Monksdream
11 months ago
PHARMA
Vertex Made a Fortune Curing Rare Diseases. Its Next Bet Is Developing a Non-Addictive Opioid Alternative.
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There’s more to pharma than just COVID vaccines and weight-loss medications. Thirty-five years ago, Vertex Pharmaceuticals (NASDAQ:VRTX) took a fresh approach to treating illnesses by targeting human cells and genes. Except it spent 20 years with bleak growth prospects before scoring its first blockbuster drug approval in 2012.
Since then, Vertex has generated over $20B in profits attacking cystic fibrosis at the source. And last year, it became the first company to bring a gene-editing therapy to market, a generational moment for the biopharma industry that pushed Vertex into the top 10 US pharma companies.
We feel heal your pain: Yesterday, Vertex announced positive clinical trial results from its experimental non-addictive alternative to opioid painkillers, VX-548, which blocks pain signals from reaching the brain. This opens the door for the “first new acute pain medicine in 25 years,” Vertex Chief Scientific Officer Dr. David Altshuler tells Barron’s.
While VX-548 might not match the effectiveness of Vicodin (opioid med), analysts predict it could replace addictive opioids for cases of “moderate-to-severe pain” once approved in early 2025.
The drug could cater to over 80M patients experiencing acute pain, a vast market that analysts at Leerink Partners forecast will help VX-548 generate $5.1B in sales by 2030.
Vertex’s next act
Altogether, Vertex has over a dozen treatments in research or studies — a strategy deemed “more important than ever” by RBC Capital Markets analyst Brian Abrahams, as it will drive the next leg of the company’s growth.
Vertex plans to expand its pain and blood disorder franchises but boasts a pipeline of gene-editing treatments with CRISPR Therapeutics (NASDAQ:CRSP), which could eventually treat Muscular Dystrophy and Type 1 Diabetes.
Focusing on rare diseases has also allowed Vertex to skip some costly drug filing fees and secure fast-track approval — all while charging hefty premiums for its orphan drugs.
Forward-looking: Unlike many other pharma giants facing patent expirations, Vertex doesn’t have any of its major drugs reaching a patent cliff anytime soon, leaving the company ample time to commercialize its new cures.
Monksdream
1 year ago
From Cathie Wood
Gene-Editing Therapies Could Rearrange Healthcare Expenditures In The US
ARK Invest_Illustration_Ali Urman_Final_Circle 400 px By Ali Urman | @aurmanARK
Analyst
Last week, Vertex Pharmaceuticals presented3 promising results from a Phase I/II trial focused on Type 1 Diabetes (T1D): VX-880, an allogeneic stem-cell derived islet cell therapy infused into the hepatic portal vein for T1D patients with severe hypoglycemia. Importantly, the six patients produced endogenous insulin, reducing the need for exogenous insulin and showing blood glucose control with a reduction of HbA1c.
In the US today, healthcare spending approximates $4.3 trillion,4 of which therapeutics accounts for ~15%, or $600 billion,5 as shown below.
In our view, novel gene-editing therapies could command premium pricing by shifting healthcare expenditures from chronic disease management to one-dose therapies like VX-880. Addressing diabetes, for example, therapeutics account for 30% of total direct and indirect costs, as shown below. If emerging drugs like VX-880 were to shift costs from chronic disease management to one-dose therapies, curative therapeutics could take share from chronic treatments.
Kristallweizen
3 years ago
Vertex surprised by FDA halt of diabetes cell therapy study
https://www.biopharmadive.com/news/vertex-diabetes-clinical-hold-cell-therapy/623010/
Published May 2, 2022
Dive Brief:
The Food and Drug Administration has stopped Vertex from continuing a clinical trial testing a promising cell therapy for Type 1 diabetes, judging the company has “insufficient information” to study a higher dose of the treatment as planned.
The clinical hold, which Vertex announced in a statement Monday, comes after startlingly positive results from the first patient given the therapy, known as VX-880. Nine months after the treatment, that patient no longer needed to take synthetic insulin, while blood and laboratory testing indicated his body was producing its own.
