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Pictured: Blood sample for insulin-like growth factor-1 (IGF-1)/iStock, Jarun011 Crinetics Pharmaceuticals has secured another late-stage win as its oral, once-daily investigational drug paltusotine aced a second Phase III study for acromegaly, a rare hormonal disorder. The biotech’s stock shot up over 10% Tuesday morning following the announcement. In the late-stage trial, 56% of patients taking paltusotine achieved the primary endpoint for the insulin-like growth factor 1 (IGF-1) level compared to 5% on placebo. The treatment also helped reduce symptoms of the disease—a secondary endpoint of the study. No serious adverse events were reported. Acromegaly, sometimes referred to as gigantism, is a serious hormonal disorder caused by a benign tumor in the pituitary gland that secretes growth hormone. Over 25,000 patients in the U.S. suffer from the rare disease, which results in enlargement of face, hands and feet. Left untreated, it can cause type 2 diabetes, hypertension, heart disease and other serious complications. Surgical removal of the tumor is the preferred treatment but many patients are not candidates for surgery. Current pharmacological options include painful monthly injections or Pfizer’s subcutaneous daily injection of Somavert. The Phase III data are the second positive trial results for Crinetics in acromegaly. In September 2023, the biotech shared data for a study of paltusotine in patients who switched from their current injections to Crinetics’ pill. Among treated patients, 83% were able to maintain their IGF-1 levels within the upper limit of normal, compared to only 4% of those on placebo. With these data in hand, Crinetics plans to submit its New Drug Application to the FDA in the second half of this year, while prepping for a 2025 launch. Jefferies analysts believe the market opportunity could be around $300 million for the company. Crinetics is also testing the drug candidate in carcinoid syndrome, reporting positive Phase II topline results last week. Kate Goodwin is a freelance life science writer based in Des Moines, Iowa. She can be reached at kate.goodwin@biospace.com and on LinkedIn.

New York, NY, March 14, 2024 (GLOBE NEWSWIRE) -- Lirum Therapeutics, Inc. (“Lirum”), an innovative clinical-stage biopharmaceutical company focused on the treatment of debilitating diseases, announced today the acceptance for presentation of positive data on LX-101, a novel clinical-stage targeted therapy directed to the insulin-like growth factor 1 receptor (“IGF-1R”), at the 2024 European Society for Medical Oncology (“ESMO”) Sarcoma and Rare Cancers Congress being held from March 14-16 in Lugano, Switzerland. Lirum’s data covers clinically-tested LX-101’s potent preclinical anti-tumor activity against cancers with well-established IGF-1R pathway involvement, including malignancies with known genetic alterations affecting the IGF-1R pathway and/or with high IGF-1R expression. The poster is available on the Lirum website ( ) under the Investors and Media tab. Given these promising results and the existing clinical data, Lirum is planning new clinical trials with LX-101 focused on oncologic indications, both pediatric and adult, that carry well-established ties to the IGF-1/IGF-1R pathway. Lirum is also developing LX-101 in certain autoimmune diseases, including thyroid dye disease, where IGF-1R has been clinically and commercially validated. About Lirum Therapeutics, Inc. Lirum is an innovative clinical-stage biopharmaceutical company focused on the treatment of debilitating diseases through the acquisition, development and commercialization of novel drug candidates with compelling mechanisms of action, regulatory pathways and commercial opportunities. Lirum’s lead candidate, LX-101, is a novel clinical-stage targeted therapy directed to the insulin-like growth factor-1 receptor (“IGF-1R”) with a differentiated mechanism of action. Lirum is developing LX-101 in oncology and autoimmune indications, including thyroid eye disease (“TED”). For more information on Lirum, please visit . Forward Looking Statements This press release contains certain “forward-looking statements” within the meaning of the Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, each as amended. Forward-looking statements can be identified by words such as: