daniel-gordon/biopharma-dive huggingface dataset:
To support the launch Ardelyx has secured additional debt financing from SLR Capital Partners giving it access to an initial $50 million and potentially another 50 million at a later date
The sale of the PRVs obtained as a result of the FDA approval of VYONDYS 53 and AMONDYS 45 in December 2019 and February 2021 respectively are nonrecurring events and excluded from the Companys nonGAAP results
But even then concern about potential side effects emerged. Now the FDA is asking Idorsia to outline a risk mitigation strategy for aprocitentan
If an IRR occurs consider slowing or interrupting the infusion and instituting medical management as clinically indicated
AMT130 the companys lead therapy is designed to silence the malfunctioning gene responsible for the huntingtin protein which in its mutated form builds up and damages nerve cells
In a securities filing ahead of its public offering Cargo wrote that its cash on hand and proceeds from two tranches of stock issued in March and October will not be sufficient to fund our operations for at least one year from the issuance date of our financial statements However the proceeds from the IPO should stretch its financial runway into 2025 the company said in the document
GSK also mounted somewhat of a return to neuroscience in 2021
Unlike other startups that seek to bring entirely new technologies to the market Aiolos Bio is going after a known drug target for a disease thats well understood
Shares in BioMarin traded down by nearly 10 at one point Thursday morning before gaining back some of those losses
The Agency first informed the Company of this recommendation in the CRL having not raised this at any time during development or review
Despite the setbacks Chaplin said Bavarian is still on track to meet its goal  optimism thats supported by the travel vaccine portfolio it acquired from Emergent BioSolutions earlier this year
The new cuts affect approximately 25 of staff primarily those working on early stage programs and at a gene therapy facility in New Jersey. The layoffs follow a decision by European regulators to deny PTCs request to convert Translarnas conditional clearance there to a full approval for types of Duchenne muscular dystrophy
Over the last year or so the agency has cleared for market two more ALS drugs. One Relyvrio was developed by Massachusetts based Amylyx Pharmaceuticals. It was approved for the broader ALS population  based on data from a study of about 140 patients that found it offered modest but significant benefits on function and survival
BUSINESS WIREMay 12 2023 Sarepta Therapeutics Inc NASDAQ SRPT the leader in precision genetic medicine for rare diseases today announced that the US Food and Drug Administration FDA Cellular Tissue and Gene Therapies Advisory Committee CTGTAC voted 8 to 6 in support of accelerated approval of SRP9001 delandistrogene moxeparvovec for the treatment of ambulatory patients with Duchenne muscular dystrophy with a confirmed mutation in the DMD gene
As it seeks the same success it had with Hemgenix the company expects to start clinical trials for several other gene therapies over the next year including two next quarter
Ascendis Ascendis Pharma the Ascendis Pharma logo the company logo and TransCon are trademarks owned by the Ascendis Pharma Group
It matches the profile of biotechs that have successfully pulled off IPOs this year when investors looking for surer bets have gravitated toward companies with clinical data
Supplementation at the recommended daily allowance RDA of vitamin A is advised for patients taking ONPATTRO
Eisai which led the development of Leqembi said this week that around 800 patients in the US were on the drug by the end of October and the goal is to grow that total to 10000 by March
Kinnate shares have since fallen and this year they dropped significantly further after the publication of data for exarafenib used in combination with the MEK inhibitor binimetinib
Hardy nodded to the issues he faces in BioMarins statement announcing his hiring identifying the opportunity that lies ahead in unlocking the full value of Voxzogo and Roctavian Genentech tapped Hardy as CEO in 2019 and before that he held a number of roles there and at Roche Genetechs parent company including leading patient access services and serving as head of global product strategy
Small interfering RNA siRNA the molecules that mediate RNAi and comprise Alnylams RNAi therapeutic platform function upstream of todays medicines by silencing messenger RNA mRNA  the genetic precursors  that encode for diseasecausing proteins thus preventing them from being made
The disorder is characterized by intellectual disabilities loss of communication seizures slowing andor regression of development motor and respiratory impairment and shortened life expectancy
