Malformations of Cortical Development, Group II

Enrolling Patients in Global Phase 3 CoMpass Trial in Small Choroidal Melanoma and Indeterminate Lesions; Granted SPA Agreement by FDA Data Expected Mid-2024 from Ongoing Phase 1 Trial in Bladder Cancer (Non-Muscle Invasive Bladder Cancer and Muscle Invasive Bladder Cancer) Strong Cash Position into Second Half of 2026 BOSTON--(BUSINESS WIRE)-- Aura Biosciences, Inc. (NASDAQ: AURA), a clinical-stage biotechnology company developing precision immunotherapies to treat solid tumors to preserve the function of the afflicted organ with cancer, today reported financial results for the fourth quarter and year ended December 31, 2023, and provided recent business highlights. “We’re excited to expand bel-sar into bladder cancer, as we leverage our therapy’s unique mechanism of action in additional solid cancer indications with major unmet medical need,” said Elisabet de los Pinos, Ph.D., CEO of Aura Biosciences. “Bel-sar is a potential vision-and-organ sparing therapy that we believe will change the standard of care in ocular oncology, in particular choroidal melanoma, where there are no treatment options except radiotherapy which leads to vision loss, or surgical removal of the eye. There is an urgent need to develop vision-sparing therapies, as all eye cancers represent an estimated 60,000 patients annually in the US and EU. This is a multi-billion dollar market, which includes choroidal melanoma, choroidal metastases, and ocular surface cancers. Our financial strength allows us to fund multiple clinical programs through major inflection points while enabling the flexibility to expand bel-sar into additional indications, starting with bladder cancer.” Recent Pipeline Developments Global Phase 3 CoMpass trial actively enrolling patients for the treatment of small choroidal melanoma (CM) and indeterminate lesions (ILs). The trial is a superiority trial comparing treatment with bel-sar versus a sham control arm. The trial is a global Phase 3, randomized, multi-center, masked study, and is intended to enroll approximately 100 patients randomized 2:1:2 to receive three cycles of treatment with either high or low doses of bel-sar, or a sham control. The Company received written agreement from the U.S. Food and Drug Administration (FDA) under an SPA for the overall design, statistical analysis plan and clinical endpoints. The primary endpoint is time to tumor progression when the last patient completes 15 months of follow up. The trial is actively enrolling in the U.S. with a strong endorsement from the ocular oncology community. The Company is on track to activate sites and enroll patients globally throughout 2024. Positive Phase 2 data evaluating SC administration of bel-sar for patients with small CM and ILs was presented at the American Academy of Ophthalmology (AAO) 2023 Annual Meeting. Results: Patients who received the therapeutic regimen with three cycles of therapy showed a tumor control rate of 80% (8/10) and a visual acuity preservation rate of 90% (9/10), with most of the patients (>90%) being at 12 months of follow up. Final study results with all patients at 12 months will be presented by year end 2024. The overall tolerability pro bel-sar was favorable, with no dose-limiting toxicities, treatment-related serious adverse events (SAEs) or significant adverse events (AEs) reported as of August 3, 2023. There was no posterior segment inflammation and only mild anterior inflammation (Grade 1) in approximately 18% of the patients which was self-limited or resolved with a short course of topical steroids. Treatment-related AEs were predominantly mild and resolved without sequelae. These patients match the criteria for enrollment in the ongoing Phase 3 trial which is highly powered based on the Phase 2 results. Bel-sar is being evaluated in additional ocular oncology indications with a collective incidence of approximately 60,000 patients/year in the US/EU per year. The Company’s plan is to initiate clinical development in choroidal metastasis (Cmets), an indication with a high unmet medical need where bel-sar has the potential to be the first approved therapy that is vision and organ sparing. Cmets is the second potential ocular oncology indication for bel-sar affecting over 20,000 patients in the US/EU annually. The Company is on track to initiate a Phase 2 trial in 2024. The Phase 1 trial of bel-sar for the treatment of non-muscle invasive bladder cancer (NMIBC) and muscle invasive bladder cancer (MIBC) is currently ongoing, and the Company expects to report data in mid-2024. This represents an area of high unmet need with approximately 80,000 patients diagnosed in the U.S. every year where we have the possibility to selectively treat and induce a tissue and tumor specific immune response to prevent