Several research groups and biotech startups are developing new ideas for treating Parkinson’s that capitalize on the gut-brain connection. (CC0 Creative Commons)
Over the past few years, several research groups have drawn links between the gut microbiome and central nervous system disorders, especially Parkinson’s disease. Now, scientists at the universities of Edinburgh and Dundee are going a step further and investigating a treatment strategy that capitalizes on microbes in the gut that may protect against the neurodegenerative disease.
The researchers started with roundworms engineered to produce the human version of alpha-synuclein protein, which is a major culprit in Parkinson’s. In people, alpha-synuclein misfolds and forms toxic clumps in the brain, leading to the death of nerve cells that produce dopamine. That, in turn, causes the tremors, movement difficulties and other symptoms typical of the disease.
The team fed the worms various over-the-counter probiotics and discovered that one in particular—Bacillus subtilis—cleared some alpha-synuclein clumps and improved motor symptoms. They published the findings in the journal Cell Reports.
This doesn’t mean everyone should go to the drugstore to buy probiotics, the researchers warn—the finding needs to be confirmed in mice and then in large-scale clinical trials. But they believe it does bolster previous discoveries linking gut microbes to Parkinson’s.
“The results provide an opportunity to investigate how changing the bacteria that make up our gut microbiome affects Parkinson’s,” said lead author Maria Doitsidou, Ph.D., of the Centre for Discovery Brain Sciences at the University of Edinburgh, in a statement.
Doitsidou and colleagues discovered that B. subtilis works by changing how enzymes process specific fats known as sphingolipids. The bacterium produces chemicals that spark the change, which in turn prevents alpha-synuclein protein from forming clumps.
Several research groups have turned to the gut microbiome in search of new ideas for treating Parkinson’s. Last June, Johns Hopkins University School of Medicine researchers published research showing that in mice, misfolded alpha-synuclein protein can travel from the gut to the brain. And in 2018, a team led by Michigan-based Van Andel Research Institute studied two large patient registries and found that people who have their appendixes removed lower their chances of getting Parkinson’s by up to 25%—a phenomenon they suspected was related to their discovery of alpha-synuclein clumps in appendix tissue.
There has also been entrepreneurship in biopharma centered around exploiting the gut-brain connection in Parkinson’s. In 2016, California Institute of Technology scientists showed in mouse models that some gut bacteria promote Parkinson’s progression, leading them to launch Axial Biotherapeutics to pursue microbiome-aimed treatments for the disease and other central nervous system disorders. Last February, the company raised $25 million in a series B round that was led by Seventure Partners’ microbiome-focused Health for Life Capital fund. Enterin has also snapped up capital to advance its lead drug targeting alpha-synuclein.
The new research centered around the probiotic B. subtilis was partially funded by Parkinson’s UK, which hopes that clinical trials stemming from the research will be fast-tracked. “Studies that identify bacteria that are beneficial in Parkinson’s have the potential to not only improve symptoms but could even protect people from developing the condition in the first place,” said the organization’s research manager, Beckie Port, Ph.D., in the statement.
The pact was penned back in late 2018 but has now been axed (Eli Lilly/LinkedIn).
Eli Lilly has killed off its deal with immuno-oncology biotech NextCure after spending $25 million upfront on the pact just over a year ago, with no details as to why.
That deal, which also included undisclosed milestones, was built upon a platform for scouring cell surface interactions in search of targets that affect immune function with NextCure, which is staffed by executives who led Amplimmune to a takeover by AstraZeneca.
I-O laggard Lilly was hoping this, with other recent buys, would help shore up its cancer pipeline, and saw the Big Pharma pay $25 million upfront and take out a $15 million stake in NextCure for the chance to access the fruits of the platform.
In return, Lilly bagged an option to exclusively license antibodies against targets discovered using NextCure’s platform and NextCure had the option to license antibodies, too, and could have got milestones and royalties of undisclosed size if the drugs Lilly picks up progress.
Now, that’s been axed.
In an extremely brief SEC update (and no press release at time of writing), Maryland-based NextCure said: “On January 10, 2020, Eli Lilly and Company notified NextCure of Lilly’s termination, effective as of March 3, 2020, of the Research and Development Collaboration Agreement, dated as of November 2, 2018, between the Company and Lilly.”