Consider also measuring glomerular filtration rate using an exogenous filtration marker before starting VYONDYS 53
Aiolos is also studying the monoclonal antibodys benefits in other conditions including chronic obstructive pulmonary disorder and chronic spontaneous urticaria
Idorsia said Wednesday that Johnson  Johnson plans to return most of the rights to its most closely watched experimental drug aprocitentan for patients with resistant hypertension
Elsewhere Vertex Pharmaceuticals last year agreed to pay 320 million for ViaCyte  a privately held developer of stem cellderived Therapeutics in 2019
NonGAAP research and development expenses were 1890 million and 1604 million for the three months ended June 30 2021 and 2020 respectively an increase of 286 million
More information can be found on wwwclinicaltrialsgov
BrainStorm Cell Therapeutics plans to withdraw an approval application for its experimental treatment for ALS a decision that follows significant pushback from the Food and Drug Administration
New Alzheimers drugs Eisai says new data could support more convenient form of Alzheimerʼs drug Leqembi. An under-the-skin injection of Leqembi worked about the same as the already marketed intravenous form. Eisai plans to ﬁle for approval by the end of March
Nippon Kayaku a licensing partner and Japan-based pharmaceutical manufacturer also agreed to acquire 40 million in additional shares in a concurrent private financing deal according to a regulatory filing. The offering will fund the development of a targeted cancer drug once owned by Novartis
Per terms of the deal announced by Nurix Thursday Seagen could pay as much as 34 billion more if certain research development regulatory and commercial milestones are met. Nurix retains an option to comarket and share profits on two products that emerge from the partnership
Data supporting the companys lead program currently in Phase 3 testing have been mixed. The company has changed leaders multiple times with Gould stepping down in 2021  returning as CEO in early 2023 and then giving way to new head Alex Sapir in July. And its sickle cell program has been held up since February sending shares to record lows
Diabetes and weight loss treatments like Novo Nordisks Ozempic and Eli Lillys Mounjaro are bringing in billions of dollars in sales spurring a wave of wouldbe competitors. Meanwhile new kinds of autoimmune disease drugs are being looked to as successors to current blockbusters and complex cancer treatments like radiopharmaceuticals and antibodydrug conjugates are newly in vogue
We are thrilled that APOLLOB successfully met all its major objectives which we believe for the first time validates the hypothesis that TTR silencing by an RNAi therapeutic can be an eective approach for treating the cardiomyopathy of ATTR amyloidosis said Pushkal Garg MD Chief Medical Oicer of Alnylam
Depreciation expense can vary substantially from period to period as the purchases of property and equipment may vary significantly from period to period and without any direct correlation to the Companys operating performance
About VYONDYS 53 VYONDYS 53 uses Sareptas proprietary phosphorodiamidate morpholino oligomer PMO chemistry and exonskipping technology to bind to exon 53 of dystrophin premRNA resulting in exclusion or skipping of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 53 skipping
The Food and Drug Administrations decision last month not to approve a new treatment for one of the most common forms of depression was in part due to safety concerns according to newly released documents detailing the agencys review process
Precede Biosciences has emerged from stealth with blood test technology designed to improve profiling of cancers and expand the use of liquid biopsies into other indications
That technology an implantable device dubbed Medici is a matchsticksized pump that can hold enough medication to treat a patient for up to one year
The biotech said ART5803 appears to reverse brain swelling and decrease the behavioral symptoms caused by the harmful antibodies
Idorsia also got to share revenue from a JJ drug for multiple sclerosis now sold as Ponvory. That agreement will also end with the return of rights to aprocitentan
About Hemophilia A Hemophilia A also called FVIII deficiency or classic hemophilia is an Xlinked genetic disorder caused by missing or defective FVIII a clotting protein
It takes into consideration quantitative and qualitative characteristics of each item including a nature b whether the items relate to the Companys ongoing business operations and c whether the Company expects the items to continue on a regular basis
At that time many of Romes biotech peers were using Series B funding to make the leap to the public markets
Novartis won drug approval of a drug it acquired with the buyout of the biotech Endocyte