progression and recurrence of the disease. The Company received Fast Track Designation from the Oncology Division of the FDA for NMIBC in June 2022. The ongoing Phase 1 multi-center, open-label clinical trial is expected to enroll approximately 21 adult patients. The trial is designed to assess the safety and tolerability of bel-sar as a single agent. The trial will provide histopathological evaluation after the local treatment to assess bel-sar’s biological activity which will include the evaluation of focal necrosis and immune activation. The trial has completed enrollment of the cohort that received bel-sar injection without light activation. Protocol mandated safety review found no safety issues and the study has proceeded to the bel-sar injection plus light activation cohorts. Preliminary data from the first patient in the light activated cohort of the trial, demonstrated a clinical complete response demonstrated by absence of cancer cells on histopathology with evidence of extensive necrosis and immune activation after a single administration of bel-sar followed by light activation. Recent Corporate Events Raised Gross Proceeds of $99.0 million in an underwritten public offering. In November 2023, the Company announced the pricing of an underwritten public offering of 11,000,000 shares of its common stock at a price to the public of $9.00 per share. The offering closed on November 9, 2023. Full Year and Fourth Quarter 2023 Financial Results As of December 31, 2023, Aura had cash and cash equivalents and marketable securities totaling $226.2 million. The Company believes its current cash and cash equivalents and marketable securities are sufficient to fund its operations into the second half of 2026. Research and development expenses increased to $20.3 million and $65.2 million for the three months and full year ended December 31, 2023, respectively, from $13.2 million and $42.2 million for the three months and full year ended December 31, 2022, respectively, primarily due to ongoing clinical costs associated with the progression of the Phase 2 study and contract research organization costs associated with the start of the Phase 3 global trial, and manufacturing and development costs for bel-sar. General and administrative expenses increased to $4.5 million and $19.8 million for the three months and full year ended December 31, 2023, respectively, from $4.5 million and $18.1 million for the three months and full year ended December 31, 2022, respectively. General and administrative expenses include $1.2 million and $1.1 million of stock-based compensation for the three months ended December 31, 2023 and 2022, respectively. The increase was primarily driven by personnel expenses, as well as increases in travel expenses related to growth of the Company. Net loss for the three months and full year ended December 31, 2023, was $22.1 million and $76.4 million, respectively, compared to $16.6 million and $58.8 million for the three months and full year ended December 31, 2022, respectively. About Aura Biosciences Aura Biosciences is a clinical-stage biotechnology company developing precision immunotherapies to treat solid tumors designed to preserve the function of the afflicted organ with cancer. Our lead candidate bel-sar is in late-stage clinical development for the treatment of patients with primary choroidal melanoma, and other ocular oncology indications as well as in early-stage clinical development in bladder cancer. We are evaluating the safety and efficacy of bel-sar as a potential vision-sparing therapy in an ongoing global Phase 3 CoMpass trial for the first-line treatment of adult patients with early-stage choroidal melanoma. Bel-sar is also being evaluated in additional solid cancers, including bladder cancer. Our mission is to develop vision and organ-sparing therapies to improve patient outcomes in cancer. Aura is headquartered in Boston, MA. For more information, visit aurabiosciences.com. Visit us @AuraBiosciences and on LinkedIn. Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, and other federal securities laws. Any statements that are not statements of historical fact may be deemed to be forward looking statements. Words such as “may,” “will,” “could”, “should,” “expects,” “intends,” “plans,” “anticipates,” “believes,” “estimates,” “predicts,” “projects,” “seeks,” “endeavor,” “potential,” “continue” or the negative of such words or other similar expressions that can be used to identify forward-looking statements. These forward looking statements include express or implied statements regarding Aura’s future expectations, plans and prospects, including, without limitation, statements regarding the therapeutic potential of bel-sar for the treatment of cancers including choroidal melanoma, bladder cancer and choroidal metastasis; any