BridgeBio Pharma Reports Fourth Quarter and Full Year 2023 Financial Results and Business Update

On February 22, 2024 BridgeBio Pharma, Inc. (Nasdaq: BBIO) (BridgeBio or the Company), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, reported its financial results for the fourth quarter and full year ended December 31, 2023, and provided an update on the Company’s operations (Press release, BridgeBio, FEB 24, 2024, View Source [SID1234640418]).

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"Our focus this year is executing on the launch of acoramidis for patients with ATTR cardiomyopathy," said Neil Kumar, Ph.D., founder and CEO of BridgeBio. "At the same time, we are also focused on fully enrolling three ongoing Phase 3 clinical trials by the end of 2024. Finally, we hope that reading out potentially exciting data from our Phase 1/2 trial in congenital adrenal hyperplasia later this year will let us take the next step in serving that patient community."

BridgeBio’s key programs:


Acoramidis (AG10) – Transthyretin (TTR) stabilizer for transthyretin amyloid cardiomyopathy (ATTR-CM):
o
The Company filed an NDA for acoramidis for the treatment of ATTR-CM with the US FDA; the NDA was accepted for review with a PDUFA date of November 29, 2024. The Company has also filed a Marketing Authorization Application for acoramidis with the EMA, which has been accepted for review.
o
The regulatory filings were based on data from the Phase 3 ATTRibute-CM study, which met its primary endpoint (Win Ratio of 1.8) with a highly statistically significant p-value (p<0.0001). Additional results from ATTRibute-CM include:

An 81% survival rate on acoramidis, which approaches the survival rate in the age-matched U.S. database (~85%), and a 0.29 mean annual CVH rate on acoramidis, which approaches the annual hospitalization rate observed in the broader U.S. Medicare population (~0.26);

Improvements from baseline observed for a large proportion of participants treated with acoramidis on laboratory and functional measures including n-terminal prohormone of brain natriuiretic peptide (NT-proBNP) and 6-minute walk distance;

Rapid clinical benefit on the composite endpoint of ACM and CVH in participants treated with acoramidis, demonstrated by placebo and acoramidis time-to-first event Kaplan-Meier curves for a composite of ACM and CVH that separated at Month 3 and continued to diverge steadily through Month 30 as presented at the American Heart Association Scientific Sessions in November 2023; and

Acoramidis was well-tolerated with no safety signals of potential clinical concern identified.
o
The Company also shared positive results of an open-label, single-arm Phase 3 study conducted in Japan by licensing partner Alexion, AstraZeneca Rare Disease, including that no mortality was reported over the 30 month acoramidis treatment period.
o
Additional detailed results of ATTRibute-CM are planned for presentation at 2024 medical meetings.

Low-dose infigratinib – FGFR1-3 inhibitor for achondroplasia and hypochondroplasia:
o
In December 2023, the Company announced the dosing of the first child in PROPEL 3, its global Phase 3 registrational study of infigratinib in achondroplasia.
o
In February 2024, the Company announced a partnership with Kyowa Kirin wherein the Company grants Kyowa Kirin an exclusive license to develop and commercialize infigratinib for achondroplasia, hypochondroplasia, and other skeletal dysplasias in Japan; in exchange, the Company will receive an upfront payment of $100 million as well as royalties up to the high-twenties percent on sales of infigratinib in Japan, with the potential for additional milestone-based payments.
2

o
The Company is committed to exploring the potential of infigratinib on the wider medical and functional impacts of achondroplasia, hypochondroplasia and other skeletal dysplasias, and anticipates initiating its clinical program for hypochondroplasia in 2024.

BBP-418 – Glycosylation substrate for limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9):
o
FORTIFY, the global Phase 3 registrational trial of BBP-418, continues to enroll in the U.S. with clinical trial sites planned for Europe and Australia. Full enrollment of the interim analysis population is expected in 2024. The Company believes there is potential to pursue Accelerated Approval for BBP-418 based on recent interactions with the FDA on the use of glycosylated αDG levels as a surrogate endpoint.


Encaleret – Calcium-sensing receptor (CaSR) inhibitor for autosomal dominant hypocalcemia type 1 (ADH1):
o
CALIBRATE, the Phase 3 clinical trial of encaleret, continues to enroll; the Company anticipates sharing topline data from CALIBRATE in 2025.

