Quellis Biosciences LLC

-- ALPHA-STAR Phase 1b/2 Trial in People with HAE Underway with Initial Proof-of-Concept Results Anticipated Mid-2024 -- -- Phase 1a Results Support STAR-0215’s Target Pro a Long-Acting Plasma Kallikrein Inhibitor with Estimated Half-Life of up to 117 Days -- -- Ended 4Q 2022 with Cash, Cash Equivalents, and Short-term Investments of $226M, Expected Cash Runway Through H1 2025 -- -- Webcast Today at 8:30am ET -- BOSTON--(BUSINESS WIRE)-- Astria Therapeutics, Inc. (NASDAQ:ATXS), a biopharmaceutical company developing STAR-0215 for the treatment of hereditary angioedema (HAE) and focused on life-changing therapies for rare and niche allergic and immunological diseases, today reported financial results for the fourth quarter and full year ended December 31, 2022 and provided a corporate update. “We made excellent progress with our STAR-0215 program in 2022, culminating in the promising initial Phase 1a results. These results support our vision for STAR-0215 to be the first-choice preventative therapy for HAE,” said Jill C. Milne, Ph.D., Chief Executive Officer of Astria. “The recent initiation of our ALPHA-STAR Phase 1b/2 trial marked an important step towards our goal of reducing the disease and treatment burden as we work to normalize the lives of people with HAE. We look forward to the initial proof-of-concept results from ALPHA-STAR expected in mid-2024.” STAR-0215 Clinical Development Positive preliminary results from the Phase 1a clinical trial of STAR-0215 in healthy subjects were announced in December 2022 and initial unblinded results from the first three cohorts were presented at the American Academy of Allergy Asthma, and Immunology Annual Meeting (AAAAI) in February 2023. STAR-0215 was well-tolerated at all doses studied. The results showed rapid and sustained drug levels, with an estimated half-life of up to 117 days and sustained target engagement with plasma kallikrein inhibition for at least three months. These results establish early proof of concept for STAR-0215 as a long-acting preventative therapy for HAE. The Company initiated the ALPHA-STAR Phase 1b/2 trial of STAR-0215 in people with HAE in February 2023. Initial proof-of-concept results are expected in mid-2024. The ALPHA-STAR trial is a global, open-label, proof-of-concept trial enrolling patients with HAE types I and II that is evaluating safety and tolerability, changes in HAE attack rate, pharmacokinetics, pharmacodynamics, and quality-of-life assessments. Following an initial run-in period, qualifying participants will be enrolled in either single or multiple dose cohorts. Data from up to 18 participants will evaluate efficacy and safety, and comparisons will be made against data collected in the run-in period. Pending proof-of-concept results from the ALPHA-STAR trial, Astria expects to progress directly to a pivotal trial. Astria is also evaluating the potential for administration of STAR-0215 every six months in additional cohorts in the Phase 1a trial, with preliminary results expected in the fourth quarter of 2023. These data, in conjunction with the ALPHA-STAR results, are expected to inform plans for the pivotal trial. Webcast Information The Company will host a webcast today at 8:30am ET. Interested parties may join the webcast via the Investors section of the Astria website, or with following the link . The webcast will be archived for 90 days. Fourth Quarter and Full Year 2022 Financial Results Cash Position: In December 2022, Astria closed a $115.0 million underwritten offering of common stock. As of December 31, 2022, Astria had cash, cash equivalents, and short-term investments of $226.4 million, compared to $116.6 million as of September 30, 2022. The Company expects that it has sufficient cash to fund its current operating plan through the first half of 2025. Net cash used in operating activities for the three months ended December 31, 2022, was $10.7 million, compared to $6.3 million for the three months ended December 31, 2021. Net cash used in operating activities for the full year 2022 was $43.5 million, compared to $30.2 million for the full year 2021. R&D Expenses: Research and development expenses were $9.6 million for the three months ended December 31, 2022, compared to $5.7 million for the three months ended December 31, 2021, and $34.3 million for the full year 2022, compared to $15.6 million for the full year 2021. The increase in research and development expenses was associated with STAR-0215’s advancement through IND-enabling activities into clinical trials. G&A Expenses: General and administrative expenses were $4.7 million for the three months ended December 31, 2022, compared to $3.8 million for the three months ended December 31, 2021, and $19.2 million for the full year 2022, compared to $14.8 million for the full year 2021. Operating Loss: Loss from operations was $14.3 million for the three months ended December 31, 