Rallybio Corp

Previous wording

RLYB212 is a monoclonal anti-HPA-1a antibody for the prevention of FNAIT, a potentially life-threatening rare hematological disease that impacts fetuses and newborns.

Current wording

RLYB212 is a monoclonal anti-HPA-1a antibody for the prevention of FNAIT, a potentially life-threatening rare hematological disease that impacts fetuses and newborns. In the fourth quarter of 2024, the European Medicines Agency (the "EMA") and the United Kingdom’s Medicines and Healthcare products Regulatory Agency (the "MHRA") approved our clinical trial applications ("CTAs") for the Phase 2 clinical trial of RLYB212 and we are planning to initiate the trial in the fourth quarter of 2024.The Phase 2 trial, which will be conducted in Belgium, the Netherlands, Norway, Sweden, and the UK, is designed to assess the pharmacokinetic ("PK") and safety of RLYB212, a monoclonal anti-HPA-1a antibody, in a total of eight pregnant women at higher risk for HPA-1a alloimmunization and FNAIT. Secondary objectives include assessments of pregnancy and neonatal/infant outcomes, and the occurrence of emergent HPA-1a alloimmunization. Subcutaneous administration of RLYB212 will be initiated by Gestational Week 16 and continue every 4 weeks through parturition. Following completion of the Phase 2 dose confirmation trial and consultation with regulatory authorities, we expect to initiate a Phase 3 registrational trial. Both the U.S. Food and Drug Administration (the "FDA") and EMA have designated RLYB212 as an orphan drug. Orphan drug designation offers certain incentives including tax credits, marketing exclusivity upon any approval, fee waivers, and the ability to interact with both agencies to receive specialized regulatory advice and assistance.

Previous wording

We have completed two RLYB212 clinical studies: a Phase 1 first-in-human clinical study and a Phase 1b proof of concept clinical study. The Phase 1 first-in-human clinical study was a single-blind, placebo-controlled study that investigated the safety and pharmacokinetic ("PK") of subcutaneous ("SC") administration of RLYB212 in HPA-1a negative healthy participants. The clinical study included a single dose cohort and a multiple dose cohort. In the multiple dose cohort, subjects received SC RLYB212 or placebo every 2 weeks for 12 weeks. We reported results from the multi-dose cohort in the fourth quarter of 2023. The data and our clinical pharmacology modeling predictions support a once monthly dosing regimen for the planned Phase 2 clinical trial.

Current wording

We have completed two RLYB212 clinical studies: a Phase 1 first-in-human clinical study and a Phase 1b proof of concept clinical study. The Phase 1 first-in-human clinical study was a single-blind, placebo-controlled study that investigated the safety and PK of subcutaneous ("SC") administration of RLYB212 in HPA-1a negative healthy participants. The clinical study included a single dose cohort and a multiple dose cohort. In the multiple dose cohort, subjects received SC RLYB212 or placebo every 2 weeks for 12 weeks. We reported results from the multi-dose cohort in the fourth quarter of 2023. The data and our clinical pharmacology modeling predictions support a once monthly dosing regimen for the Phase 2 clinical trial.

Previous wording

Screening in the Company's FNAIT natural history study is ongoing, with more than 12,000 pregnant women screened as of August 1, 2024. In connection with the initiation of the Phase 2 trial, we will begin to transition European sites from the natural history study to the Phase 2 trial, where sites will continue to collect natural history data in women who do not receive RLYB212. As a result, we expect to see a temporary reduction in screening rates at these sites during such transition. The North American natural history study sites will continue screening activities as currently planned. The totality of natural history data from both studies is designed to provide a contemporary dataset for HPA-1a alloimmunization frequency in a racially and ethnically diverse population that can serve as a control arm for the planned Phase 3 trial.

Current wording

Screening in the natural history study is ongoing, with more than 13,000 pregnant women screened as of November 1, 2024. In connection with the initiation of the Phase 2 trial, we have transitioned European sites from the natural history study to the Phase 2 clinical trial, where sites will continue to collect natural history data in women who do not receive RLYB212. While the North American natural history study sites have continued screening activities as planned, we have seen an anticipated reduction in screening rates at the European sites during their transition into the Phase 2 clinical trial. The totality of natural history data from both studies is designed to provide a contemporary dataset for HPA-1a alloimmunization frequency in a racially and ethnically diverse population that can serve as a control arm for the planned Phase 3 trial.