Other therapies in Neumoras pipeline include treatments for schizophrenia and amyotrophic lateral sclerosis or ALS
ALS drug development FDA staff hold ʻmajor concernsʼ with Brainstorm ALS therapy documents show A panel of expert advisers will meet Wednesday to review the stem cell treatment which has become the latest test case in a debate over regulatory ﬂexibility
Sage shares currently trade at about 20 apiece roughly half of their value at the start of the year
2Seventy CEO to step down as company cuts staff spending Citing nearterm headwinds the cancer cell therapy developer will lay oﬀ 176 employees and pare back some of its research spending
OrbiMed previously funded another company developing a treatment for infants whose bones and teeth did not mineralize properly which would later become Alexion Pharmaceuticals  enzyme replacement therapy Strensiq
Investors included one of the most prominent players in the diabetes market Eli Lilly along with Frazier Life Sciences Polaris Partners and the JDRF T1D Fund
BrainStorm said Wednesday the FDA invited the New York-based biotechnology company to request an expedited facetoface meeting to discuss potential next steps for its treatment known as NurOwn
Given Cargos progress to date and its experienced leadership team Cargo is wellpositioned to be firsttomarket with an autologous CD22 CAR Tcell therapy Reid Huber a partner at Third Rock Ventures said at the time. That projection hinges on the biotechs ability to fund its clinical trial plans
JJ sends blood pressure drug back to Idorsia. The decision is the latest in a series of setbacks for the Actelion spinout and ends a lucrative partnership that began in 2017
That includes VERVE101 an experimental medicine that uses base editing  a more precise form of CRISPR gene editing  to inactivate the PCSK9 gene and thereby lower LDL cholesterol
Companies like OrsoBio have stepped in with plans for complementary therapies for instance. OrsoBios lead program dubbed TLC3595 is already in a Phase 2 study for diabetes
The announcement came as part of Ultragenyxs investor day program  though the company didn't flag the spinout in its press release
In its statement Wednesday the Swiss biotech said its initiating activities to determine the best approach to maximize the value of aprocitentan
Gilead is paying $66 million in upfront cash and an equity investment in Tentarix and has agreed to pay an additional 80 million for each acquisition option it exercises
In addition children under the age of 2 years treated with VOXZOGO for three years had a mean height Zscore improvement of 08 to 10 standard deviations 95 CI limits from 037 to 159 and a height gain between 35 and 39 cm 95 CI limits from 157 to 616 cm
Patients also experienced adverse reactions from corticosteroid use
Headquartered in La Jolla California Rampart emerged from stealth Tuesday with 85 million in new funding to develop its lead program for a rare inherited condition known as hypophosphatasia
However the FDA could take up to three more months to review additional materials on how best to ensure the drug is used safely Idorsia said
In addition to ANRE Arialys wants to test whether its medicine can treat more common illnesses like schizophrenia and dementia tied to Alzheimers disease
The New Jersey-based company's restructuring expands an initial reprioritization in May that focused the companys resources on higher potential programs
TSHA102 is being evaluated in the REVEAL Phase 12 trial  a firstinhuman openlabel randomized doseescalation and doseexpansion study evaluating the safety and preliminary efficacy of TSHA102 in adult females with Rett syndrome due to MECP2 lossoffunction mutation
The Food and Drug Administration has denied Alnylam Pharmaceuticals an expanded approval for its RNA drug patisiran rejecting it for use in people with an uncommon genetic heart condition
This week it won Food and Drug Administration clearance for an updated version  and US health officials are recommending that similar one from Pfizer
Cargo is now one of seven cancer-focused drugmakers  and the 19th biotech overall  to price a new stock offering in 2023
Company Contact Hayleigh Collins Director Head of Corporate Communications Taysha Gene Therapies Inc hcollinstayshagtxcom Media Contact Carolyn Hawley Canale Communications carolynhawleycanalecommcom Source Taysha Gene Therapies Inc
Histogen was also working in infectious disease  a field of research thats proven difficult to succeed in for many drug developers
Radiopharm aceutical drugm aker RayzeBio signals plans to go public. The San Diego-based biotech is developing a cancer drug for the same kind of tumors as targeted by Novartisʼ approved radiopharmaceutical Lutathera
