On April 29, 2021 Syros Pharmaceuticals (NASDAQ:SYRS), a leader in the development of medicines that control the expression of genes, reported that it will host a live conference call and webcast at 8:30 a.m. ET on Thursday, May 6, 2021 to report its first quarter 2021 financial results and provide a corporate update (Press release, Syros Pharmaceuticals, APR 29, 2021, View Source [SID1234578743]).

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To access the live conference call, please dial 866-595-4538 (domestic) or 636-812-6496 (international), and refer to conference ID 5770919. A webcast of the call will also be available on the Investors & Media section of the Syros website at www.syros.com. An archived replay of the webcast will be available for approximately 30 days following the presentation.

On April 29, 2021 Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism to treat genetically defined diseases, reported business highlights and financial results for the first quarter ended March 31, 2021.

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"With the recent closing of the sale of our oncology business, we are excited to embrace a focused future in genetically defined diseases," said Jackie Fouse, Ph.D., chief executive officer at Agios. "In the weeks and months ahead, we look forward to a number of important catalysts, beginning with presentations at the EHA (Free EHA Whitepaper) 2021 Virtual Congress, where we will share data from our ACTIVATE and ACTIVATE-T studies of mitapivat in adults with pyruvate kinase (PK) deficiency, as well as data from our Phase 2 study of mitapivat in thalassemia. The PK deficiency data will form the basis of our upcoming submissions for regulatory approval in the U.S. this quarter and in the EU mid-year. Additionally, we look forward to further exploring the impact of mitapivat in thalassemia and sickle cell disease as we launch two Phase 3 studies and a Phase 2/3 study, respectively, later this year. The courage and needs of the patients and families that we seek to serve continue to be our great motivation, and we are proud of our recent educational and patient-focused efforts that aim to make a meaningful impact on rare hemolytic anemia communities."

FIRST QUARTER 2021 & RECENT HIGHLIGHTS
Closed the sale of commercial, clinical and research-stage oncology portfolio to Servier Pharmaceuticals, LLC, on March 31, 2021.

Initiated the repurchase of up to $1.2 billion of outstanding shares, as authorized by the board of directors, and entered into a definitive agreement with Bristol-Myers Squibb Company (BMS) to repurchase 7,121,658 shares of Agios common stock held by BMS and its affiliates for an aggregate purchase price of $344.5 million, or $48.38 per share. Agios expects to execute a meaningful portion of the planned repurchases by year-end through a combination of 10b5-1 plans and open market purchases.

Supported several educational and patient-focused initiatives, including the sponsorship of a health literacy program for sickle cell disease and the launch of a collaboration with 23andMe that led to addition of the very first PK deficiency carrier status report.

KEY UPCOMING MILESTONES

Finalize global regulatory filings for mitapivat in adults with PK deficiency; submit new drug application (NDA) in the U.S. in the second quarter of 2021 and marketing authorization application (MAA) in the EU in mid-2021.
Initiate two Phase 3 studies of mitapivat, ENERGIZE and ENERGIZE-T, in not regularly transfused and regularly transfused adults with thalassemia in the second half of 2021.
Initiate Phase 2/3 study of mitapivat in sickle cell disease by year-end.
Prioritize new indications for pyruvate kinase R (PKR) and pyruvate kinase M2 (PKM2) activator clinical development by year-end.

Data Presentations
Present data from the following clinical studies for presentation at the EHA (Free EHA Whitepaper) Virtual Congress, hosted June 9-17, 2021:
Phase 3 ACTIVATE study of mitapivat in adults with PK deficiency who do not receive regular transfusions
Phase 3 ACTIVATE-T study of mitapivat in adults with PK deficiency who receive regular transfusions
Phase 2 study of mitapivat in adults with α- and β-thalassemia who do not receive regular transfusions
Submit data from ongoing clinical studies of mitapivat in sickle cell disease for presentation at medical meetings in 2021.
Present data from the Phase 1 study of AG-946, the company’s next-generation PKR activator, in healthy volunteers by year-end.

FIRST QUARTER 2021 FINANCIAL RESULTS

Research and Development (R&D) Expenses: R&D expenses for continuing operations were $57.7 million for the first quarter of 2021 compared to $55.4 million for the first quarter of 2020. The year-over-year increase in R&D was driven primarily by start-up costs associated with the Phase 3 studies of mitapivat in thalassemia and sickle cell disease.

