Pyropoikilocytosis, Hereditary

Ilofotase alfa demonstrates pharmacologically relevant effect on disease specific biomarkers and a positive safety pro adult patients with hypophosphatasia; Data to be presented by Lothar Seefried, MD, at the American Society for Bone and Mineral Research’s Symposium on Rare Bone Diseases on Thursday, October 12, 2023 UTRECHT, The Netherlands--(BUSINESS WIRE)-- AM-Pharma B.V. today announced positive clinical results from the Phase 1b study evaluating the company’s proprietary recombinant alkaline phosphatase, ilofotase alfa, as potential enzyme replacement therapy in adult hypophosphatasia (HPP) patients. HPP is an inherited metabolic disease, characterized by low activity of tissue non-specific alkaline phosphatase (TNAP), caused by mutations at the ALPL gene. This low activity leads to accumulation of inorganic pyrophosphate (PPi), a strong inhibitor of mineralization, as well as pyridoxal-5’-phosphate (PLP), an important cofactor for enzymes. The disease can range widely in severity, however most adults with HPP suffer from recurrent fractures, muscle weakness, joint diseases and pain.1 In the open-label pilot trial, a total of 12 patients were randomly assigned to one of two treatment arms and received a single intravenous dose of either 0.8 mg/kg or 3.2 mg/kg. Patients were followed for 10 days after dosing to evaluate changes in the biochemical fingerprint of HPP, safety and tolerability. The primary objectives for the study were changes in serum levels of PLP and PPi as compared to baseline. For PLP, the maximum proportional reduction was 35% in the 0.8 mg/kg cohort and 66% in the 3.2 mg/kg cohort. The mean time to return to 90% of baseline was 55 hours in the low-dose cohort and 168 hours in the high-dose cohort. The maximal proportional reduction for PPi was 36% in the 0.8 mg/kg cohort and 77% in the 3.2 mg/kg cohort. The mean time to return to 90% of baseline was 9.3 hours in the low-dose cohort and 53 hours in the high-dose cohort. In addition, ilofotase alfa was well-tolerated with pharmacokinetics consistent with observations from previous clinical studies. “The pronounced effect on all HPP disease relevant biomarkers together with a positive safety and tolerability profile, consistent with previous clinical studies with ilofotase alfa, supports its further development as a treatment option for adult HPP patients who are currently underserved,” said Juliane Bernholz, PhD, Chief Executive Officer of AM-Pharma. The full dataset will be presented at the American Society for Bone and Mineral Research – Rare Bone Disease Alliance (ASBMR-RBDA) Symposium at the Vancouver Convention Centre in Vancouver, BC, Canada. The presentation by Lothar Seefried, MD, from the University of Wuerzburg, lead investigator of the trial, is scheduled for 3:00 pm PDT on Thursday, October 12, 2023, in session III: Latest in Clinical Trials. "Considering the wide range of clinical manifestations of HPP, there's an unequivocal need to expand therapeutic opportunities for these patients," added Lothar Seefried, MD. "The encouraging early results confirming dose-dependent reductions of PLP and PPi levels upon treatment with ilofotase alfa underline the compound's therapeutic potential in that indication and I'm looking forward to evaluating ilofotase alfa in next stage clinical studies." About ilofotase alfa Ilofotase alfa is a proprietary recombinant alkaline phosphatase, constructed from two human isoforms of alkaline phosphatase, that has been shown to be stable and highly active in multiple clinical trials. The recombinant enzyme displays exquisite activity towards dephosphorylating and detoxifying damage-associated molecular patterns (DAMPs) and pathogen-associated molecular patterns (PAMPs) such as lipopolysaccharide (LPS), ATP, ADP and other extracellular substrates that drive acute inflammation, coagulation and microvascular ischemia found in kidney following sepsis or ischemia-induced damage. Research has shown that ATP dephosphorylation has a double effect in protecting against kidney injury. When the pro-inflammatory ATP is dephosphorylated, the resulting adenosine further reduces inflammation through the activation of the immunosuppressive adenosine A2a receptor pathway. In hypophosphatasia, ilofotase alfa addresses elevated levels of pyridoxal-5′-phosphate (PLP), inorganic pyrophosphates (PPi), two disease related biomarkers that are related to, for example, bone mineralization and pain sensation. About hypophosphatasia HPP is a rare inherited disease characterized by a deficiency in alkaline phosphatase which is essential for bone mineralization and general functions. HPP patients of all ages can exhibit a wide variety of symptoms that worsen overtime, including bone injury, severe muscle pain and weakness. The disease can also lead to life-threatening complications in infants. The