X-Bi­otix sus­pends R&D ef­forts amid dearth of fi­nanc­ing for an­tibi­otics re­search; Ver­tex picks a pain drug for mid-stage stud­ies

Lament­ing the dearth of fi­nanc­ing avail­able for an­tibi­otics re­search de­spite a grow­ing de­mand for new prod­ucts to fight drug re­sis­tance, lit­tle Waltham, MA-based X-Bi­otix has sus­pend­ed its own R&D ef­forts in the field while con­tin­u­ing to try to hunt up fresh back­ing.

Fund­ed with a small A round in 2018, X-Bi­otix says it will now look to back up its IP and pub­lish man­u­scripts as re­search work laps­es.

An­tibi­otics play­ers have de­scribed a dire lack of fund­ing and in­ter­est in the field, as pay­ers con­tin­ue to re­ly on old, cheap gener­ics to fill most of the de­mand. X-Bi­otix CEO Ra­mani Varanasi has been work­ing with chair­man Steve Isaacs, who ran Aduro for years be­fore toss­ing in the tow­el af­ter a steady drum­beat of fail­ures.

“Pol­i­cy mak­ers must en­act mar­ket-based re­forms, in­clud­ing re­im­burse­ment re­form and com­mer­cial ‘pull’ in­cen­tives, in or­der to re­vi­tal­ize the an­tibi­otics mar­ket and dri­ve sus­tain­able in­vest­ments in an­tibi­otics R&D,” Isaacs not­ed in a state­ment. — John Car­roll

Ver­tex picks a pain drug for mid-stage stud­ies

Over a year af­ter drop­ping their sec­ond at­tempt at a drug for pain, Ver­tex is back with a third.

The big Cam­bridge biotech an­nounced to­day that it’s mov­ing a new mol­e­cule, VX-548, in­to Phase II stud­ies for vis­cer­al and non-vis­cer­al acute pain.

The mol­e­cule re­places an old­er can­di­date, VX-150, that yield­ed proof-of-con­cept da­ta for re­duc­ing pain but which Ver­tex said lacked key fea­tures — such as safe­ty and op­ti­mal dos­ing reg­i­men — they want­ed to see be­fore bring­ing a mol­e­cule in­to mid or late-stage de­vel­op­ment. They scrapped the sec­ond at­tempt, VX-961, in Jan­u­ary of 2020 af­ter dis­ap­point­ing phar­ma­co­ki­net­ic re­sults in Phase I.

VX-548 showed promis­ing PK re­sults in a Phase I study, Ver­tex said.

The se­ries of mol­e­cules are all de­signed to go af­ter Nav1.8, a re­cep­tor that drug de­vel­op­ers be­gan pur­su­ing in the mid-200s af­ter a study show­ing that peo­ple with a rare mu­ta­tion on the re­cep­tor ex­pe­ri­enced no pain. Those at­tempts, though, large­ly fell short as big phar­ma’s best at­tempts proved too weak or too tox­ic. Ver­tex’s VX-548 will be one of the first to reach mid-stage de­vel­op­ment and, if it bears out, could pro­vide a po­tent, non-ad­dic­tive al­ter­na­tive to opi­oids.  — Ja­son Mast

With clin­i­cal hold lift­ed, Voy­ager Ther­a­peu­tics preps its gene ther­a­py for a swift PhI/II launch

About six months af­ter putting a clin­i­cal hold on Voy­ager’s gene ther­a­py for Hunt­ing­ton’s dis­ease, the FDA is giv­ing the biotech a green light to en­ter the clin­ic.

The agency had re­quest­ed more in­for­ma­tion about chem­istry, man­u­fac­tur­ing and con­trols (CMC), in­clud­ing “drug de­vice com­pat­i­bil­i­ty and drug sub­stance and prod­uct char­ac­ter­i­za­tion,”  ac­cord­ing to Voy­ager’s 2020 fi­nan­cial re­port. With that cleared away, VY-HTT01 is ex­pect­ed to en­ter a Phase I/II study lat­er this year.

“Our in­ves­ti­ga­tion­al gene ther­a­py has been de­signed to achieve broad knock­down of HTT mR­NA through­out the brain via a one-time MRI-guid­ed neu­ro­sur­gi­cal de­liv­ery,” CMO Omar Khwa­ja said in a state­ment.