express or implied statements regarding the Company’s expectations for the Phase 2 and Phase 3 clinical trials of bel-sar for choroidal melanoma and indeterminate legions and the Phase 1 trial of bel-sar for non-muscle invasive bladder cancer and muscle invasive bladder cancer; and Aura’s expectations regarding the estimated patient populations and related market opportunities for bel-sar. The forward-looking statements in this press release are neither promises nor guarantees, and investors should not place undue reliance on these forward-looking statements because they involve known and unknown risks, uncertainties, and other factors, many of which are beyond Aura’s control and which could cause actual results to differ materially from those expressed or implied by these forward-looking statements, including, without limitation, an improved quality of life of patients after treatment with bel-sar; a potential paradigm shift in the approach to the treatment of choroidal melanoma; the urgent need for a vision preserving targeted therapy; the potential of bel-sar compared to the existing standard of care for patients with choroidal melanoma; uncertainties inherent in clinical trials and in the availability and timing of data from ongoing clinical trials; the expected timing for submissions for regulatory approval or review by governmental authorities; the risk that the results of Aura’s clinical trials may not be predictive of future results in connection with future clinical trials; the risk that interim data from ongoing clinical trials may not be predictive of final data from completed clinical trials; the risk that governmental authorities may disagree with Aura’s clinical trial designs, even where Aura has obtained agreement with governmental authorities on the design of such trials, such as the Phase 3 SPA agreement with FDA; whether Aura will receive regulatory approvals to conduct trials or to market products; whether Aura’s cash resources will be sufficient to fund its foreseeable and unforeseeable operating expenses and capital expenditure requirements; Aura’s ongoing and planned pre-clinical activities; and Aura’s ability to initiate, enroll, conduct or complete ongoing and planned clinical trials. These risks, uncertainties, and other factors include those risks and uncertainties described under the heading “Risk Factors” in Aura’s most recent Annual Report on Form 10-K and Quarterly Report on Form 10-Q filed with the U.S. Securities and Exchange Commission (SEC) and in subsequent filings made by Aura with the SEC, which are available on the SEC’s website at . Except as required by law, Aura disclaims any intention or responsibility for updating or revising any forward-looking statements contained in this press release in the event of new information, future developments or otherwise. These forward-looking statements are based on Aura’s current expectations and speak only as of the date hereof and no representations or warranties (express or implied) are made about the accuracy of any such forward-looking statements. Aura Biosciences, Inc. Consolidated Statement of Operations and Comprehensive Loss (in thousands, except share and per share amounts) Year Ended December 31, 2023 2022 Operating Expenses: Research and development $ 65,232 $ 42,238 General and administrative 19,759 18,057 Total operating expenses 84,991 60,295 Total operating loss (84,991 ) (60,295 ) Other income (expense): Interest income, including amortization and accretion income 8,588 1,864 Gain (loss) from disposal of assets 208 (318 ) Other income (expense) (76 ) (14 ) Total other income 8,720 1,532 Loss before income taxes (76,271 ) (58,763 ) Income tax benefit (provision), net (137 ) — Net loss (76,408 ) (58,763 ) Net loss per common share—basic and diluted (1.93 ) (1.96 ) Weighted average common stock outstanding—basic and diluted 39,620,036 29,937,228 Comprehensive loss: Net loss $ (76,408 ) $ (58,763 ) Other comprehensive items: Unrealized gain (loss) on marketable securities 611 (72 ) Total other comprehensive income (loss) 611 (72 ) Total comprehensive loss $ (75,797 ) $ (58,835 ) Aura Biosciences, Inc. Consolidated Balance Sheets (in thousands, except share and per share amounts) December 31, 2023 December 31, 2022 Assets Current assets: Cash and cash equivalents $ 41,063 $ 121,582 Marketable securities 185,087 67,229 Restricted cash and deposits 19 20 Prepaid expenses and other current assets 5,625 7,871 Total current assets 231,794 196,702 Restricted cash and deposits, net of current portion 768 768 Right of use assets - operating lease 18,854 20,671 Other long-term assets 509 423 Property and equipment, net 3,150 5,371 Total Assets $ 255,075 $ 223,935 Liabilities and Stockholders’ Equity Current liabilities: Accounts payable 1,787 2,921 Short-term operating lease liability 2,687 2,963 Accrued expenses and other current liabilities 7,883 4,573 Total current liabilities 12,357 10,457 Long-term operating lease liability 16,870 17,895 Total Liabilities 29,227 28,352 Commitments and Contingencies Stockholders’ Equity: Common stock, $0.00001 par value, 150,000,000 authorized at December 31, 2023 and December 31, 2022, and 49,350,788 and 37,771,918 shares issued and outstanding at December 31, 2023 and December 31, 2022, respectively — — Additional paid-in capital 512,617 406,555 Accumulated deficit (287,308 ) (210,900 ) Accumulated other comprehensive loss 539 (72 ) Total Stockholders’ Equity 225,848 195,583 Total Liabilities and Stockholders’ Equity $ 255,075 $ 223,935