2022, compared to $9.5 million for the three months ended December 31, 2021, and $53.5 million for the full year 2022, compared to $195.0 million for the full year 2021. The year-over-year decrease in net loss was largely attributable to the $164.6 million in acquired in-process R&D expense associated with acquiring Quellis Biosciences in 2021. Net Loss: Net loss was $13.3 million for the three months ended December 31, 2022, compared to a net loss of $9.5 million for the three months ended December 31, 2021, and $51.8 million, or $3.55 per share for the full year 2022, compared to $194.9 million, or $21.84 per share, for the full year 2021. Net Loss Per Share Basic and Diluted: Net loss per share basic and diluted was $0.72 for the three months ended December 31, 2022, compared to a net loss basic and diluted of $0.73 per share for the three months ended December 31, 2021. About Astria Therapeutics: Astria Therapeutics is a biopharmaceutical company, and our mission is to bring life-changing therapies to patients and families affected by rare and niche allergic and immunological diseases. Our lead program, STAR-0215, is a monoclonal antibody inhibitor of plasma kallikrein in clinical development for the treatment of hereditary angioedema. Learn more about our company on our website, , or follow us on Twitter and Instagram @AstriaTx and on Facebook and LinkedIn. About STAR-0215: STAR-0215 is a monoclonal antibody inhibitor of plasma kallikrein in development for the treatment of HAE. Our vision is for STAR-0215 to be the first-choice preventative treatment for people with HAE dosed once every three or six months and to make substantial progress towards normalizing their lives. The Phase 1b/2 ALPHA-STAR trial evaluating STAR-0215 in HAE patients is ongoing, with proof-of-concept results expected in mid-2024. Forward Looking Statements This press release contains forward-looking statements within the meaning of applicable securities laws and regulations including, but not limited to, statements regarding: expectations regarding the potential significance of the preliminary results from the Phase 1a STAR-0215 trial and the anticipated nature and timing of receipt of the data from the two additional cohorts in such trial; expectations regarding the timing and nature of the anticipated proof of concept results from the ALPHA-STAR Phase 1b/2 clinical trial of STAR-0215; the longer term development plans for STAR-0215, including the plan, assuming positive results, to move directly from the ALPHA-STAR trial into a pivotal trial; the potential attributes and differentiated pro STAR-0215 as a treatment for HAE, including those suggested by the preliminary and initial results from the STAR-0215 Phase 1a trial and market research, and our goals and vision for STAR-0215; the potential commercial opportunity for STAR-0215 in HAE and the likelihood that it can effectively compete in HAE, assuming its approved; the need for effective treatments for HAE; the potential for six-month dosing of STAR-0215; the Company’s anticipated cash runway; and the Company’s goal to meet the unmet needs of patients with rare and niche allergic and immunological diseases. The use of words such as, but not limited to, “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “goals,” “intend,” “may,” “might,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “will,” or “would” and similar words expressions are intended to identify forward-looking statements. Forward-looking statements are neither historical facts nor assurances of future performance. Instead, they are based on the Company’s current beliefs, expectations and assumptions regarding the future of its business, future plans and strategies, future financial performance, results of pre-clinical and clinical results of the Company’s product candidates and other future conditions. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including risks and uncertainties: changes in applicable laws or regulations; the possibility that we may be adversely affected by other economic, business, and/or competitive factors, including the COVID-19 pandemic; risks inherent in pharmaceutical research and development, such as: adverse results in our drug discovery, preclinical and clinical development activities, the risk that the results of pre-clinical studies may not be replicated in clinical studies, that the preliminary results from the Phase 1a trial may not be indicative of the final results, that the results of early stage clinical studies, such as the preliminary results from the Phase 1a trial, may not be replicated in later stage clinical studies, including the ALPHA-STAR trial, the risk that we may not be able to enroll sufficient patients in our clinical trials on a timely basis, and the risk that any of our clinical trials may not commence, continue or be completed on time, or at all; decisions made by, and feedback received from, the