Estimates hold that sickle cell affects more than 100000 people in the US and millions more in the rest of the world with the disease most commonly found in those with African ancestry
Upfront payments are made at the commencement of a collaborative relationship or a license agreement anticipated to continue for a multiyear period and provide the Company with intellectual property rights option rights and other rights with respect to particular programs
Use of the terms nonGAAP research and development expenses nonGAAP selling general and administrative expenses nonGAAP other income and lossadjustments nonGAAP income tax benefit expense nonGAAP net loss and nonGAAP basic and diluted net loss per share may differ from similar measures reported by other companies which may limit comparability and are not based on any comprehensive set of accounting rules or principles
The decision puts Bluebird on a different regulatory path than rival Vertex Pharmaceuticals which developed a competing candidate called exacel with partner CRISPR Therapeutics
Information on BioMarins website is not incorporated by reference into this press release
This EAP which remains Page 2 of 3 open for enrollment allows US physicians to request access to investigational TransCon PTH for their eligible patients
Apellis estimates the restructuring will save up to 300 million through 2024 70 million of which is associated with the layoffs and the remainder due to elimination of planned expenses
The company believes its choice of target a gene called TRPV4 could also play a rose in serious bone diseases like metatropic dysplasia
According to Evercore ISI analyst Jonathan Miller that research is the primary focus of investors who dont put much value in the companys COVID program. But the BARDA contract with Gritstone makes the program basically free for investors he wrote in a note to clients adding that it will also extend the companys cash runway by about one quarter
About 100000 people in the US have sickle cell disease a progressive and debilitating disease that significantly shortens the lifespan of its victims. There are currently no therapies that address the underlying genetic cause of the condition
Patisiran is the established name for ONPATTRO which is approved in the United States and Canada for the treatment of the polyneuropathy of hATTR amyloidosis in adults
The company which was staked with about 6 million from Syncona in April 2022 has so far conducted testing of its approach in mice
While the Phase 2 trial of VTX958 in plaque psoriasis met the primary and key secondary endpoints we are disappointed by the magnitude of efficacy observed despite having achieved target levels of drug exposure in the trial said Ventyx CEO Raju Mohan in a statement
We now have the largest travel commercial portfolio in the globe and that has led us to become a fully integrated profitable company he said of the Emergent deal adding that a resurgence in travel demand postpandemic bodes well for bringing in new travel vaccines like chikungunya Here Chaplin explains the impact of Bavarians Phase 3 failures the promise of its chikungunya vaccine candidate and the companys strategy to become a major vaccine player
Bioluminescence Ventures a San Franciscobased life sciences venture capital firm made its official debut on Wednesday with a 350 million first fund aimed at faster development of experimental drugs In total the firm has 477 million in assets under management including a smaller second fund
Apellis had a net loss of 122 million on drug sales of 95 million in the second quarter
While the agency isn't required to follow its advisers advice it typically does making the negative FDA decision unusual
We look forward to sharing full results at an upcoming conference in September and based on these positive results we plan to submit a supplemental NDA for patisiran with the US Food and Drug Administration in late 2022 APOLLOB is a Phase 3 randomized doubleblind placebocontrolled multicenter global study designed and powered to evaluate the eects of patisiran on functional capacity and quality of life in patients with ATTR amyloidosis with cardiomyopathy
Its hard to not also wonder whether the sluggish launch of Roctavian and the broader ups and downs for BioMarin over recent years made it logical to move on to a successor sooner versus later Still BioMarin said sales are ramping up better than expected for its Voxzogo therapy for a condition called achondroplasia that affects bone growth
Its caused by mutations that make red blood cells crescentshaped and less flexible which can in turn block the flow of blood through the body
The pharma has reshuffled its organization laid off staff and moved ahead with plans to separate out its Sandoz division in the year since then
Founded in 1989 the investment firm had made a name for itself as one of the biggest investors in biotechnology medtech and healthcare companies. Since its founding the firm has expanded its reach globally with offices in China Israel London and Hong Kong