No explanation was given; NextCure saw its shares down 6% shortly after the SEC filing was released Monday morning.
A new multiyear pact with Charles River will help Takeda gain new clinical candidates centered around its core pipeline focus. (Charles River)
SAN FRANCISCO—At the annual J.P. Morgan Healthcare Conference, major CRO Charles River Laboratories and Japanese Big Pharma Takeda announced a new preclinical tie-up.
The pair will “launch multiple integrated programs across Takeda’s four core therapeutic areas,” they said in a statement, namely: oncology, gastroenterology, neuroscience and rare disease, with Charles River being tasked with moving its preclinical candidates into the clinic and ultimately into the hands of patients.
This will be done by tapping Charles River’s drug discovery expertise and scientific bench, with the CRO using its end-to-end drug discovery and safety assessment platform to “explore potential therapeutic approaches and progress these programs towards candidate status.”
Takeda, which has been ramping up the deals with biotechs in recent years to help shore up its pipeline and progress with its core focus, will then have the option to push on with these early candidates through their clinical development pipeline.
Takeda will pay Charles River a one-time, upfront fee to establish the collab, though both stayed mum on what that dollar figure was. They did say Charles River will be in line to nab development payments with a potential value of over $50 million per program in preclinical and clinical milestones for those that will be put forward to a regulator.
The pact also includes additional potential commercial milestones of up to $120 million plus royalties on launched products, Takeda said.
James Foster, chairman, president and CEO at Charles River, said: “We are pleased to expand our relationship with Takeda, who shares our commitment to bring innovative, safe, and effective medicines to patients as quickly and efficiently as possible. We expect the expertise of Charles River and Takeda will prove to be a powerful combination in delivering novel drug candidates.”
Shares in Ultragenyx were up 25% Friday morning on news of positive data from its gene therapy trial. (science photo/Shutterstock)
Ultragenyx Pharmaceutical saw its shares jump around 25% in trading Friday after announcing positive top-line data out of its gene therapy trial.
It’s a small number, just three patients that form part of a third cohort for the phase 1/2 study, as well as another small test but a longer-term look from the second cohort.
In cohort three testing the biotech’s drug DTX301, an adeno-associated virus gene therapy for the treatment of ornithine transcarbamylase (OTC) deficiency, there were two confirmed female responders as well a third potential male responder who requires longer-term follow-up to confirm response status.
Meanwhile, in cohort two, one female patient saw a new response after a year. The biotech added that the two previously disclosed responders in cohort one and two “also remain clinically and metabolically stable at 104 and 78 weeks, respectively. Across all nine patients dosed in the study, up to six patients have demonstrated a response,” it said in a statement.
OTC deficiency is a rare X-linked genetic disorder characterized by complete or partial lack of the enzyme OTC. Excess ammonia, which is a neurotoxin, travels to the central nervous system through the blood,
According to the National Organization for Rare Disorders, the severity and age of onset of OTC deficiency varies from person to person, even within the same family. “A severe form of the disorder affects some infants, typically males, shortly after birth (neonatal period). A milder form of the disorder affects some children later in infancy. Both males and females may develop symptoms of OTC deficiency during childhood. Most carrier females are healthy, but may be prone to severe headaches following protein intake.”
Analysts at Jefferies said the data “looked better than expected” and could “be a positive spark to help turn the stock heading into 2020 events.” It certainly did in the immediate term, with the biotech’s shares up by 25% in mid-morning trading Friday.
“We are encouraged to see a more uniform response at the higher doses including three female responders. To date, three patients in the study have discontinued alternate pathway medication and liberalized their diets while remaining clinically and metabolically stable,” said Eric Crombez, M.D., chief medical officer of the Ultragenyx Gene Therapy development unit.
“We are moving to prophylactic steroid use in the next cohort as we believe this could further enhance the level and consistency of expression that we have demonstrated so far.”
New fundraising comes in the wake of Blackstone’s acquisition of Clarus. (Blackstone)
Blackstone has raised $3.4 billion for its life science fund. The financing, which could grow by another $1.2 billion, sets Blackstone up to continue placing bets such as its ventures with Novartis and Ferring.
U.S. private equity firm Blackstone disclosed the funding in a regulatory filing, details of which were reported by Reuters. The filing shows that Blackstone Life Sciences V has pulled in $3.4 billion out of a targeted $4.6 billion so far. Even at the current close, the fund has significant financial firepower, reflecting Blackstone’s interest in getting involved in big, late-phase projects.
