Core Insights - Q32 Bio Inc. announced topline results from the SIGNAL-AA Phase 2a trial for bempikibart in alopecia areata, planning to expand the trial by enrolling additional patients [1][4] - The trial did not meet its primary endpoint in Part B, leading to a review of results and exclusion of one site from efficacy analysis due to protocol violations [2] - A post-hoc analysis indicated that bempikibart showed a 16% reduction in SALT score at week 24 compared to 2% in placebo, with 9% of patients achieving SALT-20 [3] SIGNAL-AA Trial Results - Bempikibart was safe and well-tolerated, with no serious adverse events reported [4] - The company plans to enroll approximately 20 additional patients in the Part B expansion of the SIGNAL-AA trial [4] Future Plans - Enrollment for the planned Phase 2 trial of ADX-097 in ANCA-Associated Vasculitis has been deferred to focus on ongoing trials for bempikibart and ADX-097 renal basket [5] SIGNAL-AD Trial Update - In the SIGNAL-AD trial, patients treated with bempikibart showed significant improvements in EASI scores, with 58% improvement at 2mg/kg and 84% at 3mg/kg compared to 38% in placebo [6] - Part B of the SIGNAL-AD trial showed a 74% improvement in EASI for bempikibart, compared to 76% for placebo, which was not statistically significant [7] Pharmacokinetics and Biomarkers - Bempikibart demonstrated favorable pharmacokinetics and target engagement, with substantial reductions in Th2 and Th1 biomarkers [8] Stock Performance - Q32 Bio Inc.'s stock price decreased by 67.1%, trading at $8.04 [9]

Product Development - Legacy Q32 has multiple product candidates targeting autoimmune and inflammatory diseases, with clinical readouts for two lead programs expected in 2024 and 2025[161] - Bempikibart (ADX-914) is in two Phase 2 trials for atopic dermatitis and alopecia areata, with topline data expected in Q4 2024[162] - ADX-097, a complement inhibitor, is in a Phase 1 trial and will enter a Phase 2 renal basket program, with initial data anticipated in H1 2025[163] - Following the termination of the Horizon Agreements, Legacy Q32 retained full development and commercial rights to bempikibart[167] - The company has exclusive licenses for the development and commercialization of ADX-097, with potential milestone payments of up to $2.2 million per licensed product[228] Financial Performance - As of September 30, 2024, Legacy Q32 had cash and cash equivalents of $89.1 million, expected to fund operations until mid-2026[174] - The company does not expect to generate revenue from product sales in the foreseeable future[177] - The net loss for the three months ended September 30, 2024, was $17.6 million, compared to a net loss of $14.0 million for the same period in 2023[192] - The net loss for the nine months ended September 30, 2024, was $33.5 million, compared to a net loss of $26.7 million in 2023, an increase of $6.9 million[200] - Total operating expenses for the three months ended September 30, 2024, were $18.8 million, up from $9.7 million in the same period in 2023, reflecting a loss from operations of $18.8 million[192] Expenses - Research and development expenses for the three months ended September 30, 2024, were $14.3 million, an increase of $6.9 million from $7.5 million in the same period in 2023[194] - The increase in research and development expenses was primarily due to a $6.1 million rise in clinical spending related to the bempikibart program, including a $4.0 million milestone payment to Bristol-Myers Squibb[194] - General and administrative expenses rose to $4.4 million for the three months ended September 30, 2024, compared to $2.2 million in the same period in 2023, driven by increased stock-based compensation and consulting costs[196] - Research and development expenses rose to $37.6 million in 2024, up from $23.4 million in 2023, an increase of $14.2 million[201] - General and administrative expenses increased to $14.0 million in 2024 from $7.1 million in 2023, reflecting a change of $6.9 million[203] Cash Flow and Financing - Net cash used in operating activities was $56.3 million for the nine months ended September 30, 2024, compared to $8.0 million in 2023[210] - The company raised $111.4 million in aggregate cash proceeds from various financing activities since inception[207] - The company may need to raise additional capital through private or public equity or debt financings, which could dilute existing stockholders' ownership[223] - The company expects to fund its operating expenses and capital expenditure requirements into mid-2026 based on current cash, cash equivalents, and short-term investments[222] Obligations and Commitments - The company is obligated to pay Horizon up to $75.1 million contingent on regulatory and sales milestones[180] - The company is obligated to pay Bristol-Myers Squibb up to $215 million in commercial milestone payments based on net sales of licensed products, along with tiered royalties ranging from mid-single digit percentages to up to 10%[232] - The total operating lease obligation as of September 30, 2024, is $13.229 million, with $5.259 million due in 1 to 3 years, $3.672 million in 3 to 5 years, and $4.298 million due after 5 years[225] - The company has received $55.0 million in initial consideration and staged development funding from Horizon for the bempikibart program[236] Accounting and Reporting - The company accounts for stock-based compensation based on grant date fair values, expensing awards over their service periods[258] - The company has determined that recently issued accounting pronouncements will not have a material impact on its financial position and results of operations[266] - The company is classified as a smaller reporting company and is not required to provide certain market risk disclosures[268] Market and Economic Conditions - The company may face challenges in raising additional capital due to potential worsening global economic conditions and disruptions in financial markets[223] - Management anticipates substantial increases in expenses related to ongoing research and development activities and public company operations[219]