Recent Corporate Updates:


Secured up to $1.25 billion of capital from Blue Owl and CPP Investments: The raise includes $500 million in cash from Blue Owl and CPP Investments available upon FDA approval of acoramidis in exchange for a 5% royalty on future global net sales of acoramidis, as well as a $450 million credit facility from Blue Owl that refinanced existing senior secured credit, extending maturity from 2026 to 2029 subject to certain conditions.

Fourth Quarter and Full Year 2023 Financial Results:

Cash, Cash Equivalents, Marketable Securities and Short-term Restricted Cash

Cash, cash equivalents and short-term restricted cash, totaled $392.6 million as of December 31, 2023, compared to cash, cash equivalents, marketable securities and short-term restricted cash of $466.2 million as of December 31, 2022. The net decrease of $73.6 million in cash, cash equivalents, marketable securities and short-term restricted cash was primarily attributable to net cash used in operating activities of $527.7 million and $6.9 million in repurchase of shares to satisfy tax withholdings, primarily offset by net proceeds received of $449.8 million from various equity financings, $6.0 million from stock option exercises, and $3.4 million from common stock issuances under our employee stock purchase plan during the year ended December 31, 2023.

Revenue

Revenue for the three months and year ended December 31, 2023 were $1.7 million and $9.3 million, respectively, as compared to $1.9 million and $77.6 million for the same periods in the prior year, respectively. The net decreases of $0.2 million and $68.3 million for the three months and year ended December 31, 2023, respectively, compared to the same periods in the prior year, were primarily due to license revenue recognized in 2022 upon the transfer of the license in accordance with the Navire-BMS License Agreement which was entered into in May 2022.

3

Operating Costs and Expenses

Operating costs and expenses for the three months and year ended December 31, 2023 were $179.2 million and $616.7 million, respectively, compared to $131.1 million and $589.9 million, for the same periods in the prior year, respectively.

The overall increase of $48.1 million in operating costs and expenses for the three months ended December 31, 2023, compared to the same period in the prior year, was primarily due to an increase of $39.3 million in research and development and other expenses (R&D) to advance the Company’s pipeline of development programs, an increase of $15.7 million in selling, general and administrative (SG&A) expenses to support commercialization readiness efforts, offset by a decrease of $6.9 million in restructuring, impairment and related charges given that the majority of the restructuring initiatives occurred in the prior year.

The overall increase of $26.8 million in operating costs and expenses for the year ended December 31, 2023 , compared to the same period in the prior year, was primarily due to an increase of $55.2 million in R&D expenses to advance the Company’s pipeline of development programs, an increase of $7.4 million in SG&A expenses to support commercialization readiness efforts, offset by a decrease of $35.8 million in restructuring, impairment and related charges given that the majority of the restructuring initiatives occurred in the prior year.

Restructuring, impairment and related charges for the three months and year ended December 31, 2023, amounted to $0.8 million and $7.9 million, respectively. These charges primarily consisted of winding down, exit costs, and severance and employee-related costs. Restructuring, impairment and related charges for the same periods in the prior year were $7.7 million and $43.8 million, respectively. These charges primarily consisted of impairments and write-offs of long-lived assets, severance and employee-related costs, and exit and other related costs.

Stock-based compensation expenses included in operating costs and expenses for the three months ended December 31, 2023 were $37.1 million, of which $22.5 million is included in R&D expenses, and $14.6 million is included in SG&A expenses. Stock-based compensation expenses included in operating costs and expenses for the same period in the prior year were $22.6 million, of which $8.9 million is included in R&D expenses, and $13.6 million is included in SG&A expenses.

Stock-based compensation expenses included in operating costs and expenses for the year ended December 31, 2023 were $115.0 million, of which $61.6 million is included in R&D expenses, and $53.4 million is included in SG&A expenses. Stock-based compensation expenses included in operating costs and expenses for the same period in the prior year were $93.8 million, of which $38.0 million is included in R&D expenses, $54.7 million is included in SG&A expenses, and $1.2 million is included in restructuring, impairment and related charges.