Blackstone has revealed the type of biopharma bets it wants to place through its investments so far. Last year, the private equity group teamed up with Novartis to create Anthos Therapeutics, committing $250 million to fund work on a midphase anti-thrombotic. Later in the year, Blackstone put up $400 million to help Ferring-spinout FerGene challenge for the U.S. bladder cancer market.
The Novartis and Ferring deals came in the wake of Blackstone’s acquisition of Clarus, an investor that helped push late-phase programs to market. Clarus made bigger bets than many life science investors, for example by leading Galera Therapeutics’ $150 million series C, but still lacked the scale to take on the biggest opportunities that came its way.
Selling up to Blackstone has given Clarus that extra scale. At $400 million, Blackstone’s commitment to FerGene amounts to almost 45% of the size of the late-phase fund operated by Clarus. With $3.4 billion in the pot for new investments, Blackstone is positioned to place a series of bets of that size and larger, enabling it to help out biotechs that fly solo deep into the clinic and Big Pharma companies that have more assets than they can manage.
Nasdaq-listed biotech Solid saw it shares hit again this morning on news of layoffs. (Nasdaq)
Amid clinical holds, manufacturing concerns, safety issues and downsized IPOs, Solid Biosciences is bloodletting from its staffers.
First, the history: Toward the end of last year, a new clinical hold following serious adverse events formed just part of a long line of setbacks for Solid Bio and the development of its gene therapy for Duchenne muscular dystrophy.
In 2018, the company resolved partial and full clinical holds for its phase 1/2 study of SGT-001, its single-dose, AAV-mediated microdystrophin therapy, after addressing safety and manufacturing concerns.
This looked like a positive, but then that trial was put on hold again in November after the sixth and latest patient saw decreases in red blood cell and platelet counts, activation of the complement immune system response, acute kidney injury and cardio-pulmonary insufficiency. This saw its shares take a nosedive.
In January 2018, Solid Bio revealed the trial’s first partial clinical hold, remarkably, at the same time as the company’s IPO and debut on the Nasdaq. It moved forward with an inevitable cut-price offering for an upsized stake, after receiving heavy criticism for not disclosing the notice earlier in its December prospectus or subsequent filings.
Things then got worse, as the partial hold was upgraded to a full clinical hold later that spring over manufacturing concerns related to higher doses. The company was able to resume enrollment by mid-June 2018 after amending its study protocol.
But the company’s stock took another hit this past February after SGT-001 demonstrated low levels of microdystrophin expression in an analysis of the trial’s lower dose.
In December of last year, the company reported biomarker data from two patients it said “provide evidence that SGT-001 is biologically active with differentiated properties,” which it believes “warrants further evaluation.”
So, to the present: In a statement this morning, and not really a surprise given the above, the biotech announced swingeing cuts: “Going forward, the company will focus on conducting its analyses of SGT-001 to determine how to address the clinical hold and resume dosing.
“The activities supporting the company’s other research and development programs will be curtailed as Solid establishes a path forward for SGT-001. As part of the organizational changes, the company will reduce its work force by approximately one third. In conjunction, Chief Operating Officer Alvaro Amorrortu, M.B.A. and Chief Medical Officer Jorge Quiroz, M.D., M.B.A. will depart the company but will continue as advisors to Solid.”
The biotech hopes these cuts will save cash and give it enough to keep going until next year.
“We believe SGT-001 holds great potential for the treatment of Duchenne, and in order to effectively evaluate its potential for patients, we made some difficult choices to focus our resources and help extend our cash runway,” said Ilan Ganot, CEO, president and co-founder of Solid Biosciences.
“We are grateful for the efforts and contributions of our employees who have worked tirelessly to advance our mission to improve the lives of patients with Duchenne. We are committed to supporting all employees during this transition.”
Shares in the company were down 17% premarket on the news this morning.
A Boehringer facility in Denmark (Boehringer Ingelheim)
Boehringer Ingelheim has struck another deal to expand its pipeline of nonalcoholic steatohepatitis (NASH) prospects. The latest deal sees Boehringer swallow Enleofen’s anti-IL-11 antibody platform, putting it on the hook for up to $1 billion in milestones per product.