Selling, General and Administrative (SG&A) Expenses: SG&A expenses for continuing operations were $33.6 million for the first quarter of 2021 compared to $31.7 million for the first quarter of 2020. The year-over-year increase in SG&A expenses was primarily attributable to one-time workforce expense.

Discontinued Operations: Due to the sale of the oncology business during the first quarter of 2021, we have reclassified the results of the oncology business as discontinued operations, including total revenue of $41.4 million, TIBSOVO net sales of $37 million, and operating expenses of $50.2 million related to discontinued operations.
Net Income: Net income was $1.9 billion for the first quarter of 2021 compared to a net loss of $40.3 million for the first quarter of 2020.

Cash Position and Guidance: Cash, cash equivalents and marketable securities as of March 31, 2021 were $2.4 billion compared to $613.1 million as of March 31, 2020. The company expects that its cash, cash equivalents and marketable securities as of March 31, 2021, together with anticipated interest income, future product sales and TIBSOVO royalties, will enable the company to fund its planned operating expenses and capital expenditure requirements through major catalysts and to cash-flow positivity without the need to raise additional equity.

CONFERENCE CALL INFORMATION

Agios will host a conference call and live webcast with slides today at 8:00 a.m. ET to discuss first quarter 2021 financial results and recent business activities. To participate in the conference call, please dial 1-877-377-7098 (domestic) or 1-631-291-4547 (international) and refer to conference ID 4497151. The live webcast can be accessed under "Events & Presentations" in the Investors section of the company’s website at www.agios.com. The archived webcast will be available on the company’s website beginning approximately two hours after the event.

On April 29, 2021 Acorda Therapeutics, Inc. (NASDAQ: ACOR) reported that it will host a conference call and webcast in conjunction with its first quarter 2021 update and financial results on Thursday, May 6 at 4:30 p.m. ET (View Source). To participate in the Webcast/Conference Call, please note there is a new pre-registration process.

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To register for the Webcast, use the link below:
View Source

To register for the Conference Call, use the link below:
View Source

**When registering please type your phone number with no special characters**
Once you have registered, you will receive a confirmation email with Webcast/Conference Call details. For the Webcast you will receive an email 2 hours prior to the start of the call with the link to join. The presentation will be available on the Investors section of www.acorda.com.

A replay of the call will be available from 7:30 p.m. ET on May 6, 2021 until 11:59 p.m. ET on June 3, 2021. To access the replay, please dial (800) 585-8367 (domestic) or (416) 621-4642 (international); reference code 2996776. The archived webcast will be available in the Investor Relations section of the Acorda website at www.acorda.com.

Forge Biologics, a gene therapy-focused contract development and manufacturing organization, reported that the closing of a $120 million Series B financing (Press release, Forge Biologics, APR 29, 2021, View Source [SID1234578742]). The financing was led by RA Capital Management with participation from Perceptive Advisors and related affiliates, Surveyor Capital (a Citadel company), Octagon Capital, and Marshall Wace. Existing investors Perceptive Xontogeny Venture Fund and Drive Capital also participated. In connection with the financing, Matthew Hammond, Ph.D., of RA Capital, and Fred Callori of the Perceptive Xontogeny Venture Fund will join the Company’s Board of Directors.

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This press release features multimedia. View the full release here: View Source

"We are very pleased to be working with RA Capital and a strong syndicate of top-tier life sciences investors who share our vision as we now advance our growth as a global gene therapy manufacturing and development company," said Timothy J. Miller, Ph.D., CEO, President and Co-Founder of Forge Biologics. "We have built Forge into a focused gene therapy development engine with a firm commitment to helping our clients provide potentially life-saving gene therapies to patients. We believe that focusing entirely on gene therapy will allow us to best serve our clients and patients by efficiently delivering high quality product."

Forge will use the proceeds of this Series B financing to accelerate the expansion of its AAV manufacturing CDMO capabilities with cGMP production capacity, as well as operate its subsidiaries that are advancing novel AAV gene therapy programs. Forge brings a patients-first approach to accelerate the development of transformative medicines for those who need them most and is addressing the growing demand for gene therapy manufacturing capacity. Through its currently-operational 175,000 square foot cGMP facility in Columbus, Ohio, dedicated to AAV viral vector manufacturing, Forge offers end-to-end manufacturing services, including research and toxicology grade AAV production, to accelerate gene therapy programs from preclinical through clinical and commercial stage manufacturing.