"Coming off of our recent royalty financing, we find ourselves well capitalized to launch acoramidis this year alongside strong new partners who share our confidence in acoramidis’ potential in the ATTR-CM market," said Brian Stephenson, Ph.D., CFA, Chief Financial Officer of BridgeBio. "We are excited for this launch, as well as for the continued advancement of our late stage pipeline, which we hope will allow us to serve patients with genetic diseases both directly with the advancement of those medicines towards the market as well as by diversifying our top line revenue and enabling reinvestment into the R&D and business development opportunities that will allow us to be sustainable in the long term."

4

BRIDGEBIO PHARMA, INC.

Condensed Consolidated Statements of Operations

(in thousands, except shares and per share amounts)

Three Months Ended December 31,

Year Ended December 31,

2023

2022

2023

2022

(Unaudited)

(Unaudited)

(1)

Revenue

$

1,745

$

1,870

$

9,303

$

77,648

Operating costs and expenses:

Research, development and other expenses

130,824

91,549

458,157

402,896

Selling, general and administrative

47,583

31,862

150,590

143,189

Restructuring, impairment and related charges

754

7,691

7,926

43,765

Total operating costs and expenses

179,161

131,102

616,673

589,850

Loss from operations

(177,416

)

(129,232

)

(607,370

)

(512,202

)

Other income (expense), net:

Interest income

5,578

4,092

18,038

7,542

Interest expense

(20,268

)

(19,990

)

(81,289

)

(80,438

)

Gain from sale of priority review voucher, net

107,946

Other income (expense), net

21,778

4,560

17,370

(7,500

)

Total other income (expense), net

7,088

(11,338

)

(45,881

)

27,550

Net loss

(170,328

)

(140,570

)

(653,251

)

(484,652

)

Net loss attributable to redeemable convertible
noncontrolling interests and noncontrolling interests

2,180

2,979

10,049

3,469

Net loss attributable to common stockholders
of BridgeBio

$

(168,148

)

$

(137,591

)

$

(643,202

)

$

(481,183

)

Net loss per share, basic and diluted

$

(0.96

)

$

(0.92

)

$

(3.95

)

$

(3.26

)

Weighted-average shares used in computing net
loss per share, basic and diluted

174,462,332

149,344,380

162,791,511

147,473,076

Three Months Ended December 31,

Year Ended December 31,

Stock-based Compensation

2023

2022

2023

2022

(Unaudited)

(Unaudited)

(1)

Research, development and others

$

22,495

$

8,941

$

61,647

$

37,987

Selling, general and administrative

14,638

13,643

53,369

54,669

Restructuring, impairment and related charges

1,172

Total stock-based compensation

$

37,133

$

22,584

$

115,016

$

93,828

(1)

The condensed consolidated financial statements as of and for the year ended December 31, 2022 are derived from the audited consolidated financial statements as of that date.

5

BRIDGEBIO PHARMA, INC.

Condensed Consolidated Balance Sheets

(In thousands)

December 31,

December 31,

2023

2022

(Unaudited)

(1)

Assets

Cash, cash equivalents and marketable securities

$

375,935

$

428,269

Investment in equity securities

58,949

43,653

Receivable from licensing and collaboration agreements

1,751

17,079

Short-term restricted cash

16,653

37,930

Prepaid expenses and other current assets

24,305

21,922

Property and equipment, net

11,816

14,569

Operating lease right-of-use assets

8,027

10,678

Intangible assets, net

26,319

28,712

Other assets

22,625

20,224

Total assets

$

546,380

$

623,036

Liabilities, Redeemable Convertible Noncontrolling Interests and Stockholders’ Deficit

Accounts payable

$

10,655

$

11,558

Accrued and other liabilities

129,061

106,195

Operating lease liabilities

13,109

15,949

2029 Notes, net

736,905

734,988

2027 Notes, net

543,379

541,634

Term loan, net

446,445

430,993

Other long-term liabilities

9,361

26,643

Redeemable convertible noncontrolling interests

478

(1,589

)

Total BridgeBio stockholders’ deficit

(1,354,257

)

(1,254,617

)

Noncontrolling interests

11,244

11,282

Total liabilities, redeemable convertible noncontrolling interests and stockholders’ deficit

$

546,380

$

623,036

(1)

The condensed consolidated financial statements as of and for the year ended December 31, 2022 are derived from the audited consolidated financial statements as of that date.

