ascendis granted VISEN exclusive rights to develop and commercialize TransCon hGH (lonapegsomatropin), TransCon PTH and TransCon CNP in Greater China and received 50 percent ownership of VISEN’s issued and outstanding shares. Following VISEN’s Series B financing, Ascendis retains approximately 44 percent of VISEN’s issued and outstanding shares. As a result, Ascendis expects to recognize a non-cash gain in the first quarter of 2021.

Concurrent with the closing of the Series B financing, Michael Wolff Jensen, Chairman, Senior Vice President and Chief Legal Officer of Ascendis, became a member of VISEN’s board and serves as Chairman. Mr. Mikkelsen also will continue to serve on VISEN’s board.

"I look forward to joining the VISEN board and to working with the talented management team to accelerate the clinical pipeline of investigational TransCon drug candidates," said Mr. Jensen.

About VISEN Pharmaceuticals

VISEN Pharmaceuticals is committed to the treatment of endocrine-related diseases, introducing the world’s leading treatment methods and drugs into the China market and hoping to provide more Chinese patients quick access to the world’s most advanced and reliable treatment solutions.

In 2018, VISEN Pharmaceuticals (VISEN) was formed by Ascendis Pharma A/S (Nasdaq: ASND) and an investor syndicate led by Vivo Capital (along with participation by Sofinnova Ventures), to develop and commercialize endocrinology therapies in Greater China, which includes mainland China, Hong Kong, Macau, and Taiwan.

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 optimize therapeutic effect, including efficacy and safety and through 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 technology can be applied broadly to proteins, peptides or small molecules in multiple therapeutic areas, and can be designed for systemic or localized release.

On January 8, 2021 Blacksmith Medicines, Inc. (Blacksmith), a biotechnology company focused on creating novel medicines for immuno-oncology and inflammatory diseases by targeting human metalloenzymes, reported that it has secured seed funding and has entered into a research collaboration with Eli Lilly and Company (Lilly) (Press release, Blacksmith Medicines, JAN 8, 2021, View Source [SID1234643541]). Lilly joins existing Blacksmith investors Evotec A.G., MP Healthcare Partners, MagnaSci Ventures, and Alexandria Venture Investments. Blacksmith is a spin-out of Forge Therapeutics, a leading antibiotic discovery company discovering therapies to combat the rise of drug resistance.

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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Under the terms of the research collaboration agreement, Blacksmith and Lilly will collaborate on up to 5 human metalloenzyme targets, with Blacksmith advancing novel small molecule inhibitors from fragment to ‘hit’ stage after which Lilly has the right to continue research, development, and commercialization. Blacksmith will receive an upfront payment and an investment from Lilly in exchange for a convertible note and will be eligible for up to approximately $60 million per target in potential research, development and commercial milestones for a total potential deal size up to approximately $300 million.

The Blacksmith metalloenzyme platform is a unique drug discovery engine purpose-built to target the largest and most diverse group of enzymes in the human genome. Blacksmith’s proprietary platform has several distinct advantages over traditional drug discovery approaches including:

A large proprietary fragment library of metal-binding pharmacophores (MBPs);
A comprehensive database containing a full characterization of the metalloenzyme genome including functions, metal cofactors, and associations to disease;
A first-of-its-kind metallo-CRISPR library of custom single guide RNAs;
An industry-leading metalloenzyme computational toolkit for docking, modeling and structure-based drug design; and
A robust and blocking intellectual property estate covering bioinorganic, medicinal, and computational chemistry approaches for metalloenzyme-targeted medicines.
"We are excited to launch Blacksmith Medicines with our investors and enter this research collaboration with Lilly, who collectively share our commitment to advancing novel chemistry approaches to create new medicines for patients in need," said Zachary Zimmerman, Ph.D., CEO and co-founder. "With the creation of Blacksmith, we are now able to expand our metalloenzyme platform into other therapeutic areas outside of anti-infectives, deepening our expertise and creating increased value for our shareholders, strategic partners and patients. We look forward to broadening the reach of our technology as we focus our efforts on identifying novel targets for immuno-oncology and inflammatory diseases."