aim,” “anticipate,” “assume,” “believe,” “contemplate,” “continue,” “could,” “design,” “due,” “estimate,” “expect,” “goal,” “intend,” “may,” “objective,” “plan,” “positioned,” “potential,” “predict,” “seek,” “should,” “target,” “will,” “would” and other similar expressions. We have based these forward-looking statements largely on our current expectations and projections about future events that we believe may affect our financial condition, results of operations, business strategy and financial needs. Because forward-looking statements relate to the future, they are subject to inherent uncertainties, risks and changes in circumstances that are difficult to predict and many of which are outside of our control. Actual results and outcomes may differ materially from those indicated in the forward-looking statements. Important factors that could cause actual results and outcomes to differ materially from those indicated in the forward-looking statements include, among others: our ability to successfully develop our product candidates, including obtaining positive results from planned clinical trials; expectations for the clinical development, manufacturing, regulatory approval and commercialization of our product candidates or other products we may acquire or in-license; expectations for incurring capital expenditures and generating revenue; estimates of the sufficiency of our existing cash and cash equivalents and investments to finance operations; changes in applicable laws or regulations; the possibility that we may be adversely affected by other economic, business, and/or competitive factors; the impact of health epidemics, including the COVID-19 pandemic, on our business and the actions we may take in response thereto; and other risks and uncertainties indicated from time to time. Although we believe that the plans and expectations reflected in any forward-looking statements are based on reasonable assumptions, we can give no assurance that our plans and expectations will be attained, and therefore actual outcomes and results may differ materially from what is expressed or forecasted in such forward-looking statements. These forward-looking statements are subject to numerous risks and uncertainties, including those identified elsewhere in this communication and those included in the “Risk Factors” section in our most recently filed Registration Statement on Form S-1 and in other documents that we may the SEC, which are available at . These cautionary statements qualify all of our forward-looking statements, and you are cautioned not to place undue reliance on these forward-looking statements. Our forward-looking statements speak only as of the date they are made and should not be relied upon as representing our plans and expectations as of any subsequent date. While we may elect to update or revise forward-looking statements at some time in the future, we specifically disclaim any obligation to publicly release the results of any revisions to our forward-looking statements, except as required by law. Company contact: Matt Hoberman Investor Relations IR@lirumtx.com Phone: (646) 389-6015 Lirum Therapeutics, Inc. 590 Madison Avenue 21st Floor New York, NY 10022

- VRDN-001 Phase 3 THRIVE and THRIVE-2 topline clinical data readouts are expected for mid-year 2024 and year-end 2024, respectively - - Subcutaneous VRDN-003 pivotal program in thyroid eye disease expected to start mid-year 2024 pending regulatory authority alignment, as previously shared - - Fc receptor (FcRn) inhibitors are on track with VRDN-006 Investigational New Drug Application (IND) submission anticipated by year-end 2024 and VRDN-008 non-human primate data expected in the second half of 2024 - - Year-end 2023 cash balance of approximately $477.4 million; in January 2024, raised approximately $150 million in gross proceeds from a public offering which extended the company’s operating runway into the second half of 2026 - WALTHAM, Mass.