Singapore-based Enleofen began operations in 2017, starting out with an exclusive license to patents and antibodies from the SingHealth Duke-NUS Academic Medical Centre (AMC). Enleofen built on those licensed resources, creating an anti-IL-11 antibody platform and hustling the most promising assets toward the clinic.
Now, Boehringer has stepped in to run the next stage of the program. The German drugmaker has bought exclusive rights to the IL-11 platform, setting it up to advance drugs against fibrotic disorders including NASH and interstitial lung diseases.
Boehringer will continue to work with AMC, the originator of the science, but will take responsibility for clinical, regulatory and commercial development of the licensed therapies. In return for rights to the IL-11 assets, Boehringer has put together a package that is worth up to $1 billion (€900 million) per product in upfront fees and milestones. The deal is likely to be heavily backloaded.
Research into the role of IL-11 has led Boehringer to foresee applications for the antibodies in a wide range of diseases. Studies suggest blocking the action of the IL-11 cytokine can prevent and reverse fibrosis and inflammation across multiple organs. Reflecting its pipeline priorities, Boehringer will initially explore the antibodies in NASH and interstitial lung diseases.
Boehringer’s focus on the role of IL-11 in NASH and interstitial lung diseases, an umbrella term that covers indications including idiopathic pulmonary fibrosis, is in keeping with its recent deal-making. Last year, Boehringer paid Yuhan $40 million and committed to up to $830 million in milestones for the rights to a dual GLP1R/FGF21R agonist with applications in NASH.
Further back, Boehringer has struck deals with companies including Dicerna Pharmaceuticals, MiNA Therapeutics and Pharmaxis to secure rights to potential NASH treatments. Not all the bets have paid off, though. Late last year, Boehringer dropped the drug it acquired from Pharmaxis amid concerns about drug interactions.
Some segments of genes shared between human, mouse and rat genomes could serve as new targets for developing treatments against cancer, scientists at Fred Hutchison have found. (Kim Carney/Fred Hutch News Service)
In 2004, scientists found 481 segments of DNA that are shared between human, rat and mouse genomes. These ultra-conserved bits must be essential if they could survive millions of years of evolution and remain unchanged, they suspected. But how?
Researchers at Fred Hutchison Cancer Research Center have used the CRISPR-Cas9 gene-editing tool to demonstrate that some of those DNA elements are key for cell growth and suppressing tumors. Therefore, targeting them may represent a new approach for developing anti-cancer treatments, they argue.
The research, published in the journal Nature Genetics, is the “first study finding large-scale importance of these highly conserved [DNA] elements,” senior author Rob Bradley said in a statement.
Bradley and colleagues focused their research on parts of DNA called poison exons. Cells can sometimes skip exons to make RNA during the protein-production step known as RNA splicing. But poison exons can act as a “kill switch” to put an end to protein production. It’s usually a beneficial process that helps avoid the production of unwanted proteins.
Previous studies have found wide overlaps between ultra-conserved DNA pieces and poison exons, but efforts to understand exactly what that signifies have been largely fruitless.
Rather than focusing on one specific ultra-conserved DNA element, first author James Thomas, a postdoc fellow in Bradly’s lab, designed a CRISPR-based tool that allowed the researchers to remove and examine hundreds of poison exons at the same time.
The researchers compared 465 highly conserved poison exons to 91 others that are not shared between species. They found that many poison exons are essential for the growth of cells cultured in lab dishes, as removing them led to cell death.
To validate the findings, the team tested mice with lung cancer xenografts. They observed that some poison exons helped promote cell growth, but a subset had anti-tumor effects. Many of the anti-tumor poison exons were within the genes regulating RNA splicing factors, which are produced at increased levels in cancer.
“These data suggest that many RNA splicing factors are proto-oncoproteins whose pro-tumorigenic effects are constrained by poison exons,” the researchers wrote in the study.
By examining poison exons in lung cancer patients, the scientists confirmed that their tumor-suppressing capacity was clinically relevant, as low inclusion of tumor-fighting poison exons was linked to significantly worse survival rates, they reported.
Drugs that target RNA mis-splicing are in use now to treat some diseases. Biogen’s antisense oligonucleotide Spinraza (nusinersen), for example, treats spinal muscular atrophy by correcting SMN2 exon 7 splicing to compensate for the loss of SMN1.