Back in 2019, Sanofi Gen­zyme de­sert­ed pro­grams in an up to $845 mil­lion part­ner­ship with Voy­ager, walk­ing away from an op­tion to ac­quire US co-com­mer­cial­iza­tion rights and ex-US de­vel­op­ment and com­mer­cial­iza­tion rights to mul­ti­ple can­di­dates in­clud­ing VY-HTT01.

The Phase I/II dose es­ca­la­tion study, dubbed VY­TAL, will eval­u­ate the safe­ty and tol­er­a­bil­i­ty of VY-HTT01 in pa­tients with ear­ly man­i­fest Hunt­ing­ton’s dis­ease. Sec­ondary end­points in­clude dis­ease bio­mark­ers and clin­i­cal out­come mea­sures. — Nicole De­Feud­is 

I-Mab reads of­fers first look at how IL-6 in­hibitor per­formed in PhII ul­cer­a­tive col­i­tis pa­tients

Ul­cer­a­tive col­i­tis pa­tients who re­ceived I-Mab’s IL-6 in­hibitor olam­ki­cept saw sig­nif­i­cant­ly high­er re­sponse rates than those who re­ceived a place­bo, the com­pa­ny said on Mon­day.

The 12-week re­sults come from a Phase II study, which hit both pri­ma­ry and key sec­ondary end­points, ac­cord­ing to I-Mab. While it hasn’t re­leased any hard da­ta, the biotech al­so said that more pa­tients in the 600 mg group achieved clin­i­cal re­mis­sion and mu­cos­al heal­ing than pa­tients in the place­bo group, with a p-val­ue of less than 0.001.

“This is the first demon­stra­tion that IL-6 block­ade through the trans-sig­nal­ing path­way plays a sig­nif­i­cant ther­a­peu­tic role in UC,” prin­ci­pal in­ves­ti­ga­tor Min­hu Chen said in a state­ment.

A more de­tailed read­out is com­ing dur­ing Di­ges­tive Dis­ease Week and at the Eu­ro­pean Crohn’s and Col­i­tis Or­gan­i­sa­tion (EC­CO) meet­ing in Ju­ly 2021. — Nicole De­Feud­is 

Caris­ma Ther­a­peu­tics strikes CAR-M deal with Uni­ver­si­ty of Min­neso­ta

A month af­ter dos­ing the first pa­tients with its CAR-M ther­a­py for HER2-over­ex­press­ing sol­id tu­mors, Caris­ma Ther­a­peu­tics is join­ing forces with pro­fes­sor Bruze Blazar at the Uni­ver­si­ty of Min­neso­ta to take its macrophage-based cell ther­a­py re­search be­yond on­col­o­gy.

“The col­lab­o­ra­tion with Dr. Blazar marks the ini­ti­a­tion of the de­vel­op­ment of al­lo­gene­ic, uni­ver­sal donor de­rived mono­cyte and macrophage cell ther­a­pies at CARIS­MA,” Michael Klichin­sky, Caris­ma’s sci­en­tif­ic co-founder and se­nior VP of re­search, said in a state­ment.

“The fo­cus of this mul­ti-year col­lab­o­ra­tion will be op­ti­miz­ing and de­vel­op­ing iP­SC de­rived al­lo­gene­ic chimeric anti­gen re­cep­tor macrophages, fur­ther ex­pand­ing the po­ten­tial of macrophage-based cell ther­a­py for can­cer and oth­er dis­eases,” he said.

The com­pa­ny is keep­ing the fi­nan­cial terms of the deal un­der wraps. — Nicole De­Feud­is 

Bris­tol My­ers Squibb ex­pands on pro­tein degra­da­tion deal with Evotec

Bris­tol My­ers Squibb is wad­ing deep­er in­to the pro­tein degra­da­tion field by ex­pand­ing a part­ner­ship with Evotec to iden­ti­fy new can­di­dates for treat­ing sol­id tu­mors.

By ex­tend­ing the part­ner­ship, struck back in 2018, BMS trig­gered an undis­closed pay­ment to Evotec to ex­pand its screen­ing ef­forts. The col­lab­o­ra­tion makes use of Evotec’s Pa­nOmics plat­form, which com­bines en­hanced through­put pro­teomics, high through­put tran­scrip­tomics and cell imag­ing with the com­pa­ny’s da­ta analy­sis plat­form Pan­Hunter.