US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) granted AM-Pharma Orphan Drug Designation (ODD) for ilofotase alfa in HPP. In preclinical models, ilofotase alfa was able to achieve improved overall survival of severe disease, as well as restoration of phenotypes associated with HPP. About AM-Pharma AM-Pharma strives to develop medicines for patients confronted with severe medical conditions. Our proprietary asset, ilofotase alfa, is being developed for the treatment of patients with acute kidney injury and has been granted FDA fast-track status. We also develop ilofotase alfa in the severe rare disease hypophosphatasia where ilofotase alfa has orphan drug status in the US and EU. With approximately 1,000 subjects evaluated to date in clinical trials, ilofotase alfa has a proven safety pro a demonstrated kidney benefit. We are a dedicated team driven to bring treatment options to severely ill patients, their families and acute care professionals. Find out more about us online at: . 1 Tournis, S., Yavropoulou, M. P., Polyzos, S. A., & Doulgeraki, A. (2021). Hypophosphatasia. Journal of Clinical Medicine, 10(23), 5676. View source version on businesswire.com: Contacts Investors: Juliane Bernholz, Chief Executive Officer j.bernholz@am-pharma.com Media: Trophic Communications Eva Mulder or Priscillia Perrin +31 6 52 33 15 79 am-pharma@trophic.eu Source: AM-Pharma View this news release online at:

Advancing ilofotase alfa into a Phase 2 study for lead indication cardiac surgery-associated renal damage; Phase 1b study of ilofotase alfa as enzyme replacement therapy in hypophosphatasia patients being finalized, results to be announced before end of 2023; Juliane Bernholz, AM-Pharma’s Chief Operating Officer since 2019, succeeds Erik van den Berg as Chief Executive Officer. Erik van den Berg continues as board member and strategic advisor UTRECHT, The Netherlands--(BUSINESS WIRE)-- AM-Pharma B.V. today announced an updated clinical development strategy to evaluate ilofotase alfa in two indications. The company’s proprietary compound ilofotase alfa is a recombinant alkaline phosphatase with an extensive clinical record and proven safety profile. Based on the significant improvements in Major Adverse Kidney Events by day 90 (MAKE90) observed in the company’s Phase 2 and Phase 3 studies with ilofotase alfa in sepsis-associated acute kidney injury (AKI), AM-Pharma advances ilofotase alfa as a potential preventive treatment for Cardiac Surgery-Associated Renal Damage (CSA-RD). In addition, based on preclinical data showing improved overall survival and restored phenotypes associated with hypophosphatasia (HPP), the company develops ilofotase alfa as a potential enzyme replacement therapy in HPP. AM-Pharma also announced the appointment of Juliane Bernholz, Ph.D., as Chief Executive Officer. Juliane previously served as AM-Pharma’s Chief Operating Officer. She will succeed Erik van den Berg, Chief Executive Officer since 2011, who will join the company’s Supervisory Board and remain as strategic advisor to the company. To align the organization with ongoing activities, AM-Pharma has completed a reduction of its workforce. “With the updated development strategy and funding in place for the CSA-RD Phase 2 study and awaiting imminent results from the Phase 1b in HPP, I am excited to pass on the baton to Juliane after serving 12 years as AM-Pharma’s CEO,” said Erik van den Berg, Supervisory Board member and former Chief Executive Officer of AM-Pharma. “Juliane’s expertise in clinical and corporate development, together with her experience as AM-Pharma’s COO over the past four years, will be extremely useful as the company pursues its development strategy. I am grateful to all at AM-Pharma and the clinical site staff who have contributed to the development of ilofotase alfa from initial studies to the current clinical stage of the program.” “Ilofotase alfa has reduced MAKE90 consistently in two large clinical studies involving over 1,000 subjects with sepsis and AKI. These findings together with an outstanding tolerability pro a potential reno-protective effect, which we aim to demonstrate in the upcoming Phase 2 study evaluating ilofotase alfa in CSA-RD. Together with our recently initiated clinical program studying ilofotase alfa as enzyme replacement therapy in adult patients with HPP, AM-Pharma is focusing on helping patients with severe illness, who are medically underserved, and I am working closely with the AM-Pharma team to achieve this goal,” said Juliane Bernholz, Chief Executive Officer of AM-Pharma. The planned double-blind, randomized, placebo-controlled Phase 2 clinical trial will evaluate the safety and efficacy of ilofotase alfa in 150 patients, who are at risk of developing renal damage based on pre-operative kidney function, scheduled for complex open-heart surgery performed with the use of a cardiopulmonary bypass pump. In parallel, the company is currently