Previous wording

Based on the data generated in the MAD portion of the study, we initiated additional manufacturing activities and biomarker analyses. The manufacturing work on RLYB116 is on track to be completed in the third quarter of 2024. Drug substance characterization data indicates that Rallybio's efforts to enhance the manufacturing process have been successful. In addition, we have conducted additional complement biomarker analyses that when taken together with the MAD data lead us to believe that there is an opportunity to pursue indications beyond gMG including PNH and APS at doses tested in the Phase 1 MAD study. We plan to provide an update on future plans for RLYB116 later this year.

Current wording

Based on the data generated in the MAD portion of the study, we initiated additional manufacturing activities and biomarker analyses. The manufacturing work on RLYB116 was completed in the third quarter of 2024 and drug substance characterization data indicates that Rallybio's efforts to enhance the manufacturing process have been successful. In addition, we have conducted additional complement biomarker analyses that when taken together with the MAD data indicate that RLYB116 led to greater sustained reductions in free C5 than initially indicated and lead us to believe that there is an opportunity to pursue indications beyond gMG such as PNH and APS at doses tested in the Phase 1 MAD study. In December 2024, we expect to provide updates on the manufacturing process enhancements and biomarker characterization, as well as future plans for RLYB116.

Previous wording

In May 2022, we obtained worldwide exclusive rights to RLYB331, a preclinical, monoclonal antibody that is designed to inhibit Matriptase-2 ("MTP-2"). The inhibition of MTP-2 significantly increases levels of hepcidin, decreases iron load and treats ineffective erythropoiesis. In the first quarter of 2024, we completed nonclinical studies that demonstrated favorable tolerability, dose-dependent PK and sustained PD effects with RLYB332, a long-acting version of the RLYB331 anti-Matriptase-2 antibody. These findings support the continued development of RLYB332 as a potentially best-in-class therapeutic for treating diseases of iron overload. We anticipate presentation of this data in the fourth quarter of 2024. We continue to evaluate non-dilutive options to further advance this program, including potential partnerships.

Current wording

In May 2022, we obtained worldwide exclusive rights to RLYB331, a preclinical, monoclonal antibody that is designed to inhibit Matriptase-2 ("MTP-2"). The inhibition of MTP-2 significantly increases levels of hepcidin, decreases iron load and treats ineffective erythropoiesis. In the first quarter of 2024, we completed nonclinical studies that demonstrated favorable tolerability, dose-dependent PK and sustained PD effects with RLYB332, a long-acting version of the RLYB331 anti-Matriptase-2 antibody. These data will be presented at the American Society of Hematology ("ASH") Annual Meeting which will be held December 7 – 10 in San Diego, CA. These findings support the continued development of RLYB332 as a potentially best-in-class therapeutic for treating diseases of iron overload. We continue to evaluate non-dilutive options to further advance this program, including potential partnerships.

Previous wording

In collaboration with Exscientia Limited ("Exscientia"), we continue to work toward the selection of a small molecule development candidate to advance into the clinic targeting Ectonucleotide Pyrophosphatase/ Phosphodiesterase 1 ("ENPP1") for the treatment of patients with hypophosphatasia ("HPP"). Proof of mechanism studies are in progress with a leading global HPP expert. We expect to achieve development candidate nomination of a small molecule inhibitor of ENPP1 for the treatment of patients with HPP in the fourth quarter of 2024. In addition, data from an early lead compound in a nonclinical model of HPP will be presented at the American Society for Bone and Mineral Research ("ASBMR") meeting which is being held from September 27 – 30, 2024 in Toronto, ON, Canada.

Current wording

In collaboration with Exscientia Limited ("Exscientia"), we continue to work toward the selection of a small molecule development candidate to advance into the clinic targeting Ectonucleotide Pyrophosphatase/ Phosphodiesterase 1 ("ENPP1") for the treatment of patients with hypophosphatasia ("HPP"). Proof of mechanism studies are in progress with a leading global HPP expert. We expect to achieve development candidate nomination of a small molecule inhibitor of ENPP1 for the treatment of patients with HPP in the fourth quarter of 2024. In addition, data from an early lead compound in a nonclinical model of HPP was presented at the American Society for Bone and Mineral Research meeting in September 2024. The data demonstrated that oral dosing of REV101 to adult HPP mice lowered inorganic pyrophosphate (PPi) by 30%, leading to improvements in mineralization of long and vertebrate bones. Furthermore, data showed that ENPP1 inhibition was safe and generally well-tolerated. Data also showed, for the first time, that ENPP1 is a druggable target for later-onset HPP.