U.S. Food and Drug Administration and other regulatory authorities on our regulatory and clinical trial submissions and other feedback from potential clinical trial sites, including investigational review boards at such sites, and other review bodies with respect to STAR-0215 and any other future development candidates; our ability to manufacture sufficient quantities of drug substance and drug product for STAR-0215 and any other future product candidates on a cost-effective and timely basis, and to develop dosages and formulation for STAR-0215 and any other future product candidates that are patient-friendly and competitive; our ability to develop biomarker and other assays, along with the testing protocols therefore; our ability to obtain, maintain and enforce intellectual property rights for STAR-0215 and any other future product candidates; our potential dependence on collaboration partners; competition with respect to STAR-0215 or any of our other future product candidates; the risk that survey results and market research may not be accurate predictors of the commercial landscape for HAE, the ability of STAR-0215 to compete in HAE and the anticipated position and attributes of STAR-0215 in HAE based on its clinical data to date, pre-clinical profile, pharmacokinetic modeling, market research and other data; our ability to manage our cash usage and the possibility of unexpected cash expenditures; our ability to obtain necessary financing to conduct our planned activities and to manage unplanned cash requirements; the risks and uncertainties related to our ability to recognize the benefits of any additional acquisitions, licenses or similar transactions; and general economic and market conditions; as well as the risks and uncertainties discussed in the “Risk Factors” section of our Annual Report on Form 10-K for the period ended December 31, 2021 and in other filings that we make with the Securities and Exchange Commission (“SEC”), including our Current Report on Form 8-K filed on December 15, 2022. New risks and uncertainties may emerge from time to time, and it is not possible to predict all risks and uncertainties. The Company may not actually achieve the forecasts or expectations disclosed in our forward-looking statements, and investors and potential investors should not place undue reliance on the Company’s forward-looking statements. Neither the Company, nor its affiliates, advisors or representatives, undertake any obligation to publicly update or revise any forward-looking statement, whether as 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. Astria Therapeutics, Inc. Consolidated Statements of Operations (In thousands, except share and per share data) (Audited) Three Months Ended December 31, Year Ended December 31, 2022 2021 2022 2021 Operating expenses: Research and development $ 9,591 $ 5,693 $ 34,264 $ 15,552 General and administrative 4,699 3,816 19,239 14,807 Acquired in-process research and development - - 164,617 Total operating expenses 14,290 9,509 53,503 194,976 Loss from operations (14,290) (9,509) (53,503) (194,976) Other income (expense): Interest and investment income 1,018 32 1,724 122 Other expense, net 9 (16) (55) (58) Total other income, net 1,027 16 1,669 64 Net loss (13,263) (9,493) (51,834) (194,912) Dividend on convertible preferred stock related to beneficial conversion feature and issuance costs - - - (24,437) Net loss attributable to common shareholders $ (13,263) $ (9,493) $ (51,834) $ (219,349) Net loss per share attributable to common shareholders - basic and diluted $ (0.72) $ (0.74) $ (3.55) $ (24.58) Weighted-average common shares outstanding used in net loss per share - basic and diluted 18,417,203 12,830,782 14,620,618 8,925,173 Astria Therapeutics, Inc. Selected Consolidated Balance Sheets Data (In thousands) (Audited) December 31, December 31, 2022 2021 Assets Cash and cash equivalents $ 20,525 $ 86,508 Short-term investments 205,912 39,000 Right-of-use asset 948 394 Other current and long-term assets 3,248 1,612 Total assets 230,633 127,514 Liabilities and stockholders’ equity Current portion of operating lease liabilities 582 365 Long-term portion of operating lease liabilities 357 - Other current and long-term liabilities 8,478 4,838 Total liabilities 9,417 5,203 Total stockholders’ equity $ 221,216 $ 122,311 Astria Therapeutics, Inc. Selected Consolidated Statements of Cash Flows Data (In thousands) (Audited) Year Ended December 31, 2022 2021 Net cash used in operating activities $ (43,533) $ (30,151) Net cash used in by investing activities (167,129) (12,555) Net cash provided by financing activities 144,721 104,284 Net (decrease) increase in cash, cash equivalents and restricted cash $ (65,941) $ 61,578