exploring the potential of ilofotase alfa as an enzyme replacement therapy in HPP, a rare disorder impairing bone metabolism and muscle strength, in a Phase 1b study in adult patients. The company anticipates announcing the first clinical results before the end of 2023. AM-Pharma’s revised clinical strategy is backed by existing investors who have provided additional funding. “AM-Pharma remains committed to demonstrate the potential of ilofotase alfa for critically ill kidney and HPP patients,” continued Mark Altmeyer, Chairman of the Board at AM-Pharma. “We look forward to collaborating with Juliane and thank Erik for his unyielding dedication over the past 12 years and we are excited to have him join the Board.” About ilofotase alfa Ilofotase alfa is a proprietary recombinant alkaline phosphatase, constructed from two human isoforms of alkaline phosphatase, that has been shown to be stable and highly active in multiple clinical trials. The recombinant enzyme displays exquisite activity towards dephosphorylating and detoxifying damage-associated molecular patterns (DAMPs) and pathogen-associated molecular patterns (PAMPs) such as lipopolysaccharide (LPS), ATP, ADP and other extracellular substrates that drive acute inflammation, coagulation and microvascular ischemia found in kidney following sepsis or ischemia-induced damage. Research has shown that ATP dephosphorylation has a double effect in protecting against kidney injury. When the pro-inflammatory ATP is dephosphorylated, the resulting adenosine further reduces inflammation through the activation of the immunosuppressive adenosine A2a receptor pathway. In hypophosphatasia, ilofotase alfa addresses elevated levels of pyridoxal-5′-phosphate (PLP), inorganic pyrophosphates (PPi), two disease related biomarkers that are related to, for example, bone mineralization and pain sensation. About cardiac surgery-associated renal damage and hypophosphatasia CSA-RD is a clinical complication that arises from acute kidney injury following open heart surgery performed with the use of a cardiopulmonary bypass pump. AKI occurs in about 30% of patients undergoing this surgical procedure, and there are currently no therapeutic options to prevent the long-term renal impairment caused by CSA-RD. HPP is a rare inherited disease characterized by a deficiency in alkaline phosphatase which is essential for bone mineralization and general functions. HPP patients of all ages can exhibit a wide variety of symptoms that worsen overtime, including bone injury, severe muscle pain and weakness. The disease can also lead to life-threatening complications in infants. The US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) granted AM-Pharma Orphan Drug Designation (ODD) for ilofotase alfa in HPP. In preclinical models, ilofotase alfa was able to achieve improved overall survival of severe disease, as well as restoration of phenotypes associated with HPP. About AM-Pharma AM-Pharma strives to develop medicines for patients confronted with severe medical conditions. Our proprietary asset, ilofotase alfa, is being developed for the treatment of patients with acute kidney injury and has been granted FDA fast-track status. We also develop ilofotase alfa in the severe rare disease hypophosphatasia where ilofotase alfa has orphan drug status in the US and EU. With approximately 1,000 subjects evaluated to date in clinical trials, ilofotase alfa has a proven safety pro a demonstrated kidney benefit. We are a dedicated team driven to bring treatment options to severely ill patients, their families and acute care professionals. Find out more about us online at: . View source version on businesswire.com: Contacts Investors: Juliane Bernholz, Chief Executive Officer j.bernholz@am-pharma.com Media: Trophic Communications Eva Mulder or Priscillia Perrin +31 6 52 33 15 79 am-pharma@trophic.eu Source: AM-Pharma View this news release online at:

-- Proof-of-Concept Achieved for RLYB212; Showed Rapid and Complete Elimination of Transfused HPA-1a Positive Platelets in HPA-1a Negative Subjects -- -- Phase 1 Multiple Dose Cohort RLYB212 Study Initiated -- -- Phase 1 Multiple Ascending Dose Study of RLYB116 Continues to Progress; Safety, PK and PD Data Expected in 4Q 2023 -- -- $169.0 million cash, cash equivalents and marketable securities as of December 31, 2022; Provides Runway into 1Q 2025 -- NEW HAVEN, Conn.