Previous wording

In April 2024, we entered into a Securities Purchase Agreement with JJDC pursuant to which we sold to JJDC, in an unregistered offering, 3,636,363 shares of our common stock at a price of $1.82 per share, which represents a 10% premium on the Company’s closing stock price on April 9, 2024, for aggregate gross proceeds of approximately $6.6 million, before deducting offering expenses. We agreed, among other things, to file with the Securities and Exchange Commission (the "SEC") a registration statement covering the resale of the shares (the "Resale Registration Statement") within 120 days following the closing of the offering. We filed the Resale Registration Statement on May 10, 2024.

Current wording

In April 2024, we entered into a Securities Purchase Agreement with JJDC pursuant to which we sold to JJDC, in an unregistered offering, 3,636,363 shares of our common stock at a price of $1.82 per share, which represents a 10% premium on the Company’s closing stock price on April 9, 2024, for aggregate gross proceeds of approximately $6.6 million, before deducting offering expenses. We agreed, among other things, to file with the Securities and Exchange Commission (the "SEC") a registration statement covering the resale of the shares, which we filed on May 10, 2024.

Previous wording

As of June 30, 2024, we had cash, cash equivalents and marketable securities of $88.6 million. We believe that our existing cash, cash equivalents and marketable securities will be sufficient to fund our operating expenses and capital expenditure requirements into the middle of 2026. This estimate and our expectation to advance the preclinical and clinical development of RLYB212, RLYB116, and any other product candidates are based on assumptions that may prove to be wrong, and we could exhaust our available capital resources sooner than we expect, or our clinical trials may be more expensive, time consuming or difficult to design or implement than we currently anticipate. See “—Liquidity and Capital Resources.”

Current wording

As of September 30, 2024, we had cash, cash equivalents and marketable securities of $75.1 million. We believe that our existing cash, cash equivalents and marketable securities will be sufficient to fund our operating expenses and capital expenditure requirements into the middle of 2026. This estimate and our expectation to advance the preclinical and clinical development of RLYB212, RLYB116, and any other product candidates are based on assumptions that may prove to be wrong, and we could exhaust our available capital resources sooner than we expect, or our clinical trials may be more expensive, time consuming or difficult to design or implement than we currently anticipate. See “—Liquidity and Capital Resources.”

Previous wording

We have incurred significant operating losses since inception, including net losses of $16.2 million and $35.3 million for the three and six months ended June 30, 2024, respectively, and $18.6 million and $35.9 million for the three and six months ended June 30, 2023, respectively. As of June 30, 2024, we had an accumulated deficit of $270.5 million. These losses have resulted primarily from costs incurred in connection with research and development activities and general and administrative costs associated with our operations. We have not commercialized any products and have never generated revenue from the commercialization of any product. We expect to incur significant additional operating losses in the foreseeable future as we advance our programs through preclinical and clinical development, expand our research and development activities, acquire and develop new product candidates, complete preclinical studies and clinical trials, finance our business development strategy, seek regulatory approval for the commercialization of our product candidates and commercialize our products, if approved. Our expenses will increase substantially over time if and as we:

Current wording

We have incurred significant operating losses since inception, including net losses of $11.5 million and $46.7 million for the three and nine months ended September 30, 2024, respectively, and $18.4 million and $54.3 million for the three and nine months ended September 30, 2023, respectively. As of September 30, 2024, we had an accumulated deficit of $282.0 million. These losses have resulted primarily from costs incurred in connection with research and development activities and general and administrative costs associated with our operations. We have not commercialized any products and have never generated revenue from the commercialization of any product. We expect to incur significant additional operating losses in the foreseeable future as we advance our programs through preclinical and clinical development, expand our research and development activities, acquire and develop new product candidates, complete preclinical studies and clinical trials, finance our business development strategy, seek regulatory approval for the commercialization of our product candidates and commercialize our products, if approved. Our expenses will increase substantially over time if and as we:

Previous wording

(1) Data collection & submission revenue – derived from Rallybio’s ongoing management of the studies including the maintenance of a minimum site footprint, the license to utilize, and timely, semi-annual submission of the anonymized data, in the required formats.