Vertex treated the first and second patients in its trial with a half dose and, following the recommendation of independent trial monitors, treated a third with a full dose in the second phase of the study, which is meant to enroll four other participants. That progression seems to be what the FDA is questioning with its hold, which Vertex said it would work to address.
Dive Insight:
From Vertex’s perspective, the results it’s obtained from the first two patients in its study represent “proof of concept” for a treatment that’s designed to replace the insulin-producing cells destroyed by Type 1 diabetes with ones made from stem cells.
Initial results from the first patient, released last October, helped to validate the company’s approach, which was developed over decades by the laboratory of Doug Melton, a Harvard University professor who recently joined Vertex as a distinguished fellow. (Melton founded a biotech company called Semma Therapeutics, which Vertex bought in 2019 for nearly $1 billion.)
That patient no longer needs to inject himself with synthetic insulin, and further follow-up data indicates his body is now able to control blood sugar levels. According to Vertex, a second patient has been able to reduce insulin use by about a third and shows signs that treatment is restoring insulin production as well.
A third patient, who was given a full dose of VX-880, is only 29 days out from treatment and will be more comprehensively examined after three months.
No serious adverse reactions have been reported, while the most commonly reported side effects were judged unrelated to treatment.
However, the FDA, at least according to Vertex, doesn’t agree the company has enough information to proceed with “dose escalation,” an industry term for evaluating progressively higher doses of a treatment.
“We are surprised by the clinical hold placed on the study,” said Carmen Bozic, Vertex’s chief medical officer, in a statement.
The company said it will work with the agency to understand and address its concerns “so that the trial can resume at U.S. sites as soon as possible.” Nine of the 10 study sites are located in the U.S., while the 10th is in Montreal, according to a federal database of clinical trials.
Overall, Vertex intends to enroll 17 patients in the early-stage trial, which is divided into three phases.
VX-880 consists of pancreatic islet cells that are derived from stem cells and differentiated into their mature form. It’s administered by an infusion into a vein that conveys blood to the liver. Patients treated with VX-880 must take immunosuppressants so their immune system doesn’t destroy the islet cells.
Vertex is also working on another version of VX-880 “encapsulated” in an immunoprotective device that’s surgically implanted. The company aims to ask the FDA later this year for clearance to begin human testing.
The hold is an unexpected roadblock, however, and it’s unclear what impact the delay might have on Vertex’s plans. According to Michael Yee, an analyst at Jefferies, recent clinical holds for cell therapies have been lifted quickly, generally within four to five months.
“We’re hopeful for a speedy resolution and [management] also indicated they are being very transparent [with the] FDA,” Yee wrote in a May 2 note to clients.
protagonist12
9 years ago
New $2.6B deal between Boston-area biotechs a first for gene editing
Cambridge startup CRISPR Therapeutics is getting $105 million up front, and as much as $2.6 billion in coming years, in a new partnership with Vertex Pharmaceuticals that CRISPR CEO Rodger Novak called “the first really big deal” in the burgeoning field of gene editing.
CRISPR, a privately-held company — which, coincidentally, just moved into a building formerly occupied by Vertex(Nasdaq: VRTX) over the summer — said the deal could help develop cures for diseases including sickle cell and cystic fibrosis. While all research in gene editing — treatments designed to fix genes that cause disease — is still very early stage, Nozak said in an interview today it’s “very likely” that a treatment for sickle cell could begin human trials sometime before the end of the four-year research agreement.
If successful, the collaboration could result in a method to correct the underlying gene mutations that cause cystic fibrosis. While the approach is yet unproven, the hope is that a one-time treatment with gene editing would render the drugs now marketed by Vertex for that disease — including Kalydeco and Orkambi — obsolete.
“The main interest of both Vertex and ours is to help patients with cystic fibrosis. Their therapies are helping patients tremendously now ... but there is room for improvement,” said Novak.
Currently, almost all the treatments in development at Vertex are small-molecule, or pill-based, drugs, so the CRISPR deal represents a new type of research for the 30-year-old company. Vertex will have the option to exclusively license up to six potential treatments (including for cystic fibrosis), and for most of them it would pay for all development and commercialization costs — up to $420 million each, plus sales royalties. The only exception would be in sickle cell, where CRISPR will pay half the R&D costs and would lead any commercialization efforts.