"The success of complex biologics like AAV is exhausting existing manufacturing capacity around the industry. This financing will help to address this industry-wide capacity shortage by properly capitalizing an emerging gene therapy-focused CDMO capable of producing high quality cGMP product for its clients," said Matthew Hammond, Ph.D., principal at RA Capital Management. "We are confident that Forge’s experienced team will become the trusted partner of innovative therapeutics companies, working collaboratively with clients to successfully deliver AAV manufacturing solutions."

Through its subsidiaries, Forge is also working to advance a proprietary pipeline of novel gene therapies, including its lead program FBX-101 for the treatment of patients with Krabbe disease, a first-in-human gene therapy utilizing an adeno-associated virus (AAV) to deliver a functioning copy of the GALC gene intravenously to cells in the central nervous system (CNS) and peripheral organs.

Chardan Capital Markets acted as exclusive placement agent for the offering, with Ice Miller acting as legal advisor.

Adcendo, a biotech company developing antibody-drug conjugates (ADCs) for the treatment of cancers, reported that the successful closing of a EUR 51 million (US$ 62 million) Series A financing (Press release, ADCendo, APR 29, 2021, View Source [SID1234578741]). The investment was led by Novo Seeds, the early-stage investment and company creation team of Novo Holdings, and Ysios Capital, along with RA Capital Management, HealthCap and Gilde Healthcare. The company was initially incubated and funded at the BioInnovation Institute (BII).

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The new financing, which is the largest Series A financing for a Danish biotech company, will be used to establish a pipeline of ADCs directed at novel cancer targets and to bring the lead program targeting the novel cancer target uPARAP/Endo180 to proof of concept in patients.

Commenting on the financing, Henrik Stage, Chief Executive Officer of Adcendo, said: "In the last few years the ADC modality has delivered promising approvals of new drugs as well as significant commercial transactions. We are excited that we have secured this major financing from top tier investors and are looking forward to delivering on our vision of bringing new innovative treatments to cancer patients."

John Haurum, Chairman of the Board of Adcendo, added: "With this funding we will be able to bring our first program – the uPARAP program – all the way to proof of concept in patients, translating the basic research done at The Finsen Laboratory benches, incubated through the BII’s Creation House Program, to new, real-life treatments."

uPARAP is a unique novel cancer target overexpressed on the cell surface of several cancers. Being a collagen scavenger receptor that possesses constitutively active and highly efficient internalization and recycling properties, it has been demonstrated to play a role in tumor invasion. The expression and biological mechanisms of uPARAP makes it ideal for an ADC approach as it may be used as a cancer-associated "drug internalization pump" to bring conjugated drugs directly into the cancer cells.

The uPARAP collagen scavenger receptor has been found to be overexpressed by cancer cells in several indications with high unmet needs including soft tissue sarcoma, glioblastoma multiforme, triple-negative breast cancers, leukemia and osteosarcoma, as well as by stromal cells in several high prevalence cancers with substantial stromal tissue content, such as prostate, breast and pancreatic cancer.

In addition to the uPARAP program, Adcendo will build a pipeline of additional novel cancer targets ideally suited to ADC approaches.

Jeroen Bakker, Principal at Novo Seeds, commented: "Novo Seeds is focused on building world class companies that are developing innovative treatments for patients with unmet medical needs. We are proud to have been involved with the company since its early days and are very impressed with the progress achieved to date. We are very pleased to now co-lead this strong investment, building on the founders’ early-stage research at The Finsen Laboratory to develop Adcendo into a world leading ADC player with a pipeline of exciting novel ADC drugs."

Joël Jean-Mairet, Managing Partner at Ysios Capital, added: "Adcendo is taking the ADC approach to the next level by focusing on targets that are professional internalizers and have exquisite tumor selectivity. We are very pleased to support this financing."

The Board of Adcendo includes: John Haurum, Chair; Jeroen Bakker, Novo Seeds; Joël Jean-Mairet, Ysios Capital; Sanne de Jongh, Gilde Healthcare; Reza Halse, RA Capital Management; Mårten Steen, HealthCap and Lars Engelholm of Adcendo.