--(BUSINESS WIRE)-- Rallybio Corporation (Nasdaq: RLYB), a clinical-stage biotechnology company committed to identifying and accelerating the development of life-transforming therapies for patients with severe and rare diseases, today reported financial results for the fourth quarter and full year ended December 31, 2022 and provided an update on recent program and corporate developments. “In 2023, we will fortify our position as a leader in the development of transformative medicines for underserved rare diseases,” said Martin Mackay, Ph.D., Chief Executive Officer of Rallybio. “Earlier today, we announced proof-of-concept for RLYB212, our product candidate for the prevention of fetal and neonatal alloimmune thrombocytopenia (FNAIT). These data show that RLYB212 was able to markedly accelerate the elimination of transfused HPA-1a positive platelets in HPA-1a negative subjects, supporting our development of RLYB212 as a potential first-in-class medicine to prevent HPA-1a alloimmunization and, ultimately, eliminate FNAIT. We are now enrolling the multiple dose cohort of our Phase 1 trial and look forward to sharing additional data in the fourth quarter of 2023.” Dr. Mackay continued, “In parallel, we are advancing our efforts across complement dysregulation, hematology and metabolic disorders. In the fourth quarter of 2022, we initiated a multiple ascending dose Phase 1 study of RLYB116, our inhibitor of complement component 5 (C5), and we look forward to sharing initial data from this study in the fourth quarter of 2023. In addition, we entered into a collaboration with AbCellera, under which we will co-develop programs for up to five rare disease targets. Following our offering of common stock and pre-funded warrants in November 2022, we believe our capital will enable us to advance our growing portfolio through upcoming milestones, and opportunistically invest in strategic business development transactions that may allow us to broaden our impact and deliver life-transforming therapies to patients globally.” Recent Business Highlights and Upcoming Milestones: Maternal Fetal Blood Disorders Rallybio announced today that proof-of-concept has been achieved in a Phase 1b study for RLYB212, an anti-HPA-1a monoclonal antibody for the prevention of fetal and neonatal alloimmune thrombocytopenia. Results showed that one week after a single subcutaneous dose, RLYB212 was able to rapidly and completely eliminate transfused, HPA-1a positive platelets in HPA-1a negative subjects. RLYB212 was observed to be well-tolerated with no serious adverse events reported. The Company believes that the broad range of pharmacokinetic and pharmacodynamic data in the Phase 1b study will allow for substantive modeling to inform dose selection for a future registrational study. The Company expects to report data from the Phase 1b clinical study of RLYB212 at a scientific conference in 2023. Rallybio also announced today that testing in the multiple dose cohort of its Phase 1 trial in Europe initiated in the first quarter of 2023. This portion of the Phase 1 study will evaluate safety and pharmacokinetics of RLYB212 based on repeat dosing over 12 weeks in healthy male and female participants. The Company expects results from this cohort of subjects in the fourth quarter of 2023. The Company continues to advance the FNAIT natural history alloimmunization study. This non-interventional study is designed to inform on the frequency of women at higher risk for FNAIT across a broad population of pregnant women of different racial and ethnic characteristics, and the occurrence of HPA-1a alloimmunization in these women. The Company expects that the data from this study will contribute to a control dataset for a future single armed registration trial for RLYB212. Given the favorable development pro RLYB212 as compared to RLYB211 to date, the data generated to date for RLYB212, and the expected manufacturing and supply efficiencies for RLYB212, the Company will not continue development of RLYB211, a plasma-derived polyclonal anti-HPA-1a antibody. Complement Dysregulation Rallybio initiated dosing in the first multiple ascending dose cohort of a Phase 1 study of RLYB116 in the fourth quarter of 2022. RLYB116 is a novel, potentially long-acting, subcutaneously administered inhibitor of C5 in development for the treatment of patients with complement-related diseases. The single-blind, dose escalation, placebo-controlled study is designed to evaluate the safety, pharmacokinetics, and pharmacodynamics of RLYB116 in healthy participants. The Company expects initial data from the multiple ascending dose cohort in the fourth quarter of 2023. Rallybio continues to advance development of RLYB114, formulated for intravitreal injection, for the treatment of ophthalmic disorders. Rallybio and EyePoint Pharmaceuticals, Inc. announced in February 2023 a research collaboration to explore and assess the viability of utilizing Rallybio’s inhibitor of complement component 5 (C5) in EyePoint’s Durasert®technology for sustained intraocular delivery. The initial focus will be on geographic atrophy, an advanced form of age-related macular degeneration that leads to irreversible vision loss. Metabolic Disorders Rallybio, together with its partner Exscientia, continues to work toward the selection of a small molecule development candidate to advance into the clinic targeting ENPP1 for the treatment of patients with