--(BUSINESS WIRE)-- Viridian Therapeutics Inc. (NASDAQ: VRDN), a biotechnology company focused on discovering and developing potential best-in-class medicines for serious and rare diseases, today reported recent business highlights and financial results for the fourth quarter and full year ended December 31, 2023. “Throughout 2023, we delivered important clinical results across our TED portfolio, marking significant progress for the company,” said Steve Mahoney, Viridian President and Chief Executive Officer. “We also unveiled our FcRn inhibitor portfolio in October 2023. I would like to thank the Viridian team for their commitment throughout this past year as we work towards our mission of delivering potential best-in-class medicines for patients with serious rare and autoimmune diseases. The momentum we generated throughout 2023 has placed us in a strong position to execute on our upcoming 2024 milestones across our TED and FcRn inhibitor portfolios.” RECENT PROGRESS Thyroid Eye Disease Portfolio: VRDN-001 and VRDN-003 VRDN-001: Viridian’s lead product candidate is an intravenously-delivered monoclonal antibody that acts as a full antagonist of the insulin-like growth factor-1 receptor (IGF-1R). Two ongoing registrational Phase 3 clinical trials, THRIVE and THRIVE-2, are continuing to enroll patients with active and chronic TED, respectively. Topline data readouts are expected mid-year for THRIVE and year-end for THRIVE-2. VRDN-003: In December 2023, Viridian reported positive data for VRDN-003 from a Phase 1 clinical study in healthy volunteers and selected VRDN-003 to move forward as its subcutaneous anti-IGF-1R product candidate with the potential to become a best-in-class anti-IGF-1R. VRDN-003 has the same binding domain as its parent VRDN-001 and was engineered to have a longer half-life. The Phase 1 results showed a VRDN-003 half-life of 40-50 days which is 4-5x the half-life of VRDN-001. Further, pharmacokinetic modeling predicted that convenient dosing regimens of VRDN-003 (e.g., a subcutaneous injection once every two, four, or eight weeks) could achieve exposure levels of VRDN-003 that are equivalent to the exposure levels of VRDN-001 that produced clinically meaningful results in TED. Viridian expects to initiate a global pivotal program with VRDN-003 mid-year, with planned trials in both active and chronic TED patients, pending regulatory authority alignment. FcRn Inhibitor Portfolio: VRDN-006 and VRDN-008 In October 2023, Viridian unveiled its development of a portfolio of engineered FcRn inhibitors, including VRDN-006 and VRDN-008. FcRn inhibitors have the potential to treat a broad array of autoimmune diseases. Viridian’s multi-pronged engineering approach has resulted in a portfolio of FcRn-targeting molecules that leverage the clinically and commercially validated mechanism of FcRn inhibition while potentially addressing the limitations of current agents. VRDN-006: VRDN-006 is a highly selective Fc fragment designed to be a convenient subcutaneous and self-administered option for patients. Viridian anticipates submitting an IND for VRDN-006 by year-end 2024. VRDN-008: VRDN-008 is designed to be a half-life extended FcRn inhibitor with the goal to prolong IgG suppression and provide a potentially best-in-class subcutaneous option for patients. The VRDN-008 program is on track to deliver pharmacokinetic and pharmacodynamic non-human primate data in the second half of the year. Recent Financing Activity & Runway Extension On January 17, 2024, the company announced a public offering of common stock with gross proceeds of approximately $150.0 million, which consisted of the sale of 7,142,858 shares of common stock at a public offering price of $21.00 per share. In December 2023 and January 2024, Viridian sold an aggregate of 2,245,868 shares of common stock through its at-the-market (ATM) facility resulting in net proceeds of approximately $14.8 million in December, which is included in the company’s year-end reported financials, and approximately $35.2 million in January 2024. The company believes that its current cash, cash equivalents, and short-term investments, including proceeds from the ATM and January financing, will be sufficient to fund its operations into the second half of 2026. UPCOMING PRESENTATIONS AT NANOS Viridian plans to present encore VRDN-001 Phase 2 clinical data and Phase 3 clinical trial designs via two abstracts at the 50th Annual Meeting of the North American Neuro-Ophthalmology Society (NANOS) to be held March 2-7, 2024 in Honolulu, Hawaii. Following NANOS, the presentations will be available at . Oral Presentation Title: Clinical Activity and Safety of VRDN-001, a Full Antagonist Antibody to Insulin-like Growth Factor-1 Receptor, in Active and Chronic Thyroid Eye Disease Session: Scientific platform Session III Date: Tuesday, March 5, 2024 Time: 10:15 – 10:30 am HST Poster Presentation Title: THRIVE and THRIVE-2: Phase 3 Trials of VRDN-001 in Thyroid Eye Disease: Next Generation Insulin-like Growth Factor-1 Receptor Blockade Session: Analytical Studies Poster Reception Date: Tuesday, March 5, 2024 Time: 7:00 – 9:00 pm HST CORPORATE HIGHLIGHTS In January 2024, the company promoted Dr. Thomas Ciulla to Chief Medical Officer. In February 2024, the company appointed Jennifer Tousignant as Chief Legal Officer. FINANCIAL RESULTS Cash Position: Cash, cash equivalents, and short-term investments were $477.4 million as of December 31, 2023, compared with $424.6 million as of December 31, 2022. As of January 31, 2024, Viridian’s cash, cash equivalents, and short-term investments were approximately $645.7 million, which includes January proceeds from the public offering and its ATM. This is a preliminary number that has not been audited and is subject to change pending completion of the company’s financial statements for the quarter ended March 31, 2024, which Viridian anticipates reporting on in its first quarter earnings release. R&D Expenses: Research and development expenses were $159.8 million during the year ended December 31, 2023, compared to $100.9 million during the year ended December 31, 2022. The increase in research and development expenses was driven by increased costs associated with our ongoing THRIVE and THRIVE-2 clinical trials, increased costs associated with the selection of our subcutaneous product candidate VRDN-003, as well as increased personnel-related costs as a result of increased headcount. G&A Expenses: General and administrative expenses were $95.0 million during the year ended December 31, 2023, compared to $35.2 million during the year ended December 31, 2022. The increase in general and administrative expenses was driven by personnel-related costs, including share-based compensation and $31.0 million of one-time severance costs, as well as professional, accounting and licensing fees to support a growing organization and preparation for commercial activities. Net Loss: The company’s net loss was $237.7 million for the year ended December 31, 2023, compared with $129.9 million for the same period last year. About Viridian Therapeutics Viridian is a biopharmaceutical company focused on engineering and developing potential best-in-class medicines for patients with serious and rare diseases. Viridian’s expertise in antibody discovery and protein engineering enables the development of differentiated therapeutic candidates for previously validated drug targets in commercially established disease areas. Viridian is advancing multiple candidates in the clinic for the treatment of patients with thyroid eye disease (TED). The company is conducting two global Phase 3 clinical trials (THRIVE and THRIVE-2) to evaluate the safety and efficacy of VRDN-001 in patients with active and chronic TED. Viridian’s goal is to advance VRDN-001 as a potential best-in-class intravenous therapy followed by VRDN-003 as a potential first- and best-in-class subcutaneous therapy for the treatment of TED. In addition to its TED portfolio, Viridian is advancing a novel portfolio of neonatal Fc receptor (FcRn) inhibitors, including VRDN-006 and VRDN-008, which has the potential to be developed in multiple autoimmune diseases. Viridian is based in Waltham, Massachusetts. For more information, please visit . Follow Viridian on LinkedIn and X. Forward Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may be identified by the use of words such as, but not limited to, “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “might,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “will,” or “would” or other similar terms or expressions that concern our expectations, plans and intentions. Forward-looking statements are neither historical facts nor assurances of future performance. Instead, they are based on our current beliefs, expectations, and assumptions. Forward-looking statements include, without limitation, statements regarding: preclinical and clinical development of Viridian’s product candidates VRDN-001, VRDN-003, VRDN-006 and VRDN-008; anticipated start dates of studies, including those related to the VRDN-003 pivotal program; alignment with regulatory authorities and anticipated regulatory submissions; enrollment in Viridian’s clinical studies, including the THRIVE and THRIVE-2 Phase 3 clinical studies; upcoming milestones and anticipated data results, including topline results; the potential utility, efficacy, potency, safety, clinical benefits, clinical response and convenience of VRDN-001, VRDN-003, VRDN-006 and VRDN-008; Viridian’s product candidates potentially being