The Fred Hutch team figured the new findings could open up the possibility of targeting ultra-conserved poison exons with drug treatments. “Once you find a target, it’s easy to build upon the previous technology that exists for therapeutics,” Thomas said in the statement.
Verastem Oncology has signed a pact to license a drug targeting KRAS-mutation positive cancers from Chugai.
Under the deal, which is worth only $3 million upfront, Verastem nabs worldwide development and sales rights to RAF/MEK inhibitor CH5126766 (CKI27) from Chugai, which is in tests for KRAS mutant solid tumors. The company will host an investor call to discuss the opportunity and a development update today (details below).
The drug is already in a combo phase 1 trial with Verastem’s focal adhesion kinase inhibitor, defactinib, in KRAS-positive cancers, including ovarian, non-small cell lung and colorectal cancers.
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Verastem is also testing Copiktra in relapsed and refractory blood cancers and as a first-line treatment for younger patients with chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL).
Now, Verastem gets its hands on the med and will be responsible for the development and selling of CH5126766. Along with the $3 million upfront, it will also pay royalties to Chugai.
Verastem added that “given the potential of the opportunity, the company will be evaluating various partnering strategies,” but gave no further details.
“Based on the single-agent defactinib results in KRAS mutant NSCLC, we conducted an internal pre-clinical effort to identify drug classes that were synergistic with defactinib and saw the highest level of synergy in combination with MEK inhibitors and, specifically, with CH5126766,” said Dan Paterson, president and chief operating officer at Verastem.
“The exciting early clinical results led to our decision to enter into a partnership with Chugai for CH5126766 and accelerate the combination development program for patients with KRAS mutant cancers, which are highly aggressive and recurrent. We plan to initiate discussions with regulatory authorities about our development plans and to define the registration path early this year.”
This comes around six months after Verastem took on a new leader: Brian Stuglik, the Eli Lilly veteran who sat on Verastem’s board of directors. Stuglik’s appointment came a month after Robert Forrester stepped down from the CEO post. He soon took the chop to 40 positions from the company in an attempt to lower costs.
Stuglik’s predecessor, Forrester, joined Verastem as COO in 2011 and replaced Christoph Westphal as CEO in 2013. He steered the company through turbulent times marked by the foundering of its lead asset and the subsequent regrouping around the AbbVie and Infinity castoff duvelisib.
Now known as Copiktra and after a troubled path, duvelisib earned an FDA nod in 2018 as a third-line treatment for CLL, SLL and follicular lymphoma. But that approval came with a black box warning that could limit use of the drug. The company predicted Copiktra to log about $10 million in sales, but analysts expect revenues to rise beyond that. Peak sales for the drug have been forecast as high as $850 million.
Verastem is also testing Copiktra in relapsed and refractory blood cancers and as a first-line treatment for younger patients with CLL and SLL. Last year, Sanofi signed up to sell the drug in certain regions.
Verastem is now hoping to pull off a similar feat with its Chugai deal, although there is a long way to go and many hurdles to trip up on.
The biotech was up 12% premarket with a market cap of just under $100 million.
Boston biotech Verastem Oncology has seen cuts to staff and sluggish sales of its cancer drug but hopes a pipeline boost can help shore up its future. (Sean Pavone/Getty Images via Newscred)
Verastem Oncology has signed a pact to license a drug targeting KRAS-mutation positive cancers from Chugai.
Under the deal, which is worth only $3 million upfront, Verastem nabs worldwide development and sales rights to RAF/MEK inhibitor CH5126766 (CKI27) from Chugai, which is in tests for KRAS mutant solid tumors. The company will host an investor call to discuss the opportunity and a development update today (details below).
The drug is already in a combo phase 1 trial with Verastem’s focal adhesion kinase inhibitor, defactinib, in KRAS-positive cancers, including ovarian, non-small cell lung and colorectal cancers.
Verastem is also testing Copiktra in relapsed and refractory blood cancers and as a first-line treatment for younger patients with chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL).
Now, Verastem gets its hands on the med and will be responsible for the development and selling of CH5126766. Along with the $3 million upfront, it will also pay royalties to Chugai.
Verastem added that “given the potential of the opportunity, the company will be evaluating various partnering strategies,” but gave no further details.