Financial Position - Cash and cash equivalents were $89.1 million as of September 30, 2024, expected to fund operations into mid-2026[8] - Total assets increased to $104,542,000 as of September 30, 2024, compared to $47,057,000 on December 31, 2023, representing a growth of 122%[19] - Cash and cash equivalents rose significantly to $89,078,000 from $25,617,000, marking an increase of 248%[19] - Stockholders' equity improved to $18,295,000, recovering from a deficit of $(182,921,000) as of December 31, 2023[19] - Accounts payable and accrued expenses decreased to $10,170,000 from $13,231,000, a reduction of 23%[19] - Venture debt increased to $12,604,000 from $5,459,000, reflecting a rise of 130%[19] - The company reported a CVR liability of $2,680,000, which was not present in the previous period[19] - Right-of-use asset for operating leases slightly decreased to $5,869,000 from $6,301,000, a decline of 7%[19] - Restricted cash and equivalents dropped significantly to $647,000 from $5,647,000, a decrease of 89%[19] - Other assets decreased to $6,348,000 from $9,492,000, a decline of 33%[19] - Convertible preferred stock was eliminated, down from $111,445,000 in the previous period[19] Expenses - Research and development expenses increased to $14.3 million for Q3 2024, up from $7.5 million in Q3 2023, primarily due to higher clinical trial costs[9] - General and administrative expenses rose to $4.5 million for Q3 2024, compared to $2.2 million in Q3 2023, driven by increased stock-based compensation and consulting costs[10] Net Loss - Net loss for Q3 2024 was $17.6 million, or $1.46 per share, compared to a net loss of $14.0 million, or $40.52 per share, in Q3 2023[11] Clinical Trials - Bempikibart Phase 2 trials for atopic dermatitis and alopecia areata are on track, with topline results expected in December 2024[2] - Enrollment in the ADX-097 Phase 2 basket trial for complement mediated renal diseases is ongoing, with initial open-label data expected in 1H 2025[6] - A total of 121 patients were enrolled in the Bempikibart SIGNAL-AD Phase 2 trial, exceeding the initial target of approximately 100 patients due to high demand[4] - The primary endpoint for the Bempikibart SIGNAL-AD trial is the mean percent change in the Eczema Area and Severity Index (EASI) score from baseline to week 14[3] - The company plans to commence the ADX-097 Phase 2 trial in ANCA-Associated Vasculitis in the first half of 2025, with topline results expected in the second half of 2025[6] - Positive Phase 1 results for ADX-097 were presented at ASN Kidney Week 2024, demonstrating a favorable safety profile and desired pharmacokinetic properties[7]

Core Insights - Q32 Bio Inc. is a clinical stage biotechnology company focused on developing biologic therapeutics aimed at restoring immune homeostasis [3][4] - The company will participate in a fireside chat at Guggenheim's Inaugural Healthcare Innovation Conference on November 12, 2024 [1] Company Overview - Q32 Bio develops biologic therapeutics targeting regulators of the innate and adaptive immune systems to address autoimmune and inflammatory diseases [3] - The lead programs focus on the IL-7 / TSLP receptor pathways and the complement system, which are crucial for re-balancing immunity [3] Product Pipeline - The lead product, bempikibart (ADX-914), is a fully human anti-IL-7Rα antibody being evaluated in two Phase 2 trials for atopic dermatitis and alopecia areata [4] - The second product, ADX-097, targets the complement system with a novel platform that allows tissue-targeted regulation without long-term systemic blockade, differentiating it from current therapies [4] - ADX-097 has completed a Phase 1 ascending dose clinical study in healthy volunteers [4]

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended June 30, 2024 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from ___________________ to ___________________ Commission File Number: 001-38433 Q32 Bio Inc. (Exact name of registrant as specified in its charter) Delaware 47-3468 ...

Exhibit 99.1 Q32 Bio Reports Second Quarter 2024 Financial Results and Provides Corporate Update -- Completed enrollment in bempikibart atopic dermatitis (AD) Phase 2 clinical trial and increased trial size to 121 patients based on Part B patient demand -- -- Bempikibart Phase 2 topline results in AD and alopecia areata (AA) remain on-track for Q4'24 -- -- Enrolling patients in the Phase 2 basket trial of ADX-097 for complement mediated renal diseases; initial open-label data expected by year-end 2024 and t ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2024 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from ___________________ to ___________________ Commission File Number: 001-38433 Q32 Bio Inc. (Exact name of registrant as specified in its charter) Delaware 47-346 ...

Exhibit 99.1 Q32 Bio Reports First Quarter 2024 Financial Results and Provides Corporate Update -- Completed enrollment in bempikibart alopecia areata (AA) Phase 2 clinical trial, with topline results expected in Q4'24; enrollment in bempikibart atopic dermatitis (AD) Phase 2 clinical trial remains ontrack, with topline results expected in Q4'24 -- -- ADX-097 continues to advance, with anticipated Phase 2 clinical trial initiations on-track: renal basket in 1H'24 and ANCA-associated vasculitis (AAV) in 1H'2 ...

Table of Contents As filed with the Securities and Exchange Commission on April 19, 2024 Registration No. 333- UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM S-1 REGISTRATION STATEMENT UNDER THE SECURITIES ACT OF 1933 Q32 BIO INC. (Exact name of registrant as specified in its charter) (State or other jurisdiction of incorporation or organization) (Primary Standard Industrial Classification Code No.) Delaware 2834 47-3468154 (I.R.S. Employer Identification Number) 830 Winter Str ...

Table of Contents As filed with the Securities and Exchange Commission on April 19, 2024 Registration No. 333- UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM S-1 REGISTRATION STATEMENT UNDER THE SECURITIES ACT OF 1933 Q32 BIO INC. (Exact name of registrant as specified in its charter) (State or other jurisdiction of incorporation or organization) (Primary Standard Industrial Classification Code No.) Delaware 2834 47-3468154 (I.R.S. Employer Identification Number) 830 Winter Str ...