The facility can serve as a critical link that enables ground breaking stem cell discoveries to be taken all the way to proofof concept in human chronic disease trials said Mads Krogsgaard Thomsen the foundations CEO who was formerly the drugmakers science chief in a statement
23andMe also sells the first FDA authorized developing side effects from specific drugs Wojcicki hopes all the data theyve collected will come full circle to help the very people who contributed their information to the drug discovery process
NASH or nonalcoholic steatohepatitis is a disease connected with obesity and its believed to be growing widely in prevalence
The centers initial focus is diseases of the brain such as ALS Alzheimers and Parkinsons
Amyloidtargeting drugs like Leqembi are also known to cause ARIA a class of side effects characterized by swelling and micro bleeding in the brain
By contrast Vertex and its partner are vying to market the first therapy based on CRISPR gene editing technology
Patisiran has not been approved by any regulatory agency for the treatment of ATTR amyloidosis with cardiomyopathy
Apellis Pharmaceuticals facing safety questions about its potential blockbuster eye drug Syfovre indicated Tuesday that a needle used to prepare injections may be to blame
AMONDYS 45 is approved under accelerated review based on an increase in dystrophin production in skeletal muscle of patients amenable to exon 45 skipping
Revenues For the three months ended June 30 2021 the Company recorded net product revenues of 1418 million compared to net product revenues of 1113 million for the same period of 2020 an increase of 305 million
Its aimed at a target called hematopoietic progenitor kinase 1 or HPK1 immune system and suppresses the bodys response to cancer Keiper said
A Chinese biotechnology startup promising breakthrough medicine with the use of a hot new gene modulation technology has raised 32 million in a Series A financing
The company will stop work on a very early-stage program targeting hepatitis B and seek a partner for that as well as its ex vivo CART cell therapies
The contract does give some validation for Gritstone company's approach and is certainly a nice signal of continued government support for COVID research Miller continued. Shares in the company rose by onefourth in Thursday morning trading
But that chapter closed quite some time ago and weve written a completely new chapter for Laronde that builds on a lot of scientific foundation that was established earlier Its merger with Senda which launched three years ago and raised 266 million in private financing before the merger creates a biotech that can draw on two Flagship platforms
The initial round of layoffs was expected to impact about 8 of its workers and came after the study failure of an experimental drug called vatiquinone in Friedreich ataxia an inherited disorder affecting the nerves
Its discovery has been heralded as a major scientific breakthrough that happens once every decade or so and was recognized with the award of the 2006 Nobel Prize for Physiology or Medicine
For the entire population while we saw separation at every time point between the active and placebo cohorts Study 102 did not achieve statistical significance on the primary functional endpoint
Transthyretin-mediated ATTR amyloidosis is a rare rapidly progressive debilitating disease caused by misfolded transthyretin TTR proteins which accumulate as amyloid fibrils in multiple tissues including the nerves heart and gastrointestinal GI tract
Now however Eisais Biogenpartnered drug Leqembi is approved in the US and Lillys donanemab is under Food and Drug Administration review. Roche hasnt fully ended its antiamyloid drug work yet continuing tests of one called crenezumab in people who have an inherited form of Alzheimers
The CEO called the finding too important not to pursue, but the work and funding needed to develop an Alzheimers drug is well outside of Ultragenyxs rare disease focus. So Ultragenyx is forming a new company called Amlogenyx to capitalize on the highrisk high return discovery. The spinout will be cash neutral for Ultragenyx the CEO said
We have our immunooncology programs in house and we have a dedicated discovery team focused on inflammation and immunology and looking at how we can go through datasets pull out interesting and novel insights and translate those into meaningful discoveries and cures potentially for our customers
The company was incubated at GV  which joined in the Series B extension financing and where Kapeller is a fellow
One other Phase 3 study is ongoing for pamrevlumab in pancreatic cancer. Initial results are expected by the end of the year according to a federal database