6

BRIDGEBIO PHARMA, INC.

Condensed Consolidated Statements of Cash Flows

(In thousands)

Year Ended December 31,

2023

2022

(Unaudited)

(1)

Operating activities:

Net loss

$

(653,251

)

$

(484,652

)

Adjustments to reconcile net loss to net cash used in operating activities:

Stock-based compensation

108,710

91,559

Depreciation and amortization

6,494

6,771

Noncash lease expense

4,032

5,172

Accrual of payment-in-kind interest on term loan

10,207

13,562

Loss on deconsolidation of PellePharm

1,241

(Gain) loss from investment in equity securities, net

(18,314

)

8,222

Fair value of shares issued under a license agreement

4,567

Accretion of debt

8,907

8,570

Fair value adjustment of warrants

(984

)

1,571

Loss on sale of certain assets

6,261

Impairment of long-lived assets

12,720

Gain from sale of priority review voucher, excluding transaction costs

(110,000

)

Gain from recognition of receivable from licensing and collaboration agreement

(12,500

)

Other noncash adjustments

181

604

Changes in operating assets and liabilities:

Receivable from licensing and collaboration agreements

15,328

15,169

Prepaid expenses and other current assets

(2,702

)

7,671

Other assets

(1,546

)

10,971

Accounts payable

2,780

(349

)

Accrued compensation and benefits

7,802

(2,362

)

Accrued research and development liabilities

(9,855

)

(4,309

)

Operating lease liabilities

(4,829

)

(6,245

)

Deferred revenue

(5,438

)

15,262

Accrued professional and other liabilities

3,517

(7,729

)

Net cash used in operating activities

(527,720

)

(419,494

)

Investing activities:

Purchases of marketable securities

(29,726

)

(137,493

)

Maturities of marketable securities

82,550

479,688

Purchases of investment in equity securities

(107,538

)

(55,562

)

Sales of investment in equity securities

110,556

52,835

Decrease in cash and cash equivalents resulting from deconsolidation of PellePharm

(503

)

Payment for intangible asset

(1,500

)

Proceeds from sale of priority review voucher

110,000

Proceeds from sale of certain assets

10,000

Purchases of property and equipment

(1,306

)

(4,821

)

Net cash provided by investing activities

54,033

453,147

Financing activities:

Proceeds from issuance of common stock through Private Placement offering, net

240,796

Proceeds from issuance of common stock through Follow-on offering, net

144,049

Proceeds from issuance of common stock through ATM offering, net

64,965

4,852

Transactions with noncontrolling interests

(801

)

Repayment of term loan

(20,486

)

Proceeds from BridgeBio common stock issuances under ESPP

3,398

2,558

Repurchase of RSU shares to satisfy tax withholding

(6,880

)

(1,561

)

Proceeds from stock option exercises, net of repurchases

6,008

666

Other financing activities

837

Net cash provided by (used in) financing activities

451,535

(13,134

)

Net increase (decrease) in cash, cash equivalents and restricted cash

(22,152

)

20,519

Cash, cash equivalents and restricted cash at beginning of period

416,884

396,365

Cash, cash equivalents and restricted cash at end of period

$

394,732

$

416,884

7

Year Ended December 31,

2023

2022

(Unaudited)

(1)

Supplemental Disclosure of Cash Flow Information:

Cash paid for interest

$

61,108

$

54,443

Supplemental Disclosures of Noncash Investing and Financing Information:

Unpaid property and equipment

$

100

$

47

Recognized intangible asset recorded in "Other accrued and other long-term liabilities"

$

$

11,000

Transfers (to) from noncontrolling interests

$

(10,534

)

$

(3,512

)

Payment-in-kind interest added to principal of term loan

$

$

1,763

Reconciliation of Cash, Cash Equivalents and Restricted Cash:

Cash and cash equivalents

$

375,935

$

376,689

Short-term restricted cash

16,653

37,930

Restricted cash — Included in "Other assets"

2,144

2,265

Total cash, cash equivalents and restricted cash at end of periods

$

394,732

$

416,884

(1)

The condensed consolidated financial statements as of and for the year ended December 31, 2022 are derived from the audited consolidated financial statements as of that date.