When Chris Garabedian set out early last year to raise money for a second Perceptive Advisors fund focused on early-stage biotech startups, it took just a matter of months to draw interest from investors willing to commit more than $1 billion total. The fund could have closed with that much money, maybe more, said Garabedian, the portfolio manager. But it ended up with a little more than half of that, which he described as “the right size.” Garabedian acknowledges the large sums raised by large venture capital firms, many of them pumping money into platform technologies with potential for a broad range of applications. Then he points out that that’s exactly where Perceptive Xontogeny Ventures (PXV) doesn’t want to be. Rather than looking at platforms, Garabedian said PXV is scouting products. He’s looking for biotech companies that have an asset showing a promising signal and a path to reach clinical testing. “We’re very product focused,” he said. “We’re not trying to do next-generation CRISPR, which requires a couple of hundred million dollars before a candidate gets to the clinic.” On Wednesday, Perceptive Advisors announced its second fund to invest in biotech startups with promising potential products. PXV II closed with $515 million in financial commitments. The new fund, which will invest in 10 to 12 startups, comes about 18 months after the firm unveiled PXV I, which totaled $210 million. The new Perceptive fund starts with lessons learned from the first fund. Garabedian said that PXV I was targeting up to a dozen investments in drug companies raising Series A rounds ranging from $15 million to $25 million—enough to get them through late-preclinical research, perhaps through early clinical testing in healthy volunteers. However, Garabedian said that founders wanted to go for bigger Series A financings to advance their technologies to proof-of-concept testing. The larger rounds meant that PXV I didn’t have much left over to deploy in Series B rounds. The larger fund size of PXV II is an acknowledgement that the Series A rounds may be larger, ranging from $25 million to $40 million. The fund size also affords the flexibility to participate in Series B financings. PXV II is not starting from scratch. Based on the outreach and the diligence done for the first fund, Garabedian and his team already have connections and a pipeline of potential investments. Some of those startups could come from Xontogeny, a biotech accelerator that Garabedian also runs. Launched in 2017, Xontogeny provides seed financing and other support to these startups, some of which are led by first-time founders from academia. Garabedian, a former executive at Gilead Sciences, Celgene, and Sarepta Therapeutics, mentors those companies. Though Xontogeny and PXV operate separately, there are connections between them. Xontogeny’s first investor was Perceptive Advisors. Fred Callori, Xontogeny’s senior vice president of corporate development, and Ben Askew, the accelerator’s head of R&D, are partners in PXV Fund II. Also, startups in the accelerator may be considered for Series A investment from the PXV fund. Two Xontogeny graduates secured PXV I Series A funding and went on to exit events earlier this year. Quellis Biosciences was acquired by Catabasis Pharmaceuticals in late January; days later, Landos Biopharma priced its IPO. Most of PXV I’s investments were in drug developers, though the fund did invest in one medical device company and one contract development and manufacturing organization. PXV II will continue the first fund’s drug focus, but Garabedian said that the life sciences industry is changing and innovation can also be found in drug discovery tools. He added that companies developing artificial intelligence and machine learning technologies offer additional investment opportunities, along with startups working to digitize health care and bring treatment into the home. Separate from the close of the second PXV fund, Xontogeny announced five new portfolio companies that have joined the accelerator. They are: • Nephraegis Therapeutics is developing potential first-in-class drug for renal disease. The company’s lead program, NPH-022, is for the prevention of acute kidney injury following surgical procedures, such as heart valve replacement, abdominal surgery, and liver transplant. The drug, a next-generation epoxyeicosatraenoic acid (EET) analog, is being developed in both intravenous and oral formulations. • NephroDI Therapeutics is also focusing on kidney disorders. The company’s lead compound, NDI-5033, is an experimental treatment for Nephrogenic Diabetes Insipidus, a rare, congenital disease in which the kidneys do not respond to the anti-diuretic hormone vasopressin, leading patients to produce extremely large amounts of dilute urine. The small molecule is an oral AMPK activator. • Peroxitech is developing a treatment for acute lung injury. The company’s lead program is a peptide that works through a novel pathway to eliminate molecules that build up in the lung leading to respiratory failure. The startup is a University of Pennsylvania spinout. • Shifa Biomedical