– Introduced preliminary outcomes of a Section 1 scientific trial of losmapimod in FSHD sufferers –
– Initiated IND-enabling research for FTX-6058 for the potential remedy of sickle cell illness and beta-thalassemia and introduced pre-clinical information displaying improve in HbF ranges to ~30% of whole hemoglobin –
CAMBRIDGE, Mass., Nov. 14, 2019 (GLOBE NEWSWIRE) — Fulcrum Therapeutics, Inc. (Nasdaq: FULC), a clinical-stage biopharmaceutical firm centered on enhancing the lives of sufferers with genetically outlined uncommon ailments, right this moment supplied a enterprise replace and reported monetary outcomes for the third quarter of 2019.
“This previous quarter, we made vital progress in advancing our pipeline and executing on our objective of making therapeutics focused at genetically outlined uncommon ailments. We introduced encouraging Section 1 scientific outcomes from our lead program, losmapimod in sufferers with FSHD, which assist the design and dose collection of our ongoing Section 2 scientific trials. Moreover, we introduced our plans to advance our second program, FTX-6058 for the potential remedy of sickle cell illness and beta-thalassemia,” stated Robert J. Gould, Ph.D., Fulcrum’s president and chief govt officer. “We sit up for advancing these two packages by means of our ongoing Section 2 trials with losmapimod and our IND-enabling research with FTX-6058 and using our product engine to determine and validate drug targets to handle ailments brought on by the mis-expression of sure genes.”
Third Quarter 2019 and Latest Enterprise Highlights
- In October 2019, Fulcrum introduced Section 1 information from its scientific trial of losmapimod in facioscapulohumeral dystrophy (FSHD) sufferers and associated plans for utilizing molecular biomarkers indicative of the basis reason for illness within the Firm’s ongoing Section 2 scientific trials of losmapimod on the Worldwide Annual Congress of the World Muscle Society in Copenhagen. The outcomes confirmed that losmapimod was effectively tolerated with no critical hostile occasions (SAEs) reported, and that remedy with losmapimod demonstrated dose-dependent pharmacokinetics (PK) and goal engagement (TE) in blood. These information assist the collection of the 15 mg dose of losmapimod taken orally twice every day within the Firm’s ongoing Section 2b placebo-controlled 24-week scientific trial, known as ReDUX4, in addition to its ongoing Section 2 open label-study, of losmapimod for the remedy of sufferers with FSHD.
- In October 2019, Fulcrum introduced progress within the Firm’s plan to advance the event program for FTX-6058 for the potential remedy of sickle cell illness and beta-thalassemia. Fulcrum has initiated IND-enabling research for FTX-6058 and anticipates submitting an IND in mid-2020. In pre-clinical analysis, remedy with FTX-6058 was proven to extend HbF ranges to ~30% of whole hemoglobin as measured by HPLC and mass spectrometry strategies in human erythroid progenitor cells from a number of donors.
- In September 2019, Fulcrum introduced the appointment of Pamela Strode as senior vp, regulatory affairs and high quality assurance. She beforehand served as senior vp, regulatory affairs and high quality assurance at Epizyme, Inc.
Third Quarter 2019 Monetary Outcomes
- Money Place: As of September 30, 2019, money and money equivalents have been $101.6 million, as in comparison with $72.eight million as of December 31, 2018. Based mostly on its present plans, the Firm expects that its present money and money equivalents, together with the proceeds from its July 2019 preliminary public providing, might be enough to allow it to fund its working bills and capital expenditure necessities into the third quarter of 2021.
- R&D Bills: Analysis and growth bills have been $13.5 million for the third quarter of 2019, as in comparison with $7.zero million for the third quarter of 2018. The rise of $6.5 million was primarily as a result of a $2.5 million improve related to the achievement of a milestone due below the best of reference and license settlement with GlaxoSmithKline plc through the third quarter of 2019, elevated prices associated to the development of losmapimod for the remedy of FSHD, together with elevated exterior prices to assist Fulcrum’s ongoing and deliberate scientific trials, in addition to elevated personnel-related prices as a result of further headcount to assist the expansion of Fulcrum’s analysis and growth group.