Current wording

(1) Data collection & submission revenue – derived from Rallybio’s ongoing management of our studies including the maintenance of a minimum site footprint, the license to utilize, and timely, semi-annual submission of the anonymized data, in the required formats to J&J.

Previous wording

(2) Dissemination of J&J materials & participant revenue – derived from Rallybio’s dissemination of content, information or materials related to the J&J-Sponsored Studies that are developed by J&J and are provided by Rallybio for the purpose of disseminating such content, information, or materials to staff at Rallybio study sites to provide to potential eligible participants regarding J&J’s independent study.

Current wording

(2) Dissemination of J&J materials & participant revenue – derived from Rallybio’s dissemination of content, information or materials that are developed by J&J and related to the J&J-Sponsored Studies and are provided by Rallybio to staff at Rallybio study sites for the purpose of disseminating such content, information, or materials to provide to potential eligible participants regarding J&J’s independent study.

Previous wording

In April 2024, we also entered into a Securities Purchase Agreement with JJDC. Under the terms of the Securities Purchase Agreement, JJDC made an equity investment purchasing 3,636,363 shares of common stock with a par value of $0.0001 per share for a share purchase price of $1.82 per share which includes a 10% premium for an aggregate purchase price of $6.6 million. The Securities Purchase Agreement contains provisions related to the registration of the shares and the restriction on the sale or transfer of the shares for a period of time. We determined the Collaboration Agreement and Securities Purchase Agreement represented combined agreements. In accordance with ASC 606 and ASC Topic 820, Fair Value Measurement (“ASC 820”), total consideration of $1.2 million for the shares of common stock from the Securities Purchase Agreement, which represents the premium of $0.7 million and discount for lack of marketability of $0.5 million, has been allocated to revenue and will be recognized over the two year expected performance period.

Current wording

In April 2024, we also entered into a Securities Purchase Agreement with JJDC. Under the terms of the Securities Purchase Agreement, JJDC made an equity investment purchasing 3,636,363 shares of common stock with a par value of $0.0001 per share for a share purchase price of $1.82 per share which includes a 10% premium for an aggregate purchase price of $6.6 million. The Securities Purchase Agreement contains provisions related to the registration of the shares and the restriction on the sale or transfer of the shares for a period of time. We determined the Collaboration Agreement and Securities Purchase Agreement represented combined agreements. In accordance with ASC 606 and ASC Topic 820, total consideration of $1.2 million for the shares of common stock from the Securities Purchase Agreement, which represents the premium of $0.7 million and discount for lack of marketability of $0.5 million, has been allocated to revenue and will be recognized over the two year expected performance period.

Previous wording

Costs for certain activities are recognized based on an evaluation of the progress to completion of each specific contract using information and data provided to us by our vendors and analyzing the progress of our research studies or other services performed. Significant judgments and estimates are made in determining the expenses incurred balances at the end of any reporting period.

Current wording

Costs for certain activities are recognized based on an evaluation of the progress to completion of each specific contract using information and data provided to us by our vendors and analyzing the progress of our research studies or other services performed. Significant judgments and estimates are made in determining the expenses incurred at the end of any reporting period.

Previous wording

Research and development expenses were $12.9 million for the three months ended June 30, 2024, compared to $13.1 million for the three months ended June 30, 2023. The decrease of $0.2 million in 2024 as compared to 2023 was primarily due to:

Current wording

Research and development expenses were $8.2 million for the three months ended September 30, 2024, compared to $13.3 million for the three months ended September 30, 2023. The decrease of $5.0 million in 2024 as compared to 2023 was primarily due to:

Previous wording

▪a $0.7 million increase in costs related to the development of RLYB212, primarily related to an increase in clinical development costs, which was largely offset by a decrease in manufacturing and other research and development costs; and

Current wording

▪a $0.5 million decrease in RLYB212 development costs, primarily related to a decrease in manufacturing costs, which was largely offset by an increase in clinical and other research and development costs.