aims to treat dyslipidemia, which is a high level of lipids in the blood. The company is developing an oral small molecule intended to block PCSK9, an enzyme that plays a role in cholesterol metabolism. The biotech’s drug, P-21, has data showing it could provide an alternative to currently available PCSK9 inhibitors, antibodies that are injectable drugs. The technology was developed by the Malvern, Pennsylvania-based biotech with National Institute of Health grant funding. • Tellus Therapeutics, based on research from Duke University, is developing a treatment for babies born at risk of brain injury. The company’s lead program, TT-20, is made from compounds found in breast milk that have been shown to repair brain damage in animal models. The treatment will be delivered on top of parenteral nutrition given to infants in neonatal intensive care units. Photo by Perceptive Advisors

Progressed lead programs VRDN-001 and VRDN-002 towards clinical trials in Thyroid Eye Disease (TED); both IND filings expected by the end of 2021 Completed integration and name change to Viridian Therapeutics following October 2020 merger and concurrent financing Ended 2020 with a strong cash position of $127.6 million, and runway into the second half of 2023 Strengthened leadership team with the appointment of Dr. Jonathan Violin as CEO and additions of Dr. Barrett Katz as CMO and Dr. Vahe Bedian as Chief Scientist BOULDER, Colo., March 25, 2021 (GLOBE NEWSWIRE) -- Viridian Therapeutics, Inc. (NASDAQ: VRDN), a biopharmaceutical company advancing new treatments for patients suffering from serious diseases but underserved by today’s therapies, today announced financial results for the fourth quarter and year ended December 31, 2020 and provided corporate updates. “2020 was a transformative year for Viridian. We completed the acquisition of private Viridian Therapeutics and a concurrent financing, revamped our business and added $115.0 million to our balance sheet in the fourth quarter. We have also strengthened our management team and now have multiple product candidates with the potential to become meaningful treatments for patients suffering from TED,” said Jonathan Violin, Ph.D., President and Chief Executive Officer of Viridian. “As we enter 2021, we look forward to reaching important R&D milestones in our TED programs, continuing to build out our team and expanding our pipeline beyond TED.” Recent Highlights Jonathan Violin, Ph.D. appointed President, CEO and Director– Dr. Violin co-founded privately-held Viridian Therapeutics, which was acquired by the Company in October 2020. Dr. Violin also founded and served as CEO of two drug discovery companies, Quellis Biosciences and Dianthus Therapeutics, and co-founded and held several executive positions at Trevena, Inc. Barrett Katz M.D. joined Viridian as Chief Medical Officer (CMO)– Dr. Katz is an internationally recognized neuro-ophthalmologist. He comes to Viridian from BridgeBio Pharma, Inc. where he developed therapeutics to treat orphan eye diseases. Prior to BridgeBio, he was CMO at GenSight Biologics where he oversaw early- and late-stage clinical programs. Vahe Bedian, Ph.D. appointed as Chief Scientist– Dr. Bedian co-founded privately-held Viridian Therapeutics. Dr. Bedian also co-founded and served as Chief Scientific Officer of Quellis Biosciences and Dianthus Therapeutics. He also previously held leadership positions in therapeutic antibody research and development at AstraZeneca and Pfizer. Changed Company name to Viridian Therapeutics–The new name reflects the evolution of the Company and its patient-centric model of innovation. Development Pipeline Overview and Update Viridian is developing multiple product candidates to treat patients who suffer from thyroid eye disease (TED), a debilitating auto-immune disease that causes inflammation and fibrosis of the orbit and tissues surrounding the eye which can lead to proptosis, or bulging of the eyes, redness and swelling, double vision, pain, and potential blindness. TED significantly impacts quality of life, imposing a high physical and mental burden on patients. There is currently one Food and Drug Administration (FDA)-approved treatment for TED, which is an intravenously administered monoclonal antibody that targets the insulin-like growth factor-1 receptor (IGF-1R). Viridian’s most advanced product candidate is VRDN-001, an intravenously administered anti-IGF-1R monoclonal antibody licensed from ImmunoGen, Inc. This antibody had previously been developed in oncology as AVE-1642 and studied in over 100 patients. The pharmacokinetics, pharmacodynamics, safety, and tolerability data from that clinical program has informed the Company’s plans to further evaluate VRDN-001 in TED. Manufacturing is underway and the Company expects to IND in the fourth quarter of 2021, with initial proof of concept data in patients expected in the second quarter of 2022. Viridian’s second