So far, two can­di­dates gen­er­at­ed through the part­ner­ship have tran­si­tioned to the lead op­ti­miza­tion phase, ac­cord­ing to Evotec. — Nicole De­Feud­is 

Deer­field joins forces with ac­cel­er­a­tor to sup­port aca­d­e­m­ic re­search

Through a part­ner­ship with a non­prof­it ac­cel­er­a­tor, Deer­field Man­age­ment is in­ject­ing more cash in­to aca­d­e­m­ic re­search.

The com­pa­ny an­nounced on Mon­day that it’s join­ing forces with the Em­pire Dis­cov­ery In­sti­tute (EDI), a non­prof­it that works in part­ner­ship with re­search labs at the Uni­ver­si­ty of Rochester, the Uni­ver­si­ty at Buf­fa­lo, and Roswell Park Com­pre­hen­sive Can­cer Cen­ter. Deer­field says it plans on in­vest­ing $65 mil­lion over the next five years.

A new­ly launched com­pa­ny called Em­pire Deer­field Dis­cov­ery & De­vel­op­ment (ED3), will pro­vide fund­ing for projects in high-need ar­eas, in­clud­ing hard-to-treat and rare dis­eases, ac­cord­ing to a state­ment.

“EDI was found­ed to help over­come the key chal­lenges of­ten faced by life sci­ence re­searchers in acad­e­mia, name­ly a lack of ex­ter­nal fund­ing to ad­vance pre-clin­i­cal de­vel­op­ment and ac­cess to phar­ma­ceu­ti­cal in­dus­try ex­per­tise to ad­vance pro­grams in an ef­fi­cient man­ner to the clin­ic,” EDI CEO Mar­tin Gra­ham said in a state­ment.

Deer­field has struck mul­ti­ple aca­d­e­m­ic part­ner­ships over the years, in­clud­ing with the Broad In­sti­tute of MIT, Har­vard, and the Uni­ver­si­ty of North Car­oli­na, Chapel Hill. — Nicole De­Feud­is 

*This ar­ti­cle pre­vi­ous­ly stat­ed more pa­tients in the treat­ment arm I-Mab’s Phase II tri­al achieved clin­i­cal re­mis­sion and mu­cos­al heal­ing than pa­tients in the place­bo group, with a p-val­ue of less than 0.01. The p-val­ue is less than 0.001.

Biotech Half­time Re­port: Af­ter a bumpy year, is biotech ready to re­bound?

The biotech sector has come down firmly from the highs of February as negative sentiment takes hold. The sector had a major boost of optimism from the success of the COVID-19 vaccines, making investors keenly aware of the potential of biopharma R&D engines. But from early this year, clinical trial, regulatory and access setbacks have reminded investors of the sector’s inherent risks.

RBC Capital Markets recently surveyed investors to take the temperature of the market, a mix of specialists/generalists and long-only/ long-short investment strategies. Heading into the second half of the year, investors mostly see the sector as undervalued (49%), a large change from the first half of the year when only 20% rated it as undervalued. Around 41% of investors now believe that biotech will underperform the S&P500 in the second half of 2021. Despite that view, 54% plan to maintain their position in the market and 41% still plan to increase their holdings.

How to col­lect and sub­mit RWD to win ap­proval for a new drug in­di­ca­tion: FDA spells it out in a long-await­ed guid­ance

Real-world data is messy. There can be differences in the standards used to collect different types of data, differences in terminologies and curation strategies, and even in the way data is exchanged.

While acknowledging this somewhat controlled chaos, the FDA is now explaining how biopharma companies can submit study data derived from real-world data (RWD) sources in applicable regulatory submissions, including new drug indications.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

David Lockhart, ReCode Therapeutics CEO

Pfiz­er throws its weight be­hind LNP play­er eye­ing mR­NA treat­ments for CF, PCD

David Lockhart did not see the meteoric rise of messenger RNA and lipid nanoparticles coming.