hypophosphatasia (HPP). In vivo efficacy data is expected in the second half of 2023. Following those results, we expect to commence investigational new drug (IND)-enabling studies. In December 2022, Rallybio announced a strategic alliance with AbCellera to discover, develop, and commercialize novel antibody-based therapeutics for rare diseases. Under the terms of the agreement, AbCellera and Rallybio will co-develop up to five rare disease therapeutic targets, which will be chosen together by both companies. The partnership’s first program will focus on addressing the significant unmet therapeutic needs of patients with rare metabolic diseases. Hematological Disorders Rallybio continues to conduct IND-enabling activities for RLYB331, a preclinical, potentially first-in-class therapeutic monoclonal antibody that inhibits Matriptase-2 (MTP-2), to support the transition of this asset into clinical development. Corporate Updates In January 2023, Rallybio announced the appointment of Jonathan I. Lieber as Chief Financial Officer (CFO), effective February 1, 2023. Mr. Lieber succeeds Jeffrey Fryer, CPA, Rallybio’s Co-Founder and former CFO. The Company announced Mr. Fryer’s retirement in June 2022, and following a transition period with Mr. Lieber, Mr. Fryer departed the company on February 15, 2023. In November 2022, Rallybio consummated a follow-on offering of common stock and pre-funded warrants that raised approximately $55.0 million in gross proceeds. Fourth Quarter and Full Year 2021 Financial Results: Research & Development (R&D) Expenses: R&D expenses were $10.8 million for the fourth quarter of 2022, compared to $6.1 million for the same period in 2021. R&D expenses for the fourth quarter 2022 increased primarily due to an increase in costs to advance RLYB212 and an increase in R&D related headcount costs as compared to the prior year. R&D expenses were $40.7 million for the year ended December 31, 2022 compared to $26.9 million for the year ended December 31, 2021. R&D expenses for the year ended December 31, 2022 increased primarily due to an increase in costs to advance RLYB212, the asset acquisition in-process research and development expense and development expenses of RLYB331 and additional R&D related headcount costs as compared to the prior year including an increase of $2.4 million in share-based compensation expense. General & Administrative (G&A) Expenses: G&A expenses were $6.3 million for the fourth quarter of 2022, compared to $6.2 million for the same period in 2021. G&A expenses were $27.2 million for the year ended December 31, 2022 compared to $18.7 million for the year ended December 31, 2021. The increase in general and administrative expenses for the full year was primarily due to additional G&A related payroll and personnel-related costs, including an increase of $3.5 million in share-based compensation expense, other professional fees, and costs associated with operating as a public company for full fiscal year as compared to 2021. Net Loss and Net Loss Per Common Share: Net loss was $16.2 million for the fourth quarter of 2022, or net loss per common share of $0.46 compared to a net loss of $12.4 million, or net loss per common share of $0.42 for the fourth quarter of 2021. Net loss was $66.7 million, or net loss per share of $2.09, for the year ended December 31, 2022 compared to $47.0 million, or net loss per share of $1.84, for the year ended December 31, 2021. Cash Position: As of December 31, 2022, cash, cash equivalents and marketable securities were $169.0 million. About Rallybio Rallybio is a clinical-stage biotechnology company committed to identifying and accelerating the development of life-transforming therapies for patients with severe and rare diseases. Since its launch in January 2018, Rallybio has built a portfolio of promising product candidates, which are now in development to address rare diseases in the areas of hematology, immuno-inflammation, maternal fetal health, and metabolic disorders. The Company’s mission is being advanced by a team of highly experienced biopharma industry leaders with extensive research, development, and rare disease expertise. Rallybio is headquartered in New Haven, Connecticut, with an additional facility at the University of Connecticut’s Technology Incubation Program in Farmington, Connecticut. For more information, please visit . Forward-Looking Statements This press release contains forward-looking statements that are based on our management’s beliefs and assumptions and on currently available information. All statements, other than statements of historical facts contained in this press release are forward-looking statements. In some cases, forward-looking statements can be identified by terms such as “may,” “will,” “should,” “expect,” “plan,” “anticipate,” “could,” “intend,” “target,” “project,” “contemplate,” “believe,” “estimate,” “predict,” “potential” or “continue” or the negative of these terms or other similar expressions, although not all forward-looking statements contain these words. Forward-looking statements in this press release include, but are not limited to, statements concerning, initiation, substance, design and timing of our planned or ongoing studies for RLYB212 and RLYB116, the timing of the availability of data from such studies, our expectations regarding reporting of data from such studies, and our expectations regarding the usefulness of data from such studies, the potential markets for RLYB212, RLYB114, RLYB116 and RLYB331, our estimates of our capital requirements and the sufficiency thereof, our ability to advance our portfolio, our ability to invest in business development activities, our ability to reach and potential impact on patients globally, our plans for development of RLYB114 for the treatment of ophthalmic disorders, our plans for development activities with our strategic collaboration partners, including Exscientia and Abcellera, the initiation and timing of and plans for our pre-IND enabling studies for our ENPP1 inhibitor, the timing of the availability of data from such studies, the initiation and timing of and plans for our pre-IND enabling studies of RLYB331, and the therapeutic effects of RLYB331. The forward-looking statements in this press release are only predictions and are based largely on management’s current expectations and projections about future events and financial trends that management believes may affect Rallybio’s business, financial condition and results of operations. These forward-looking statements speak only as of the date of this press release and are subject to a number of known and unknown risks, uncertainties and assumptions, including, but not limited to, our ability to successfully initiate and conduct our planned clinical trials, including the FNAIT natural history study, and the Phase 1 and 1b clinical trials for RLYB212 and the Phase 1 study for RLYB116, and complete such clinical trials and obtain results on our expected timelines, or at all, whether our cash resources will be sufficient to fund our operating expenses and capital expenditure requirements and whether we will be successful raising additional capital, our ability to identify new product candidates and successfully acquire such product candidates from third parties, our ability to enter into strategic partnerships or other arrangements, including the development of RLYB114, competition from other biotechnology and pharmaceutical companies, and those risks and uncertainties described in Rallybio’s filings with the U.S. Securities and Exchange Commission (SEC), including Rallybio’s Quarterly Report on Form 10-Q for the period ended September 30, 2022, and subsequent filings with the SEC. The events and circumstances reflected in our forward-looking statements may not be achieved or occur and actual future results, levels of activity, performance and events and circumstances could differ materially from those projected in the forward-looking statements. Except as required by applicable law, we are not obligated to publicly update or revise any forward-looking statements contained in this press release, whether as a result of any new information, future events, changed circumstances or otherwise. Financial Tables RALLYBIO CORPORATION SELECTED CONDENSED CONSOLIDATED FINANCIAL INFORMATION Condensed Consolidated Statements of Operations and Comprehensive Loss (Unaudited) FOR THE THREE MONTHS ENDED DECEMBER 31, FOR THE YEAR ENDED DECEMBER 31, (in thousands, except share and per share amounts) 2022 2021 2022 2021 Operating expenses: Research and development $ 10,793 $ 6,094 $ 40,689 $ 26,909 General and administrative 6,298 6,219 27,195 18,739 Total operating expenses 17,091 12,313 67,884 45,648 Loss from operations (17,091 ) (12,313 ) (67,884 ) (45,648 ) Other income (expenses): Interest income 1,071 12 1,963 54 Interest expense — — — (10 ) Other income 31 109 342 96 Total other income, net 1,102 121 2,305 140 Loss from continuing operations (15,989 ) (12,192 ) (65,579 ) (45,508 ) Loss on investment in joint venture 214 223 1,075 1,505 Net loss $ (16,203 ) $ (12,415 ) $ (66,654 ) $ (47,013 ) Net loss per common share, basic and diluted $ (0.46 ) $ (0.42 ) $ (2.09 ) $ (1.84 ) Weighted-average common shares outstanding, basic and diluted 35,516,630 29,789,974 31,821,311 25,519,114 Other comprehensive loss (income): Net unrealized loss (income) on marketable securities (220 ) — 214 — Other comprehensive (loss) income 220 — (214 ) — Comprehensive loss $ (15,983 ) $ (12,415 ) $ (66,868 ) $ (47,013 ) Condensed Consolidated Balance Sheets (Unaudited) (in thousands) DECEMBER 31, 2022 DECEMBER 31, 2021 Cash, cash equivalents and marketable securities $ 168,994 $ 175,334 Total assets 180,435 182,185 Total liabilities 11,118 6,583 Total stockholders' equity 169,317 175,602