GAL-201 specifically targets and binds to misfolded Aβ monomers, preventing their aggregation to toxic Aβ oligomers and protofibrilsAs a consequence, consistent beneficial effects have been demonstrated on synaptic spine density and on cognitive performance after single injection of GAL-201 in transgenic models of Alzheimer’s diseaseThese results warrant initiation of an IND-enabling program as next step towards clinical development KENSINGTON, Md., March 08, 2024 (GLOBE NEWSWIRE) -- Galimedix Therapeutics, Inc. (“Galimedix”), a Phase 2 clinical-stage biotechnology company developing novel oral and topical neuroprotective therapies with the potential to revolutionize the treatment of serious eye and brain diseases, announces encouraging data with orally available small molecule GAL-201, showing its neuroprotective and symptomatic alleviation potential in models of Alzheimer’s disease (AD). These new findings strongly support previous studies where GAL-201 was characterized as a promising development candidate for the treatment of AD (Russ et. al. 2022). The data was presented this week at the International Conference on Alzheimer’s and Parkinson’s Diseases and related neurological disorders (AD/PD™ 2024: advances in science and therapy). “The small molecule GAL-201 belongs to a new pharmacological class of amyloid beta aggregation modulators that acts upstream of other known Aβ-targeting agents,” explained Hermann Russ, M.D., Ph.D., Co-founder, and Chief Scientific Officer of Galimedix. “The positive results presented at AD/PD™ 2024 demonstrate how treatment with GAL-201 has a potential neuroprotective effect and may also improve cognitive function. Furthermore, they validate that toxic Aβ oligomers and protofibrils are a major underlying cause of this devastating disease, and not the deposited forms of Aβ. We look forward to advancing this promising new drug candidate into the clinic.” Data from the poster, entitled “The amyloid beta aggregation modulator GAL-201 is under development for oral AD treatment: Cognitive improvement in a transgenic AD model”, showed a high affinity of GAL-201 to misfolded Aβ monomers, the precursors of the neurotoxic forms of Aβ oligomers and protofibrils. Binding of GAL-201 to the Aβ monomers significantly prevented Aβ-induced suppression of long-term potentiation (LTP), considered a neuronal correlate of learning and memory. These results mirrored morphological changes observed in neuronal cells (dendrites), where Aβ-induced spine loss was also prevented. Preserving dendritic spines is essential, as they are correlated with synaptic plasticity. Additionally, in behavioral experiments using AD animal models, a single injection of GAL-201 resulted in improved cognitive performance, both in duration (escape latency) and accuracy (left and right decisions) performing a task (water-cross maze) and were comparable to healthy mice. The presented results further substantiate GAL-201’s role as a promising future Aβ-targeting treatment for AD, with an advanced mechanism of action and a patient-friendly application route as an oral tablet. The next step is to move GAL-201 towards IND submission and clinical development. The poster can be accessed here: “The amyloid beta aggregation modulator GAL-201 is under development for oral AD treatment: Cognitive improvement in a transgenic AD model” About GAL-201GAL-201 is an orally administered small molecule specifically designed to target amyloid beta (Aβ) and suppress the formation of toxic forms of Aβ aggregates in the brain of people with AD. GAL-201 has been shown to be able to bind with high affinity to the misfolded forms of Aβ, and thereby prevent their aggregation to toxic Aβ oligomers and protofibrils. GAL-201 is believed to be a promising development candidate for the treatment of AD with the potential to slow or stop disease progression and is currently in pre-clinical testing. Galimedix holds global rights to GAL-201. About Galimedix Therapeutics, Inc. Galimedix is a Phase 2 clinical-stage private company developing novel oral and topical neuroprotective therapies with