Previous wording

General and administrative expenses were $4.4 million and $7.0 million for the three months ended June 30, 2024 and 2023, respectively. The decrease of $2.6 million in 2024 as compared to 2023 was primarily due to:

Current wording

General and administrative expenses were $4.1 million and $6.1 million for the three months ended September 30, 2024 and 2023, respectively. The decrease of $2.0 million in 2024 as compared to 2023 was primarily due to:

Previous wording

▪a $1.2 million decrease in other related general and administrative expenses, including a reduction in our consulting fees, director and officer insurance premiums, professional fees and other related general and administrative expenses.

Current wording

▪a $1.1 million decrease in other related general and administrative expenses, including a reduction in consulting fees; and

Previous wording

▪a $0.6 million decrease in payroll and personnel-related costs, primarily related to the workforce reduction, effective March 6, 2024, in addition to lower ongoing headcount in 2024 as compared to 2023.

Current wording

▪a $0.9 million decrease in payroll and personnel-related costs, primarily related to the workforce reduction, effective March 6, 2024, in addition to lower ongoing headcount in 2024 as compared to 2023.

Previous wording

Total other income, net, for the three months ended June 30, 2024 was $1.3 million compared to $1.7 million for the three months ended June 30, 2023. The change was primarily related to a decrease in interest income from marketable securities.

Current wording

Total other income, net, for the three months ended September 30, 2024 was $1.2 million compared to $1.6 million for the three months ended September 30, 2023. The change was primarily related to a decrease in interest income from marketable securities due to a lower cash balance.

Previous wording

Collaboration and license revenue was $0.3 million during the six months ended June 30, 2024. There was no collaboration and license revenue during the six months ended June 30, 2023. The increase of $0.3 million in 2024 as compared to 2023 was related to our entrance into the Collaboration Agreement with J&J in the second quarter of 2024 and the recognition of revenue related to the collaboration performance obligations.

Current wording

Collaboration and license revenue was $0.6 million during the nine months ended September 30, 2024. There was no collaboration and license revenue during the nine months ended September 30, 2023. The increase of $0.6 million in 2024 as compared to 2023 was related to our entrance into the Collaboration Agreement with J&J in the second quarter of 2024 and the recognition of revenue related to the collaboration performance obligations.

Previous wording

Research and development expenses were $25.9 million for the six months ended June 30, 2024, compared to $24.3 million for the six months ended June 30, 2023. The increase of $1.6 million in 2024 as compared to 2023 was primarily due to:

Current wording

Research and development expenses were $34.1 million for the nine months ended September 30, 2024, compared to $37.6 million for the nine months ended September 30, 2023. The decrease of $3.5 million in 2024 as compared to 2023 was primarily due to:

Previous wording

▪a $0.6 million increase in costs related to the development of RLYB116, primarily related to an increase in manufacturing costs, which was largely offset by a decrease in clinical development and other research and development costs.

Current wording

▪a $2.1 million decrease in RLYB116 development costs, primarily related to a decrease in clinical and other research and development costs, which was partially offset by an increase in manufacturing costs; and

Previous wording

General and administrative expenses were $11.2 million and $14.1 million for the six months ended June 30, 2024 and 2023, respectively. The decrease of $2.9 million in 2024 as compared to 2023 was primarily due to:

Current wording

General and administrative expenses were $15.4 million and $20.2 million for the nine months ended September 30, 2024 and 2023, respectively. The decrease of $4.8 million in 2024 as compared to 2023 was primarily due to:

Previous wording

▪a $2.3 million decrease that included a reduction in our consulting fees, director and officer insurance premiums, professional fees and other related general and administrative expenses; and

Current wording

▪a $3.1 million decrease in consulting fees, director and officer insurance premiums, professional fees and other related general and administrative expenses; and

Previous wording

▪a $1.4 million decrease in payroll and personnel-related costs, primarily related to the workforce reduction, effective March 6, 2024, in addition to lower ongoing headcount in 2024 as compared to 2023; and

Current wording

▪a $1.7 million decrease in payroll and personnel-related costs, primarily related to the workforce reduction, effective March 6, 2024, in addition to lower ongoing headcount in 2024 as compared to 2023.