- G&A Bills: Common and administrative bills have been $three.5 million for the third quarter of 2019, as in comparison with $2.1 million for the third quarter of 2018. The rise of $1.four million was primarily as a result of elevated personnel-related prices as a result of further headcount, in addition to elevated consulting and professional charges.
- Web Loss: Web loss was $16.5 million for the third quarter of 2019, as in comparison with a internet lack of $eight.9 million for the third quarter of 2018.
FSHD is characterised by progressive skeletal muscle loss that originally causes weak spot in muscle mass within the face, shoulders, arms and trunk, and progresses to weak spot all through the decrease physique. Skeletal muscle weak spot leads to vital bodily limitations, together with an incapability to smile and problem utilizing arms for actions, with many sufferers in the end changing into dependent upon the usage of a wheelchair for every day mobility.
FSHD is brought on by mis-expression of DUX4 in skeletal muscle, ensuing within the presence of DUX4 proteins which can be poisonous to muscle tissue. Usually, DUX4-driven gene expression is restricted to early embryonic growth, after which era the DUX4 gene is silenced. In folks with FSHD, the DUX4 gene is turned “on” on account of a genetic mutation. The result’s loss of life of muscle and its substitute by fats, resulting in skeletal muscle weak spot and progressive incapacity. There aren’t any authorized therapies for FSHD, some of the frequent types of muscular dystrophy, with an estimated affected person inhabitants of 16,000 to 38,000 in america alone.
Losmapimod is a selective p38α/β mitogen activated protein kinase (MAPK) inhibitor that was solely in-licensed by Fulcrum Therapeutics following Fulcrum’s discovery of the function of p38α/β inhibitors within the discount of DUX4 expression and an in depth overview of identified compounds. Using its inside product engine, Fulcrum found that inhibition of p38α/β lowered expression of the DUX4 gene in muscle cells derived from sufferers with FSHD. Though losmapimod has by no means beforehand been explored in muscular dystrophies, it has been evaluated in additional than three,500 topics in scientific trials throughout a number of different indications, together with in a number of Section 2 trials and a Section three trial. No security alerts have been attributed to losmapimod in any of those trials. Fulcrum is at the moment conducting Section 2 trials investigating the protection, tolerability, and efficacy of losmapimod to deal with the basis reason for FSHD.
About Sickle Cell Illness
Sickle cell illness (SCD) is a genetic dysfunction of the purple blood cells brought on by a mutation within the HBB gene. This gene encodes a protein that could be a key element of hemoglobin, a protein complicated whose operate is to move oxygen within the physique. The results of the mutation is much less environment friendly oxygen transport and the formation of purple blood cells which have a sickle form. These sickle formed cells are a lot much less versatile than wholesome cells and might block blood vessels or rupture cells. SCD sufferers sometimes endure from critical scientific penalties, which can embrace anemia, ache, infections, stroke, coronary heart illness, pulmonary hypertension, kidney failure, liver illness and lowered life expectancy.
Beta-thalassemia is a uncommon blood dysfunction brought on by genetic mutations within the HBB gene, that are related to the absence or lowered manufacturing of beta-globin – one of many two proteins that comprise grownup hemoglobin. This leads to an abnormally low stage of hemoglobin in addition to an extra of alpha-globin chains, inflicting destruction of purple blood cells.
Beta-thalassemia has been clinically characterised into three types, relying on illness severity: main, intermedia and minor. Essentially the most extreme type is usually identified shortly after start and is characterised by life-threatening anemia. Pediatric sufferers don’t develop and achieve weight on the typical charges, and infrequently have liver, coronary heart and bone issues. Many sufferers with beta-thalassemia main require power blood transfusions as a result of extreme anemia that outcomes from low hemoglobin ranges. Beta-thalassemia intermedia is a much less extreme type of the illness that leads to delicate to reasonable anemia. These sufferers typically require blood transfusions relying on the severity of their signs. Sufferers with beta-thalassemia minor endure from very delicate anemia and customarily don’t require remedy.