TG Therapeutics to Host Conference Call on Fourth Quarter and Year-End 2023 Financial Results and Business Update

On February 23, 2024 TG Therapeutics, Inc. (NASDAQ: TGTX), reported that a conference call will be held on Wednesday, February 28, 2024, at 8:30 AM ET to discuss results for the fourth quarter and year-end 2023 and provide a business outlook for 2024. Michael S. Weiss, Chairman and Chief Executive Officer, will host the call (Press release, TG Therapeutics, FEB 23, 2024, https://ir.tgtherapeutics.com/news-releases/news-release-details/tg-therapeutics-host-conference-call-fourth-quarter-and-year-3 [SID1234640422]).

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In order to participate in the conference call, please call 1-877-407-8029 (U.S.), 1-201-689-8029 (outside the U.S.), Conference Title: TG Therapeutics Fourth Quarter and Year End Update Call. A live webcast of this presentation will be available on the Events page, located within the Investors & Media section, of the Company’s website at www.tgtherapeutics.com. An audio recording of the conference call will also be available for replay at www.tgtherapeutics.com, for a period of 30 days after the call.

TG Therapeutics will announce its financial results for this period in a press release to be issued prior to the call.

Silence Therapeutics Achieves $10 Million Milestone Payment from AstraZeneca Collaboration Following the Initiation of Phase 1 Trial

On February 23, 2024 Silence Therapeutics plc, Nasdaq: SLN ("Silence" or "the Company"), an experienced and innovative biotechnology company committed to transforming people’s lives by silencing diseases through precision engineered medicines, reported that the initiation by AstraZeneca of a phase 1 clinical trial of the first product candidate under its siRNA (short interfering RNA) collaboration, has triggered a $10.0 million milestone payment to Silence (Press release, Silence Therapeutics, FEB 23, 2024, View Source [SID1234640421]).

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"This represents the first clinical milestone under our collaboration with AstraZeneca and the third program to enter the clinic from our mRNAi GOLD platform," said Craig Tooman, President and CEO of Silence. "This is a very exciting time for Silence as we are beginning to establish ourselves as a platform company progressing multiple clinical programs targeting both rare and common genetic diseases. In addition to this great achievement under our collaboration with AstraZeneca, we are also pleased with the continued advancement of our proprietary pipeline with encouraging clinical data now emerging from our zerlasiran program in high Lp(a) and divesiran program in polycythemia vera."

"We are pleased to have achieved this important clinical milestone through our collaboration with Silence Therapeutics. Our goal in working together is to advance these novel programs and develop the next wave of innovative therapies that address cardiovascular, renal and metabolic diseases," said Regina Fritsche Danielson, SVP, Early Cardiovascular, Renal and Metabolism, BioPharmaceuticals R&D, AstraZeneca.

Silence and AstraZeneca initiated a multi-target collaboration in March 2020 focused on using Silence’s proprietary mRNAi GOLD platform to develop siRNA therapeutics for cardiovascular, renal, metabolic and respiratory diseases. Under the agreement, AstraZeneca will pay Silence an option fee of $10 million for each selected target at the point of candidate nomination. The deal covers up to ten targets. For each target selected, Silence is eligible for up to $140 million in development milestones and up to $250 million in commercialization milestones as well as tiered royalties on net sales ranging from high single digit to low double digit.

Biodexa Announces Positive Top Line Phase I Clinical Trial Results for Diffuse Midline Glioma and Provides R&D Update

On February 23, 2024 Biodexa Pharmaceuticals PLC, a clinical stage biopharmaceutical company developing a pipeline of innovative products for the treatment of diseases with unmet medical needs including Type 1 diabetes and rare / orphan brain cancers, reported an R&D update including positive top-line clinical trial results of a recently completed Phase 1 study of MTX110 in patients with diffuse midline glioma, or DMG, and results of a preclinical experiment designed to demonstrate tolimidone’s potential for beta cell proliferation in an in vitro model (Press release, Midatech Pharma, FEB 23, 2024, View Source [SID1234640419]).