Previous wording

Total other income, net, for the six months ended June 30, 2024 was $2.7 million compared to $3.3 million for the six months ended June 30, 2023. The change was primarily related to a decrease in interest income from marketable securities.

Current wording

Total other income, net, for the nine months ended September 30, 2024 was $4.0 million compared to $4.9 million for the nine months ended September 30, 2023. The change was primarily related to a decrease in interest income from marketable securities due to a lower excess cash balance.

Previous wording

Loss on investment in joint venture for the three months ended June 30, 2024 was $0.5 million compared to $0.2 million for the three months ended June 30, 2023.

Current wording

Loss on investment in joint venture for the nine months ended September 30, 2024 was $1.8 million compared to $1.4 million for the nine months ended September 30, 2023.

Previous wording

As of June 30, 2024, we had $88.6 million of cash, cash equivalents and marketable securities.

Current wording

As of September 30, 2024, we had $75.1 million of cash, cash equivalents and marketable securities.

Previous wording

During the six months ended June 30, 2024, net cash used in operating activities was $25.9 million as compared to $32.1 million for the six months ended June 30, 2023. The decrease in net cash used in operating activities for the six months ended June 30, 2024 was primarily related to changes in working capital.

Current wording

During the nine months ended September 30, 2024, net cash used in operating activities was $39.4 million as compared to $48.6 million for the nine months ended September 30, 2023. The decrease in net cash used in operating activities for the nine months ended September 30, 2024 was primarily related to a decrease in research and development and general and administrative activities and changes in working capital.

Previous wording

Net cash provided by investing activities was $12.9 million for the six months ended June 30, 2024 as compared to $7.7 million of net cash provided by investing activities for the six months ended June 30, 2023. The increase of $5.2 million in net cash provided by investing activities was primarily related to proceeds of $44.3 million from maturities of highly-rated debt securities, partially offset by purchases of highly-rated debt securities of $29.9 million during the six months ended June 30, 2024, as compared to proceeds from maturities of highly-rated debt securities of $70.0 million, partially offset by purchases of highly-rated debt securities of $61.6 million during the six months ended June 30, 2023.

Current wording

Net cash provided by investing activities was $35.1 million for the nine months ended September 30, 2024 as compared to $10.7 million of net cash provided by investing activities for the nine months ended September 30, 2023. The increase of $24.3 million in net cash provided by investing activities was primarily related to proceeds of $68.7 million from maturities of highly-rated debt securities, partially offset by purchases of highly-rated debt securities of $31.6 million during the nine months ended September 30, 2024, as compared to proceeds from maturities of highly-rated debt securities of $107.9 million, partially offset by purchases of highly-rated debt securities of $95.7 million during the nine months ended September 30, 2023.

Previous wording

Net cash provided by financing activities during the six months ended June 30, 2024 was $5.2 million, representing proceeds from the issuance of common stock pursuant to the Securities Purchase Agreement with JJDC, after deducting offering costs and accounting for the total consideration allocation related to the Collaboration Agreement of $1.2 million. Net cash provided by financing activities during the six months ended June 30, 2023 was $0.1 million, representing proceeds from the issuance of common stock under the Rallybio Corporation 2021 Employee Stock Purchase Plan, partially offset by offering cost payments made related to our November 2022 follow-on offering.

Current wording

Net cash provided by financing activities during the nine months ended September 30, 2024 was $5.2 million, representing proceeds from the issuance of common stock pursuant to the Securities Purchase Agreement with JJDC, after deducting offering costs and accounting for the total consideration allocation related to the Collaboration Agreement of $1.2 million. Net cash provided by financing activities during the nine months ended September 30, 2023 was $0.1 million, representing proceeds from the issuance of common stock under the Rallybio Corporation 2021 Employee Stock Purchase Plan, partially offset by offering cost payments made related to our November 2022 follow-on offering.

Previous wording

There have been no other material changes in our contractual obligations and commitments during the six months ended June 30, 2024 from those described under the heading “Management’s Discussion and Analysis of Financial Condition and Results of Operations—Contractual Obligations” in our Annual Report.

Current wording

There have been no other material changes in our contractual obligations and commitments during the nine months ended September 30, 2024 from those described under the heading “Management’s Discussion and Analysis of Financial Condition and Results of Operations—Contractual Obligations” in our Annual Report.