About Fulcrum Therapeutics
Fulcrum Therapeutics is a clinical-stage biopharmaceutical firm centered on enhancing the lives of sufferers with genetically outlined uncommon ailments in areas of excessive unmet medical want. Fulcrum’s proprietary product engine identifies drug targets which may modulate gene expression to deal with the identified root reason for gene mis-expression. Please go to www.fulcrumtx.com.
This press launch comprises “forward-looking statements” inside the that means of the Personal Securities Litigation Reform Act of 1995 that contain substantial dangers and uncertainties, together with statements relating to the event standing of the Firm’s product candidates, the timing of availability of scientific trial information and the Firm’s potential to fund its operations with money available. All statements, aside from statements of historic info, contained on this press launch, together with statements relating to the Firm’s technique, future operations, future monetary place, prospects, plans and targets of administration, are forward-looking statements. The phrases “anticipate,” “imagine,” “proceed,” “might,” “estimate,” “count on,” “intend,” “could,” “plan,” “potential,” “predict,” “challenge,” “ought to,” “goal,” “will,” “would” and related expressions are meant to determine forward-looking statements, though not all forward-looking statements comprise these figuring out phrases. Any forward-looking statements are based mostly on administration’s present expectations of future occasions and are topic to quite a few dangers and uncertainties that might trigger precise outcomes to vary materially and adversely from these set forth in, or implied by, such forward-looking statements. These dangers and uncertainties embrace, however will not be restricted to, dangers related to Fulcrum’s potential to acquire and preserve vital approvals from the FDA and different regulatory authorities; proceed to advance its product candidates in scientific trials; replicate in later scientific trials optimistic outcomes present in preclinical research and early-stage scientific trials of losmapimod and its different product candidates; advance the event of its product candidates below the timelines it anticipates in present and future scientific trials; receive, preserve or shield mental property rights associated to its product candidates; handle bills; and lift the substantial further capital wanted to realize its enterprise targets. For a dialogue of different dangers and uncertainties, and different essential components, any of which might trigger the Firm’s precise outcomes to vary from these contained within the forward-looking statements, see the “Threat Elements” part, in addition to discussions of potential dangers, uncertainties and different essential components, within the Firm’s most up-to-date filings with the Securities and Alternate Fee. As well as, the forward-looking statements included on this press launch signify the Firm’s views as of the date hereof and shouldn’t be relied upon as representing the Firm’s views as of any date subsequent to the date hereof. The Firm anticipates that subsequent occasions and developments will trigger the Firm’s views to vary. Nevertheless, whereas the Firm could elect to replace these forward-looking statements sooner or later sooner or later, the Firm particularly disclaims any obligation to take action.
Fulcrum Therapeutics, Inc.
Chosen Consolidated Stability Sheet Information
|Money and money equivalents||$||101,597||$||72,797|
|Convertible most popular inventory||—||139,670|
|Whole stockholders’ fairness (deficit)||102,128||(63,670||)|
(1) We outline working capital as present belongings minus present liabilities.
Fulcrum Therapeutics, Inc.
Consolidated Statements of Operations and Complete Loss
(In hundreds, besides per share information)
|Three Months Ended
|9 Months Ended
|Analysis and growth||$||13,496||$||6,963||$||58,985||$||18,324|
|Common and administrative||three,510||2,125||eight,742||5,923|
|Whole working bills||$||17,006||$||9,088||$||67,727||$||24,247|
|Loss from operations||(17,006||)||(9,088||)||(67,727||)||(24,247||)|
|Different earnings, internet:|
|Curiosity earnings, internet||457||138||1,151||133|
|Web loss and complete loss||$||(16,542||)||$||(eight,943||)||$||(66,554||)||$||(23,729||)|
|Cumulative convertible most popular inventory dividends||(796||)||(1,858||)||(7,128||)||(three,736||)|
|Web loss attributable to frequent stockholders||$||(17,338||)||$||(10,801||)||$||(73,682||)||$||(27,465||)|
|Web loss per share attributable to frequent stockholders, primary and diluted||$||(zero.97||)||$||(eight.08||)||$||(10.33||)||$||(23.28||)|
|Weighted common variety of frequent shares utilized in internet loss per share attributable to frequent stockholders, primary and diluted||17,785||1,337||7,133||1,180|
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