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MTX110

In an investigator initiated study conducted by Columbia University Irving Medical Center, patients newly diagnosed with DMG were administered MTX110 via convection enhanced delivery ("CED") using a subcutaneous pump connected to a catheter directly implanted into the pons in a 3+3 dose-escalating design (NCT 04264143). As this was the first ever study of repeated infusions to the pons via an implanted CED catheter, the primary objective of the study was safety and tolerability and, accordingly, the number of infusions was limited to two, each of 48 hours, 7 days apart. Nine patients were treated in the study (30 M group, n=3; 60 M group, n=4; 90 M group (optimal dose), n=2). One patient in the 60 M group suffered a severe adverse event assessed by the investigators as not related to the study drug but related to the infusion and tumor anatomy. Although the study was not powered to reliably demonstrate efficacy, median overall survival (OS) of patients in the study was 16.5 months. This compares favourably with median survival rate in a cohort of 316 cases of 10.0 months (Jansen et al, 2015. Neuro-Oncology 17(1):160-166).

Study investigators are planning to present detailed results of the trial at the 21st International Symposium on Pediatric Neuro-Oncology (ISPNO 2024) being held on June 28-July 2, 2024 in Philadelphia, PA.

Tolimidone

On the Company’s behalf, a CRO conducted an in vitro experiment designed to demonstrate tolimidone’s potential for beta cell proliferation using reaggregated pancreatic islets. The results of the experiment were inconclusive in that they did not correlate with the results previously seen in in vitro and in vivo studies of tolimidone. The Company believes there are a few possible explanations to the outcome of this in vitro study and accordingly, plans to move ahead rapidly with an in vivo preclinical study with similar objectives while continuing preparations for its planned Phase IIa open-label study of tolimidone in patients with Type 1 diabetes due to start recruitment later this year.

Biodexa Announces Positive Top Line Phase I Clinical Trial Results for Diffuse Midline Glioma and Provides R&D Update

On February 23, 2024 Biodexa Pharmaceuticals PLC, a clinical stage biopharmaceutical company developing a pipeline of innovative products for the treatment of diseases with unmet medical needs including Type 1 diabetes and rare / orphan brain cancers, reported an R&D update including positive top-line clinical trial results of a recently completed Phase 1 study of MTX110 in patients with diffuse midline glioma, or DMG, and results of a preclinical experiment designed to demonstrate tolimidone’s potential for beta cell proliferation in an in vitro model (Press release, Midatech Pharma, FEB 23, 2024, View Source [SID1234640419]).

Schedule your 30 min Free 1stOncology Demo!
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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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MTX110

In an investigator initiated study conducted by Columbia University Irving Medical Center, patients newly diagnosed with DMG were administered MTX110 via convection enhanced delivery ("CED") using a subcutaneous pump connected to a catheter directly implanted into the pons in a 3+3 dose-escalating design (NCT 04264143). As this was the first ever study of repeated infusions to the pons via an implanted CED catheter, the primary objective of the study was safety and tolerability and, accordingly, the number of infusions was limited to two, each of 48 hours, 7 days apart. Nine patients were treated in the study (30 M group, n=3; 60 M group, n=4; 90 M group (optimal dose), n=2). One patient in the 60 M group suffered a severe adverse event assessed by the investigators as not related to the study drug but related to the infusion and tumor anatomy. Although the study was not powered to reliably demonstrate efficacy, median overall survival (OS) of patients in the study was 16.5 months. This compares favourably with median survival rate in a cohort of 316 cases of 10.0 months (Jansen et al, 2015. Neuro-Oncology 17(1):160-166).

Study investigators are planning to present detailed results of the trial at the 21st International Symposium on Pediatric Neuro-Oncology (ISPNO 2024) being held on June 28-July 2, 2024 in Philadelphia, PA.

Tolimidone

On the Company’s behalf, a CRO conducted an in vitro experiment designed to demonstrate tolimidone’s potential for beta cell proliferation using reaggregated pancreatic islets. The results of the experiment were inconclusive in that they did not correlate with the results previously seen in in vitro and in vivo studies of tolimidone. The Company believes there are a few possible explanations to the outcome of this in vitro study and accordingly, plans to move ahead rapidly with an in vivo preclinical study with similar objectives while continuing preparations for its planned Phase IIa open-label study of tolimidone in patients with Type 1 diabetes due to start recruitment later this year.