“Based on the single-agent defactinib results in KRAS mutant NSCLC, we conducted an internal pre-clinical effort to identify drug classes that were synergistic with defactinib and saw the highest level of synergy in combination with MEK inhibitors and, specifically, with CH5126766,” said Dan Paterson, president and chief operating officer at Verastem.
“The exciting early clinical results led to our decision to enter into a partnership with Chugai for CH5126766 and accelerate the combination development program for patients with KRAS mutant cancers, which are highly aggressive and recurrent. We plan to initiate discussions with regulatory authorities about our development plans and to define the registration path early this year.”
This comes around six months after Verastem took on a new leader: Brian Stuglik, the Eli Lilly veteran who sat on Verastem’s board of directors. Stuglik’s appointment came a month after Robert Forrester stepped down from the CEO post. He soon took the chop to 40 positions from the company in an attempt to lower costs.
Stuglik’s predecessor, Forrester, joined Verastem as COO in 2011 and replaced Christoph Westphal as CEO in 2013. He steered the company through turbulent times marked by the foundering of its lead asset and the subsequent regrouping around the AbbVie and Infinity castoff duvelisib.
Now known as Copiktra and after a troubled path, duvelisib earned an FDA nod in 2018 as a third-line treatment for CLL, SLL and follicular lymphoma. But that approval came with a black box warning that could limit use of the drug. The company predicted Copiktra to log about $10 million in sales, but analysts expect revenues to rise beyond that. Peak sales for the drug have been forecast as high as $850 million.
Verastem is also testing Copiktra in relapsed and refractory blood cancers and as a first-line treatment for younger patients with CLL and SLL. Last year, Sanofi signed up to sell the drug in certain regions.
Verastem is now hoping to pull off a similar feat with its Chugai deal, although there is a long way to go and many hurdles to trip up on.
The biotech was up 12% premarket with a market cap of just under $100 million.
Apellis’ shares rose more than 50% before giving up some of the gains. (Getty Images)
Apellis Pharmaceuticals’ pegcetacoplan has beaten Alexion’s blockbuster Soliris in a head-to-head phase 3 trial. The result suggests Apellis can challenge Alexion’s dominance in paroxysmal nocturnal hemoglobinuria (PNH), but the biotech needs to talk to the FDA before deciding on the next steps.
For the phase 3, investigators enrolled 80 PNH patients who were anemic despite taking Soliris and randomized then to receive either Alexion’s blockbuster or Apellis’ rival complement inhibitor.
After 16 weeks, hemoglobin levels in the pegcetacoplan arm had increased by 3.8 g/dL over the Soliris group, resulting in the trial hitting its primary endpoint. The trial also hit some key secondary endpoints. Notably, 85% of patients on pegcetacoplan were transfusion-free at week 16, compared to 15% of their peers in the Soliris cohort.
Analysts at Cantor Fitzgerald talked up the significance of data against both endpoints, noting that Apellis had outperformed expectations and delivered results that physicians will view as clinically meaningful.
“We think investors were looking for a hemoglobin increase of 2g/dL+ vs. Soliris,” the analysts wrote in a note to investors. “We think the benefit on transfusion independence is highly meaningful. Transfusions are a key endpoint of focus for physicians where our checks suggested a ~20% difference is seen as highly meaningful vs. this is a 70% difference.”
Those positives led investors to drive Apellis’ share price up by more than 50% shortly after the data dropped. However, enthusiasm had waned somewhat by the time the market opened, when Apellis’ shares traded up around 30% over the closing price yesterday.
Exactly why investors cooled on the data is unclear, but the information released by Apellis features potential areas of concern. Rates of injection site reaction, diarrhea and discontinuation were higher in the pegcetacoplan arm, and Apellis’ drug failed to show non-inferiority versus Soliris in terms of its effect on lactate dehydrogenase.
There are also outstanding questions about when Apellis will be able to file for approval of the drug. Apellis set out the current situation in a regulatory filing.
“The FDA has advised us that, for the approval of a new treatment for PNH, hemoglobin stabilization in conjunction with change in transfusion dependence constitute accepted clinical benefit, but that a rise in hemoglobin levels may not translate to clinical benefit in patients who entered the trial with high hemoglobin levels, such as permitted by the inclusion criteria of the PEGASUS trial, and who do not require transfusions,” the company wrote.
Apellis plans to meet with regulatory agencies in the coming months to discuss the next steps for the drug.
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