product candidate, VRDN-002, is a distinct anti-IGF-1R antibody that incorporates half-life extension technology and is intended for subcutaneous administration. VRDN-002 manufacturing is underway, and the Company expects to IND before the end of 2021. The Company expects to initiate clinical development with a Phase 1 single ascending dose trial to explore safety, tolerability, pharmacokinetics, and target engagement of VRDN-002 in healthy volunteers. Data from this trial is expected in mid-year 2022 and the Company expects to initiate the dosing of patients later in 2022. Viridian is also pursuing multiple hypotheses within the VRDN-003 program that may offer additional improvements to the class of IGF-1R targeted therapeutic antibodies. In addition, the Company continues to advance its efforts beyond IGF-1R and TED and is focused on opportunities that will leverage validated mechanisms, technologies, and modalities to bring new therapeutic options to patients underserved by today’s options. The most advanced of these programs is VRDN-004, a therapeutic antibody program currently in discovery stage. The Company also continues to evaluate other targets and indications for a future VRDN-005 program. 2020 Financial Results Cash Position and Runway: Cash, cash equivalents and short-term investments were $127.6 million as of December 31, 2020, compared to $30.1 million as of September 30, 2020 and $26.8 million as of December 31, 2019. Viridian believes that its current cash, cash equivalents and short-term investments will be sufficient to fund its operations into the second half of 2023. Revenue: Revenue was $0.1 million during the fourth quarter and $1.1 million for the year ended December 31, 2020, compared to $0.9 million and $4.5 million, respectively for the comparable periods in 2019. The decrease in revenue was primarily due to a decrease in research and development activities related to legacy microRNA assets that were reimbursable to the Company under a prior collaboration agreement. Research and Development Expenses: Research and development expenses were $15.3 million for the fourth quarter of 2020 and $28.3 million for the year ended December 31, 2020, compared to $8.4 million and $34.8 million, respectively for the comparable periods in 2019. The increase in research and development expenses during the fourth quarter was primarily attributable to increased license fees related to the Company’s license agreement with Xencor. The year over year decrease in research and development expenses was primarily attributable to a decrease in clinical and related manufacturing development activities primarily associated with the Company’s legacy microRNA programs and decreases in personnel related costs including restructuring charges in 2020. These decreases were partially offset by an increase in licensing fees primarily attributable to the Company’s license agreement with Xencor. Acquired In-Process Research and Development (IPR&D) Expense: Acquired IPR&D expense was $69.9 million during the fourth quarter of 2020 and for the year ended December 31, 2020. IPR&D expense resulted from the acquisition of private Viridian Therapeutics in October 2020. The acquisition cost allocated to acquired IPR&D with no alternative future use was recorded as expense at the acquisition date. No acquired IPR&D expenses were incurred in 2019. General and Administrative Expenses: General and administrative expenses were $5.5 million for the fourth quarter of 2020 and $13.3 million for the year ended December 31, 2020, compared to $2.5 million and $11.6 million, respectively for the comparable periods in 2019. The fourth quarter and year over year increases in general and administrative expenses were due primarily to increases in professional and personnel related costs, including consulting and contract labor. Net Loss: The Company’s net loss was $90.7 million, or $23.07 per share, for the fourth quarter of 2020, and $110.7 million, or $31.13 per share for the year ended December 31, 2020, compared to $10.1 million, or $4.69 per share, for the fourth quarter of 2019 and $41.9 million, or $20.04 per share for the year ended December 31, 2019. Shares Outstanding: As of March 15, 2021, Viridian had 30,886,700 shares of common stock outstanding on an as-converted basis, which included 7,230,651 shares of common stock and 23,656,049 shares of common stock issuable upon the conversion of 354,823 shares of preferred stock. Conference Call Information The Viridian Therapeutics management team will host a conference call and webcast today at 4:30 p.m. ET to provide corporate updates and discuss the Company’s financial results for the fourth quarter and year ended December 31, 2020. To access the call, please dial 877-407-0789 (domestic) or 201-689-8562 (international) and provide the passcode 13717079. A live webcast of the call will be available on the Investors section of the Viridian