The deal appears to be exactly the kind touted as a benefit of Cambridge’s life sciences cluster, where two local companies decide to join forces on a project that would advance human health. Novak said that the physical proximity of the two companies (Vertex is in South Boston; CRISPR is in East Cambridge) played a part, but was also helped by an existing relationship between the top scientists at both companies, Vertex’s David Altshuler and CRISPR’s Bill Lundberg.
The focus on sickle cell makes CRISPR a potential competitor with another big local biotech, bluebird bio (Nasdaq: BLUE). Cambridge-based bluebird has a promising gene therapy treatment (which seeks to replace, rather than edit, a faulty gene) in early-stage clinical development to address the disease. Novak said in this disease, gene editing has the potential to be as effective as gene therapy, but with fewer side effects, though he added that the technology still needs to prove it works.
CRISPR is one of three local biotech firms now pursuing gene editing using a technique known as CRISPR/Cas9, which allows for multiple cuts, or “edits,” to a single gene. Cambridge-based Editas Medicine made a deal in May potentially worth more than $700 million with Seattle-based Juno Therapeutics (Nasdaq: JUNO), which last year held the sector’s biggest initial public offering of of $265 million. Cambridge-based Intellia Therapeutics, which has yet to announce a major deal with a more established company, just got a $70 million investment last month.
http://www.bizjournals.com/boston/blog/bioflash/2015/10/new-2-6b-deal-between-boston-area-biotechs-a.html
KingDMC
10 years ago
Food & Drug Administration Advisory Panel Voted 12 to 1 to Recommend Approval of ORKAMBI™ (lumacaftor/ivacaftor) to Treat P...
Source: Business Wire
-FDA decision expected by July 5, 2015 PDUFA date-
-Approximately 8,500 people with cystic fibrosis in the U.S. have two copies of the F508del mutation and are ages 12 and older-
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration’s (FDA) Pulmonary-Allergy Drugs Advisory Committee (PADAC) voted 12 to 1 to recommend that the FDA approve ORKAMBITM (lumacaftor/ivacaftor) for use in people with cystic fibrosis (CF) ages 12 and older who have two copies of the F508del mutation in the CFTR gene. Advisory committees provide the FDA with independent scientific and medical advice on safety, effectiveness and appropriate use of potential new medicines. The FDA is expected to make a decision on the approval of ORKAMBI by July 5, 2015 under the Prescription Drug User Fee Act (PDUFA). The FDA is not bound by the committee's recommendation but often follows its advice. If approved, ORKAMBI will be the first and only medicine to treat the underlying cause of CF for eligible people with CF ages 12 and older with two copies of the F508del mutation in the CFTR gene. People with two copies of the F508del mutation represent the largest group of people with CF. There are approximately 8,500 people ages 12 and older with two copies of the F508del mutation in the U.S.
“Today’s positive recommendation brings the cystic fibrosis community one step closer to potential approval of the first medicine to treat the underlying cause of this disease for many more people,” said Jeffrey Chodakewitz, M.D., Executive Vice President and Chief Medical Officer at Vertex. “We look forward to continuing to work with the FDA and other regulatory agencies throughout the world to make ORKAMBI available to eligible patients as soon as possible.”
Cystic fibrosis is a rare genetic disease that is caused by defective or missing CFTR proteins resulting from mutations in the CFTR gene. The defective or missing proteins result in poor flow of salt and water into and out of the cell in a number of organs, including the lungs. In people with two copies of the F508del mutation, the CFTR protein is not processed and trafficked normally within the cell, resulting in little to no CFTR protein at the cell surface.
ORKAMBI is a combination of lumacaftor, which is designed to increase the amount of functional protein at the cell surface by addressing the processing and trafficking defect of the protein, and ivacaftor, which is designed to enhance the function of the CFTR protein once it reaches the cell surface. ORKAMBI is an oral medicine that, if approved, would be taken as fully co-formulated tablets that contain both lumacaftor and ivacaftor.
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