the potential to revolutionize the treatment of serious eye and brain diseases. Founded by a seasoned and highly dedicated team of bio-entrepreneurs, pharmaceutical executives, and scientists, Galimedix’s groundbreaking small molecules offer the hope of changing the course of diseases where amyloid beta (Aβ) plays a role, such as in dry age-related macular degeneration (AMD), glaucoma and Alzheimer’s disease - Galimedix’s initial areas of focus. The Company’s approach targets toxic Aβ oligomers and protofibrils. Many studies have indicated that these oligomers and protofibrils are an underlying cause of neurodegenerative diseases of the eye. Recent approvals and promising Phase 3 results of anti-Aβ drugs also have validated them as a key target in Alzheimer’s disease. Compelling pre-clinical data support the potential of Galimedix’s product candidates to slow or stop neurodegeneration and restore lost neuronal function. A Phase 2 proof-of-concept study in dry AMD with lead program, topical GAL-101, is in preparation with strong support from partner, Théa Open Innovation (TOI). Clinical studies in other indications are planned. Contact Alexander Gebauer, MD, PhD Galimedix Therapeutics, Inc. Co-founder and Executive Chairman info@galimedix.com Media inquiries: Anne Hennecke / Lydia Robinson-GarciaU.S.MC Services AGLaurie DoyleTel: +49 (0)170 7134018Tel: +1-339-832-0752galimedix@mc-services.eu

Bristol Myers Squibb has agreed to spend at least $100 million to expand a partnership aimed at creating RNA-based medicines for cardiovascular diseases.The deal, announced Tuesday, shows Bristol Myers is willing to bet more on drugmaking technology developed by Avidity Biosciences. Founded in 2012, Avidity is trying to combine elements from two kinds of medicines monoclonal antibodies and oligonucleotide therapies to make new treatments that are both precise and target the root cause of diseases.The most advanced of Aviditys so-called AOCs, or antibody oligonucleotide conjugates, are in early-stage human trials, where researchers are evaluating their ability to treat three kinds of rare, muscle-eroding diseases. The company has other experimental therapies in preclinical testing, and is partnered with Eli Lilly on AOCs for immune system disorders and other select indications.Those programs are separate from the Bristol Myers collaboration, which will now include the discovery, development and commercialization of AOCs for as many as five cardiovascular targets. Deal terms hold that Avidity will initially receive $60 million in cash and an approximately $40 million equity investment. Bristol Myers has also agreed to pay as much as $2.2 billion more, provided Avidity's programs hit certain milestones.The pharmaceutical giant said it will fund all future clinical development, regulatory and commercialization activities tied to the deal. Avidity, meanwhile, could earn royalties up to low double-digits on net sales from any resulting products.This strategic collaboration solidifies our commitment in cardiology as we continue to advance our own research and development programs in cardiac indications,“ said Sarah Boyce, Aviditys CEO, in a statement.The two companies originally linked up in late 2020, when Avidity entered a research partnership with the Bristol Myers subsidiary MyoKardia to demonstrate that AOCs can be useful in cardiac tissue. That deal came just after Bristol Myers completed its $13 billion acquisition of MyoKardia, which was advancing medicines for genetic forms of heart disease. One of those medicines has since received regulatory approval, and is sold under the brand name Camzyos.To Steven Seedhouse, an analyst at the investment firm Raymond James, the new collaboration is positive for Avidity in several ways. First, it provides a meaningful extension to the companys already very strong cash runway, which was at $542 million by the end of September.Second, its a vote of confidence in Aviditys platform from a large, established drugmaker. Bristol Myers must have liked what it saw preclinically over the last two or so years from the existing MyoKardia collaboration, Seedhouse wrote in a note to clients.Avidity shares rose more than 11% Tuesday morning, to trade at $6.87 apiece. The companys stock was valued at more than $25 per share in February, but has ticked down in the months since.In September 2022, the Food and Drug Administration placed a partial clinical hold on a trial testing one of Aviditys muscular dystrophy medicines, due to a rare serious adverse event. Following discussions between the agency and the company, the hold was eased in May.In his note, Seedhouse wrote that his team believes the newly expanded collaboration implies a high likelihood (or at least Bristols lack of concern about) the partial clinical hold ... is lifted soon. '