Therapeutics website at and a replay of this conference call will be available approximately one hour after its completion. About Viridian Therapeutics, Inc. Viridian Therapeutics is a biotechnology company advancing new treatments for patients suffering from serious diseases but underserved by today’s therapies. Viridian’s most advanced program, VRDN-001, is an anti-IGF-1R monoclonal antibody in development for thyroid eye disease (TED), a debilitating auto-immune disease that causes inflammation and fibrosis within the orbit of the eye which can cause double vision, pain, and potential blindness. Patients with severe disease often require multiple remedial surgeries to the orbit, eye muscles and eyelids Viridian is based in Boulder, Colorado, and Waltham, Massachusetts. Learn more about Viridian and our programs at Follow us on Twitter @ViridianThera and on LinkedIn. Note Regarding 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 Viridian’s expectation, strategy, plans and intentions. Forward-looking statements include, without limitation, statements regarding the Company’s expectations and guidance regarding its business plans and objectives for its product candidates, including the therapeutic potential and clinical benefits thereof, and pipeline, projected cash runway, the timing, progress and plans for the Company’s ongoing and future research and clinical development programs, future regulatory interactions, expectations regarding the timing for data, and the timing of the Company’s IND filings for VRDN-001 and VRDN-002. Forward-looking statements are neither historical facts nor assurances of future performance. Instead, they are based on our current beliefs, expectations and assumptions regarding the future of our business, future plans and strategies, our clinical results and other future conditions. 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. We may not actually achieve the forecasts disclosed in our forward-looking statements, and you should not place undue reliance on our forward-looking statements. Such forward-looking statements are subject to a number of material risks and uncertainties including but not limited to the effects from the COVID-19 pandemic on the company’s clinical activities, business and operating results; uncertainty and potential delays related to clinical drug development; smaller than anticipated market opportunities for the company’s product candidates; manufacturing risks; competition from other therapies or products; other matters that could affect the sufficiency of existing cash, cash equivalents and short-term investments to fund operations; the company’s future operating results and financial performance; the timing of pre-clinical and clinical trial activities and reporting results from same; and those risks set forth under the caption “Risk Factors” in the Company’s Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) on November 12, 2020 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 we, nor our 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 our views as of any date subsequent to the date hereof. Viridian Contacts: Investors: Dan Ferry LifeSci Advisors 617-430-7576 IR@viridiantherapeutics.com Media: Darby Pearson Verge Scientific Communications 703-587-0831 PR@viridiantherapeutics.com Viridian Therapeutics, Inc. Condensed Consolidated Statements of Operations and Comprehensive Loss (in thousands, except share and per share data) (unaudited) Three Months Ended December 31, Year Ended December 31, 2020 2019 2020 2019 Revenue: Collaboration revenue $ 54 $ 837 $ 735 $ 4,308 Grant revenue — 43 315 153 Total revenue 54 880 1,050 4,461 Operating expenses: Research and development 15,254 8,417 28,304 34,794 General and administrative 5,537 2,534 13,265 11,646 Acquired in-process research and development 69,861 — 69,861 — Total operating expenses 90,652 10,951 111,430 46,440 Loss from operations (90,598 ) (10,071 ) (110,380 ) (41,979 ) Other income (expense): Interest and other income 36 123 173 941 Interest and other expense (180 ) (170 ) (508 ) (835 ) Net loss (90,742 ) (10,118 ) (110,715 ) (41,873 ) Change in unrealized gain (loss) on investments — (3 ) (8 ) 3 Comprehensive loss $ (90,742 ) $ (10,121 ) $ (110,723 ) $ (41,870 ) Net loss $ (90,742 ) $ (10,118 ) $ (110,715 ) $ (41,873 ) Net loss per share, basic and diluted $ (23.07 ) $ (4.69 ) $ (31.13 ) $ (20.04 ) Weighted-average shares used to compute basic and diluted net loss per share 3,932,917 2,158,695 3,557,065 2,089,094 Viridian Therapeutics, Inc. Selected Financial Information Condensed Consolidated Balance Sheet Data (amounts in thousands) (unaudited) December 31, 2020 2019 Cash and cash equivalents $ 45,897 $ 24,846 Short-term investments $ 81,742 $ 1,999 Total assets $ 131,255 $ 30,262 Notes payable, inclusive of current portion $ — $ 8,304 Total liabilities $ 11,218 $ 14,508 Total stockholders’ equity $ 120,037 $ 15,754