Thanks to the worldwide fight against Covid-19, mRNA — the genetic code that can be engineered to turn the body into a mini protein factory — and LNPs, those tiny bubbles of fat carrying those instructions, have found their way into hundreds of millions of people. Within the biotech world, pioneers like Alnylam and Intellia have demonstrated just how versatile LNPs can be as a delivery vehicle for anything from siRNA to CRISPR/Cas9.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 120,300+ biopharma pros reading Endpoints daily — and it's free.

Leen Kawas (L) has resigned as CEO of Athira and will be replaced by COO Mark Litton

Ex­clu­sive: Athi­ra CEO Leen Kawas re­signs af­ter in­ves­ti­ga­tion finds she ma­nip­u­lat­ed da­ta

Leen Kawas, CEO and founder of the Alzheimer’s upstart Athira Pharma, has resigned after an internal investigation found she altered images in her doctoral thesis and four other papers that were foundational to establishing the company.

Mark Litton, the company’s COO since June 2019 and a longtime biotech executive, has been named full-time CEO. Kawas, meanwhile, will no longer have ties to the company except for owning a few hundred thousand shares.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Lian­Bio an­nounces terms for IPO next week; NIH and White House of­fi­cials dis­cuss hy­po­thet­i­cal bio-med re­search agency

LianBio, a biotech that has operations in both the US and China, announced the terms yesterday for its initial public offering.

The biotech plans to raise $325 million by offering 20.3 million shares priced between $15 and $17.

At the midpoint of the proposed range, LianBio could command a fully diluted market value of $1.8 billion, based on a number of expected outstanding shares.

The two year old biotech has focused on in-licensing and commercialization of therapeutics in China, Hong Kong, Taiwan, Macau and other Asian markets. The company currently has nine programs across five therapeutic areas, including oncology, cardiovascular, and inflammatory diseases.

Sen. Richard Durbin (D-IL, foreground) and Sen. Richard Blumenthal (D-CT) (Patrick Semansky/AP Images)

Sen­a­tors back FDA's plan to re­quire manda­to­ry pre­scriber ed­u­ca­tion for opi­oids

Three Senate Democrats are backing an FDA plan to require mandatory prescriber education for opioids as overdose deaths have risen sharply over the past decade, with almost 97,000 American opioid-related overdose deaths in the past year alone.

While acknowledging a decline in overall opioid analgesic dispensing in recent years, the FDA said it’s reconsidering the need for mandatory prescriber training through a REMS given the current situation with overdoses, and is seeking input on the aspects of the opioid crisis that mandatory training could potentially mitigate.

Bris­tol My­ers pledges to sell its Ac­celeron shares as ac­tivist in­vestors cir­cle Mer­ck­'s $11.5B buy­out — re­port

Just as Avoro Capital’s campaign to derail Merck’s proposed $11.5 billion buyout of Acceleron gains steam, Bristol Myers Squibb is leaning in with some hefty counterweight.

The pharma giant is planning to tender its Acceleron shares, Bloomberg reported, which add up to a sizable 11.5% stake. Based on the offer price, the sale would net Bristol Myers around $1.3 billion.

To complete its deal, Merck needs a majority of shareholders to agree to sell their shares.

Boost­er bo­nan­za: FDA en­dors­es 'mix-and-match' scheme, and Mod­er­na and J&J too

The FDA late Wednesday signed off on authorizing the use of heterologous — or what FDA calls a “mix and match” of a primary vaccine series and different booster doses — for all currently available Covid-19 vaccines, in addition to separately authorizing Moderna and J&J boosters.

On the mix-and-match approach, which FDA officials insisted isn’t too confusing in a press conference, the agency offered the example of an 18-year-old who received the J&J shot at least two months ago and may now receive a single booster of the J&J, a half dose of the Moderna, or the Pfizer-BioNTech booster.

David Livingston (Credit: Michael Sazel for CeMM)

Renowned Dana-Far­ber sci­en­tist, men­tor and bio­phar­ma ad­vi­sor David Liv­ingston has died

David Livingston, the Dana-Farber/Harvard Med scientist who helped shine a light on some of the key molecular drivers of breast and ovarian cancer, died unexpectedly last Sunday.

One of the senior leaders at Dana-Farber during his nearly half century of work there, Livingston was credited with shedding light on the genes that regulate cell growth, with insights into inherited BRCA1 and BRCA2 mutations that helped lay the scientific foundation for targeted therapies and earlier detection that have transformed the field.