best-in-class; that pharmacokinetic modeling predicts that convenient dosing regimens of VRDN-003 (e.g., a subcutaneous injection once every two, four, or eight weeks) could achieve exposure levels of VRDN-003 that are equivalent to the exposure levels of VRDN-001 that produced clinically meaningful results in TED; potential dosing schedules and trial designs; the Company’s expected cash, cash equivalents and short-term investments of $645.7 million as of January 31, 2024; and that the company’s cash, cash equivalents and short-term investments will be sufficient to fund its operations into the second half of 2026. New risks and uncertainties may emerge from time to time, and it is not possible to predict all risks and uncertainties. No representations or warranties (expressed or implied) are made about the accuracy of any such forward-looking statements. Such forward-looking statements are subject to a number of material risks and uncertainties including but not limited to: potential utility, efficacy, potency, safety, clinical benefits, clinical response and convenience of Viridian’s product candidates; the relationship between the results from the positive data from completed or ongoing clinical trials and the results of ongoing or future clinical trials; that preliminary data may not be representative of final data; the timing, progress and plans for our ongoing or future research, preclinical and clinical development programs; trial protocols for ongoing clinical trials; expectations regarding the timing for regulatory filings; expectations regarding the timing for enrollment and data; uncertainty and potential delays related to clinical drug development; the duration and impact of regulatory delays in our clinical programs; the timing of and our ability to obtain and maintain regulatory approvals for our therapeutic candidates; manufacturing risks; competition from other therapies or products; estimates of market size; other matters that could affect the sufficiency of existing cash, cash equivalents and short-term investments to fund operations; our financial position and projected cash runway; our future operating results and financial performance; Viridian’s intellectual property position; the timing of preclinical and clinical trial activities and reporting results from same, including those risks set forth under the caption “Risk Factors” in our most recent quarterly report on Form 10-Q filed with the Securities and Exchange Commission (SEC) on November 13, 2023 and other subsequent disclosure documents filed with the SEC. Any forward-looking statement speaks only as of the date on which it was made. Neither the company, nor its affiliates, advisors, or representatives, undertake any obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law. These forward-looking statements should not be relied upon as representing the company’s views as of any date subsequent to the date hereof. CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS (amounts in thousands, except share and per share data) (unaudited) Three Months Ended December 31, Twelve Months Ended December 31, 2023 2022 2023 2022 Revenue: Collaboration Revenue - related party $ 72 $ 105 $ 314 $ 1,772 Total revenue 72 105 314 1,772 Operating Expenses: Research and development 38,558 39,317 159,765 100,894 General and administrative 32,993 9,854 94,999 35,182 Total operating expenses 71,551 49,171 254,764 136,076 Loss from operations (71,479 ) (49,066 ) (254,450 ) (134,304 ) Other income Interest and other income 5,535 3,449 18,563 4,916 Interest and other expense (916 ) (168 ) (1,847 ) (486 ) Net loss (66,860 ) (45,785 ) (237,734 ) (129,874 ) Change in unrealized gain (loss) on investments 403 300 728 (233 ) Comprehensive loss $ (66,457 ) $ (45,485 ) $ (237,006 ) $ (130,107 ) Net loss $ (66,860 ) $ (45,785 ) $ (237,734 ) $ (129,874 ) Net loss per share, basic and diluted $ (1.35 ) $ (1.13 ) $ (5.31 ) $ (4.05 ) Weighted-average shares used to compute basic and diluted loss per share 49,681,803 40,541,507 44,755,475 32,087,293 Viridian Therapeutics, Inc. Selected Financial Information Condensed Condolidated Balance Sheets (amounts in thousands) (unaudited) December 31, 2023 2022 Cash, cash equivalents and short-term investments $ 477,370 $ 424,550 Other assets 13,054 10,541 Total assets $ 490,424 $ 435,091 Total liabilities 48,402 40,027 Total stockholders’ equity 442,022 395,064 Total liabilities and stockholders’ equity $ 490,424 $ 435,091